Mobilization of Stem Cells With AMD3100 (Plerixafor) in Multiple Myeloma Patients

• Sponsor: Genzyme, a Sanofi Company (Industry)
• Overall Status: Completed
• CT.gov ID: NCT00103662
• Collaborator: (none)
• Enrollment: 302
• Locations: 39
• Arms: 2
• Duration (Months): 36
• Patients Per Site: 7.7
• Patients Per Site Per Month: 0.2

Study Details

Study Description

Brief Summary

The purpose of this study is to determine whether the combination of AMD3100 (plerixafor) and granulocyte colony-stimulating factor (G-CSF, generic name of filgrastim) is better than G-CSF alone to mobilize and collect the optimal number of stem cells in multiple myeloma patients for autologous transplantation.

Condition or Disease	Intervention/Treatment	Phase
Multiple Myeloma	Drug: Granulocyte colony-stimulating factor plus plerixafor; Drug: Granulocyte colony-stimulating factor plus placebo	Phase 3

Detailed Description

A peripheral stem cell transplant may be able to replace blood-forming cells that were destroyed by chemotherapy. Currently filgrastim (G-CSF), a colony stimulating factor, is used to cause the growth and mobilization of stem cells from bone marrow to peripheral blood, which can then be collected from the peripheral blood by a process called apheresis. Plerixafor aids in the release of the stem cells from the bone marrow into the peripheral blood, possibly allowing for a more rapid collection of a larger number of stem cells from the peripheral blood. Larger stem cell doses for transplantation correlate to faster recovery times after high dose chemotherapy followed with stem cell transplantation. This study is intended to determine whether the combination of plerixafor with filgrastim (G-CSF)is better than filgrastim (G-CSF) alone in helping multiple myeloma patients collect at least 6 million stem cells in two or less apheresis sessions.

This study was previously posted by AnorMED, Inc. In November 2006, AnorMED, Inc. was acquired by Genzyme Corporation. Genzyme Corporation is the sponsor of the trial.

Study Design

• Study Type: Interventional
• Actual Enrollment: 302 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
• Primary Purpose: Treatment
• Official Title: A Multicenter, Randomized, Double-Blind, Placebo-Controlled, Comparative Trial of AMD3100 Plus G-CSF Versus G-CSF Plus Placebo to Mobilize and Collect ≥ 6*10^6 CD34+ Cells/kg in Multiple Myeloma Patients for Autologous Transplantation
• Study Start Date: Jan 1, 2005
• Actual Primary Completion Date: Oct 1, 2006
• Actual Study Completion Date: Jan 1, 2008

Arms and Interventions

Arm	Intervention/Treatment
Experimental: G-CSF plus plerixafor	Drug: Granulocyte colony-stimulating factor plus plerixafor; Participants underwent mobilization with granulocyte colony-stimulating factor (G-CSF) (10 µg/kg/day) for 4 days, administered by subcutaneous (SC) injection. On the evening of Day 4, participants received plerixafor (240 µg/kg), administered by SC injection. On Day 5, participants received a morning dose of G-CSF (10 µg/kg) and underwent apheresis approx. 10 to 11 hours after the dose of plerixafor (within 60 minutes of G-CSF administration). Participants continued to receive an evening dose of plerixafor followed by a morning dose of G-CSF and apheresis for up to 4 aphereses or until ≥ 6*10^6 CD34+ cells/kg were collected. Participants who participated in the rescue procedure underwent an additional daily treatment with plerixafor (240 µg/kg) and apheresis for up to 4 days.; Other Names: Mozobil AMD3100
Placebo Comparator: G-CSF plus placebo	Drug: Granulocyte colony-stimulating factor plus placebo; Participants underwent mobilization with granulocyte colony-stimulating factor (G-CSF) (10 µg/kg/day) for 4 days, administered by subcutaneous (SC) injection. On the evening of Day 4, participants received placebo, administered by SC injection. On Day 5, participants received a morning dose of G-CSF (10 µg/kg) and underwent apheresis approx. 10 to 11 hours after the dose of placebo (within 60 minutes of G-CSF administration). Participants continued to receive an evening dose of placebo followed by a morning dose of G-CSF and apheresis for up to 4 aphereses or until ≥ 6*10^6 CD34+ cells/kg were collected. Participants who participated in the rescue procedure underwent an additional daily treatment with plerixafor (240 µg/kg) and apheresis for up to 4 days.

Outcome Measures

Primary Outcome Measures

1. Proportion of Participants Achieving a Target of ≥ 6*10^6 CD34+ Cells/kg in 2 or Fewer Days of Apheresis. [up to Day 6]

   Proportion of participants achieving a target of ≥ 6*10^6 CD34+ cells/kg in 2 or fewer days of apheresis. Central lab data were taken from Days 5 to 6 of the Treatment/Apheresis period. Each participant's value was calculated as the sum of all daily values collected over the 2 apheresis days.

Secondary Outcome Measures

1. Number of Participants With Adverse Events [up to Day 38]

   Number of participants with treatment emergent adverse events (AEs). The timeframe for treatment emergent AEs is defined as Day 1 (start of G-CSF Mobilization) to the day before starting chemotherapy (approximately 38 days later). AEs were reported regardless of relationship to study treatment. The investigator graded each AE using the World Health Organization (WHO) Adverse Event Grading Scale. AEs of Grade 3 were considered severe and Grade 4 were considered life-threatening.

2. Proportion of Participants Achieving a Target of ≥ 6*10^6 CD34+ Cells/kg in 4 or Fewer Days of Apheresis. [up to Day 8]

   Proportion of participants achieving a target of ≥ 6*10^6 CD34+ cells/kg in 4 or fewer days of apheresis. Central lab data were taken from Days 5 to 8 of the Treatment/Apheresis period. Each participant's value was calculated as the sum of all daily values collected over the 4 apheresis days.

3. Proportion of Participants Achieving a Target of ≥ 2*10^6 CD34+ Cells/kg in 4 or Fewer Days of Apheresis. [up to Day 8]

   Proportion of participants achieving a target of ≥ 2*10^6 CD34+ cells/kg in 4 or fewer days of apheresis. Central lab data were taken from Days 5 to 8 of the Treatment/Apheresis period. Each participant's value was calculated as the sum of all daily values collected over the 4 apheresis days.

4. Median Number of Days to ≥6*10^6 CD34+ Cells/kg [up to Day 8]

   The Kaplan Meier estimate of median number of days (number of days at which 50% of participants have experienced the event, accounting for censored values) in each treatment arm to collect an optimum number of cells (≥6*10^6 CD34+ cells/kg) for transplantation.

5. Median Number of Days to Polymorphonuclear (PMN) Cell Engraftment [Up to Month 13]

   The Kaplan Meier estimate of median number of days to PMN engraftment (number of days at which 50% of participants have experienced the event, accounting for censored values) was a secondary efficacy endpoint. Engraftment was defined as PMN counts ≥ 0.5*10^9/L for 3 consecutive days or ≥ 1.0*10^9/L for 1 day. Time to engraftment corresponded to the first day that the criteria were met and was evaluated up to 12 months post transplant.

6. Median Number of Days to Platelet (PLT) Engraftment [Up to Month 13]

   The Kaplan Meier estimate of median number of days to PLT engraftment (number of days at which 50% of participants have experienced the event, accounting for censored values) was a secondary efficacy endpoint. Engraftment was defined as ≥ 20*10^9/L without transfusion for the preceding 7 days. Time to engraftment corresponded to the first day that the criteria were met and was evaluated up to 12 months post transplant.

7. Graft Durability at 100 Days Post Transplantation [approximately Day 138]

   The proportion of participants maintaining a durable graft at 100 days post-transplantation by at least 2 of the following criteria (without erythropoietin (EPO), G-CSF, or transfusions): (1) a platelet count >50000/µL without transfusion for at least 2 weeks, (2) hemoglobin >=10g/dL for at least 1 month, (3) and absolute neutrophil count >1000/µL for at least 1 week.

8. Graft Durability at 6 Months Post Transplantation [approximately Month 7]

   The proportion of participants maintaining a durable graft at 6 months post-transplantation by at least 2 of the following criteria (without erythropoietin (EPO), G-CSF, or transfusions): (1) a platelet count >50000/µL without transfusion for at least 2 weeks, (2) hemoglobin >=10g/dL for at least 1 month, (3) and absolute neutrophil count >1000/µL for at least 1 week.

9. Graft Durability at 12 Months Post Transplantation [approximately Month 13]

   The proportion of participants maintaining a durable graft at 12 months post-transplantation by at least 2 of the following criteria (without erythropoietin (EPO), G-CSF, or transfusions): (1) a platelet count >50000/µL without transfusion for at least 2 weeks, (2) hemoglobin >=10g/dL for at least 1 month, (3) and absolute neutrophil count >1000/µL for at least 1 week.

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years to 78 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• Diagnosis of multiple myeloma in first or second complete or partial remission

• = 4 weeks since last cycle of chemotherapy (thalidomide, dexamethasone, and Velcade were not considered prior chemotherapy for the purpose of this study)

• Recovered from all acute toxic effects of prior chemotherapy

• Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1

• White Blood Cell count (WBC) > 2.5*10^9/L

• Absolute polymorphonuclear leukocytes (PMN) count > 1.5*10^9/L

• Platelet (PLT) > 100*10^9/L

• Serum creatinine <=2.2 mg/dL

• Cardiac and pulmonary status sufficient to undergo apheresis and transplantation

• Negative for HIV

Exclusion Criteria):

• Failed previous stem cell collection

• Previous stem cell transplantation

• Brain metastases or myelomatous meningitis

• Radiation to ≥ 50% of the pelvis

• Abnormal electrocardiogram (ECG) with rhythm disturbance (ventricular arrhythmias) or other conduction abnormality

• Received bone-seeking radionuclides (e.g. holmium)

• A residual acute medical condition resulting from prior chemotherapy

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	City of Hope Samaritan Bone Marrow Transplant Program	Phoenix	Arizona	United States	85006
2	Myeloma Institute for Research and Therapy, University of Arkansas for Medical Sciences	Little Rock	Arkansas	United States	72205
3	City of Hope National Medical Center	Duarte	California	United States	91010
4	Cedars-Sinai	Los Angeles	California	United States	90048
5	University of California	Los Angeles	California	United States	90095
6	Rocky Mountain Cancer Center	Denver	Colorado	United States	80218
7	Yale University School of Medicine	New Haven	Connecticut	United States	06520
8	University of Florida	Gainesville	Florida	United States	32611
9	H. Lee Moffitt Cancer Center and Research Institute	Tampa	Florida	United States	33612
10	Emory University	Atlanta	Georgia	United States	30322
11	Loyola University Medical Center	Maywood	Illinois	United States	60153
12	Indiana Blood and Marrow Transplantation Center	Beech Grove	Indiana	United States	46107
13	University of Iowa Hosptials and Clinics	Iowa City	Iowa	United States	52242
14	Fairview-University Medical Center, University of Minnesota	Minneapolis	Minnesota	United States	55455
15	Mayo Clinic	Rochester	Minnesota	United States	55905
16	Kansas City Cancer Center	Kansas City	Missouri	United States	64111
17	Washington University School of Medicine, Division of Bone Marrow Transplantation and Leukemia	Saint Louis	Missouri	United States	63110
18	The Cancer Center at Hackensack University Medical Center	Hackensack	New Jersey	United States	07601
19	Roswell Park Cancer Institute	Buffalo	New York	United States	14263
20	St. Vincent's Comprehensive Cancer Center	New York	New York	United States	10011
21	New York Hospital	New York	New York	United States	10032
22	Memorial Sloan Kettering	New York	New York	United States	10065
23	University of Rochester Medical Center	Rochester	New York	United States	14642
24	Duke University Medical Center	Durham	North Carolina	United States	27705
25	Case Western Reserve University	Cleveland	Ohio	United States	44106
26	Cleveland Clinic Foundation	Cleveland	Ohio	United States	44195
27	Ohio State University	Columbus	Ohio	United States	43210
28	Oregon Health & Science University	Portland	Oregon	United States	97239
29	Hospital of the University of Pennsylvania	Philadelphia	Pennsylvania	United States	19104
30	Thomas Jefferson University	Philadelphia	Pennsylvania	United States	19107
31	The University of Texas MD Anderson Cancer Center	Houston	Texas	United States	77030
32	Wilford Hall Medical Center	Lackland AFB	Texas	United States	78236
33	Texas Transplant Institute	San Antonio	Texas	United States	78229
34	University of Texas Health Science Center	San Antonio	Texas	United States	78229
35	Utah Blood and Marrow Transplant Program, University of Utah	Salt Lake City	Utah	United States	84132
36	Virginia Commonwealth University	Richmond	Virginia	United States	23298
37	Fred Hutchinson Cancer Research Center	Seattle	Washington	United States	98109
38	Vancouver General Hospital	Vancouver	British Columbia	Canada	V5Z 1M9
39	Universitätsklinikum Heidelberg,	Heidelberg		Germany	69120

Sponsors and Collaborators

• Genzyme, a Sanofi Company

Investigators

• Study Director: Medical Monitor, Genzyme, a Sanofi Company

Study Documents (Full-Text)

None provided.

More Information

Additional Information:

• Further information on multiple myeloma from the National Cancer Institute

Publications

None provided.

• ClinicalTrials.gov Identifier: NCT00103662
• Other Study ID Numbers: AMD3100-3102; 2005-003599-39; NCT00248417
• First Posted: Feb 14, 2005
• Last Update Posted: Mar 13, 2014
• Last Verified: Feb 1, 2014

• Additional relevant MeSH terms: Multiple Myeloma; Neoplasms, Plasma Cell; Plerixafor; Lenograstim

Study Results

Participant Flow

Recruitment Details	Participants with multiple myeloma (MM) eligible for autologous hematopoietic stem cell transplant were recruited from 40 centers (38 in the U.S., 1 in Germany, 1 in Canada). The first participant was randomized on 04 February 2005 and the last participant's last study visit occurred on 22 January 2008. A total of 302 participants were randomized.

Arm/Group Title	G-CSF Plus Plerixafor	G-CSF Plus Placebo
Arm/Group Description	Participants underwent mobilization with G-CSF for 4 days. On the evening of Day 4, participants received a dose of plerixafor. On each subsequent day, participants received a morning dose of G-CSF followed by apheresis and an evening dose of plerixafor for a maximum of 4 aphereses or until ≥ 6*10^6 CD34+ cells/kg were collected.	Participants underwent mobilization with G-CSF for 4 days. On the evening of Day 4, participants received a dose of placebo. On each subsequent day, participants received a morning dose of G-CSF followed by apheresis and an evening dose of placebo for a maximum of 4 aphereses or until ≥ 6*10^6 CD34+ cells/kg were collected.
Period Title: Overall Study
STARTED	148	154
COMPLETED	129	121
NOT COMPLETED	19	33

Baseline Characteristics

Arm/Group Title	G-CSF Plus Plerixafor	G-CSF Plus Placebo	Total
Arm/Group Description	Participants underwent mobilization with G-CSF for 4 days. On the evening of Day 4, participants received a dose of plerixafor. On each subsequent day, participants received a morning dose of G-CSF followed by apheresis and an evening dose of plerixafor for a maximum of 4 aphereses or until ≥ 6*10^6 CD34+ cells/kg were collected.	Participants underwent mobilization with G-CSF for 4 days. On the evening of Day 4, participants received a dose of placebo. On each subsequent day, participants received a morning dose of G-CSF followed by apheresis and an evening dose of placebo for a maximum of 4 aphereses or until ≥ 6*10^6 CD34+ cells/kg were collected.	Total of all reporting groups
Overall Participants	148	154	302
Age (years) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [years]	58.2 (8.4)	58.4 (8.6)	58.3 (8.5)
Sex: Female, Male (Count of Participants)
Female	48 32.4%	47 30.5%	95 31.5%
Male	100 67.6%	107 69.5%	207 68.5%
Race/Ethnicity, Customized (participants) [Number]
Caucasian	117 79.1%	128 83.1%	245 81.1%
African-American	18 12.2%	14 9.1%	32 10.6%
Asian	1 0.7%	3 1.9%	4 1.3%
Hispanic/Latino	11 7.4%	4 2.6%	15 5%
Other	1 0.7%	5 3.2%	6 2%

Outcome Measures

1. Primary Outcome

Title	Proportion of Participants Achieving a Target of ≥ 6*10^6 CD34+ Cells/kg in 2 or Fewer Days of Apheresis.
Description	Proportion of participants achieving a target of ≥ 6*10^6 CD34+ cells/kg in 2 or fewer days of apheresis. Central lab data were taken from Days 5 to 6 of the Treatment/Apheresis period. Each participant's value was calculated as the sum of all daily values collected over the 2 apheresis days.
Time Frame	up to Day 6

Outcome Measure Data

Analysis Population Description

Intent-to-Treat Population

Arm/Group Title	G-CSF Plus Plerixafor	G-CSF Plus Placebo
Arm/Group Description	Participants underwent mobilization with G-CSF for 4 days. On the evening of Day 4, participants received a dose of plerixafor. On each subsequent day, participants received a morning dose of G-CSF followed by apheresis and an evening dose of plerixafor for a maximum of 4 aphereses or until ≥ 6*10^6 CD34+ cells/kg were collected.	Participants underwent mobilization with G-CSF for 4 days. On the evening of Day 4, participants received a dose of placebo. On each subsequent day, participants received a morning dose of G-CSF followed by apheresis and an evening dose of placebo for a maximum of 4 aphereses or until ≥ 6*10^6 CD34+ cells/kg were collected.
Measure Participants	148	154
Proportion achieving target in ≤2 days	0.716 0.5%	0.344 0.2%
Proportion not achieving target in ≤2 days	0.284 0.2%	0.656 0.4%

2. Secondary Outcome

Title	Number of Participants With Adverse Events
Description	Number of participants with treatment emergent adverse events (AEs). The timeframe for treatment emergent AEs is defined as Day 1 (start of G-CSF Mobilization) to the day before starting chemotherapy (approximately 38 days later). AEs were reported regardless of relationship to study treatment. The investigator graded each AE using the World Health Organization (WHO) Adverse Event Grading Scale. AEs of Grade 3 were considered severe and Grade 4 were considered life-threatening.
Time Frame	up to Day 38

Outcome Measure Data

Analysis Population Description

Primary Safety population of all participants who received at least 1 mobilization dose of G-CSF or study treatment (plerixafor or placebo). Four participants did not receive G-CSF or any study treatment and were excluded from the safety analyses.

Arm/Group Title	G-CSF Plus Plerixafor	G-CSF Plus Placebo
Arm/Group Description	Participants underwent mobilization with G-CSF for 4 days. On the evening of Day 4, participants received a dose of plerixafor. On each subsequent day, participants received a morning dose of G-CSF followed by apheresis and an evening dose of plerixafor for a maximum of 4 aphereses or until ≥ 6*10^6 CD34+ cells/kg were collected.	Participants underwent mobilization with G-CSF for 4 days. On the evening of Day 4, participants received a dose of placebo. On each subsequent day, participants received a morning dose of G-CSF followed by apheresis and an evening dose of placebo for a maximum of 4 aphereses or until ≥ 6*10^6 CD34+ cells/kg were collected.
Measure Participants	147	151
Adverse Events (AEs)	140 94.6%	140 90.9%
Related AEs	95 64.2%	67 43.5%
AEs Leading to early treatment termination	1 0.7%	2 1.3%
AEs Leading to early termination	3 2%	0 0%
Grade 3 (severe) or 4 (life-threatening) AEs	11 7.4%	11 7.1%
Serious Adverse Events (SAEs)	4 2.7%	6 3.9%

3. Secondary Outcome

Title	Proportion of Participants Achieving a Target of ≥ 6*10^6 CD34+ Cells/kg in 4 or Fewer Days of Apheresis.
Description	Proportion of participants achieving a target of ≥ 6*10^6 CD34+ cells/kg in 4 or fewer days of apheresis. Central lab data were taken from Days 5 to 8 of the Treatment/Apheresis period. Each participant's value was calculated as the sum of all daily values collected over the 4 apheresis days.
Time Frame	up to Day 8

Outcome Measure Data

Analysis Population Description

Intent-to-Treat Population

Arm/Group Title	G-CSF Plus Plerixafor	G-CSF Plus Placebo
Arm/Group Description	Participants underwent mobilization with G-CSF for 4 days. On the evening of Day 4, participants received a dose of plerixafor. On each subsequent day, participants received a morning dose of G-CSF followed by apheresis and an evening dose of plerixafor for a maximum of 4 aphereses or until ≥ 6*10^6 CD34+ cells/kg were collected.	Participants underwent mobilization with G-CSF for 4 days. On the evening of Day 4, participants received a dose of placebo. On each subsequent day, participants received a morning dose of G-CSF followed by apheresis and an evening dose of placebo for a maximum of 4 aphereses or until ≥ 6*10^6 CD34+ cells/kg were collected.
Measure Participants	148	154
Proportion achieving target in ≤4 days	0.757 0.5%	0.513 0.3%
Proportion not achieving target in ≤4 days	0.243 0.2%	0.487 0.3%

4. Secondary Outcome

Title	Proportion of Participants Achieving a Target of ≥ 2*10^6 CD34+ Cells/kg in 4 or Fewer Days of Apheresis.
Description	Proportion of participants achieving a target of ≥ 2*10^6 CD34+ cells/kg in 4 or fewer days of apheresis. Central lab data were taken from Days 5 to 8 of the Treatment/Apheresis period. Each participant's value was calculated as the sum of all daily values collected over the 4 apheresis days.
Time Frame	up to Day 8

Outcome Measure Data

Analysis Population Description

Intent-to-Treat Population

Arm/Group Title	G-CSF Plus Plerixafor	G-CSF Plus Placebo
Arm/Group Description	Participants underwent mobilization with G-CSF for 4 days. On the evening of Day 4, participants received a dose of plerixafor. On each subsequent day, participants received a morning dose of G-CSF followed by apheresis and an evening dose of plerixafor for a maximum of 4 aphereses or until ≥ 6*10^6 CD34+ cells/kg were collected.	Participants underwent mobilization with G-CSF for 4 days. On the evening of Day 4, participants received a dose of placebo. On each subsequent day, participants received a morning dose of G-CSF followed by apheresis and an evening dose of placebo for a maximum of 4 aphereses or until ≥ 6*10^6 CD34+ cells/kg were collected.
Measure Participants	148	154
Proportion achieving target in ≤4 days	0.953 0.6%	0.883 0.6%
Proportion not achieving target in ≤4 days	0.047 0%	0.117 0.1%

5. Secondary Outcome

Title	Median Number of Days to ≥6*10^6 CD34+ Cells/kg
Description	The Kaplan Meier estimate of median number of days (number of days at which 50% of participants have experienced the event, accounting for censored values) in each treatment arm to collect an optimum number of cells (≥6*10^6 CD34+ cells/kg) for transplantation.
Time Frame	up to Day 8

Outcome Measure Data

Analysis Population Description

Intent-to-treat population

Arm/Group Title	G-CSF Plus Plerixafor	G-CSF Plus Placebo
Arm/Group Description	Participants underwent mobilization with G-CSF for 4 days. On the evening of Day 4, participants received a dose of plerixafor. On each subsequent day, participants received a morning dose of G-CSF followed by apheresis and an evening dose of plerixafor for a maximum of 4 aphereses or until ≥ 6*10^6 CD34+ cells/kg were collected.	Participants underwent mobilization with G-CSF for 4 days. On the evening of Day 4, participants received a dose of placebo. On each subsequent day, participants received a morning dose of G-CSF followed by apheresis and an evening dose of placebo for a maximum of 4 aphereses or until ≥ 6*10^6 CD34+ cells/kg were collected.
Measure Participants	144	150
Median (Inter-Quartile Range) [Days]	1.0	4.0

6. Secondary Outcome

Title	Median Number of Days to Polymorphonuclear (PMN) Cell Engraftment
Description	The Kaplan Meier estimate of median number of days to PMN engraftment (number of days at which 50% of participants have experienced the event, accounting for censored values) was a secondary efficacy endpoint. Engraftment was defined as PMN counts ≥ 0.5*10^9/L for 3 consecutive days or ≥ 1.0*10^9/L for 1 day. Time to engraftment corresponded to the first day that the criteria were met and was evaluated up to 12 months post transplant.
Time Frame	Up to Month 13

Outcome Measure Data

Analysis Population Description

Participants who received a stem cell transplant.

Arm/Group Title	G-CSF Plus Plerixafor	G-CSF Plus Placebo
Arm/Group Description	Participants underwent mobilization with G-CSF for 4 days. On the evening of Day 4, participants received a dose of plerixafor. On each subsequent day, participants received a morning dose of G-CSF followed by apheresis and an evening dose of plerixafor for a maximum of 4 aphereses or until ≥ 6*10^6 CD34+ cells/kg were collected.	Participants underwent mobilization with G-CSF for 4 days. On the evening of Day 4, participants received a dose of placebo. On each subsequent day, participants received a morning dose of G-CSF followed by apheresis and an evening dose of placebo for a maximum of 4 aphereses or until ≥ 6*10^6 CD34+ cells/kg were collected.
Measure Participants	142	136
Median (Inter-Quartile Range) [Days]	11.0	11.0

7. Secondary Outcome

Title	Median Number of Days to Platelet (PLT) Engraftment
Description	The Kaplan Meier estimate of median number of days to PLT engraftment (number of days at which 50% of participants have experienced the event, accounting for censored values) was a secondary efficacy endpoint. Engraftment was defined as ≥ 20*10^9/L without transfusion for the preceding 7 days. Time to engraftment corresponded to the first day that the criteria were met and was evaluated up to 12 months post transplant.
Time Frame	Up to Month 13

Outcome Measure Data

Analysis Population Description

Participants who received a stem cell transplant

Arm/Group Title	G-CSF Plus Plerixafor	G-CSF Plus Placebo
Arm/Group Description	Participants underwent mobilization with G-CSF for 4 days. On the evening of Day 4, participants received a dose of plerixafor. On each subsequent day, participants received a morning dose of G-CSF followed by apheresis and an evening dose of plerixafor for a maximum of 4 aphereses or until ≥ 6*10^6 CD34+ cells/kg were collected.	Participants underwent mobilization with G-CSF for 4 days. On the evening of Day 4, participants received a dose of placebo. On each subsequent day, participants received a morning dose of G-CSF followed by apheresis and an evening dose of placebo for a maximum of 4 aphereses or until ≥ 6*10^6 CD34+ cells/kg were collected.
Measure Participants	142	136
Median (Inter-Quartile Range) [Days]	18.0	18.0

8. Secondary Outcome

Title	Graft Durability at 100 Days Post Transplantation
Description	The proportion of participants maintaining a durable graft at 100 days post-transplantation by at least 2 of the following criteria (without erythropoietin (EPO), G-CSF, or transfusions): (1) a platelet count >50000/µL without transfusion for at least 2 weeks, (2) hemoglobin >=10g/dL for at least 1 month, (3) and absolute neutrophil count >1000/µL for at least 1 week.
Time Frame	approximately Day 138

Outcome Measure Data

Analysis Population Description

Participants who received a stem cell transplant and were evaluable at 100 days post-transplant

Arm/Group Title	G-CSF Plus Plerixafor	G-CSF Plus Placebo
Arm/Group Description	Participants underwent mobilization with G-CSF for 4 days. On the evening of Day 4, participants received a dose of plerixafor. On each subsequent day, participants received a morning dose of G-CSF followed by apheresis and an evening dose of plerixafor for a maximum of 4 aphereses or until ≥ 6*10^6 CD34+ cells/kg were collected.	Participants underwent mobilization with G-CSF for 4 days. On the evening of Day 4, participants received a dose of placebo. On each subsequent day, participants received a morning dose of G-CSF followed by apheresis and an evening dose of placebo for a maximum of 4 aphereses or until ≥ 6*10^6 CD34+ cells/kg were collected.
Measure Participants	142	136
Proportion of participants with a durable graft	0.986 0.7%	0.978 0.6%
Proportion of participants without a durable graft	0.014 0%	0.022 0%

9. Secondary Outcome

Title	Graft Durability at 6 Months Post Transplantation
Description	The proportion of participants maintaining a durable graft at 6 months post-transplantation by at least 2 of the following criteria (without erythropoietin (EPO), G-CSF, or transfusions): (1) a platelet count >50000/µL without transfusion for at least 2 weeks, (2) hemoglobin >=10g/dL for at least 1 month, (3) and absolute neutrophil count >1000/µL for at least 1 week.
Time Frame	approximately Month 7

Outcome Measure Data

Analysis Population Description

Participants who received a stem cell transplant and were evaluable at 6 months post-transplant

Arm/Group Title	G-CSF Plus Plerixafor	G-CSF Plus Placebo
Arm/Group Description	Participants underwent mobilization with G-CSF for 4 days. On the evening of Day 4, participants received a dose of plerixafor. On each subsequent day, participants received a morning dose of G-CSF followed by apheresis and an evening dose of plerixafor for a maximum of 4 aphereses or until ≥ 6*10^6 CD34+ cells/kg were collected.	Participants underwent mobilization with G-CSF for 4 days. On the evening of Day 4, participants received a dose of placebo. On each subsequent day, participants received a morning dose of G-CSF followed by apheresis and an evening dose of placebo for a maximum of 4 aphereses or until ≥ 6*10^6 CD34+ cells/kg were collected.
Measure Participants	135	127
Proportion of participants with a durable graft	0.985 0.7%	0.984 0.6%
Proportion of participants without a durable graft	0.015 0%	0.016 0%

10. Secondary Outcome

Title	Graft Durability at 12 Months Post Transplantation
Description	The proportion of participants maintaining a durable graft at 12 months post-transplantation by at least 2 of the following criteria (without erythropoietin (EPO), G-CSF, or transfusions): (1) a platelet count >50000/µL without transfusion for at least 2 weeks, (2) hemoglobin >=10g/dL for at least 1 month, (3) and absolute neutrophil count >1000/µL for at least 1 week.
Time Frame	approximately Month 13

Outcome Measure Data

Analysis Population Description

Participants who received a stem cell transplant and were evaluable at 12 months post-transplant

Arm/Group Title	G-CSF Plus Plerixafor	G-CSF Plus Placebo
Arm/Group Description	Participants underwent mobilization with G-CSF for 4 days. On the evening of Day 4, participants received a dose of plerixafor. On each subsequent day, participants received a morning dose of G-CSF followed by apheresis and an evening dose of plerixafor for a maximum of 4 aphereses or until ≥ 6*10^6 CD34+ cells/kg were collected.	Participants underwent mobilization with G-CSF for 4 days. On the evening of Day 4, participants received a dose of placebo. On each subsequent day, participants received a morning dose of G-CSF followed by apheresis and an evening dose of placebo for a maximum of 4 aphereses or until ≥ 6*10^6 CD34+ cells/kg were collected.
Measure Participants	128	120
Proportion of participants with a durable graft	0.992 0.7%	0.992 0.6%
Proportion of participants without a durable graft	0.008 0%	0.008 0%

Adverse Events

Time Frame	Day 1 (start of G-CSF Mobilization plus Treatment/Apheresis) to the day before starting chemotherapy. Chemotherapy typically started within 30 days of the last apheresis (which may have occurred on Day 5, 6, 7, or 8).
Adverse Event Reporting Description	Four participants did not receive any study treatment and were excluded from the safety analyses. In the event a participant experienced both a serious and a non-serious form of the same AE, they were included in the numerator of both AE tables. Each AE table includes all events, regardless of reported relationship to study treatment or grade.
Arm/Group Title	G-CSF Plus Plerixafor		G-CSF Plus Placebo
Arm/Group Description	Participants underwent mobilization with G-CSF for 4 days. On the evening of Day 4, participants received a dose of plerixafor. On each subsequent day, participants received a morning dose of G-CSF followed by apheresis and an evening dose of plerixafor for a maximum of 4 aphereses or until ≥ 6*10^6 CD34+ cells/kg were collected.		Participants underwent mobilization with G-CSF for 4 days. On the evening of Day 4, participants received a dose of placebo. On each subsequent day, participants received a morning dose of G-CSF followed by apheresis and an evening dose of placebo for a maximum of 4 aphereses or until ≥ 6*10^6 CD34+ cells/kg were collected.
All Cause Mortality
	G-CSF Plus Plerixafor		G-CSF Plus Placebo
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	/ (NaN)		/ (NaN)
Serious Adverse Events
	G-CSF Plus Plerixafor		G-CSF Plus Placebo
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	4/147 (2.7%)		6/151 (4%)
Cardiac disorders
Atrial fibrillation	1/147 (0.7%)		0/151 (0%)
Gastrointestinal disorders
Nausea	0/147 (0%)		2/151 (1.3%)
Vomiting	0/147 (0%)		1/151 (0.7%)
Infections and infestations
Enterobacter bacteraemia	0/147 (0%)		1/151 (0.7%)
Metabolism and nutrition disorders
Dehydration	0/147 (0%)		1/151 (0.7%)
Musculoskeletal and connective tissue disorders
Bone pain	1/147 (0.7%)		1/151 (0.7%)
Nervous system disorders
Hemiparesis	1/147 (0.7%)		0/151 (0%)
Muscle spasticity	0/147 (0%)		1/151 (0.7%)
Psychiatric disorders
Agitation	0/147 (0%)		1/151 (0.7%)
Respiratory, thoracic and mediastinal disorders
Pneumothorax	0/147 (0%)		1/151 (0.7%)
Vascular disorders
Deep vein thrombosis	1/147 (0.7%)		0/151 (0%)
Jugular vein thrombosis	0/147 (0%)		1/151 (0.7%)
Other (Not Including Serious) Adverse Events
	G-CSF Plus Plerixafor		G-CSF Plus Placebo
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	140/147 (95.2%)		140/151 (92.7%)
Blood and lymphatic system disorders
Anaemia	2/147 (1.4%)		4/151 (2.6%)
Lymphadenopathy	1/147 (0.7%)		0/151 (0%)
Lymphopenia	1/147 (0.7%)		1/151 (0.7%)
Splenomegaly	0/147 (0%)		1/151 (0.7%)
Thrombocytopenia	2/147 (1.4%)		4/151 (2.6%)
Cardiac disorders
Angina pectoris	0/147 (0%)		1/151 (0.7%)
Arrhythmia	0/147 (0%)		1/151 (0.7%)
Atrioventricular block first degree	1/147 (0.7%)		0/151 (0%)
Extrasystoles	0/147 (0%)		1/151 (0.7%)
Myocardial ischaemia	0/147 (0%)		1/151 (0.7%)
Palpitations	2/147 (1.4%)		4/151 (2.6%)
Sinus tachycardia	1/147 (0.7%)		1/151 (0.7%)
Supraventricular tachycardia	0/147 (0%)		1/151 (0.7%)
Tachycardia	2/147 (1.4%)		4/151 (2.6%)
Ventricular extrasystoles	1/147 (0.7%)		0/151 (0%)
Congenital, familial and genetic disorders
Atrial septal defect	1/147 (0.7%)		0/151 (0%)
Ear and labyrinth disorders
Deafness	1/147 (0.7%)		0/151 (0%)
Ear pain	0/147 (0%)		2/151 (1.3%)
Tinnitus	1/147 (0.7%)		0/151 (0%)
Eye disorders
Eye irritation	0/147 (0%)		1/151 (0.7%)
Eye pruritus	0/147 (0%)		1/151 (0.7%)
Ocular hyperaemia	1/147 (0.7%)		0/151 (0%)
Vision blurred	3/147 (2%)		0/151 (0%)
Gastrointestinal disorders
Abdominal distension	3/147 (2%)		5/151 (3.3%)
Abdominal mass	0/147 (0%)		1/151 (0.7%)
Abdominal pain	3/147 (2%)		3/151 (2%)
Abdominal pain upper	0/147 (0%)		4/151 (2.6%)
Constipation	9/147 (6.1%)		5/151 (3.3%)
Dental discomfort	1/147 (0.7%)		0/151 (0%)
Diarrhoea	47/147 (32%)		29/151 (19.2%)
Dry mouth	6/147 (4.1%)		6/151 (4%)
Duodenogastric reflux	0/147 (0%)		1/151 (0.7%)
Dyspepsia	2/147 (1.4%)		1/151 (0.7%)
Dysphagia	2/147 (1.4%)		1/151 (0.7%)
Eructation	1/147 (0.7%)		0/151 (0%)
Faeces discoloured	1/147 (0.7%)		0/151 (0%)
Flatulence	9/147 (6.1%)		4/151 (2.6%)
Frequent bowel movements	6/147 (4.1%)		5/151 (3.3%)
Gastritis	0/147 (0%)		1/151 (0.7%)
Gastrooesophageal reflux disease	0/147 (0%)		1/151 (0.7%)
Haemorrhoids	0/147 (0%)		1/151 (0.7%)
Hypoaesthesia oral	3/147 (2%)		1/151 (0.7%)
Localised intraabdominal fluid collection	1/147 (0.7%)		1/151 (0.7%)
Mouth ulceration	0/147 (0%)		1/151 (0.7%)
Nausea	51/147 (34.7%)		39/151 (25.8%)
Oral soft tissue disorder	0/147 (0%)		1/151 (0.7%)
Paraesthesia oral	11/147 (7.5%)		13/151 (8.6%)
Retching	1/147 (0.7%)		0/151 (0%)
Stomach discomfort	2/147 (1.4%)		1/151 (0.7%)
Stomatitis	3/147 (2%)		0/151 (0%)
Tongue haematoma	1/147 (0.7%)		0/151 (0%)
Toothache	1/147 (0.7%)		0/151 (0%)
Vomiting	17/147 (11.6%)		9/151 (6%)
General disorders
Asthenia	5/147 (3.4%)		3/151 (2%)
Catheter related complication	1/147 (0.7%)		5/151 (3.3%)
Catheter site discharge	1/147 (0.7%)		1/151 (0.7%)
Catheter site erythema	1/147 (0.7%)		4/151 (2.6%)
Catheter site haematoma	2/147 (1.4%)		0/151 (0%)
Catheter site haemorrhage	6/147 (4.1%)		6/151 (4%)
Catheter site inflammation	1/147 (0.7%)		0/151 (0%)
Catheter site oedema	0/147 (0%)		1/151 (0.7%)
Catheter site pain	14/147 (9.5%)		19/151 (12.6%)
Catheter site pruritus	1/147 (0.7%)		1/151 (0.7%)
Catheter site related reaction	7/147 (4.8%)		4/151 (2.6%)
Chest discomfort	3/147 (2%)		2/151 (1.3%)
Chest pain	2/147 (1.4%)		2/151 (1.3%)
Chills	4/147 (2.7%)		4/151 (2.6%)
Discomfort	0/147 (0%)		1/151 (0.7%)
Fatigue	40/147 (27.2%)		41/151 (27.2%)
Feeling abnormal	0/147 (0%)		1/151 (0.7%)
Feeling cold	2/147 (1.4%)		2/151 (1.3%)
Feeling hot	0/147 (0%)		1/151 (0.7%)
Gait disturbance	0/147 (0%)		1/151 (0.7%)
Influenza like illness	3/147 (2%)		1/151 (0.7%)
Infusion site mass	1/147 (0.7%)		0/151 (0%)
Infusion site thrombosis	0/147 (0%)		1/151 (0.7%)
Injection site bruising	3/147 (2%)		2/151 (1.3%)
Injection site discharge	1/147 (0.7%)		0/151 (0%)
Injection site erythema	32/147 (21.8%)		5/151 (3.3%)
Injection site haemorrhage	4/147 (2.7%)		1/151 (0.7%)
Injection site induration	1/147 (0.7%)		0/151 (0%)
Injection site irritation	3/147 (2%)		3/151 (2%)
Injection site pain	3/147 (2%)		1/151 (0.7%)
Injection site paraesthesia	1/147 (0.7%)		0/151 (0%)
Injection site pruritus	5/147 (3.4%)		1/151 (0.7%)
Injection site rash	2/147 (1.4%)		0/151 (0%)
Injection site reaction	1/147 (0.7%)		0/151 (0%)
Injection site swelling	1/147 (0.7%)		1/151 (0.7%)
Irritability	1/147 (0.7%)		0/151 (0%)
Malaise	5/147 (3.4%)		1/151 (0.7%)
Mucosal inflammation	0/147 (0%)		1/151 (0.7%)
Non-cardiac chest pain	1/147 (0.7%)		0/151 (0%)
Oedema peripheral	12/147 (8.2%)		10/151 (6.6%)
Pain	11/147 (7.5%)		11/151 (7.3%)
Peripheral coldness	0/147 (0%)		1/151 (0.7%)
Pitting oedema	0/147 (0%)		1/151 (0.7%)
Pyrexia	8/147 (5.4%)		11/151 (7.3%)
Sensation of pressure	0/147 (0%)		1/151 (0.7%)
Vessel puncture site haematoma	0/147 (0%)		1/151 (0.7%)
Infections and infestations
Bacteraemia	0/147 (0%)		1/151 (0.7%)
Bronchopneumonia	1/147 (0.7%)		0/151 (0%)
Bronchopulmonary aspergillosis	1/147 (0.7%)		0/151 (0%)
Catheter site infection	0/147 (0%)		1/151 (0.7%)
Herpes zoster	1/147 (0.7%)		0/151 (0%)
Nasopharyngitis	2/147 (1.4%)		0/151 (0%)
Oral candidiasis	1/147 (0.7%)		0/151 (0%)
Pneumonia fungal	1/147 (0.7%)		0/151 (0%)
Respiratory tract infection	0/147 (0%)		1/151 (0.7%)
Rhinitis	1/147 (0.7%)		0/151 (0%)
Sinusitis	1/147 (0.7%)		0/151 (0%)
Tinea manuum	0/147 (0%)		1/151 (0.7%)
Tinea pedis	1/147 (0.7%)		0/151 (0%)
Tooth abscess	0/147 (0%)		1/151 (0.7%)
Upper respiratory tract infection	3/147 (2%)		4/151 (2.6%)
Urinary tract infection	0/147 (0%)		2/151 (1.3%)
Injury, poisoning and procedural complications
Arthropod bite	1/147 (0.7%)		0/151 (0%)
Citrate toxicity	6/147 (4.1%)		4/151 (2.6%)
Contusion	4/147 (2.7%)		5/151 (3.3%)
Post procedural discomfort	0/147 (0%)		1/151 (0.7%)
Procedural hypertension	1/147 (0.7%)		0/151 (0%)
Procedural nausea	1/147 (0.7%)		0/151 (0%)
Procedural pain	1/147 (0.7%)		1/151 (0.7%)
Skin laceration	0/147 (0%)		1/151 (0.7%)
Tooth fracture	0/147 (0%)		1/151 (0.7%)
Tooth injury	1/147 (0.7%)		0/151 (0%)
Investigations
Aspartate aminotransferase increased	1/147 (0.7%)		0/151 (0%)
Blood alkaline phosphatase increased	3/147 (2%)		3/151 (2%)
Blood calcium decreased	0/147 (0%)		1/151 (0.7%)
Blood glucose decreased	0/147 (0%)		1/151 (0.7%)
Blood magnesium decreased	1/147 (0.7%)		1/151 (0.7%)
Blood potassium decreased	2/147 (1.4%)		1/151 (0.7%)
Blood pressure increased	0/147 (0%)		1/151 (0.7%)
Blood uric acid increased	6/147 (4.1%)		6/151 (4%)
Body temperature increased	1/147 (0.7%)		0/151 (0%)
C-reactive protein increased	1/147 (0.7%)		0/151 (0%)
Cardiac murmur	1/147 (0.7%)		0/151 (0%)
Culture positive	0/147 (0%)		1/151 (0.7%)
Haemoglobin decreased	0/147 (0%)		1/151 (0.7%)
Heart rate increased	0/147 (0%)		1/151 (0.7%)
Heart rate irregular	1/147 (0.7%)		4/151 (2.6%)
Lymph node palpable	1/147 (0.7%)		0/151 (0%)
Oxygen saturation decreased	0/147 (0%)		2/151 (1.3%)
Platelet count decreased	0/147 (0%)		1/151 (0.7%)
Weight decreased	1/147 (0.7%)		0/151 (0%)
Weight increased	1/147 (0.7%)		1/151 (0.7%)
Metabolism and nutrition disorders
Anorexia	6/147 (4.1%)		6/151 (4%)
Decreased appetite	6/147 (4.1%)		5/151 (3.3%)
Hyperuricaemia	0/147 (0%)		3/151 (2%)
Hypocalcaemia	6/147 (4.1%)		6/151 (4%)
Hypokalaemia	19/147 (12.9%)		29/151 (19.2%)
Hypomagnesaemia	9/147 (6.1%)		16/151 (10.6%)
Hyponatraemia	1/147 (0.7%)		0/151 (0%)
Hypophosphataemia	0/147 (0%)		1/151 (0.7%)
Tetany	1/147 (0.7%)		0/151 (0%)
Musculoskeletal and connective tissue disorders
Arthralgia	16/147 (10.9%)		17/151 (11.3%)
Back pain	23/147 (15.6%)		34/151 (22.5%)
Bone pain	53/147 (36.1%)		64/151 (42.4%)
Flank pain	0/147 (0%)		1/151 (0.7%)
Jaw disorder	0/147 (0%)		1/151 (0.7%)
Joint swelling	1/147 (0.7%)		0/151 (0%)
Muscle disorder	1/147 (0.7%)		0/151 (0%)
Muscle spasms	5/147 (3.4%)		8/151 (5.3%)
Muscle twitching	1/147 (0.7%)		0/151 (0%)
Muscular weakness	1/147 (0.7%)		0/151 (0%)
Musculoskeletal chest pain	8/147 (5.4%)		5/151 (3.3%)
Musculoskeletal discomfort	0/147 (0%)		1/151 (0.7%)
Musculoskeletal pain	7/147 (4.8%)		5/151 (3.3%)
Musculoskeletal stiffness	3/147 (2%)		2/151 (1.3%)
Myalgia	2/147 (1.4%)		0/151 (0%)
Neck pain	5/147 (3.4%)		0/151 (0%)
Pain in extremity	8/147 (5.4%)		11/151 (7.3%)
Pain in jaw	1/147 (0.7%)		0/151 (0%)
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Bone neoplasm malignant	1/147 (0.7%)		0/151 (0%)
Lentigo	0/147 (0%)		1/151 (0.7%)
Prostate cancer	1/147 (0.7%)		0/151 (0%)
Nervous system disorders
Balance disorder	1/147 (0.7%)		1/151 (0.7%)
Burning sensation	1/147 (0.7%)		0/151 (0%)
Coordination abnormal	1/147 (0.7%)		0/151 (0%)
Dizziness	17/147 (11.6%)		10/151 (6.6%)
Dysgeusia	7/147 (4.8%)		2/151 (1.3%)
Dyskinesia	0/147 (0%)		1/151 (0.7%)
Headache	30/147 (20.4%)		35/151 (23.2%)
Hypoaesthesia	8/147 (5.4%)		7/151 (4.6%)
Lethargy	0/147 (0%)		1/151 (0.7%)
Neuropathy peripheral	0/147 (0%)		1/151 (0.7%)
Paraesthesia	33/147 (22.4%)		34/151 (22.5%)
Parosmia	1/147 (0.7%)		0/151 (0%)
Restless legs syndrome	2/147 (1.4%)		2/151 (1.3%)
Sensory disturbance	1/147 (0.7%)		0/151 (0%)
Sinus headache	0/147 (0%)		1/151 (0.7%)
Somnolence	0/147 (0%)		1/151 (0.7%)
Tremor	3/147 (2%)		3/151 (2%)
Psychiatric disorders
Abnormal dreams	0/147 (0%)		1/151 (0.7%)
Anticipatory anxiety	1/147 (0.7%)		0/151 (0%)
Anxiety	9/147 (6.1%)		6/151 (4%)
Confusional state	1/147 (0.7%)		0/151 (0%)
Depression	1/147 (0.7%)		1/151 (0.7%)
Emotional distress	0/147 (0%)		1/151 (0.7%)
Insomnia	10/147 (6.8%)		11/151 (7.3%)
Restlessness	2/147 (1.4%)		0/151 (0%)
Renal and urinary disorders
Haematuria	0/147 (0%)		2/151 (1.3%)
Nocturia	1/147 (0.7%)		0/151 (0%)
Pollakiuria	2/147 (1.4%)		1/151 (0.7%)
Proteinuria	1/147 (0.7%)		0/151 (0%)
Reproductive system and breast disorders
Pelvic pain	0/147 (0%)		1/151 (0.7%)
Vaginal pain	0/147 (0%)		1/151 (0.7%)
Respiratory, thoracic and mediastinal disorders
Cough	1/147 (0.7%)		3/151 (2%)
Dyspnoea	3/147 (2%)		4/151 (2.6%)
Dyspnoea exertional	2/147 (1.4%)		4/151 (2.6%)
Nasal congestion	1/147 (0.7%)		0/151 (0%)
Nasal mucosal disorder	1/147 (0.7%)		0/151 (0%)
Paranasal sinus hypersecretion	0/147 (0%)		3/151 (2%)
Pharyngolaryngeal pain	3/147 (2%)		6/151 (4%)
Postnasal drip	1/147 (0.7%)		1/151 (0.7%)
Productive cough	1/147 (0.7%)		1/151 (0.7%)
Rhinorrhoea	1/147 (0.7%)		2/151 (1.3%)
Rhonchi	1/147 (0.7%)		0/151 (0%)
Sinus congestion	2/147 (1.4%)		0/151 (0%)
Wheezing	0/147 (0%)		1/151 (0.7%)
Skin and subcutaneous tissue disorders
Actinic keratosis	1/147 (0.7%)		0/151 (0%)
Alopecia	1/147 (0.7%)		0/151 (0%)
Cold sweat	2/147 (1.4%)		0/151 (0%)
Dermatitis contact	1/147 (0.7%)		1/151 (0.7%)
Dry skin	1/147 (0.7%)		2/151 (1.3%)
Ecchymosis	2/147 (1.4%)		2/151 (1.3%)
Eczema	1/147 (0.7%)		0/151 (0%)
Ephelides	1/147 (0.7%)		0/151 (0%)
Erythema	3/147 (2%)		1/151 (0.7%)
Erythema nodosum	0/147 (0%)		1/151 (0.7%)
Hyperhidrosis	4/147 (2.7%)		5/151 (3.3%)
Hypoaesthesia facial	5/147 (3.4%)		0/151 (0%)
Ingrowing nail	0/147 (0%)		1/151 (0.7%)
Night sweats	4/147 (2.7%)		2/151 (1.3%)
Pruritus	4/147 (2.7%)		2/151 (1.3%)
Rash	3/147 (2%)		2/151 (1.3%)
Rash generalised	0/147 (0%)		1/151 (0.7%)
Scar	0/147 (0%)		1/151 (0.7%)
Skin irritation	4/147 (2.7%)		1/151 (0.7%)
Skin lesion	0/147 (0%)		1/151 (0.7%)
Vascular disorders
Flushing	2/147 (1.4%)		1/151 (0.7%)
Hot flush	2/147 (1.4%)		1/151 (0.7%)
Hypertension	2/147 (1.4%)		5/151 (3.3%)
Hypotension	2/147 (1.4%)		5/151 (3.3%)
Orthostatic hypotension	1/147 (0.7%)		0/151 (0%)
Pallor	0/147 (0%)		1/151 (0.7%)

Limitations/Caveats

[Not Specified]

More Information

Certain Agreements

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

In multi-site studies, PI can publish after Genzyme publishes or 18 months after study completion. PI gives Genzyme a draft 60 days before publication. Genzyme can ask that confidential information be removed, and can defer publication another 60 days upon notifying PI that it will file a patent application on inventions contained in the draft.