MTD, Safety, and Efficacy of Pomalidomide (CC-4047) Alone or With Low-dose Dexamethasone in Patients With Relapsed and Refractory Multiple Myeloma

Sponsor

Celgene Corporation (Industry)

Overall Status

Completed

CT.gov ID

NCT00833833

Collaborator

(none)

259

Enrollment

Locations

Arms

Duration (Months)

13.6

Patients Per Site

0.2

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

The purpose of this study is to determine the maximum tolerated dose and effectiveness of the study drug (CC-4047) Alone Or in Combination With Low-dose Dexamethasone as treatment for patients with relapsed and refractory multiple myeloma

Condition or Disease	Intervention/Treatment	Phase
Multiple Myeloma	Drug: Pomalidomide Drug: Dexamethasone Drug: Aspirin	Phase 1/Phase 2

Detailed Description

The Phase 1 segment of the study was designed to determine the maximum tolerated dose (MTD) of single-agent pomalidomide, which was to be determined in the first cycle of treatment. Following completion of the first cycle, participants were allowed to continue the study at their assigned dose of pomalidomide.

Participants who developed progressive disease (PD) at any time, or who had not achieved at least a 25% reduction of serum myeloma (M)-protein levels (if measurable) and a 50% reduction of urine M-protein (if measurable) compared with baseline after completion of 4 cycles of pomalidomide, had the option to receive oral dexamethasone 40 mg on days 1, 8, 15, and 22 of each 28-day treatment cycle in addition to their current dose of pomalidomide. Participants with PD who chose not to add dexamethasone to pomalidomide therapy were to be discontinued from the study. Participants who chose to add dexamethasone were allowed to continue study treatment until PD developed again, unacceptable toxicity or participant withdrew consent, at which time they were to be discontinued.

Based on results from the phase 1 portion, the Data Monitoring Committee confirmed 4 mg/day as MTD of pomalidomide. Therefore, the recommended starting dose of pomalidomide for Phase 2 was 4 mg/day on Days 1-21 of each 28-day cycle. In the combination treatment arm, the starting dose of dexamethasone was 40 mg once per day. For subjects who were > 75 years of age, the starting dose of dexamethasone was 20 mg once per day. To prevent blood clots, all participants were to be given aspirin 81-100 mg daily (commercial supply) unless contraindicated. If contraindicated, another form of anti-thrombotic therapy was provided.

Participants in the Phase II combination treatment arm could continue study treatment until PD developed, at which time they were to be discontinued. Participants in the single agent pomalidomide treatment arm who developed PD, confirmed by the IRAC, at any time had the option to receive oral dexamethasone in addition to their current dose of pomalidomide at the starting dose described above. Participants with PD who chose not to add dexamethasone to pomalidomide therapy were discontinued from study treatment. Participants who chose to add dexamethasone to pomalidomide therapy could continue study treatment until PD developed again, unacceptable toxicity or participant withdrew consent, at which point they were discontinued.

Upon discontinuation from study treatment for PD or any other reason, participants were to be assessed two times per year, up to five years, for survival, second primary malignancy and subsequent anti-myeloma therapies.

Two analyses were planned during the course of the Phase 2 segment: one interim analysis (at 50% information of progression-free survival (PFS) events) and one final analysis. The Data Monitoring Commmittee recommended that Celgene personnel be unblinded based on the strength of the data. Subsequently, Celgene decided to file an application based on more current study data. The product was approved by the FDA in February 2013.

Since no further analyses are required per protocol nor to support the marketing application, the study was amended to remove undue burden from patients who remain on active treatment by ending the treatment segments of this study and transferring all active patients tothe Long-term Follow-up Phase. These patients who continue to be treated with pomalidomide will be provided Pomalyst through the Celgene Patient Support Program (Pomalyst REMSTM) until disease progression, unacceptable toxicity, the investigator decides to change therapy or patient decision. Investigators will treat their patients according to the local standard of care and follow the assessments required for patients in the Long-term Follow-up Phase. These assessments include subsequent myeloma therapies, second primary malignancies and survival.

The study continues. A final analysis will be performed when the study is completed and results reported as available.

Study Design

Study Type:

Interventional

Actual Enrollment :

259 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

A Phase I/II Multicenter, Randomized, Open Label, Dose-Escalation Study To Determine The Maximum Tolerated Dose, Safety, And Efficacy Of CC-4047 Alone Or In Combination With Low-Dose Dexamethasone In Patients Wth Relapsed And Refractory Multiple Myeloma Who Have Received Prior Treatment That Includes Lenalidomide And Bortezomib

Study Start Date :

Jun 1, 2008

Actual Primary Completion Date :

Apr 1, 2011

Actual Study Completion Date :

Sep 1, 2015

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Phase 1: 2 mg pomalidomide Pomalidomide 2 mg daily on days 1-21 of each 28-day cycle. Participants with progressive disease (PD) had the option of adding dexamethasone 40 mg on Days 1, 8, 15, 22 of each 28-day cycle to the pomalidomide treatment, or discontinuing study treatment.	Drug: Pomalidomide 1 mg, 2 mg, and 5 mg capsules for oral administration packaged in bottles containing a 21-day supply Other Names: CC-4047 Pomalyst Drug: Dexamethasone oral dexamethasone Other Names: dexamethasone sodium phosphate Drug: Aspirin As prophylactic anti-thrombotic treatment, all participants were given aspirin 81-100 mg daily (commercial supply) unless contraindicated. If aspirin was contraindicated, participants were given another form of anti-thrombotic therapy according to hospital guidelines or physician preference.
Experimental: Phase 1: 3 mg pomalidomide Pomalidomide 3 mg daily on days 1-21 of each 28-day cycle. Participants with progressive disease (PD) had the option of adding dexamethasone 40 mg on Days 1, 8, 15, 22 of each 28-day cycle to the pomalidomide treatment, or discontinuing study treatment.	Drug: Pomalidomide 1 mg, 2 mg, and 5 mg capsules for oral administration packaged in bottles containing a 21-day supply Other Names: CC-4047 Pomalyst Drug: Dexamethasone oral dexamethasone Other Names: dexamethasone sodium phosphate Drug: Aspirin As prophylactic anti-thrombotic treatment, all participants were given aspirin 81-100 mg daily (commercial supply) unless contraindicated. If aspirin was contraindicated, participants were given another form of anti-thrombotic therapy according to hospital guidelines or physician preference.
Experimental: Phase 1: 4 mg pomalidomide Pomalidomide 4 mg daily on days 1-21 of each 28-day cycle. Participants with progressive disease (PD) had the option of adding dexamethasone 40 mg on Days 1, 8, 15, 22 of each 28-day cycle to the pomalidomide treatment, or discontinuing study treatment.	Drug: Pomalidomide 1 mg, 2 mg, and 5 mg capsules for oral administration packaged in bottles containing a 21-day supply Other Names: CC-4047 Pomalyst Drug: Dexamethasone oral dexamethasone Other Names: dexamethasone sodium phosphate Drug: Aspirin As prophylactic anti-thrombotic treatment, all participants were given aspirin 81-100 mg daily (commercial supply) unless contraindicated. If aspirin was contraindicated, participants were given another form of anti-thrombotic therapy according to hospital guidelines or physician preference.
Experimental: Phase 1: 5 mg pomalidomide Pomalidomide 5 mg daily on days 1-21 of each 28-day cycle. Participants with progressive disease (PD) had the option of adding dexamethasone 40 mg on Days 1, 8, 15, 22 of each 28-day cycle to the pomalidomide treatment, or discontinuing study treatment.	Drug: Pomalidomide 1 mg, 2 mg, and 5 mg capsules for oral administration packaged in bottles containing a 21-day supply Other Names: CC-4047 Pomalyst Drug: Dexamethasone oral dexamethasone Other Names: dexamethasone sodium phosphate Drug: Aspirin As prophylactic anti-thrombotic treatment, all participants were given aspirin 81-100 mg daily (commercial supply) unless contraindicated. If aspirin was contraindicated, participants were given another form of anti-thrombotic therapy according to hospital guidelines or physician preference.
Experimental: Phase 2: pomalidomide + dexamethasone Combination therapy of 4 mg pomalidomide given once per day on Days 1-21 of each 28-day cycle and the starting dose of dexamethasone (determined by age) on days 1, 8, 15, and 22 of each 28-day cycle. The starting dose of dexamethasone was 40 mg for participants who were ≤ 75 years of age and 20 mg for participants who were > 75 years of age. Dose reduction steps for dexamethasone were provided for drug-related toxicities.	Drug: Pomalidomide 1 mg, 2 mg, and 5 mg capsules for oral administration packaged in bottles containing a 21-day supply Other Names: CC-4047 Pomalyst Drug: Dexamethasone oral dexamethasone Other Names: dexamethasone sodium phosphate Drug: Aspirin As prophylactic anti-thrombotic treatment, all participants were given aspirin 81-100 mg daily (commercial supply) unless contraindicated. If aspirin was contraindicated, participants were given another form of anti-thrombotic therapy according to hospital guidelines or physician preference.
Experimental: Phase 2: pomalidomide 4 mg pomalidomide was given once per day on Days 1-21 of each 28-day cycle until PD. Participants in the single agent pomalidomide treatment arm who developed confirmed PD at any time had the option to receive oral dexamethasone on days 1, 8, 15, and 22 of each 28-day cycle at the starting dose of 20 or 40 mg depending on age in addition to their current dose of pomalidomide, or to discontinue treatment.	Drug: Pomalidomide 1 mg, 2 mg, and 5 mg capsules for oral administration packaged in bottles containing a 21-day supply Other Names: CC-4047 Pomalyst Drug: Dexamethasone oral dexamethasone Other Names: dexamethasone sodium phosphate Drug: Aspirin As prophylactic anti-thrombotic treatment, all participants were given aspirin 81-100 mg daily (commercial supply) unless contraindicated. If aspirin was contraindicated, participants were given another form of anti-thrombotic therapy according to hospital guidelines or physician preference.

Outcome Measures

Primary Outcome Measures

Phase 1: Participants With Dose-Limiting Toxicities (DLT) in Cycle 1 [Up to Day 28 (Cycle 1)]
The maximum tolerated dose was defined as the highest dose level at which no more than 1 of 6 participants experiences a DLT within the first 28-day cycle. DLTs were defined as: Grade 4 neutropenia or thrombocytopenia Febrile neutropenia Grade 3 or 4 nausea, vomiting or diarrhea despite optimal symptomatic treatment Serum transaminase > 20 * upper limit of normal (ULN) Serum transaminase > 5 * ULN for >= 7 days Delay of the start of cycle 2 by >7 days due to pomalidomide-related adverse event
Phase 2: Kaplan-Meier Estimates of Progression-free Survival (PFS) as of the 01 April 2011 Cut-off [up to 67 weeks]
Progression free survival (PFS) is the time from randomization to the first documentation of disease progression or death from any cause during study, whichever occurs earlier. Disease progression was assessed by the Independent Response Adjudication Committee (IRAC). For the primary PFS analysis, participants who withdrew for any reason or received another antimyeloma therapy (except adding dexamethasone to the Phase 2: Pomalidomide arm) without documented PD (as determined by the IRAC review) were censored on the date of their last adequate response assessment, prior to receiving any other anti-myeloma therapy. Subjects who were still active at the time of the data cut-off date without PD (as determined by the IRAC) were censored on the date of their last adequate response assessment. Data collection is ongoing and future data results will be included as available.
Phase 2: Percentage of Participants With Progression-Free Survival (PFS) Events as of the 01 April 2011 Cut-off [up to 67 weeks]
Percentage of participants with the progression-free survival events: disease progression and death. Disease progression was assessed by the Independent Response Adjudication Committee (IRAC). Data collection is ongoing and future data results will be included as available.

Secondary Outcome Measures

Phase 1: Percentage of Participants With Treatment-Emergent Adverse Events (TEAEs) While on Single-Agent Pomalidomide as of the 01 April 2011 Cut-off [Up to week 104]
Relation to study drug was assessed by the Investigator as either suspected or not suspected. Counts represent the suspected relationship. Severity was assessed using National Cancer Institute Common Toxicity Terminology Criteria for Adverse Events version 3.0 (NCI CTCAE v3.0): 1= Mild 2= Moderate 3= Severe 4= Life-threatening and 5= Death related to AE. Serious AEs (SAEs) are those that resulted in death, were life-threatening, required or prolonged inpatient hospitalization, resulted in persistent or significant disability/incapacity, congenital anomaly, or resulted in an important medical event that may have jeopardized the patient or required medical or surgical intervention to prevent one of the outcomes listed above. Data collection is ongoing and future data results will be included as available.
Phase 1: Percentage of Participants With Treatment-Emergent Adverse Events (TEAEs) While on Both Pomalidomide and Dexamethasone as of the 01 April 2011 Cut-off [Up to week 126]
TEAEs that occurred during Phase 1 after dexamethasone was added to pomalidomide treatment. Relation to study drug was assessed by the Investigator as either suspected or not suspected. Counts represent the suspected relationship. Severity was assessed using National Cancer Institute Common Toxicity Terminology Criteria for Adverse Events version 3.0 (NCI CTCAE v3.0): 1= Mild 2= Moderate 3= Severe 4= Life-threatening and 5= Death related to AE. Serious AEs (SAEs) are those that resulted in death, were life-threatening, required or prolonged inpatient hospitalization, resulted in persistent or significant disability/incapacity, congenital anomaly, or resulted in an important medical event that may have jeopardized the patient or required medical or surgical intervention to prevent one of the outcomes listed above. Data collection is ongoing and future data results will be included as available.
Phase 2: Percentage of Participants With Treatment-Emergent Adverse Events (TEAEs) as of the 01 April 2011 Cut-off [Up to week 70]
Relation to study drug was assessed by the Investigator as either suspected or not suspected. Counts represent the suspected relationship. Severity was assessed using National Cancer Institute Common Toxicity Terminology Criteria for Adverse Events version 3.0 (NCI CTCAE v3.0): 1= Mild 2= Moderate 3= Severe 4= Life-threatening and 5= Death related to AE. Serious AEs (SAEs) are those that resulted in death, were life-threatening, required or prolonged inpatient hospitalization, resulted in persistent or significant disability/incapacity, congenital anomaly, or resulted in an important medical event that may have jeopardized the patient or required medical or surgical intervention to prevent one of the outcomes listed above. Data collection is ongoing and future data results will be included as available.
Phase 2: Summary of Best Myeloma Response As Assessed by Independent Response Adjudication Committee (IRAC) Using European Group for Blood and Bone Marrow Transplant (EBMT) Criteria as of the 01 April 2011 Cut-off [up to 70 weeks]
IRAC used EBMT criteria to assess myeloma response: Complete Response (CR)-absence of serum and urine monoclonal paraprotein for 6 weeks, plus no increase in size or number of lytic bone lesions, plus other factors) Partial Response (PR)-not all CR criteria, plus >=50% reduction in serum monoclonal paraprotein plus others Minimal Response (MR)- 25-49% reduction in serum monoclonal paraprotein plus others Stable Disease (SD)- not MR or progressive disease (PD) Progressive Disease (PD)- reappearance of monoclonal paraprotein, lytic bone lesions, other Not Evaluable (NE). Data collection is ongoing and future data results will be included as available.
Phase 2: Kaplan-Meier Estimates of Duration of Response as of the 01 April 2011 Cut-off [up to 70 weeks]
Duration of myeloma response is defined as the time from when the response criteria are first met for partial response (PR) or better, until the first date the response criteria are met for progressive disease (PD) or until the participant dies from any cause, whichever occurs first. Duration of response for participants last known to be alive with no progression after a complete response (CR) or PR was censored at the date of last adequate response assessment. Participants with confirmed responses that occur after receiving any other anti-myeloma therapy (except for adding dexamethasone to the pomalidomide treatment arm), including radiation therapy initiated after baseline, was censored at the last adequate assessment prior to the initiation of such treatment. Response was assessed by the Independent Response Adjudication Committee (IRAC) using European Group for Blood and Bone Marrow Transplant (EBMT) criteria as described in the previous outcome.
Phase 2: Time to Response as of the 01 April 2011 Cut-off [up to 70 weeks]
Time to myeloma response is defined as the time from randomization to the time the response criteria for complete response (CR) or partial response (PR) are first met. Response was assessed by the Independent Response Adjudication Committee (IRAC) using European Group for Blood and Bone Marrow Transplant (EBMT) criteria as described previously. Data collection is ongoing and future data results will be included as available.
Phase 2: Kaplan-Meier Estimates of Overall Survival as of the 01 April 2011 Cut-off [up to 70 weeks]
Overall survival was defined as the time between randomization and death. Participants who die, regardless of the cause of the death, were considered to have had an event. All participants who were lost to follow-up prior to the end of the trial or who were withdrawn from the trial were censored at the time of last contact. Participants who were still being treated were censored at the last available date the subject was known to be alive, or clinical cut-off date if it was earlier. Data collection is ongoing and future data results will be included as available.

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Must be greater than or equal to 18 years at the time of signing the informed consent form
Must be able to adhere to the study visit schedule and other protocol requirements
Have a documented diagnosis of multiple myeloma and have relapsed and refractory disease. Patients must have received at least 2 prior therapies. Patients must have relapsed after having achieved at least stable disease for at least one cycle of treatment to at least one prior regimen and then developed PD. Patients must also have documented evidence of PD during or within 60 days (measured from the end of the last cycle) of completing treatment with the last anti-myeloma drug regimen used just prior to study entry (refractory disease)
Patients must have also undergone prior treatment with at least 2 cycles of lenalidomide and at least 2 cycles of bortezomib (either in separate regimens or within the same regimen)
Measurable levels of myeloma paraprotein in serum (greater than or equal to 0.5 g/dL) or urine (greater than or equal 0.2 g/dL excreted in a 24 hour collection sample)
Eastern Cooperative Oncology Group (ECOG) performance status score of 0, 1, or 2
Females of childbearing potential (FCBP) [An FCBP is a sexually mature woman who: 1) has not undergone a hysterectomy or bilateral oophorectomy; or 2) has not been naturally postmenopausal for at least 24 consecutive months (i.e., who has had menses at any time in the preceding 24 consecutive months)] must agree to refrain from becoming pregnant for 28 days prior to initiation of study drug, while on study drug and for 28 days after discontinuation of study drug and must agree to regular pregnancy testing during this timeframe.
All patients must also agree to refrain from donating blood while on study drug and for 28 days after discontinuation from this study
Males must agree to use a latex condom during any sexual contact with FCBP while participating in the study and for 28 days following discontinuation from this study even if he has undergone a successful vasectomy. Males must also agree to refrain from donating blood, semen or sperm during the above referenced timeframe.
All patients must agree not to share medication with another person.

Exclusion Criteria:

Any serious medical condition, laboratory abnormality, or psychiatric illness that would prevent the patient from signing the informed consent form.
Any serious concurrent medical conditions that may make the patient non-evaluable or put the patient's safety at risk.
Pregnant or lactating females
Any of the following laboratory abnormalities:
Absolute neutrophil count (ANC) < 1,000 cells/mm3
Platelet count < 75,000/mm3 for patients in whom < 50% of bone marrow nucleated cells are plasma cells; or a platelet count < 30,000/mm3 for patients in whom ≥ 50% of bone marrow nucleated cells are plasma cells
Serum creatinine > 3.0 mg/dL
Serum glutamic oxaloacetic transaminase/Aspartate transaminase (SGOT/AST) or Serum Glutamic Pyruvate Transaminase/Alanine transaminase (SGPT/ALT) > 3.0 X upper limit of normal (ULN)
Serum total bilirubin > 2.0 mg/dL
Prior history of malignancies, other than multiple myeloma, unless the patient has been free of the disease for ≥ 3 years. Exceptions include the following:
Basal or squamous cell carcinoma of the skin
Carcinoma in situ of the cervix or breast
Incidental histologic finding of prostate cancer (TNM stage of T1a or T1b)
Known Human Immunodeficiency Virus (HIV), Hepatitis B Virus (HBV) and/or Hepatitis C Virus (HCV) infection
Hypersensitivity to thalidomide, lenalidomide, or dexamethasone
Peripheral neuropathy ≥ Grade 2
Use of any anti-myeloma drug therapy within 14 days of the initiation of study drug treatment or use of any experimental non-drug therapy within 28 days of the initiation of study drug treatment
Radiation therapy within 14 days of initiation of study drug treatment Inability or unwillingness to comply with birth control requirements

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Mayo Clinic Arizona	Scottsdale	Arizona	United States	85259
2	Colorado Blood Cancer Institute	Denver	Colorado	United States	80218
3	Moffitt Cancer Center	Tampa	Florida	United States	33612
4	Winship Cancer Institute of Emory University	Atlanta	Georgia	United States	30322
5	Mass General Hospital	Boston	Massachusetts	United States	02114
6	Dana-Farber Cancer Institute	Boston	Massachusetts	United States	02115
7	University of Michigan Comprehensive Cancer CenterDivision of Hematology/Oncology	Ann Arbor	Michigan	United States	48109
8	Mayo Clinic - Arizona	Rochester	Minnesota	United States	55905
9	Washington University School of Medicine	St. Louis	Missouri	United States	63110
10	The Cancer Center at Hackensack University Medical Center	Hackensack	New Jersey	United States	07601
11	Roswell Park Cancer Institute Department of Medicine	Buffalo	New York	United States	14263
12	Mt. Sinai Hospital	New York	New York	United States	10029
13	Ohio State University Arthur G. James Cancer Hospital	Columbus	Ohio	United States	43210-1240
14	University of Pittsburgh	Pittsburgh	Pennsylvania	United States	15232
15	Tom Baker Cancer Center	Calgary	Alberta	Canada	T2N 2T9
16	Cross Cancer Institute	Edmonton	Alberta	Canada	T6G 1Z2
17	Vancouver General Hospital	Vancouver	British Columbia	Canada	V5Z 1M9
18	Princess Margaret Hospital and University of Toronto	Toronto	Ontario	Canada	M5G 2M9
19	Royal Victoria Hosptial	Montreal	Quebec	Canada	H3A 1A1

Sponsors and Collaborators

Celgene Corporation

Investigators

Study Director: Lars A Sternas, MD, Celgene Corporation

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

Celgene Corporation

ClinicalTrials.gov Identifier:

NCT00833833

Other Study ID Numbers:

CC-4047-MM-002

First Posted:

Feb 2, 2009

Last Update Posted:

Apr 27, 2016

Last Verified:

Mar 1, 2016

Additional relevant MeSH terms:

Multiple Myeloma

Neoplasms, Plasma Cell

Aspirin

Dexamethasone

Dexamethasone acetate

Pomalidomide

Dexamethasone 21-phosphate

BB 1101

Study Results

Participant Flow

Recruitment Details
Pre-assignment Detail	Phase 2: Participants were stratified by age (≤ 75 vs. > 75), prior number of treatments (2 vs. > 2), and prior thalidomide exposure (yes vs. no).

Arm/Group Title	Phase 1: 2 mg Pomalidomide	Phase 1: 3 mg Pomalidomide	Phase 1: 4 mg Pomalidomide	Phase 1: 5 mg Pomalidomide	Phase 2: Pomalidomide + Dexamethasone	Phase 2: Pomalidomide
Arm/Group Description	Pomalidomide 2 mg daily on days 1-21 of each 28-day cycle. Upon PD, participants had the option of discontinuing treatment or adding dexamethasone.	Pomalidomide 3 mg daily on days 1-21 of each 28-day cycle. Upon PD, participants had the option of discontinuing treatment or adding dexamethasone.	Pomalidomide 4 mg daily on days 1-21 of each 28-day cycle. Upon PD, participants had the option of discontinuing treatment or adding dexamethasone.	Pomalidomide 5 mg daily on days 1-21 of each 28-day cycle. Upon PD, participants had the option of discontinuing treatment or adding dexamethasone.	Combination therapy of 4 mg pomalidomide given once per day on Days 1-21 of each 28-day cycle and the starting dose of dexamethasone (either 40mg or 20 mg) on days 1, 8, 15, and 22 of each 28-day cycle.	4 mg pomalidomide was given once per day on Days 1-21 of each 28-day cycle until PD. Participants in the single agent pomalidomide treatment arm who developed confirmed PD at any time had the option to receive oral dexamethasone at the starting dose in addition to their current dose of pomalidomide, or to discontinue treatment.
Period Title: Phase 1 (as of 01 April 2011)
STARTED	6	8	14	10	0	0
COMPLETED	2	3	5	4	0	0
NOT COMPLETED	4	5	9	6	0	0
Period Title: Phase 1 (as of 01 April 2011)
STARTED	0	0	0	0	113	108
Safety Population	0	0	0	0	112	107
COMPLETED	0	0	0	0	58	47
NOT COMPLETED	0	0	0	0	55	61

Baseline Characteristics

Arm/Group Title	Phase 1: 2 mg Pomalidomide	Phase 1: 3 mg Pomalidomide	Phase 1: 4 mg Pomalidomide	Phase 1: 5 mg Pomalidomide	Phase 2: Pomalidomide + Dexamethasone	Phase 2: Pomalidomide	Total
Arm/Group Description	Pomalidomide 2 mg daily on days 1-21 of each 28-day cycle. Upon PD, participants had the option of discontinuing treatment or adding dexamethasone.	Pomalidomide 3 mg daily on days 1-21 of each 28-day cycle. Upon PD, participants had the option of discontinuing treatment or adding dexamethasone.	Pomalidomide 4 mg daily on days 1-21 of each 28-day cycle. Upon PD, participants had the option of discontinuing treatment or adding dexamethasone.	Pomalidomide 5 mg daily on days 1-21 of each 28-day cycle. Upon PD, participants had the option of discontinuing treatment or adding dexamethasone.	Combination therapy of 4 mg pomalidomide given once per day on Days 1-21 of each 28-day cycle and the starting dose of dexamethasone (either 40mg or 20 mg) on days 1, 8, 15, and 22 of each 28-day cycle.	4 mg pomalidomide was given once per day on Days 1-21 of each 28-day cycle until PD. Participants in the single agent pomalidomide treatment arm who developed confirmed PD at any time had the option to receive oral dexamethasone at the starting dose in addition to their current dose of pomalidomide, or to discontinue treatment.	Total of all reporting groups
Overall Participants	6	8	14	10	113	108	259
Age (years) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [years]	64.7 (6.83)	70.4 (6.63)	67.5 (8.98)	61.3 (14.06)	64.4 (9.24)	62.9 (10.35)	64.1 (9.86)
Age, Customized (participants) [Number]
<=75 years	6 100%	6 75%	11 78.6%	9 90%	99 87.6%	95 88%	226 87.3%
>75 years	0 0%	2 25%	3 21.4%	1 10%	14 12.4%	13 12%	33 12.7%
Sex: Female, Male (Count of Participants)
Female	5 83.3%	5 62.5%	4 28.6%	6 60%	51 45.1%	51 47.2%	122 47.1%
Male	1 16.7%	3 37.5%	10 71.4%	4 40%	62 54.9%	57 52.8%	137 52.9%
Race/Ethnicity, Customized (participants) [Number]
Asian	0 0%	0 0%	0 0%	2 20%	2 1.8%	3 2.8%	7 2.7%
Black or African American	1 16.7%	0 0%	0 0%	0 0%	17 15%	16 14.8%	34 13.1%
White	5 83.3%	8 100%	14 100%	8 80%	92 81.4%	86 79.6%	213 82.2%
Other	0 0%	0 0%	0 0%	0 0%	2 1.8%	3 2.8%	5 1.9%
Baseline Electrocardiogram Findings (participants) [Number]
Normal	3 50%	3 37.5%	3 21.4%	4 40%	53 46.9%	44 40.7%	110 42.5%
Abnormal, not clinically significant	3 50%	5 62.5%	11 78.6%	6 60%	56 49.6%	59 54.6%	140 54.1%
Abnormal, clinically significant	0 0%	0 0%	0 0%	0 0%	0 0%	1 0.9%	1 0.4%
Missing	0 0%	0 0%	0 0%	0 0%	4 3.5%	4 3.7%	8 3.1%
Eastern Cooperative Oncology Group Performance Status (participants) [Number]
0	1 16.7%	0 0%	3 21.4%	2 20%	32 28.3%	24 22.2%	62 23.9%
1	1 16.7%	8 100%	10 71.4%	5 50%	68 60.2%	71 65.7%	163 62.9%
2	4 66.7%	0 0%	1 7.1%	3 30%	13 11.5%	11 10.2%	32 12.4%
3	0 0%	0 0%	0 0%	0 0%	0 0%	2 1.9%	2 0.8%
Baseline Multiple Myeloma Stage (participants) [Number]
Stage I	1 16.7%	1 12.5%	3 21.4%	2 20%	8 7.1%	8 7.4%	23 8.9%
Stage II	0 0%	1 12.5%	4 28.6%	1 10%	29 25.7%	29 26.9%	64 24.7%
Stage III	5 83.3%	6 75%	7 50%	7 70%	76 67.3%	71 65.7%	172 66.4%
Prior Anti-Myeloma Therapies (participants) [Number]
Yes	6 100%	8 100%	14 100%	10 100%	113 100%	108 100%	259 100%
No	0 0%	0 0%	0 0%	0 0%	0 0%	0 0%	0 0%
Prior Thalidomide Exposure (participants) [Number]
Yes	4 66.7%	6 75%	11 78.6%	9 90%	76 67.3%	72 66.7%	178 68.7%
No	2 33.3%	2 25%	3 21.4%	1 10%	37 32.7%	36 33.3%	81 31.3%
Prior Stem Cell Transplant (participants) [Number]
Yes	4 66.7%	4 50%	11 78.6%	6 60%	84 74.3%	82 75.9%	191 73.7%
No	2 33.3%	4 50%	3 21.4%	4 40%	29 25.7%	26 24.1%	68 26.3%

Outcome Measures

1. Primary Outcome

Title	Phase 1: Participants With Dose-Limiting Toxicities (DLT) in Cycle 1
Description	The maximum tolerated dose was defined as the highest dose level at which no more than 1 of 6 participants experiences a DLT within the first 28-day cycle. DLTs were defined as: Grade 4 neutropenia or thrombocytopenia Febrile neutropenia Grade 3 or 4 nausea, vomiting or diarrhea despite optimal symptomatic treatment Serum transaminase > 20 * upper limit of normal (ULN) Serum transaminase > 5 * ULN for >= 7 days Delay of the start of cycle 2 by >7 days due to pomalidomide-related adverse event
Time Frame	Up to Day 28 (Cycle 1)

Outcome Measure Data

Analysis Population Description
Safety population

Arm/Group Title	Phase 1: 2 mg Pomalidomide	Phase 1: 3 mg Pomalidomide	Phase 1: 4 mg Pomalidomide	Phase 1: 5 mg Pomalidomide
Arm/Group Description	Pomalidomide 2 mg daily on days 1-21 of each 28-day cycle. Upon PD, participants had the option of discontinuing treatment or adding dexamethasone.	Pomalidomide 3 mg daily on days 1-21 of each 28-day cycle. Upon PD, participants had the option of discontinuing treatment or adding dexamethasone.	Pomalidomide 4 mg daily on days 1-21 of each 28-day cycle. Upon PD, participants had the option of discontinuing treatment or adding dexamethasone.	Pomalidomide 5 mg daily on days 1-21 of each 28-day cycle. Upon PD, participants had the option of discontinuing treatment or adding dexamethasone.
Measure Participants	6	8	14	10
Number [participants]	1 16.7%	1 12.5%	2 14.3%	4 40%

2. Primary Outcome

Title	Phase 2: Kaplan-Meier Estimates of Progression-free Survival (PFS) as of the 01 April 2011 Cut-off
Description	Progression free survival (PFS) is the time from randomization to the first documentation of disease progression or death from any cause during study, whichever occurs earlier. Disease progression was assessed by the Independent Response Adjudication Committee (IRAC). For the primary PFS analysis, participants who withdrew for any reason or received another antimyeloma therapy (except adding dexamethasone to the Phase 2: Pomalidomide arm) without documented PD (as determined by the IRAC review) were censored on the date of their last adequate response assessment, prior to receiving any other anti-myeloma therapy. Subjects who were still active at the time of the data cut-off date without PD (as determined by the IRAC) were censored on the date of their last adequate response assessment. Data collection is ongoing and future data results will be included as available.
Time Frame	up to 67 weeks

Outcome Measure Data

Analysis Population Description
Intent to treat population. Data collection is ongoing and future data results will be included as available.

Arm/Group Title	Phase 2: Pomalidomide + Dexamethasone	Phase 2: Pomalidomide
Arm/Group Description	Combination therapy of 4 mg pomalidomide given once per day on Days 1-21 of each 28-day cycle and the starting dose of dexamethasone (either 40mg or 20 mg) on days 1, 8, 15, and 22 of each 28-day cycle.	4 mg pomalidomide was given once per day on Days 1-21 of each 28-day cycle until PD. Participants in the single agent pomalidomide treatment arm who developed confirmed PD at any time had the option to receive oral dexamethasone at the starting dose in addition to their current dose of pomalidomide, or to discontinue treatment.
Measure Participants	113	108
Median (95% Confidence Interval) [weeks]	16.6	10.7

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Phase 1: 2 mg Pomalidomide, Phase 1: 3 mg Pomalidomide
	Comments	With a 12-month accrual period and 12-month follow-up after the study closed to accrual, assuming a 10% drop out rate, 96 participants in each treatment arm would have had 85% power to detect a hazard rate ratio of 1.67 using a one-sided log rank test with an overall significance level of 0.025 adjusted for one interim analysis) and a significance level of 0.0245 for the final analysis.
	Type of Statistical Test	Superiority or Other
	Comments

Statistical Test of Hypothesis	p-Value
	Comments
	Method
	Comments

Method of Estimation	Estimation Parameter	Cox Proportional Hazard
	Estimated Value	0.73
	Confidence Interval	(2-Sided) 95% 0.54 to 0.99
	Parameter Dispersion	Type: Value:
	Estimation Comments

3. Secondary Outcome

Title	Phase 1: Percentage of Participants With Treatment-Emergent Adverse Events (TEAEs) While on Single-Agent Pomalidomide as of the 01 April 2011 Cut-off
Description	Relation to study drug was assessed by the Investigator as either suspected or not suspected. Counts represent the suspected relationship. Severity was assessed using National Cancer Institute Common Toxicity Terminology Criteria for Adverse Events version 3.0 (NCI CTCAE v3.0): 1= Mild 2= Moderate 3= Severe 4= Life-threatening and 5= Death related to AE. Serious AEs (SAEs) are those that resulted in death, were life-threatening, required or prolonged inpatient hospitalization, resulted in persistent or significant disability/incapacity, congenital anomaly, or resulted in an important medical event that may have jeopardized the patient or required medical or surgical intervention to prevent one of the outcomes listed above. Data collection is ongoing and future data results will be included as available.
Time Frame	Up to week 104

Outcome Measure Data

Analysis Population Description
Safety population. Data collection is ongoing and future data results will be included as available.

Arm/Group Title	Phase 1: 2 mg Pomalidomide	Phase 1: 3 mg Pomalidomide	Phase 1: 4 mg Pomalidomide	Phase 1: 5 mg Pomalidomide
Arm/Group Description	Pomalidomide 2 mg daily on days 1-21 of each 28-day cycle. Upon PD, participants had the option of discontinuing treatment or adding dexamethasone.	Pomalidomide 3 mg daily on days 1-21 of each 28-day cycle. Upon PD, participants had the option of discontinuing treatment or adding dexamethasone.	Pomalidomide 4 mg daily on days 1-21 of each 28-day cycle. Upon PD, participants had the option of discontinuing treatment or adding dexamethasone.	Pomalidomide 5 mg daily on days 1-21 of each 28-day cycle. Upon PD, participants had the option of discontinuing treatment or adding dexamethasone.
Measure Participants	6	8	14	10
1 or more (1+) AE	100.0 1666.7%	100.0 1250%	100.0 714.3%	100.0 1000%
1+ AE related to pomalidomide	66.7 1111.7%	75.0 937.5%	85.7 612.1%	100.0 1000%
1+ severity grade 3-4 AE	83.3 1388.3%	37.5 468.8%	78.6 561.4%	80.0 800%
1+ severity grade 3-4 AE related to pomalidomide	33.3 555%	25.0 312.5%	42.9 306.4%	70.0 700%
1+ serious AE (SAE)	50.0 833.3%	12.5 156.3%	42.9 306.4%	30.0 300%
1+ SAE related to pomalidomide	0.0 0%	12.5 156.3%	7.1 50.7%	10.0 100%
1+ AE leading to discontinuation of pomalidomide	16.7 278.3%	0.0 0%	21.4 152.9%	0.0 0%
1+AE-dose reduction/interruption of pomalidomide	16.7 278.3%	75.0 937.5%	42.9 306.4%	80.0 800%
1+related AE-reduction/interruption of pomalidomid	0.0 0%	37.5 468.8%	28.6 204.3%	70.0 700%

4. Secondary Outcome

Title	Phase 1: Percentage of Participants With Treatment-Emergent Adverse Events (TEAEs) While on Both Pomalidomide and Dexamethasone as of the 01 April 2011 Cut-off
Description	TEAEs that occurred during Phase 1 after dexamethasone was added to pomalidomide treatment. Relation to study drug was assessed by the Investigator as either suspected or not suspected. Counts represent the suspected relationship. Severity was assessed using National Cancer Institute Common Toxicity Terminology Criteria for Adverse Events version 3.0 (NCI CTCAE v3.0): 1= Mild 2= Moderate 3= Severe 4= Life-threatening and 5= Death related to AE. Serious AEs (SAEs) are those that resulted in death, were life-threatening, required or prolonged inpatient hospitalization, resulted in persistent or significant disability/incapacity, congenital anomaly, or resulted in an important medical event that may have jeopardized the patient or required medical or surgical intervention to prevent one of the outcomes listed above. Data collection is ongoing and future data results will be included as available.
Time Frame	Up to week 126

Outcome Measure Data

Analysis Population Description
Safety population. Data collection is ongoing and future data results will be included as available.

Arm/Group Title	Phase 1: 2 mg Pomalidomide	Phase 1: 3 mg Pomalidomide	Phase 1: 4 mg Pomalidomide	Phase 1: 5 mg Pomalidomide
Arm/Group Description	Pomalidomide 2 mg daily on days 1-21 of each 28-day cycle. Upon PD, participants had the option of discontinuing treatment or adding dexamethasone.	Pomalidomide 3 mg daily on days 1-21 of each 28-day cycle. Upon PD, participants had the option of discontinuing treatment or adding dexamethasone.	Pomalidomide 4 mg daily on days 1-21 of each 28-day cycle. Upon PD, participants had the option of discontinuing treatment or adding dexamethasone.	Pomalidomide 5 mg daily on days 1-21 of each 28-day cycle. Upon PD, participants had the option of discontinuing treatment or adding dexamethasone.
Measure Participants	1	4	10	7
1 or more (1+) AE	100.0 1666.7%	100.0 1250%	90.0 642.9%	100.0 1000%
1+ AE related to pomalidomide	100.0 1666.7%	75.0 937.5%	80.0 571.4%	100.0 1000%
1+ AE related to dexamethasone	100.0 1666.7%	75.0 937.5%	70.0 500%	85.7 857%
1+ severity grade 3-4 AE	0.0 0%	75.0 937.5%	70.0 500%	57.1 571%
1+ severity grade 3-4 AE related to pomalidomide	0.0 0%	75.0 937.5%	20.0 142.9%	42.9 429%
1+ severity grade 3-4 AE related to dexamethasone	0.0 0%	75.0 937.5%	20.0 142.9%	0.0 0%
1+ serious AE (SAE)	0.0 0%	75.0 937.5%	40.0 285.7%	28.6 286%
1+ SAE related to pomalidomide	0.0 0%	25.0 312.5%	20.0 142.9%	0.0 0%
1+ SAE related to dexamethasone	0.0 0%	50.0 625%	10.0 71.4%	0.0 0%
1+ AE leading to discontinuation of pomalidomide	0.0 0%	25.0 312.5%	20.0 142.9%	0.0 0%
1+ AE -- discontinuation of dexamethasone	0.0 0%	25.0 312.5%	20.0 142.9%	0.0 0%
1+AE -dose reduction/interruption of pomalidomide	0.0 0%	50.0 625%	40.0 285.7%	71.4 714%
1+ AE-dose reduction/interruption of dexamethasone	0.0 0%	75.0 937.5%	60.0 428.6%	71.4 714%
1+related AE-reduction/interruption of pomalidomid	0.0 0%	25.0 312.5%	20.0 142.9%	57.1 571%
1+related AE-reduction/interruption of dexamethaso	0.0 0%	75.0 937.5%	20.0 142.9%	42.9 429%

5. Secondary Outcome

Title	Phase 2: Percentage of Participants With Treatment-Emergent Adverse Events (TEAEs) as of the 01 April 2011 Cut-off
Description	Relation to study drug was assessed by the Investigator as either suspected or not suspected. Counts represent the suspected relationship. Severity was assessed using National Cancer Institute Common Toxicity Terminology Criteria for Adverse Events version 3.0 (NCI CTCAE v3.0): 1= Mild 2= Moderate 3= Severe 4= Life-threatening and 5= Death related to AE. Serious AEs (SAEs) are those that resulted in death, were life-threatening, required or prolonged inpatient hospitalization, resulted in persistent or significant disability/incapacity, congenital anomaly, or resulted in an important medical event that may have jeopardized the patient or required medical or surgical intervention to prevent one of the outcomes listed above. Data collection is ongoing and future data results will be included as available.
Time Frame	Up to week 70

Outcome Measure Data

Analysis Population Description
Safety population. Data collection is ongoing and future data results will be included as available.

Arm/Group Title	Phase 2: Pomalidomide + Dexamethasone	Phase 2: Pomalidomide (Pom Only)	Phase 2: Pomalidomide (Pom + Dex Only)	Phase 2: Pomalidomide (Overall)
Arm/Group Description	Combination therapy of 4 mg pomalidomide given once per day on Days 1-21 of each 28-day cycle and the starting dose of dexamethasone (either 40mg or 20 mg) on days 1, 8, 15, and 22 of each 28-day cycle.	Pomalidomide 4 mg daily on days 1-21 of each 28-day cycle. Upon PD, participants had the option of discontinuing treatment or adding dexamethasone. Data are reported during the time when participants were on pomalidomide alone, before dexamethasone was added.	Pomalidomide 4 mg daily on days 1-21 of each 28-day cycle. Upon PD, participants had the option of discontinuing treatment or adding dexamethasone. Data are reported during the time after PD when participants were on both pomalidomide and dexamethasone.	Pomalidomide 4 mg daily on days 1-21 of each 28-day cycle. Upon PD, participants had the option of discontinuing treatment or adding dexamethasone. Data are reported during the entire study up to the data cut-off, thus including both the time participants were only on pomalidomide and the time after PD when participants were on pomalidomide and dexamethasone.
Measure Participants	112	107	61	107
1 or more (1+) AE	100.0 1666.7%	99.1 1238.8%	93.4 667.1%	100.0 1000%
1+ AE related to pomalidomide	89.3 1488.3%	87.9 1098.8%	68.9 492.1%	88.8 888%
1+ severity grade 3-4 AE	88.4 1473.3%	84.1 1051.3%	70.5 503.6%	89.7 897%
1+ severity grade 3-4 AE related to pomalidomide	62.5 1041.7%	58.9 736.3%	44.3 316.4%	67.3 673%
1+ serious AE (SAE)	61.6 1026.7%	46.7 583.8%	47.5 339.3%	67.3 673%
1+ SAE related to pomalidomide	17.9 298.3%	9.3 116.3%	19.7 140.7%	20.6 206%
1+ AE leading to discontinuation of pomalidomide	8.0 133.3%	10.3 128.8%	3.3 23.6%	12.1 121%
1+related AE --discontinuation of pomalidomide	1.8 30%	2.8 35%	1.6 11.4%	3.7 37%
1+AE - reduction of pomalidomide	20.5 341.7%	25.2 315%	9.8 70%	29.9 299%
1+ AE - interruption of pomalidomide	63.4 1056.7%	47.7 596.3%	36.1 257.9%	58.9 589%
1+ related AE - interruption of pomalidomide	27.7 461.7%	24.3 303.8%	21.3 152.1%	32.7 327%
1+related AE - reduction of pomalidomide	17.9 298.3%	20.6 257.5%	8.2 58.6%	24.3 243%

6. Secondary Outcome

Title	Phase 2: Summary of Best Myeloma Response As Assessed by Independent Response Adjudication Committee (IRAC) Using European Group for Blood and Bone Marrow Transplant (EBMT) Criteria as of the 01 April 2011 Cut-off
Description	IRAC used EBMT criteria to assess myeloma response: Complete Response (CR)-absence of serum and urine monoclonal paraprotein for 6 weeks, plus no increase in size or number of lytic bone lesions, plus other factors) Partial Response (PR)-not all CR criteria, plus >=50% reduction in serum monoclonal paraprotein plus others Minimal Response (MR)- 25-49% reduction in serum monoclonal paraprotein plus others Stable Disease (SD)- not MR or progressive disease (PD) Progressive Disease (PD)- reappearance of monoclonal paraprotein, lytic bone lesions, other Not Evaluable (NE). Data collection is ongoing and future data results will be included as available.
Time Frame	up to 70 weeks

Outcome Measure Data

Analysis Population Description
Intent to treat population. Data collection is ongoing and future data results will be included as available.

Arm/Group Title	Phase 2: Pomalidomide + Dexamethasone	Phase 2: Pomalidomide
Arm/Group Description	Combination therapy of 4 mg pomalidomide given once per day on Days 1-21 of each 28-day cycle and the starting dose of dexamethasone (either 40mg or 20 mg) on days 1, 8, 15, and 22 of each 28-day cycle.	4 mg pomalidomide was given once per day on Days 1-21 of each 28-day cycle until PD. Participants in the single agent pomalidomide treatment arm who developed confirmed PD at any time had the option to receive oral dexamethasone at the starting dose in addition to their current dose of pomalidomide or to discontinue treatment.
Measure Participants	113	108
Complete response (CR)	0.9 15%	0.0 0%
Partial response (PR)	29.2 486.7%	9.3 116.3%
Minimal response (MR)	15.0 250%	15.7 196.3%
Stable disease (SD)	35.4 590%	46.3 578.8%
Progressive disease (PD)	6.2 103.3%	15.7 196.3%
Not evaluable	13.3 221.7%	13.0 162.5%

7. Secondary Outcome

Title	Phase 2: Kaplan-Meier Estimates of Duration of Response as of the 01 April 2011 Cut-off
Description	Duration of myeloma response is defined as the time from when the response criteria are first met for partial response (PR) or better, until the first date the response criteria are met for progressive disease (PD) or until the participant dies from any cause, whichever occurs first. Duration of response for participants last known to be alive with no progression after a complete response (CR) or PR was censored at the date of last adequate response assessment. Participants with confirmed responses that occur after receiving any other anti-myeloma therapy (except for adding dexamethasone to the pomalidomide treatment arm), including radiation therapy initiated after baseline, was censored at the last adequate assessment prior to the initiation of such treatment. Response was assessed by the Independent Response Adjudication Committee (IRAC) using European Group for Blood and Bone Marrow Transplant (EBMT) criteria as described in the previous outcome.
Time Frame	up to 70 weeks

Outcome Measure Data

Analysis Population Description
Responders (participants with a complete response or partial response) from the ITT population. Data collection is ongoing and future data results will be included as available.

Arm/Group Title	Phase 2: Pomalidomide + Dexamethasone	Phase 2: Pomalidomide
Arm/Group Description	Combination therapy of 4 mg pomalidomide given once per day on Days 1-21 of each 28-day cycle and the starting dose of dexamethasone (either 40mg or 20 mg) on days 1, 8, 15, and 22 of each 28-day cycle.	4 mg pomalidomide was given once per day on Days 1-21 of each 28-day cycle until PD. Participants in the single agent pomalidomide treatment arm who developed confirmed PD at any time had the option to receive oral dexamethasone at the starting dose in addition to their current dose of pomalidomide or to discontinue treatment.
Measure Participants	34	10
Median (95% Confidence Interval) [weeks]	32.1	NA

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Phase 1: 2 mg Pomalidomide, Phase 1: 3 mg Pomalidomide
	Comments
	Type of Statistical Test	Superiority or Other
	Comments

Statistical Test of Hypothesis	p-Value
	Comments
	Method
	Comments

Method of Estimation	Estimation Parameter	Cox Proportional Hazard
	Estimated Value	6.25
	Confidence Interval	(2-Sided) 95% 0.84 to 46.66
	Parameter Dispersion	Type: Value:
	Estimation Comments

8. Secondary Outcome

Title	Phase 2: Time to Response as of the 01 April 2011 Cut-off
Description	Time to myeloma response is defined as the time from randomization to the time the response criteria for complete response (CR) or partial response (PR) are first met. Response was assessed by the Independent Response Adjudication Committee (IRAC) using European Group for Blood and Bone Marrow Transplant (EBMT) criteria as described previously. Data collection is ongoing and future data results will be included as available.
Time Frame	up to 70 weeks

Outcome Measure Data

Analysis Population Description
Responders (participants achieving CR or PR) from ITT population. Data collection is ongoing and future data results will be included as available.

Arm/Group Title	Phase 2: Pomalidomide + Dexamethasone	Phase 2: Pomalidomide
Arm/Group Description	Combination therapy of 4 mg pomalidomide given once per day on Days 1-21 of each 28-day cycle and the starting dose of dexamethasone (either 40mg or 20 mg) on days 1, 8, 15, and 22 of each 28-day cycle.	4 mg pomalidomide was given once per day on Days 1-21 of each 28-day cycle until PD. Participants in the single agent pomalidomide treatment arm who developed confirmed PD at any time had the option to receive oral dexamethasone at the starting dose in addition to their current dose of pomalidomide or to discontinue treatment.
Measure Participants	34	10
Median (Full Range) [weeks]	8.1	8.9

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Phase 1: 2 mg Pomalidomide, Phase 1: 3 mg Pomalidomide
	Comments
	Type of Statistical Test	Superiority or Other
	Comments

Statistical Test of Hypothesis	p-Value	0.070
	Comments
	Method	Wilcoxon (Mann-Whitney)
	Comments

9. Secondary Outcome

Title	Phase 2: Kaplan-Meier Estimates of Overall Survival as of the 01 April 2011 Cut-off
Description	Overall survival was defined as the time between randomization and death. Participants who die, regardless of the cause of the death, were considered to have had an event. All participants who were lost to follow-up prior to the end of the trial or who were withdrawn from the trial were censored at the time of last contact. Participants who were still being treated were censored at the last available date the subject was known to be alive, or clinical cut-off date if it was earlier. Data collection is ongoing and future data results will be included as available.
Time Frame	up to 70 weeks

Outcome Measure Data

Analysis Population Description
ITT population. Data collection is ongoing and future data results will be included as available.

Arm/Group Title	Phase 2: Pomalidomide + Dexamethasone	Phase 2: Pomalidomide
Arm/Group Description	Combination therapy of 4 mg pomalidomide given once per day on Days 1-21 of each 28-day cycle and the starting dose of dexamethasone (either 40mg or 20 mg) on days 1, 8, 15, and 22 of each 28-day cycle.	4 mg pomalidomide was given once per day on Days 1-21 of each 28-day cycle until PD. Participants in the single agent pomalidomide treatment arm who developed confirmed PD at any time had the option to receive oral dexamethasone at the starting dose in addition to their current dose of pomalidomide or to discontinue treatment.
Measure Participants	113	108
Median (95% Confidence Interval) [weeks]	62.6	59.3

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Phase 1: 2 mg Pomalidomide, Phase 1: 3 mg Pomalidomide
	Comments
	Type of Statistical Test	Superiority or Other
	Comments

Statistical Test of Hypothesis	p-Value
	Comments
	Method
	Comments

Method of Estimation	Estimation Parameter	Cox Proportional Hazard
	Estimated Value	0.85
	Confidence Interval	(2-Sided) 95% 0.57 to 1.29
	Parameter Dispersion	Type: Value:
	Estimation Comments

10. Primary Outcome

Title	Phase 2: Percentage of Participants With Progression-Free Survival (PFS) Events as of the 01 April 2011 Cut-off
Description	Percentage of participants with the progression-free survival events: disease progression and death. Disease progression was assessed by the Independent Response Adjudication Committee (IRAC). Data collection is ongoing and future data results will be included as available.
Time Frame	up to 67 weeks

Outcome Measure Data

Analysis Population Description
Intent to treat population. Data collection is ongoing and future data results will be included as available.

Arm/Group Title	Phase 2: Pomalidomide + Dexamethasone	Phase 2: Pomalidomide
Arm/Group Description	Combination therapy of 4 mg pomalidomide given once per day on Days 1-21 of each 28-day cycle and the starting dose of dexamethasone (either 40mg or 20 mg) on days 1, 8, 15, and 22 of each 28-day cycle.	4 mg pomalidomide was given once per day on Days 1-21 of each 28-day cycle until PD. Participants in the single agent pomalidomide treatment arm who developed confirmed PD at any time had the option to receive oral dexamethasone at the starting dose in addition to their current dose of pomalidomide, or to discontinue treatment.
Measure Participants	113	108
Number [percentage of participants]	76.1 1268.3%	75.0 937.5%

Adverse Events

	Phase 1: 2 mg Pomalidomide		Phase 1: 3 mg Pomalidomide		Phase 1: 4 mg Pomalidomide		Phase 1: 5 mg Pomalidomide		Phase 2: Pomalidomide + Dexamethasone		Phase 2: Pomalidomide (Pom Only)		Phase 2: Pomalidomide (Pom + Dex Only)		Phase 2: Pomalidomide (Overall)
Time Frame	Data cut-off 01 April 2011 Phase 1: up to 126 weeks Phase 2: up to 70 weeks
Adverse Event Reporting Description

Arm/Group Title	Phase 1: 2 mg Pomalidomide		Phase 1: 3 mg Pomalidomide		Phase 1: 4 mg Pomalidomide		Phase 1: 5 mg Pomalidomide		Phase 2: Pomalidomide + Dexamethasone		Phase 2: Pomalidomide (Pom Only)		Phase 2: Pomalidomide (Pom + Dex Only)		Phase 2: Pomalidomide (Overall)
Arm/Group Description	Pomalidomide 2 mg daily on days 1-21 of each 28-day cycle. Upon PD, participants had the option of discontinuing treatment or adding dexamethasone.		Pomalidomide 3 mg daily on days 1-21 of each 28-day cycle. Upon PD, participants had the option of discontinuing treatment or adding dexamethasone.		Pomalidomide 4 mg daily on days 1-21 of each 28-day cycle. Upon PD, participants had the option of discontinuing treatment or adding dexamethasone.		Pomalidomide 5 mg daily on days 1-21 of each 28-day cycle. Upon PD, participants had the option of discontinuing treatment or adding dexamethasone.		Combination therapy of 4 mg pomalidomide given once per day on Days 1-21 of each 28-day cycle and the starting dose of dexamethasone (either 40mg or 20 mg) on days 1, 8, 15, and 22 of each 28-day cycle.		Pomalidomide 4 mg daily on days 1-21 of each 28-day cycle. Upon PD, participants had the option of discontinuing therapy or adding dexamethasone. Data are reported during the time when participants were on pomalidomide alone, before dexamethasone was added.		Pomalidomide 4 mg daily on days 1-21 of each 28-day cycle. Upon PD, participants had the option of discontinuing therapy or adding dexamethasone. Data are reported during the time after PD when participants were on both pomalidomide and dexamethasone.		Pomalidomide 4 mg daily on days 1-21 of each 28-day cycle. Upon PD, participants had the option of discontinuing therapy or adding dexamethasone. Data are reported during the entire study up to the data cut-off, thus including both the time participants were only on pomalidomide and the time after PD when participants were on pomalidomide and dexamethasone.
All Cause Mortality
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	/ (NaN)		/ (NaN)		/ (NaN)		/ (NaN)		/ (NaN)		/ (NaN)		/ (NaN)		/ (NaN)
Serious Adverse Events
	Phase 1: 2 mg Pomalidomide		Phase 1: 3 mg Pomalidomide		Phase 1: 4 mg Pomalidomide		Phase 1: 5 mg Pomalidomide		Phase 2: Pomalidomide + Dexamethasone		Phase 2: Pomalidomide (Pom Only)		Phase 2: Pomalidomide (Pom + Dex Only)		Phase 2: Pomalidomide (Overall)
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	3/6 (50%)		4/8 (50%)		8/14 (57.1%)		4/10 (40%)		69/112 (61.6%)		50/107 (46.7%)		29/61 (47.5%)		72/107 (67.3%)
Blood and lymphatic system disorders
Febrile neutropenia	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		1/112 (0.9%)		2/107 (1.9%)		3/61 (4.9%)		5/107 (4.7%)
Anemia	0/6 (0%)		1/8 (12.5%)		0/14 (0%)		0/10 (0%)		2/112 (1.8%)		1/107 (0.9%)		1/61 (1.6%)		2/107 (1.9%)
Neutropenia	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		2/112 (1.8%)		1/107 (0.9%)		1/61 (1.6%)		2/107 (1.9%)
Thrombocytopenia	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		2/112 (1.8%)		2/107 (1.9%)		0/61 (0%)		2/107 (1.9%)
Hyperviscosity syndrome	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		0/112 (0%)		1/107 (0.9%)		0/61 (0%)		1/107 (0.9%)
Leukopenia	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Cardiac disorders
Atrial fibrillation	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		3/112 (2.7%)		1/107 (0.9%)		1/61 (1.6%)		2/107 (1.9%)
Cardiac failure congestive	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		3/112 (2.7%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Angina pectoris	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		2/112 (1.8%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Cardio-respiratory arrest	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		0/112 (0%)		0/107 (0%)		1/61 (1.6%)		1/107 (0.9%)
Cardio-respiratory distress	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Myocardial ischaemia	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Atrioventricular block second degree	0/6 (0%)		1/8 (12.5%)		0/14 (0%)		0/10 (0%)		0/112 (0%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Gastrointestinal disorders
Constipation	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		3/112 (2.7%)		1/107 (0.9%)		0/61 (0%)		1/107 (0.9%)
Nausea	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		1/112 (0.9%)		1/107 (0.9%)		1/61 (1.6%)		2/107 (1.9%)
Diarrhoea	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		1/61 (1.6%)		1/107 (0.9%)
Intestinal obstruction	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		1/112 (0.9%)		1/107 (0.9%)		0/61 (0%)		1/107 (0.9%)
Vomiting	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		0/112 (0%)		1/107 (0.9%)		1/61 (1.6%)		2/107 (1.9%)
Abdominal pain	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Abdominal pain upper	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		0/112 (0%)		1/107 (0.9%)		0/61 (0%)		1/107 (0.9%)
Ascites	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		0/112 (0%)		1/107 (0.9%)		0/61 (0%)		1/107 (0.9%)
Caecitis	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Peptic ulcer	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Rectal haemorrhage	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		0/112 (0%)		0/107 (0%)		1/61 (1.6%)		1/107 (0.9%)
Gastrointestinal haemorrhage	0/6 (0%)		1/8 (12.5%)		0/14 (0%)		0/10 (0%)		0/112 (0%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
General disorders
Pyrexia	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		5/112 (4.5%)		3/107 (2.8%)		0/61 (0%)		3/107 (2.8%)
Fatigue	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		0/112 (0%)		2/107 (1.9%)		0/61 (0%)		2/107 (1.9%)
General physical health deterioration	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		2/112 (1.8%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Non-cardiac chest pain	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		0/112 (0%)		1/107 (0.9%)		1/61 (1.6%)		2/107 (1.9%)
Asthenia	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Catheter site haemorrhage	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		0/112 (0%)		0/107 (0%)		1/61 (1.6%)		1/107 (0.9%)
Chest pain	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Malaise	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Hepatobiliary disorders
Cholecystitis	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		0/112 (0%)		1/107 (0.9%)		0/61 (0%)		1/107 (0.9%)
Hyperbilirubinaemia	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Infections and infestations
Pneumonia	1/6 (16.7%)		0/8 (0%)		2/14 (14.3%)		0/10 (0%)		21/112 (18.8%)		10/107 (9.3%)		6/61 (9.8%)		15/107 (14%)
Sepsis	1/6 (16.7%)		2/8 (25%)		0/14 (0%)		1/10 (10%)		3/112 (2.7%)		4/107 (3.7%)		2/61 (3.3%)		6/107 (5.6%)
Urinary tract infection	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		6/112 (5.4%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Bronchopneumonia	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		1/61 (1.6%)		1/107 (0.9%)
Cellulitis	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		1/61 (1.6%)		1/107 (0.9%)
Lobar pneumonia	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		0/112 (0%)		0/107 (0%)		2/61 (3.3%)		2/107 (1.9%)
Pneumonia respiratory syncytial viral	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		2/112 (1.8%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Respiratory syncytial virus infection	0/6 (0%)		0/8 (0%)		0/14 (0%)		1/10 (10%)		1/112 (0.9%)		1/107 (0.9%)		0/61 (0%)		1/107 (0.9%)
Urosepsis	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		2/112 (1.8%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Viral infection	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		0/112 (0%)		1/107 (0.9%)		1/61 (1.6%)		2/107 (1.9%)
Arthritis baterial	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Bacteraemia	1/6 (16.7%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Bronchitis	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		0/112 (0%)		0/107 (0%)		1/61 (1.6%)		1/107 (0.9%)
Bronchopulmonary aspergillosis	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		0/112 (0%)		1/107 (0.9%)		0/61 (0%)		1/107 (0.9%)
Clostridium difficile colitis	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		0/112 (0%)		0/107 (0%)		1/61 (1.6%)		1/107 (0.9%)
Device related infection	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Escherichia bacteraemia	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Gastroenteritis	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
H1N1 influenza	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		0/112 (0%)		0/107 (0%)		1/61 (1.6%)		1/107 (0.9%)
Herpes zoster disseminated	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Herpes zoster ophthalmic	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Pneumonia fungal	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Pneumonia parainfluenzae viral	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Pneumonia streptococcal	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		0/112 (0%)		1/107 (0.9%)		0/61 (0%)		1/107 (0.9%)
Pneumonia viral	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Postoperative wound infection	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Pseudomonas infection	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		0/112 (0%)		1/107 (0.9%)		0/61 (0%)		1/107 (0.9%)
Pyelonephritis	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Septic shock	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Skin infection	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Staphylococcal bacteraemia	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		0/112 (0%)		0/107 (0%)		1/61 (1.6%)		1/107 (0.9%)
Staphylococcal infection	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		0/112 (0%)		0/107 (0%)		1/61 (1.6%)		1/107 (0.9%)
Staphylococcal sepsis	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		0/112 (0%)		0/107 (0%)		1/61 (1.6%)		1/107 (0.9%)
Abscess limb	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		0/112 (0%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Arthritis infective	0/6 (0%)		1/8 (12.5%)		0/14 (0%)		0/10 (0%)		0/112 (0%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Influenza	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		0/112 (0%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Lung infection pseudomonal	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		0/112 (0%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Meningitis bacterial	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		0/112 (0%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Pharyngeal abscess	0/6 (0%)		1/8 (12.5%)		0/14 (0%)		0/10 (0%)		0/112 (0%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Injury, poisoning and procedural complications
Compression fracture	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		2/112 (1.8%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Fall	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		0/112 (0%)		1/107 (0.9%)		1/61 (1.6%)		2/107 (1.9%)
Femur fracture	0/6 (0%)		0/8 (0%)		0/14 (0%)		1/10 (10%)		1/112 (0.9%)		1/107 (0.9%)		0/61 (0%)		1/107 (0.9%)
Hip fracture	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		0/112 (0%)		1/107 (0.9%)		1/61 (1.6%)		2/107 (1.9%)
Humerus fracture	0/6 (0%)		0/8 (0%)		0/14 (0%)		1/10 (10%)		1/112 (0.9%)		1/107 (0.9%)		0/61 (0%)		1/107 (0.9%)
Spinal compression fracture	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		1/61 (1.6%)		1/107 (0.9%)
Subdural haematoma	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		1/61 (1.6%)		1/107 (0.9%)
Accidental overdose	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Femoral neck fracture	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Pelvic fracture	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Toxicity to various agents	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		0/112 (0%)		1/107 (0.9%)		0/61 (0%)		1/107 (0.9%)
Upper limb fracture	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Investigations
Blood creatinine increased	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		1/112 (0.9%)		2/107 (1.9%)		0/61 (0%)		2/107 (1.9%)
Haemoglobin decreased	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		0/112 (0%)		2/107 (1.9%)		0/61 (0%)		2/107 (1.9%)
Blood culture positive	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Electrocardiogram QT prolonged	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Neutrophil count decreased	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		0/112 (0%)		1/107 (0.9%)		0/61 (0%)		1/107 (0.9%)
Platelet count decreased	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		0/112 (0%)		1/107 (0.9%)		0/61 (0%)		1/107 (0.9%)
Metabolism and nutrition disorders
Dehydration	0/6 (0%)		1/8 (12.5%)		0/14 (0%)		0/10 (0%)		3/112 (2.7%)		4/107 (3.7%)		1/61 (1.6%)		5/107 (4.7%)
Hypercalcaemia	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		2/112 (1.8%)		3/107 (2.8%)		2/61 (3.3%)		5/107 (4.7%)
Failure to thrive	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		0/112 (0%)		1/107 (0.9%)		1/61 (1.6%)		2/107 (1.9%)
Hyperkalaemia	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		1/112 (0.9%)		1/107 (0.9%)		0/61 (0%)		1/107 (0.9%)
Decreased appetite	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Fluid overload	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Hyponatraemia	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		0/112 (0%)		0/107 (0%)		1/61 (1.6%)		1/107 (0.9%)
Tumour lysis syndrome	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		0/112 (0%)		1/107 (0.9%)		0/61 (0%)		1/107 (0.9%)
Musculoskeletal and connective tissue disorders
Back pain	1/6 (16.7%)		0/8 (0%)		0/14 (0%)		1/10 (10%)		2/112 (1.8%)		2/107 (1.9%)		3/61 (4.9%)		4/107 (3.7%)
Bone pain	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		1/112 (0.9%)		1/107 (0.9%)		0/61 (0%)		1/107 (0.9%)
Muscular weakness	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		1/61 (1.6%)		1/107 (0.9%)
Musculoskeletal chest pain	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		1/112 (0.9%)		1/107 (0.9%)		0/61 (0%)		1/107 (0.9%)
Musculoskeletal pain	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		0/112 (0%)		0/107 (0%)		1/61 (1.6%)		1/107 (0.9%)
Neck pain	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		0/112 (0%)		1/107 (0.9%)		0/61 (0%)		1/107 (0.9%)
Pain in extremity	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Multiple myeloma	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		8/112 (7.1%)		9/107 (8.4%)		1/61 (1.6%)		10/107 (9.3%)
Cancer pain	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		0/112 (0%)		0/107 (0%)		1/61 (1.6%)		1/107 (0.9%)
Colon cancer	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Leukaemia plasmacytic	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		0/112 (0%)		1/107 (0.9%)		0/61 (0%)		1/107 (0.9%)
Metastases to meninges	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		0/112 (0%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Nervous system disorders
Cerebral haemorrhage	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		1/61 (1.6%)		1/107 (0.9%)
Brain mass	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		0/112 (0%)		1/107 (0.9%)		0/61 (0%)		1/107 (0.9%)
Cauda equina syndrome	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		0/112 (0%)		1/107 (0.9%)		0/61 (0%)		1/107 (0.9%)
Dizziness	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		0/112 (0%)		1/107 (0.9%)		0/61 (0%)		1/107 (0.9%)
Dysarthria	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		0/112 (0%)		0/107 (0%)		1/61 (1.6%)		1/107 (0.9%)
Spinal cord compression	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Subarachnoid haemorrhage	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Syncope	0/6 (0%)		1/8 (12.5%)		0/14 (0%)		0/10 (0%)		0/112 (0%)		1/107 (0.9%)		0/61 (0%)		1/107 (0.9%)
Unresponsive to stimuli	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		0/112 (0%)		0/107 (0%)		1/61 (1.6%)		1/107 (0.9%)
Headache	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		0/112 (0%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Psychiatric disorders
Confusional state	1/6 (16.7%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		2/112 (1.8%)		0/107 (0%)		2/61 (3.3%)		2/107 (1.9%)
Mental status changes	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		2/112 (1.8%)		0/107 (0%)		2/61 (3.3%)		2/107 (1.9%)
Completed suicide	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Psychotic disorder	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		0/112 (0%)		1/107 (0.9%)		0/61 (0%)		1/107 (0.9%)
Renal and urinary disorders
Renal failure acute	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		6/112 (5.4%)		5/107 (4.7%)		2/61 (3.3%)		7/107 (6.5%)
Renal failure	1/6 (16.7%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		1/112 (0.9%)		1/107 (0.9%)		1/61 (1.6%)		2/107 (1.9%)
Nephrolithiasis	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Renal failure chronic	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		0/112 (0%)		1/107 (0.9%)		0/61 (0%)		1/107 (0.9%)
Renal impairment	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		0/112 (0%)		1/107 (0.9%)		0/61 (0%)		1/107 (0.9%)
Respiratory, thoracic and mediastinal disorders
Dyspnoea	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		7/112 (6.3%)		2/107 (1.9%)		3/61 (4.9%)		5/107 (4.7%)
Bronchospasm	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		2/112 (1.8%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Pulmonary embolism	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		2/112 (1.8%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Respiratory failure	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		2/112 (1.8%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Acute respiratory failure	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Chronic obstructive pulmonary disease	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Epistaxis	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		0/112 (0%)		1/107 (0.9%)		0/61 (0%)		1/107 (0.9%)
Hypoxia	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		0/112 (0%)		0/107 (0%)		1/61 (1.6%)		1/107 (0.9%)
Interstitial lung disease	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		0/112 (0%)		1/107 (0.9%)		0/61 (0%)		1/107 (0.9%)
Pleural effusion	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Pleuritic pain	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		0/112 (0%)		1/107 (0.9%)		0/61 (0%)		1/107 (0.9%)
Pneumonia aspiration	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		0/112 (0%)		1/107 (0.9%)		0/61 (0%)		1/107 (0.9%)
Respiratory distress	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Lung infiltration	0/6 (0%)		0/8 (0%)		0/14 (0%)		1/10 (10%)		0/112 (0%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Skin and subcutaneous tissue disorders
Rash	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Vascular disorders
Deep vein thrombosis	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		1/10 (10%)		1/112 (0.9%)		0/107 (0%)		1/61 (1.6%)		1/107 (0.9%)
Hypotension	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		1/61 (1.6%)		1/107 (0.9%)
Thrombosis	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		0/112 (0%)		1/107 (0.9%)		0/61 (0%)		1/107 (0.9%)
Other (Not Including Serious) Adverse Events
	Phase 1: 2 mg Pomalidomide		Phase 1: 3 mg Pomalidomide		Phase 1: 4 mg Pomalidomide		Phase 1: 5 mg Pomalidomide		Phase 2: Pomalidomide + Dexamethasone		Phase 2: Pomalidomide (Pom Only)		Phase 2: Pomalidomide (Pom + Dex Only)		Phase 2: Pomalidomide (Overall)
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	6/6 (100%)		8/8 (100%)		14/14 (100%)		10/10 (100%)		112/112 (100%)		105/107 (98.1%)		57/61 (93.4%)		106/107 (99.1%)
Blood and lymphatic system disorders
Neutropenia	1/6 (16.7%)		5/8 (62.5%)		8/14 (57.1%)		9/10 (90%)		52/112 (46.4%)		53/107 (49.5%)		17/61 (27.9%)		55/107 (51.4%)
Anaemia	5/6 (83.3%)		4/8 (50%)		5/14 (35.7%)		3/10 (30%)		43/112 (38.4%)		36/107 (33.6%)		11/61 (18%)		41/107 (38.3%)
Thrombocytopenia	2/6 (33.3%)		3/8 (37.5%)		2/14 (14.3%)		3/10 (30%)		24/112 (21.4%)		25/107 (23.4%)		7/61 (11.5%)		27/107 (25.2%)
Leukopenia	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		19/112 (17%)		11/107 (10.3%)		2/61 (3.3%)		12/107 (11.2%)
Lymphopenia	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		17/112 (15.2%)		4/107 (3.7%)		0/61 (0%)		4/107 (3.7%)
Lymphadenopathy	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		3/112 (2.7%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Cardiac disorders
Atrial fibrillation	0/6 (0%)		1/8 (12.5%)		1/14 (7.1%)		0/10 (0%)		9/112 (8%)		3/107 (2.8%)		1/61 (1.6%)		4/107 (3.7%)
Bradycardia	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Ear and labyrinth disorders
Ear pain	0/6 (0%)		1/8 (12.5%)		1/14 (7.1%)		0/10 (0%)		0/112 (0%)		0/107 (0%)		1/61 (1.6%)		1/107 (0.9%)
Tinnitus	1/6 (16.7%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		1/112 (0.9%)		1/107 (0.9%)		0/61 (0%)		1/107 (0.9%)
Deafness	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		0/112 (0%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Endocrine disorders
Cushingoid	0/6 (0%)		0/8 (0%)		0/14 (0%)		1/10 (10%)		3/112 (2.7%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Eye disorders
Vision blurred	0/6 (0%)		1/8 (12.5%)		2/14 (14.3%)		3/10 (30%)		9/112 (8%)		4/107 (3.7%)		0/61 (0%)		4/107 (3.7%)
Dry eye	1/6 (16.7%)		0/8 (0%)		0/14 (0%)		1/10 (10%)		3/112 (2.7%)		3/107 (2.8%)		1/61 (1.6%)		4/107 (3.7%)
Cataract	0/6 (0%)		0/8 (0%)		0/14 (0%)		1/10 (10%)		1/112 (0.9%)		1/107 (0.9%)		1/61 (1.6%)		2/107 (1.9%)
Conjunctivitis	0/6 (0%)		0/8 (0%)		2/14 (14.3%)		0/10 (0%)		1/112 (0.9%)		1/107 (0.9%)		0/61 (0%)		1/107 (0.9%)
Diplopia	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		1/10 (10%)		1/112 (0.9%)		1/107 (0.9%)		0/61 (0%)		1/107 (0.9%)
Eyelid disorder	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		0/112 (0%)		1/107 (0.9%)		0/61 (0%)		1/107 (0.9%)
Ocular hyperaemia	0/6 (0%)		0/8 (0%)		0/14 (0%)		1/10 (10%)		1/112 (0.9%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Photophobia	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Periorbital oedema	0/6 (0%)		0/8 (0%)		0/14 (0%)		1/10 (10%)		0/112 (0%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Gastrointestinal disorders
Constipation	2/6 (33.3%)		1/8 (12.5%)		2/14 (14.3%)		4/10 (40%)		38/112 (33.9%)		29/107 (27.1%)		11/61 (18%)		38/107 (35.5%)
Diarrhoea	1/6 (16.7%)		1/8 (12.5%)		2/14 (14.3%)		4/10 (40%)		37/112 (33%)		22/107 (20.6%)		17/61 (27.9%)		35/107 (32.7%)
Nausea	2/6 (33.3%)		1/8 (12.5%)		3/14 (21.4%)		5/10 (50%)		24/112 (21.4%)		29/107 (27.1%)		10/61 (16.4%)		37/107 (34.6%)
Vomiting	1/6 (16.7%)		2/8 (25%)		4/14 (28.6%)		2/10 (20%)		15/112 (13.4%)		10/107 (9.3%)		4/61 (6.6%)		14/107 (13.1%)
Dry mouth	1/6 (16.7%)		0/8 (0%)		1/14 (7.1%)		1/10 (10%)		8/112 (7.1%)		9/107 (8.4%)		0/61 (0%)		9/107 (8.4%)
Abdominal distension	0/6 (0%)		1/8 (12.5%)		2/14 (14.3%)		1/10 (10%)		5/112 (4.5%)		5/107 (4.7%)		3/61 (4.9%)		8/107 (7.5%)
Abdominal pain	0/6 (0%)		1/8 (12.5%)		2/14 (14.3%)		1/10 (10%)		8/112 (7.1%)		3/107 (2.8%)		1/61 (1.6%)		4/107 (3.7%)
Dyspepsia	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		2/10 (20%)		4/112 (3.6%)		2/107 (1.9%)		1/61 (1.6%)		3/107 (2.8%)
Abdominal pain upper	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		7/112 (6.3%)		1/107 (0.9%)		0/61 (0%)		1/107 (0.9%)
Flatulence	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		1/10 (10%)		3/112 (2.7%)		1/107 (0.9%)		0/61 (0%)		1/107 (0.9%)
Faecal incontinence	0/6 (0%)		0/8 (0%)		2/14 (14.3%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		1/61 (1.6%)		1/107 (0.9%)
Gastrooesophageal reflux disease	0/6 (0%)		1/8 (12.5%)		0/14 (0%)		0/10 (0%)		1/112 (0.9%)		2/107 (1.9%)		0/61 (0%)		2/107 (1.9%)
Sensitivity of teeth	1/6 (16.7%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		1/112 (0.9%)		2/107 (1.9%)		0/61 (0%)		2/107 (1.9%)
Gingival pain	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		1/112 (0.9%)		1/107 (0.9%)		0/61 (0%)		1/107 (0.9%)
Hypoaesthesia oral	0/6 (0%)		0/8 (0%)		0/14 (0%)		1/10 (10%)		1/112 (0.9%)		1/107 (0.9%)		0/61 (0%)		1/107 (0.9%)
Haemorrhoids	1/6 (16.7%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		0/112 (0%)		0/107 (0%)		1/61 (1.6%)		1/107 (0.9%)
Haematochezia	0/6 (0%)		0/8 (0%)		0/14 (0%)		1/10 (10%)		0/112 (0%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Oesophagitis	1/6 (16.7%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		0/112 (0%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
General disorders
Fatigue	4/6 (66.7%)		5/8 (62.5%)		9/14 (64.3%)		7/10 (70%)		62/112 (55.4%)		46/107 (43%)		11/61 (18%)		53/107 (49.5%)
Pyrexia	2/6 (33.3%)		2/8 (25%)		3/14 (21.4%)		3/10 (30%)		31/112 (27.7%)		15/107 (14%)		6/61 (9.8%)		19/107 (17.8%)
Oedema peripheral	2/6 (33.3%)		1/8 (12.5%)		1/14 (7.1%)		2/10 (20%)		18/112 (16.1%)		18/107 (16.8%)		8/61 (13.1%)		25/107 (23.4%)
Asthenia	3/6 (50%)		2/8 (25%)		1/14 (7.1%)		0/10 (0%)		22/112 (19.6%)		11/107 (10.3%)		5/61 (8.2%)		15/107 (14%)
Chills	1/6 (16.7%)		0/8 (0%)		1/14 (7.1%)		1/10 (10%)		12/112 (10.7%)		9/107 (8.4%)		2/61 (3.3%)		10/107 (9.3%)
Malaise	0/6 (0%)		2/8 (25%)		2/14 (14.3%)		3/10 (30%)		4/112 (3.6%)		5/107 (4.7%)		1/61 (1.6%)		6/107 (5.6%)
Gait disturbance	1/6 (16.7%)		0/8 (0%)		1/14 (7.1%)		1/10 (10%)		4/112 (3.6%)		6/107 (5.6%)		3/61 (4.9%)		8/107 (7.5%)
Pain	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		5/112 (4.5%)		5/107 (4.7%)		1/61 (1.6%)		6/107 (5.6%)
Irritability	0/6 (0%)		0/8 (0%)		0/14 (0%)		1/10 (10%)		4/112 (3.6%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Mucosal inflammation	0/6 (0%)		1/8 (12.5%)		1/14 (7.1%)		0/10 (0%)		3/112 (2.7%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Non-cardiac chest pain	1/6 (16.7%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		3/112 (2.7%)		1/107 (0.9%)		0/61 (0%)		1/107 (0.9%)
Feeling jittery	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		3/112 (2.7%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Influenza like illness	0/6 (0%)		0/8 (0%)		0/14 (0%)		1/10 (10%)		0/112 (0%)		1/107 (0.9%)		2/61 (3.3%)		3/107 (2.8%)
Feeling abnormal	0/6 (0%)		1/8 (12.5%)		0/14 (0%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Catheter site haemorrhage	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		0/112 (0%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Feeling hot	0/6 (0%)		0/8 (0%)		0/14 (0%)		1/10 (10%)		0/112 (0%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Immune system disorders
Allergic oedema	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		0/112 (0%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Infections and infestations
Upper respiratory tract infection	0/6 (0%)		2/8 (25%)		5/14 (35.7%)		2/10 (20%)		23/112 (20.5%)		17/107 (15.9%)		10/61 (16.4%)		27/107 (25.2%)
Urinary tract infection	0/6 (0%)		1/8 (12.5%)		3/14 (21.4%)		1/10 (10%)		15/112 (13.4%)		7/107 (6.5%)		2/61 (3.3%)		8/107 (7.5%)
Pneumonia	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		1/10 (10%)		8/112 (7.1%)		6/107 (5.6%)		5/61 (8.2%)		1/107 (0.9%)
Nasopharyngitis	0/6 (0%)		1/8 (12.5%)		1/14 (7.1%)		1/10 (10%)		4/112 (3.6%)		2/107 (1.9%)		4/61 (6.6%)		6/107 (5.6%)
Sinusitis	1/6 (16.7%)		0/8 (0%)		3/14 (21.4%)		2/10 (20%)		5/112 (4.5%)		0/107 (0%)		1/61 (1.6%)		1/107 (0.9%)
Rhinitis	0/6 (0%)		1/8 (12.5%)		1/14 (7.1%)		1/10 (10%)		2/112 (1.8%)		4/107 (3.7%)		1/61 (1.6%)		5/107 (4.7%)
Candidiasis	0/6 (0%)		0/8 (0%)		0/14 (0%)		1/10 (10%)		6/112 (5.4%)		0/107 (0%)		2/61 (3.3%)		2/107 (1.9%)
Oral candidiasis	0/6 (0%)		1/8 (12.5%)		0/14 (0%)		0/10 (0%)		6/112 (5.4%)		0/107 (0%)		1/61 (1.6%)		1/107 (0.9%)
Bronchitis	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		3/112 (2.7%)		1/107 (0.9%)		2/61 (3.3%)		3/107 (2.8%)
Fungal skin infection	0/6 (0%)		1/8 (12.5%)		0/14 (0%)		1/10 (10%)		2/112 (1.8%)		1/107 (0.9%)		1/61 (1.6%)		1/107 (0.9%)
Tooth infection	0/6 (0%)		0/8 (0%)		0/14 (0%)		1/10 (10%)		1/112 (0.9%)		3/107 (2.8%)		0/61 (0%)		3/107 (2.8%)
Herpes zoster	0/6 (0%)		0/8 (0%)		0/14 (0%)		2/10 (20%)		0/112 (0%)		2/107 (1.9%)		0/61 (0%)		2/107 (1.9%)
Respiratory tract infection	0/6 (0%)		0/8 (0%)		0/14 (0%)		1/10 (10%)		1/112 (0.9%)		2/107 (1.9%)		0/61 (0%)		2/107 (1.9%)
Lower respiratory tract infection	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		1/61 (1.6%)		1/107 (0.9%)
Sepsis	0/6 (0%)		0/8 (0%)		0/14 (0%)		1/10 (10%)		1/112 (0.9%)		1/107 (0.9%)		0/61 (0%)		1/107 (0.9%)
Vulvovaginal mycotic infection	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		1/112 (0.9%)		1/107 (0.9%)		0/61 (0%)		1/107 (0.9%)
Gastroenteritis	0/6 (0%)		1/8 (12.5%)		0/14 (0%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Localised infection	0/6 (0%)		0/8 (0%)		0/14 (0%)		1/10 (10%)		1/112 (0.9%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Post procedural infection	0/6 (0%)		0/8 (0%)		0/14 (0%)		1/10 (10%)		0/112 (0%)		1/107 (0.9%)		0/61 (0%)		1/107 (0.9%)
Urethritis	0/6 (0%)		1/8 (12.5%)		1/14 (7.1%)		0/10 (0%)		0/112 (0%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Arthritis infective	0/6 (0%)		1/8 (12.5%)		0/14 (0%)		0/10 (0%)		0/112 (0%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Herpes simplex	1/6 (16.7%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		0/112 (0%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Lung infection	0/6 (0%)		1/8 (12.5%)		0/14 (0%)		0/10 (0%)		0/112 (0%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Urinary tract infection bacterial	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		0/112 (0%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Urinary tract infection fungal	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		0/112 (0%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Injury, poisoning and procedural complications
Contusion	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		5/112 (4.5%)		1/107 (0.9%)		2/61 (3.3%)		3/107 (2.8%)
Spinal compression fracture	0/6 (0%)		0/8 (0%)		0/14 (0%)		1/10 (10%)		3/112 (2.7%)		4/107 (3.7%)		3/61 (4.9%)		5/107 (4.7%)
Procedural pain	1/6 (16.7%)		1/8 (12.5%)		0/14 (0%)		1/10 (10%)		2/112 (1.8%)		2/107 (1.9%)		0/61 (0%)		2/107 (1.9%)
Excoriation	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		3/112 (2.7%)		0/107 (0%)		1/61 (1.6%)		1/107 (0.9%)
Tendon injury	1/6 (16.7%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		0/112 (0%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Wound dehiscence	0/6 (0%)		1/8 (12.5%)		0/14 (0%)		0/10 (0%)		0/112 (0%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Investigations
Blood creatinine increased	0/6 (0%)		0/8 (0%)		3/14 (21.4%)		0/10 (0%)		12/112 (10.7%)		11/107 (10.3%)		6/61 (9.8%)		14/107 (13.1%)
Weight decreased	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		9/112 (8%)		9/107 (8.4%)		5/61 (8.2%)		15/107 (14%)
Weight increased	0/6 (0%)		0/8 (0%)		0/14 (0%)		1/10 (10%)		12/112 (10.7%)		0/107 (0%)		1/61 (1.6%)		1/107 (0.9%)
Aspartate aminotransferase increased	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		7/112 (6.3%)		5/107 (4.7%)		0/61 (0%)		5/107 (4.7%)
Neutrophil count decreased	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		8/112 (7.1%)		5/107 (4.7%)		1/61 (1.6%)		5/107 (4.7%)
White blood cell count decreased	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		8/112 (7.1%)		4/107 (3.7%)		0/61 (0%)		4/107 (3.7%)
Haemoglobin decreased	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		8/112 (7.1%)		2/107 (1.9%)		0/61 (0%)		2/107 (1.9%)
Blood glucose increased	0/6 (0%)		1/8 (12.5%)		0/14 (0%)		0/10 (0%)		2/112 (1.8%)		2/107 (1.9%)		1/61 (1.6%)		3/107 (2.8%)
Platelet count decreased	0/6 (0%)		0/8 (0%)		0/14 (0%)		1/10 (10%)		5/112 (4.5%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
International normalised ratio increased	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		3/112 (2.7%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Cardiac murmur	0/6 (0%)		0/8 (0%)		0/14 (0%)		1/10 (10%)		1/112 (0.9%)		1/107 (0.9%)		0/61 (0%)		1/107 (0.9%)
Blood phosphorus decreased	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Heart rate irregular	0/6 (0%)		1/8 (12.5%)		0/14 (0%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Liver function test abnormal	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Blood potassium decreased	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		0/112 (0%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Blood urine present	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		0/112 (0%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Metabolism and nutrition disorders
Decreased appetite	1/6 (16.7%)		2/8 (25%)		3/14 (21.4%)		3/10 (30%)		19/112 (17%)		15/107 (14%)		8/61 (13.1%)		23/107 (21.5%)
Hypercalcaemia	1/6 (16.7%)		1/8 (12.5%)		0/14 (0%)		1/10 (10%)		11/112 (9.8%)		17/107 (15.9%)		3/61 (4.9%)		18/107 (16.8%)
Hyperglycaemia	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		17/112 (15.2%)		10/107 (9.3%)		4/61 (6.6%)		13/107 (12.1%)
Hyponatraemia	0/6 (0%)		0/8 (0%)		0/14 (0%)		1/10 (10%)		14/112 (12.5%)		7/107 (6.5%)		3/61 (4.9%)		10/107 (9.3%)
Hypokalaemia	1/6 (16.7%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		12/112 (10.7%)		6/107 (5.6%)		5/61 (8.2%)		11/107 (10.3%)
Hypocalcaemia	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		13/112 (11.6%)		5/107 (4.7%)		2/61 (3.3%)		6/107 (5.6%)
Dehydration	2/6 (33.3%)		1/8 (12.5%)		2/14 (14.3%)		0/10 (0%)		5/112 (4.5%)		4/107 (3.7%)		1/61 (1.6%)		4/107 (3.7%)
Hypoalbuminaemia	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		7/112 (6.3%)		4/107 (3.7%)		3/61 (4.9%)		7/107 (6.5%)
Hyperkalaemia	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		9/112 (8%)		4/107 (3.7%)		0/61 (0%)		4/107 (3.7%)
Hyperuricaemia	0/6 (0%)		1/8 (12.5%)		0/14 (0%)		0/10 (0%)		4/112 (3.6%)		7/107 (6.5%)		0/61 (0%)		7/107 (6.5%)
Hypomagnesaemia	1/6 (16.7%)		0/8 (0%)		0/14 (0%)		1/10 (10%)		5/112 (4.5%)		4/107 (3.7%)		2/61 (3.3%)		5/107 (4.7%)
Hypophosphataemia	0/6 (0%)		1/8 (12.5%)		0/14 (0%)		0/10 (0%)		8/112 (7.1%)		2/107 (1.9%)		1/61 (1.6%)		3/107 (2.8%)
Hypermagnesaemia	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		2/112 (1.8%)		1/107 (0.9%)		1/61 (1.6%)		1/107 (0.9%)
Cachexia	0/6 (0%)		1/8 (12.5%)		0/14 (0%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Musculoskeletal and connective tissue disorders
Back pain	3/6 (50%)		0/8 (0%)		3/14 (21.4%)		1/10 (10%)		32/112 (28.6%)		26/107 (24.3%)		10/61 (16.4%)		33/107 (30.8%)
Muscle spasms	2/6 (33.3%)		1/8 (12.5%)		6/14 (42.9%)		2/10 (20%)		21/112 (18.8%)		17/107 (15.9%)		7/61 (11.5%)		20/107 (18.7%)
Musculoskeletal chest pain	1/6 (16.7%)		1/8 (12.5%)		1/14 (7.1%)		2/10 (20%)		21/112 (18.8%)		16/107 (15%)		8/61 (13.1%)		22/107 (20.6%)
Arthralgia	1/6 (16.7%)		1/8 (12.5%)		1/14 (7.1%)		0/10 (0%)		17/112 (15.2%)		14/107 (13.1%)		3/61 (4.9%)		17/107 (15.9%)
Musculoskeletal pain	0/6 (0%)		1/8 (12.5%)		0/14 (0%)		3/10 (30%)		17/112 (15.2%)		8/107 (7.5%)		4/61 (6.6%)		12/107 (11.2%)
Muscular weakness	0/6 (0%)		0/8 (0%)		2/14 (14.3%)		0/10 (0%)		12/112 (10.7%)		8/107 (7.5%)		5/61 (8.2%)		13/107 (12.1%)
Pain in extremity	0/6 (0%)		1/8 (12.5%)		0/14 (0%)		2/10 (20%)		15/112 (13.4%)		4/107 (3.7%)		1/61 (1.6%)		5/107 (4.7%)
Bone pain	1/6 (16.7%)		0/8 (0%)		1/14 (7.1%)		2/10 (20%)		5/112 (4.5%)		7/107 (6.5%)		6/61 (9.8%)		12/107 (11.2%)
Myalgia	0/6 (0%)		0/8 (0%)		2/14 (14.3%)		1/10 (10%)		5/112 (4.5%)		4/107 (3.7%)		2/61 (3.3%)		6/107 (5.6%)
Neck pain	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		1/112 (0.9%)		3/107 (2.8%)		3/61 (4.9%)		6/107 (5.6%)
Pain in jaw	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		1/10 (10%)		2/112 (1.8%)		1/107 (0.9%)		2/61 (3.3%)		3/107 (2.8%)
Groin pain	0/6 (0%)		0/8 (0%)		0/14 (0%)		1/10 (10%)		2/112 (1.8%)		1/107 (0.9%)		1/61 (1.6%)		2/107 (1.9%)
Joint swelling	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		1/112 (0.9%)		2/107 (1.9%)		0/61 (0%)		2/107 (1.9%)
Flank pain	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		1/10 (10%)		1/112 (0.9%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Bone lesion	0/6 (0%)		0/8 (0%)		0/14 (0%)		1/10 (10%)		1/112 (0.9%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Bursitis	0/6 (0%)		0/8 (0%)		0/14 (0%)		1/10 (10%)		1/112 (0.9%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Musculoskeletal stiffness	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		0/112 (0%)		1/107 (0.9%)		0/61 (0%)		1/107 (0.9%)
Osteonecrosis	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Bone infarction	0/6 (0%)		0/8 (0%)		0/14 (0%)		1/10 (10%)		0/112 (0%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Musculoskeletal discomfort	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		0/112 (0%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Polymyalgia rheumatica	0/6 (0%)		1/8 (12.5%)		0/14 (0%)		0/10 (0%)		0/112 (0%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Squamous cell carcinoma of skin	0/6 (0%)		1/8 (12.5%)		0/14 (0%)		1/10 (10%)		0/112 (0%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Basal cell carcinoma	0/6 (0%)		0/8 (0%)		0/14 (0%)		1/10 (10%)		0/112 (0%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Squamous cell carcinoma	0/6 (0%)		1/8 (12.5%)		0/14 (0%)		0/10 (0%)		0/112 (0%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Nervous system disorders
Dizziness	0/6 (0%)		2/8 (25%)		4/14 (28.6%)		2/10 (20%)		19/112 (17%)		16/107 (15%)		4/61 (6.6%)		20/107 (18.7%)
Headache	0/6 (0%)		0/8 (0%)		3/14 (21.4%)		2/10 (20%)		9/112 (8%)		12/107 (11.2%)		2/61 (3.3%)		14/107 (13.1%)
Tremor	0/6 (0%)		0/8 (0%)		0/14 (0%)		2/10 (20%)		14/112 (12.5%)		7/107 (6.5%)		3/61 (4.9%)		10/107 (9.3%)
Neuropathy peripheral	1/6 (16.7%)		0/8 (0%)		1/14 (7.1%)		3/10 (30%)		8/112 (7.1%)		11/107 (10.3%)		0/61 (0%)		11/107 (10.3%)
Paraesthesia	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		2/10 (20%)		4/112 (3.6%)		4/107 (3.7%)		2/61 (3.3%)		5/107 (4.7%)
Somnolence	1/6 (16.7%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		6/112 (5.4%)		3/107 (2.8%)		1/61 (1.6%)		4/107 (3.7%)
Dysgeusia	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		8/112 (7.1%)		0/107 (0%)		1/61 (1.6%)		1/107 (0.9%)
Hypoaesthesia	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		2/112 (1.8%)		4/107 (3.7%)		2/61 (3.3%)		6/107 (5.6%)
Numb chin syndrome	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		1/10 (10%)		3/112 (2.7%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Ataxia	0/6 (0%)		0/8 (0%)		0/14 (0%)		1/10 (10%)		2/112 (1.8%)		1/107 (0.9%)		0/61 (0%)		1/107 (0.9%)
Balance disorder	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		1/112 (0.9%)		2/107 (1.9%)		0/61 (0%)		2/107 (1.9%)
Sciatica	0/6 (0%)		0/8 (0%)		0/14 (0%)		1/10 (10%)		1/112 (0.9%)		1/107 (0.9%)		1/61 (1.6%)		2/107 (1.9%)
Aphasia	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		1/61 (1.6%)		1/107 (0.9%)
Post herpetic neuralgia	0/6 (0%)		0/8 (0%)		0/14 (0%)		1/10 (10%)		2/112 (1.8%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Sensory disturbance	1/6 (16.7%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Subarachnoid haemorrhage	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		0/112 (0%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Psychiatric disorders
Insomnia	0/6 (0%)		0/8 (0%)		4/14 (28.6%)		1/10 (10%)		16/112 (14.3%)		0/107 (0%)		7/61 (11.5%)		7/107 (6.5%)
Confusional state	1/6 (16.7%)		2/8 (25%)		1/14 (7.1%)		0/10 (0%)		14/112 (12.5%)		6/107 (5.6%)		3/61 (4.9%)		9/107 (8.4%)
Anxiety	0/6 (0%)		1/8 (12.5%)		2/14 (14.3%)		0/10 (0%)		8/112 (7.1%)		9/107 (8.4%)		3/61 (4.9%)		12/107 (11.2%)
Depression	0/6 (0%)		0/8 (0%)		3/14 (21.4%)		2/10 (20%)		3/112 (2.7%)		3/107 (2.8%)		3/61 (4.9%)		6/107 (5.6%)
Mood altered	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		6/112 (5.4%)		1/107 (0.9%)		1/61 (1.6%)		2/107 (1.9%)
Agitation	0/6 (0%)		0/8 (0%)		0/14 (0%)		1/10 (10%)		3/112 (2.7%)		1/107 (0.9%)		2/61 (3.3%)		3/107 (2.8%)
Mental status changes	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		4/112 (3.6%)		1/107 (0.9%)		1/61 (1.6%)		2/107 (1.9%)
Affect lability	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		0/112 (0%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Mania	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		0/112 (0%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Renal and urinary disorders
Renal failure acute	0/6 (0%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		2/112 (1.8%)		4/107 (3.7%)		2/61 (3.3%)		6/107 (5.6%)
Urinary incontinence	1/6 (16.7%)		0/8 (0%)		2/14 (14.3%)		0/10 (0%)		3/112 (2.7%)		1/107 (0.9%)		1/61 (1.6%)		2/107 (1.9%)
Dysuria	0/6 (0%)		0/8 (0%)		0/14 (0%)		1/10 (10%)		1/112 (0.9%)		0/107 (0%)		3/61 (4.9%)		3/107 (2.8%)
Haematuria	1/6 (16.7%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		2/112 (1.8%)		1/107 (0.9%)		1/61 (1.6%)		2/107 (1.9%)
Pollakiuria	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		1/112 (0.9%)		1/107 (0.9%)		0/61 (0%)		1/107 (0.9%)
Cystitis noninfective	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		0/112 (0%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Respiratory, thoracic and mediastinal disorders
Dyspnoea	3/6 (50%)		6/8 (75%)		2/14 (14.3%)		3/10 (30%)		33/112 (29.5%)		23/107 (21.5%)		6/61 (9.8%)		26/107 (24.3%)
Cough	1/6 (16.7%)		0/8 (0%)		7/14 (50%)		2/10 (20%)		23/112 (20.5%)		14/107 (13.1%)		4/61 (6.6%)		15/107 (14%)
Epistaxis	1/6 (16.7%)		0/8 (0%)		2/14 (14.3%)		0/10 (0%)		12/112 (10.7%)		12/107 (11.2%)		4/61 (6.6%)		16/107 (15%)
Dyspnoea exertional	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		17/112 (15.2%)		9/107 (8.4%)		4/61 (6.6%)		12/107 (11.2%)
Oropharyngeal pain	0/6 (0%)		1/8 (12.5%)		2/14 (14.3%)		1/10 (10%)		11/112 (9.8%)		4/107 (3.7%)		2/61 (3.3%)		5/107 (4.7%)
Productive cough	0/6 (0%)		0/8 (0%)		0/14 (0%)		1/10 (10%)		10/112 (8.9%)		7/107 (6.5%)		1/61 (1.6%)		8/107 (7.5%)
Dysphonia	1/6 (16.7%)		1/8 (12.5%)		0/14 (0%)		1/10 (10%)		7/112 (6.3%)		4/107 (3.7%)		3/61 (4.9%)		7/107 (6.5%)
Rhinorrhoea	0/6 (0%)		1/8 (12.5%)		2/14 (14.3%)		2/10 (20%)		4/112 (3.6%)		3/107 (2.8%)		1/61 (1.6%)		4/107 (3.7%)
Nasal congestion	2/6 (33.3%)		1/8 (12.5%)		1/14 (7.1%)		0/10 (0%)		7/112 (6.3%)		1/107 (0.9%)		0/61 (0%)		1/107 (0.9%)
Pleural effusion	0/6 (0%)		1/8 (12.5%)		0/14 (0%)		0/10 (0%)		3/112 (2.7%)		2/107 (1.9%)		1/61 (1.6%)		3/107 (2.8%)
Sinus congestion	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		2/112 (1.8%)		3/107 (2.8%)		1/61 (1.6%)		4/107 (3.7%)
Rhinitis allergic	0/6 (0%)		0/8 (0%)		0/14 (0%)		1/10 (10%)		1/112 (0.9%)		3/107 (2.8%)		0/61 (0%)		3/107 (2.8%)
Upper-airway cough syndrome	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		2/112 (1.8%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Hiccups	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Sputum discoloured	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		0/112 (0%)		1/107 (0.9%)		0/61 (0%)		1/107 (0.9%)
Skin and subcutaneous tissue disorders
Rash	0/6 (0%)		2/8 (25%)		3/14 (21.4%)		1/10 (10%)		18/112 (16.1%)		22/107 (20.6%)		2/61 (3.3%)		23/107 (21.5%)
Hyperhidrosis	1/6 (16.7%)		2/8 (25%)		1/14 (7.1%)		2/10 (20%)		18/112 (16.1%)		4/107 (3.7%)		2/61 (3.3%)		6/107 (5.6%)
Pruritus	0/6 (0%)		2/8 (25%)		1/14 (7.1%)		2/10 (20%)		8/112 (7.1%)		14/107 (13.1%)		1/61 (1.6%)		15/107 (14%)
Dry skin	0/6 (0%)		0/8 (0%)		2/14 (14.3%)		0/10 (0%)		12/112 (10.7%)		9/107 (8.4%)		1/61 (1.6%)		10/107 (9.3%)
Night sweats	0/6 (0%)		1/8 (12.5%)		1/14 (7.1%)		0/10 (0%)		14/112 (12.5%)		3/107 (2.8%)		2/61 (3.3%)		5/107 (4.7%)
Alopecia	1/6 (16.7%)		0/8 (0%)		2/14 (14.3%)		2/10 (20%)		2/112 (1.8%)		1/107 (0.9%)		0/61 (0%)		1/107 (0.9%)
Ecchymosis	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		1/112 (0.9%)		3/107 (2.8%)		3/61 (4.9%)		5/107 (4.7%)
Petechiae	1/6 (16.7%)		0/8 (0%)		0/14 (0%)		1/10 (10%)		1/112 (0.9%)		3/107 (2.8%)		0/61 (0%)		3/107 (2.8%)
Urticaria	0/6 (0%)		2/8 (25%)		1/14 (7.1%)		1/10 (10%)		1/112 (0.9%)		1/107 (0.9%)		0/61 (0%)		1/107 (0.9%)
Cold sweat	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		1/112 (0.9%)		1/107 (0.9%)		0/61 (0%)		1/107 (0.9%)
Nail disorder	1/6 (16.7%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		1/112 (0.9%)		1/107 (0.9%)		0/61 (0%)		1/107 (0.9%)
Pruritus allergic	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Rash erythematous	0/6 (0%)		1/8 (12.5%)		0/14 (0%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Acne	1/6 (16.7%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		0/112 (0%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Drug eruption	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		0/112 (0%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Hirsutism	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		0/112 (0%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Ingrown nail	1/6 (16.7%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		0/112 (0%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Lichenoid karatosis	0/6 (0%)		0/8 (0%)		0/14 (0%)		1/10 (10%)		0/112 (0%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Skin disorder	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		0/112 (0%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Skin fissures	0/6 (0%)		1/8 (12.5%)		0/14 (0%)		0/10 (0%)		0/112 (0%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Vascular disorders
Hypotension	1/6 (16.7%)		0/8 (0%)		0/14 (0%)		1/10 (10%)		6/112 (5.4%)		5/107 (4.7%)		1/61 (1.6%)		6/107 (5.6%)
Hot flush	0/6 (0%)		1/8 (12.5%)		1/14 (7.1%)		0/10 (0%)		5/112 (4.5%)		3/107 (2.8%)		1/61 (1.6%)		4/107 (3.7%)
Deep vein thrombosis	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		1/112 (0.9%)		1/107 (0.9%)		1/61 (1.6%)		2/107 (1.9%)
Haematoma	1/6 (16.7%)		0/8 (0%)		0/14 (0%)		1/10 (10%)		0/112 (0%)		1/107 (0.9%)		1/61 (1.6%)		2/107 (1.9%)
Orthostatic hypotension	0/6 (0%)		0/8 (0%)		0/14 (0%)		1/10 (10%)		1/112 (0.9%)		1/107 (0.9%)		0/61 (0%)		1/107 (0.9%)
Peripheral coldness	0/6 (0%)		0/8 (0%)		1/14 (7.1%)		0/10 (0%)		1/112 (0.9%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Haemorrhage	0/6 (0%)		0/8 (0%)		0/14 (0%)		1/10 (10%)		0/112 (0%)		0/107 (0%)		0/61 (0%)		0/107 (0%)
Thrombosis	1/6 (16.7%)		0/8 (0%)		0/14 (0%)		0/10 (0%)		0/112 (0%)		0/107 (0%)		0/61 (0%)		0/107 (0%)

Limitations/Caveats

[Not Specified]

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Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

Multicenter publication must include input from investigators and Celgene, and has priority over subset (single center) publication, for 1 year after study completion. Investigators have publication right after multicenter publication is complete (or 1 year after study completion), whichever is first. Celgene has 45 days to review and comment on draft manuscript/presentation, and may ask for an additional delay (not to exceed 90 days total) to address intellectual property issues.

Results Point of Contact

Name/Title	Associate Director, Clinical Trials Disclosure
Organization	Celgene Corporation
Phone	1-888-260-1599
Email	clinicaltrialdisclosure@celgene.com

Responsible Party:

Celgene Corporation

ClinicalTrials.gov Identifier:

NCT00833833

Other Study ID Numbers:

CC-4047-MM-002

First Posted:

Feb 2, 2009

Last Update Posted:

Apr 27, 2016

Last Verified:

Mar 1, 2016

MTD, Safety, and Efficacy of Pomalidomide (CC-4047) Alone or With Low-dose Dexamethasone in Patients With Relapsed and Refractory Multiple Myeloma

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Statistical Analysis 1

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Outcome Measure Data

Outcome Measure Data

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Statistical Analysis 1

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Statistical Analysis 1

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Statistical Analysis 1

Outcome Measure Data

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