A Phase I/II Trial of Pomalidomide and Dexamethasone in Subjects With Previously-Treated AL Amyloidosis
Study Details
Study Description
Brief Summary
This study seeks to enroll patients with AL amyloidosis, for whom treatment with one of the standard melphalan chemotherapy-based regimens is either not recommended or is not their preference.
Pomalidomide (CC-4047) is a drug given by mouth, which can change or regulate the functioning of the immune system. So, in theory, it may reduce or prevent the production of the amyloid protein. Pomalidomide is not currently FDA-approved for AL Amyloidosis. Pomalidomide is chemically similar to thalidomide and lenalidomide, both of these drugs have been approved by the FDA for treatment of patients with multiple myeloma (MM), a disease similar to AL Amyloidosis.
Participants in this study will receive pomalidomide and dexamethasone. Phase I is a dose-escalation study and dose escalation will proceed through 3 dose-levels according to standard rules in which dose levels are started sequentially after complete evaluation of the occurrence of dose-limiting toxicities. In the Phase II portion, participants will receive pomalidomide and dexamethasone using the defined maximum tolerated dose.
Condition or Disease | Intervention/Treatment | Phase |
---|---|---|
Phase 1/Phase 2 |
Detailed Description
Primary objective:
Determine dose-limiting toxicity (DLT) and the maximal tolerated dose (MTD) of pomalidomide combined with dexamethasone in subjects with previously- treated light-chain (AL)-amyloidosis
Secondary objectives:
Determine the following at the MTD:
-
Hematological complete (CR) very good partial (VGPR) and partial (PR) rates
-
duration of response
-
organ response
-
Time-to-event
-
Survival
Exploratory study objective:
To investigate the relationship of changes in the levels of the biomarkers B-type natriuretic peptide (BNP) and troponin I to frequency of specific adverse events and the occurrence of DLT
Study Design
Arms and Interventions
Arm | Intervention/Treatment |
---|---|
Experimental: Phase I - cohort 1 (Pomalidomide 2mg) plus Dexamethasone Pomalidomide 2 mg/day on days 1-28 plus dexamethasone 10-20 mg on days 1-21 of a 28 day cycle |
Drug: Pomalidomide
Cohort 1 = 2 mg/day, Cohort 2 = 3 mg/day, Cohort 3 = 4 mg/day: Days 1-21 of a 28 day cycle
Other Names:
Drug: Dexamethasone
10-20 mg on days 1, 8, 15, and 22
Other Names:
|
Experimental: Phase I - cohort 2 (Pomalidomide 3mg) plus Dexamethasone Pomalidomide 3 mg/day on days 1-28 plus dexamethasone 10-20 mg on days 1-21 of a 28 day cycle |
Drug: Pomalidomide
Cohort 1 = 2 mg/day, Cohort 2 = 3 mg/day, Cohort 3 = 4 mg/day: Days 1-21 of a 28 day cycle
Other Names:
Drug: Dexamethasone
10-20 mg on days 1, 8, 15, and 22
Other Names:
|
Experimental: Phase I - cohort 3 (Pomalidomide 4mg) plus Dexamethasone Pomalidomide 4 mg/day on days 1-28 plus dexamethasone 10-20 mg on days 1-21 of a 28 day cycle |
Drug: Pomalidomide
Cohort 1 = 2 mg/day, Cohort 2 = 3 mg/day, Cohort 3 = 4 mg/day: Days 1-21 of a 28 day cycle
Other Names:
Drug: Dexamethasone
10-20 mg on days 1, 8, 15, and 22
Other Names:
|
Experimental: Phase II Expansion- (Pomalidomide 4mg) plus Dexamethasone Expansion Phase: Pomalidomide 4 mg/day on days 1-28 plus dexamethasone 10-20 mg on days 1-21 of a 28 day cycle |
Drug: Pomalidomide
Cohort 1 = 2 mg/day, Cohort 2 = 3 mg/day, Cohort 3 = 4 mg/day: Days 1-21 of a 28 day cycle
Other Names:
Drug: Dexamethasone
10-20 mg on days 1, 8, 15, and 22
Other Names:
|
Outcome Measures
Primary Outcome Measures
- Assessing Dose-limiting Toxicity to Determine Maximal Tolerated Dosage at 2 Milligram Dose [one month]
Number of patients in Phase I cohort 1 experiencing dose-limiting toxicity at the 2 milligram dose of pomalidomide combined with dexamethasone in subjects with previously- treated light-chain amyloidosis
- Assessing Dose-limiting Toxicity to Determine Maximal Tolerated Dosage at 3 Milligram Dose [One month]
Number of patients in Phase I cohort 2 experiencing dose limiting toxicity in the 3 milligram dose level, cohort 2.
- Assessing Dose-limiting Toxicity to Determine Maximal Tolerated Dosage at 4 Milligram Dose [One month]
Number of patients in Phase I cohort 3 experiencing dose-limiting toxicity at the 4 milligram dose for participants within the third dose cohort
Secondary Outcome Measures
- Response to the Maximal Tolerated Dose [one year]
Number of participants with a response to treatment at that maximal tolerated dose (including partial, very good, or complete responses)
Eligibility Criteria
Criteria
Inclusion Criteria:
-
Understand and voluntarily sign informed consent form.
-
≥18yrs old
-
Able to adhere to the study visit schedule and other protocol requirements.
-
Biopsy proven tissue amyloid deposits or positive fat aspirate
-
Proof of AL type (a or b)
-
Measurable plasma cell dyscrasia (a or b and c of the following required):
-
Monoclonal protein in the serum or urine by immunofixation electrophoresis
-
Plasmacytosis of bone marrow (<30% plasma cells) with monoclonal staining for kappa or lambda light-chain isotype
-
dFLC of 50mg/L (dFLC=difference in involved and uninvolved serum free light-chain levels)
-
Must have received ≥1 prior treatment for AL amyloidosis, if it is intensive chemotherapy and an autotransplant it must be ≥6 months prior to enrollment on this study
-
Must have recovered from the reversible side effects of any prior therapy; permanent and stable side effects/changes are acceptable. Prior treatment for AL amyloidosis with chemotherapy, thalidomide, lenalidomide or steroids is not an exclusion
-
Eastern Cooperative Group (ECOG) performance status ≤2 at study entry
-
Lab test results within these ranges:
- Neutrophil ≥1.5 x10e9/L e. Platelets ≥100x10e9/L f. Total bilirubin <1.5mg/dL g. Aspartate aminotransferase (AST or SGOT) and Alanine Aminotransferase (ALT or SGPT) < 2 x Upper limit of normal h. Serum creatinine <2.5mg/dL
-
Disease free of prior malignancies for at least 5yrs with exception of currently treated basal cell, squamous cell carcinoma of the skin, or carcinoma "in-situ" of the cervix or breast.
-
Females of childbearing potential (FCBP) (a FCBP is a sexually mature woman who has not undergone a hysterectomy or bilateral oophorectomy, or has not been naturally postmenopausal for at least 24 consecutive months) must have a negative serum or urine pregnancy test with a sensitivity ≥ 50 milli-International unit/mL 10-14 days prior to and again ≤ 24 hours of starting pomalidomide and must either commit to continued abstinence from heterosexual intercourse or begin two (2) acceptable methods of birth control, one highly effective method and one additional effective method AT THE SAME TIME, ≥ 28 days before she starts taking pomalidomide. FCBP must also agree to ongoing pregnancy testing. Men must agree to use a latex condom during sexual contact with a FCBP even if they have had a vasectomy. All subjects must be counseled at a minimum of every 28 days about pregnancy precautions and risks of fetal exposure.
-
Able to take aspirin (81 or 325 mg) daily as prophylactic anticoagulation (subjects intolerant to aspirin may use warfarin or low molecular weight heparin).
Exclusion Criteria:
-
Secondary or familial amyloidosis
-
Multiple myeloma (≥30% plasma cells in a bone marrow biopsy specimen or lytic bone lesions)
-
Cytotoxic chemo or radiation therapy ≤4 weeks of study entry or following baseline evaluation
-
Symptomatic cardiac arrhythmias or O2-dependent restrictive cardiomyopathy
-
Dialysis-dependent
-
Untreated or uncontrolled infections.
-
Serious medical conditions, laboratory abnormality, or psychiatric illness that would prevent the subject from signing the informed consent form.
-
Pregnant or breast feeding females (lactating females must agree not to breast feed while taking pomalidomide).
-
Any condition, including the presence of laboratory abnormalities, which places the subject at unacceptable risk if he/she were to participate in the study or confounds the ability to interpret data from the study.
-
Use of any other experimental drug or therapy within 28 days of baseline.
-
Known intolerance to steroids.
-
Known hypersensitivity to thalidomide or lenalidomide
-
The development of erythema nodosum if characterized by a desquamating rash while taking thalidomide or similar drugs.
-
Concurrent use of other anti-cancer agents or treatments.
-
Known HIV positivity is not an exclusion, unless cluster of differentiation 4 (CD4) counts <200/microliter and/or patient has multi-drug resistant HIV infections and/or other concurrent AIDS-defining conditions. HIV b-DNA < 75 copies/mL.
Contacts and Locations
Locations
Site | City | State | Country | Postal Code | |
---|---|---|---|---|---|
1 | Boston Medical Center | Boston | Massachusetts | United States | 02118 |
Sponsors and Collaborators
- Boston Medical Center
- Celgene Corporation
Investigators
- Principal Investigator: Vaishali Sanchorawala, MD, Boston Medical Center
Study Documents (Full-Text)
More Information
Publications
None provided.- H-31082
- PO-AMYL-PI-0024
Study Results
Participant Flow
Recruitment Details | Protocol was open to accrual between January 2012 and August 2016. Participants were recruited from within the amyloidosis clinic within Boston Medical Center. |
---|---|
Pre-assignment Detail |
Arm/Group Title | Experimental: Phase I - Cohort 1 (Pomalidomide 2mg) | Experimental: Phase I - Cohort 2 (Pomalidomide 3mg) | Experimental: Phase I - Cohort 3 (Pomalidomide 4mg) | Experimental: Phase II Expansion- Maximum Tolerated Dose |
---|---|---|---|---|
Arm/Group Description | Drug: Pomalidomide Cohort 1 = 2 mg/day, Days 1-28 Drug: Dexamethasone 10-20 mg on days 1, 8, 15, and 22 | Drug: Pomalidomide Cohort 12= 3 mg/day, Days 1-28 Drug: Dexamethasone 10-20 mg on days 1, 8, 15, and 22 | Drug: Pomalidomide Cohort 3 = 4 mg/day, Days 1-28 Drug: Dexamethasone 10-20 mg on days 1, 8, 15, and 22 | Drug: Pomalidomide Expansion Phase - Maximum Tolerated Dose = 4 mg/day, Days 1-28 Drug: Dexamethasone 10-20 mg on days 1, 8, 15, and 22 |
Period Title: Overall Study | ||||
STARTED | 6 | 3 | 6 | 12 |
Dose Limiting Toxicity | 0 | 0 | 6 | 12 |
Response to Maximal Tolerated Dose | 0 | 0 | 3 | 6 |
COMPLETED | 5 | 3 | 6 | 10 |
NOT COMPLETED | 1 | 0 | 0 | 2 |
Baseline Characteristics
Arm/Group Title | Phase I - Cohort 1 (Pomalidomide 2mg) Plus Dexam | Phase I - Cohort 2 (Pomalidomide 3mg) | Phase I - Cohort 3 (Pomalidomide 4mg) | Phase II Expansion- (Pomalidomide 4mg) Plus Dexa | Total |
---|---|---|---|---|---|
Arm/Group Description | Pomalidomide: Cohort 1 = 2 mg/day, Cohort 2 = 3 mg/day, Cohort 3 = 4 mg/day: Days 1-28 Dexamethasone: 10-20 mg on days 1, 8, 15, and 22 | Pomalidomide 3 mg/day on days 1-28 plus dexamethasone 10-20 mg on days 1-21 of a 28 day cycle | Pomalidomide 4 mg/day on days 1-28 plus dexamethasone 10-20 mg on days 1-21 of a 28 day cycle | Pomalidomide 4 mg/day (MTD) on days 1-28 plus dexamethasone 10-20 mg on days 1-21 of a 28 day cycle | Total of all reporting groups |
Overall Participants | 6 | 3 | 6 | 12 | 27 |
Age (Count of Participants) | |||||
<=18 years |
0
0%
|
0
0%
|
0
0%
|
0
0%
|
0
0%
|
Between 18 and 65 years |
3
50%
|
0
0%
|
1
16.7%
|
5
41.7%
|
9
33.3%
|
>=65 years |
3
50%
|
3
100%
|
5
83.3%
|
7
58.3%
|
18
66.7%
|
Sex: Female, Male (Count of Participants) | |||||
Female |
4
66.7%
|
1
33.3%
|
1
16.7%
|
5
41.7%
|
11
40.7%
|
Male |
2
33.3%
|
2
66.7%
|
5
83.3%
|
7
58.3%
|
16
59.3%
|
Ethnicity (NIH/OMB) (Count of Participants) | |||||
Hispanic or Latino |
0
0%
|
0
0%
|
0
0%
|
1
8.3%
|
1
3.7%
|
Not Hispanic or Latino |
6
100%
|
3
100%
|
6
100%
|
11
91.7%
|
26
96.3%
|
Unknown or Not Reported |
0
0%
|
0
0%
|
0
0%
|
0
0%
|
0
0%
|
Race (NIH/OMB) (Count of Participants) | |||||
American Indian or Alaska Native |
0
0%
|
0
0%
|
0
0%
|
0
0%
|
0
0%
|
Asian |
0
0%
|
1
33.3%
|
1
16.7%
|
0
0%
|
2
7.4%
|
Native Hawaiian or Other Pacific Islander |
0
0%
|
0
0%
|
0
0%
|
0
0%
|
0
0%
|
Black or African American |
0
0%
|
0
0%
|
0
0%
|
1
8.3%
|
1
3.7%
|
White |
6
100%
|
2
66.7%
|
5
83.3%
|
11
91.7%
|
24
88.9%
|
More than one race |
0
0%
|
0
0%
|
0
0%
|
0
0%
|
0
0%
|
Unknown or Not Reported |
0
0%
|
0
0%
|
0
0%
|
0
0%
|
0
0%
|
Prior treatments (Count of Participants) | |||||
1 to 2 prior treatments |
4
66.7%
|
3
100%
|
1
16.7%
|
8
66.7%
|
16
59.3%
|
greater than 2 prior treatments |
2
33.3%
|
0
0%
|
5
83.3%
|
4
33.3%
|
11
40.7%
|
Outcome Measures
Title | Assessing Dose-limiting Toxicity to Determine Maximal Tolerated Dosage at 2 Milligram Dose |
---|---|
Description | Number of patients in Phase I cohort 1 experiencing dose-limiting toxicity at the 2 milligram dose of pomalidomide combined with dexamethasone in subjects with previously- treated light-chain amyloidosis |
Time Frame | one month |
Outcome Measure Data
Analysis Population Description |
---|
[Not Specified] |
Arm/Group Title | Pom Plus Dex |
---|---|
Arm/Group Description | Pomalidomide dexamethasone Pomalidomide: Cohort 1 = 2 mg/day, Cohort 2 = 3 mg/day, Cohort 3 = 4 mg/day: Days 1-28 Dexamethasone: 10-20 mg on days 1, 8, 15, and 22 |
Measure Participants | 6 |
Count of Participants [Participants] |
0
0%
|
Title | Assessing Dose-limiting Toxicity to Determine Maximal Tolerated Dosage at 3 Milligram Dose |
---|---|
Description | Number of patients in Phase I cohort 2 experiencing dose limiting toxicity in the 3 milligram dose level, cohort 2. |
Time Frame | One month |
Outcome Measure Data
Analysis Population Description |
---|
[Not Specified] |
Arm/Group Title | Pom Plus Dex |
---|---|
Arm/Group Description | Pomalidomide dexamethasone Pomalidomide: Cohort 1 = 2 mg/day, Cohort 2 = 3 mg/day, Cohort 3 = 4 mg/day: Days 1-28 Dexamethasone: 10-20 mg on days 1, 8, 15, and 22 |
Measure Participants | 3 |
Count of Participants [Participants] |
0
0%
|
Title | Assessing Dose-limiting Toxicity to Determine Maximal Tolerated Dosage at 4 Milligram Dose |
---|---|
Description | Number of patients in Phase I cohort 3 experiencing dose-limiting toxicity at the 4 milligram dose for participants within the third dose cohort |
Time Frame | One month |
Outcome Measure Data
Analysis Population Description |
---|
[Not Specified] |
Arm/Group Title | Pom Plus Dex |
---|---|
Arm/Group Description | Pomalidomide dexamethasone Pomalidomide: Cohort 1 = 2 mg/day, Cohort 2 = 3 mg/day, Cohort 3 = 4 mg/day: Days 1-28 Dexamethasone: 10-20 mg on days 1, 8, 15, and 22 |
Measure Participants | 6 |
Count of Participants [Participants] |
1
16.7%
|
Title | Response to the Maximal Tolerated Dose |
---|---|
Description | Number of participants with a response to treatment at that maximal tolerated dose (including partial, very good, or complete responses) |
Time Frame | one year |
Outcome Measure Data
Analysis Population Description |
---|
Number of evaluable patients treated at the maximal tolerated dose of 4mg including the Phase I cohort 3 participants and the Phase II expansion participants |
Arm/Group Title | Pom Plus Dex |
---|---|
Arm/Group Description | Pomalidomide dexamethasone Pomalidomide: Cohort 1 = 2 mg/day, Cohort 2 = 3 mg/day, Cohort 3 = 4 mg/day: Days 1-28 Dexamethasone: 10-20 mg on days 1, 8, 15, and 22 |
Measure Participants | 18 |
Count of Participants [Participants] |
10
166.7%
|
Adverse Events
Time Frame | 6 years | |||||||
---|---|---|---|---|---|---|---|---|
Adverse Event Reporting Description | Adverse events for each participant were assessed at all visits while treatment continued through 30 days post last dose. 6 years represents the time from first participant initiating treatment though last participant completing treatment. | |||||||
Arm/Group Title | Phase I - Cohort 1 (Pomalidomide 2mg) | Phase I - Cohort 2 (Pomalidomide 3mg) | Phase I - Cohort 3 (Pomalidomide 4mg) | Phase II Expansion- Maximum Tolerated Dose | ||||
Arm/Group Description | Drug: Pomalidomide Cohort 1 = 2 mg/day, Days 1-28 Drug: Dexamethasone 10-20 mg on days 1, 8, 15, and 22 | Drug: Pomalidomide Cohort 2 = 3 mg/day, Days 1-28 Drug: Dexamethasone 10-20 mg on days 1, 8, 15, and 22 | Drug: Pomalidomide Cohort 3 = 4 mg/day, Days 1-28 Drug: Dexamethasone 10-20 mg on days 1, 8, 15, and 22 | Drug: Pomalidomide Maximum Tolerated Dose = 4 mg/day, Days 1-28 Drug: Dexamethasone 10-20 mg on days 1, 8, 15, and 22 | ||||
All Cause Mortality |
||||||||
Phase I - Cohort 1 (Pomalidomide 2mg) | Phase I - Cohort 2 (Pomalidomide 3mg) | Phase I - Cohort 3 (Pomalidomide 4mg) | Phase II Expansion- Maximum Tolerated Dose | |||||
Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
Total | 1/6 (16.7%) | 0/3 (0%) | 0/6 (0%) | 1/12 (8.3%) | ||||
Serious Adverse Events |
||||||||
Phase I - Cohort 1 (Pomalidomide 2mg) | Phase I - Cohort 2 (Pomalidomide 3mg) | Phase I - Cohort 3 (Pomalidomide 4mg) | Phase II Expansion- Maximum Tolerated Dose | |||||
Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
Total | 2/6 (33.3%) | 0/3 (0%) | 3/6 (50%) | 3/12 (25%) | ||||
Cardiac disorders | ||||||||
myocardial infarction | 1/6 (16.7%) | 1 | 0/3 (0%) | 0 | 0/6 (0%) | 0 | 0/12 (0%) | 0 |
Infections and infestations | ||||||||
Upper respiratory infection | 1/6 (16.7%) | 1 | 0/3 (0%) | 0 | 1/6 (16.7%) | 1 | 2/12 (16.7%) | 2 |
sepsis | 1/6 (16.7%) | 1 | 0/3 (0%) | 0 | 2/6 (33.3%) | 2 | 2/12 (16.7%) | 2 |
Other (Not Including Serious) Adverse Events |
||||||||
Phase I - Cohort 1 (Pomalidomide 2mg) | Phase I - Cohort 2 (Pomalidomide 3mg) | Phase I - Cohort 3 (Pomalidomide 4mg) | Phase II Expansion- Maximum Tolerated Dose | |||||
Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
Total | 6/6 (100%) | 3/3 (100%) | 6/6 (100%) | 12/12 (100%) | ||||
Cardiac disorders | ||||||||
palpitations | 2/6 (33.3%) | 2 | 1/3 (33.3%) | 1 | 0/6 (0%) | 0 | 1/12 (8.3%) | 1 |
heart failure | 1/6 (16.7%) | 1 | 0/3 (0%) | 0 | 2/6 (33.3%) | 2 | 0/12 (0%) | 0 |
chest pain | 1/6 (16.7%) | 1 | 0/3 (0%) | 0 | 0/6 (0%) | 0 | 1/12 (8.3%) | 1 |
Ear and labyrinth disorders | ||||||||
tinnitus | 0/6 (0%) | 0 | 1/3 (33.3%) | 1 | 0/6 (0%) | 0 | 2/12 (16.7%) | 2 |
hearing impaired | 0/6 (0%) | 0 | 1/3 (33.3%) | 1 | 1/6 (16.7%) | 1 | 2/12 (16.7%) | 2 |
Endocrine disorders | ||||||||
hyperthyroidism | 0/6 (0%) | 0 | 0/3 (0%) | 0 | 2/6 (33.3%) | 2 | 1/12 (8.3%) | 1 |
Eye disorders | ||||||||
visual blurring | 4/6 (66.7%) | 4 | 0/3 (0%) | 0 | 1/6 (16.7%) | 1 | 3/12 (25%) | 3 |
Gastrointestinal disorders | ||||||||
vomiting | 1/6 (16.7%) | 1 | 0/3 (0%) | 0 | 3/6 (50%) | 3 | 2/12 (16.7%) | 2 |
abdominal pain | 0/6 (0%) | 0 | 0/3 (0%) | 0 | 2/6 (33.3%) | 2 | 5/12 (41.7%) | 6 |
nausea | 2/6 (33.3%) | 2 | 2/3 (66.7%) | 3 | 3/6 (50%) | 5 | 4/12 (33.3%) | 4 |
diarrhea | 4/6 (66.7%) | 4 | 1/3 (33.3%) | 1 | 3/6 (50%) | 5 | 3/12 (25%) | 4 |
constipation | 4/6 (66.7%) | 5 | 3/3 (100%) | 3 | 3/6 (50%) | 3 | 4/12 (33.3%) | 4 |
abdominal bloating | 2/6 (33.3%) | 2 | 0/3 (0%) | 0 | 0/6 (0%) | 0 | 0/12 (0%) | 0 |
hemorrhoids | 1/6 (16.7%) | 1 | 1/3 (33.3%) | 1 | 1/6 (16.7%) | 1 | 2/12 (16.7%) | 2 |
dyspepsia | 0/6 (0%) | 0 | 1/3 (33.3%) | 1 | 1/6 (16.7%) | 1 | 0/12 (0%) | 0 |
dry mouth | 0/6 (0%) | 0 | 0/3 (0%) | 0 | 1/6 (16.7%) | 1 | 1/12 (8.3%) | 1 |
General disorders | ||||||||
weakness | 3/6 (50%) | 3 | 1/3 (33.3%) | 1 | 3/6 (50%) | 4 | 4/12 (33.3%) | 5 |
fever | 1/6 (16.7%) | 1 | 0/3 (0%) | 0 | 5/6 (83.3%) | 5 | 2/12 (16.7%) | 2 |
fatigue | 3/6 (50%) | 4 | 2/3 (66.7%) | 2 | 4/6 (66.7%) | 8 | 8/12 (66.7%) | 10 |
edema - limbs | 0/6 (0%) | 0 | 0/3 (0%) | 0 | 2/6 (33.3%) | 2 | 7/12 (58.3%) | 9 |
chills | 0/6 (0%) | 0 | 0/3 (0%) | 0 | 2/6 (33.3%) | 2 | 0/12 (0%) | 0 |
Infections and infestations | ||||||||
upper respiratory infection | 2/6 (33.3%) | 3 | 1/3 (33.3%) | 2 | 4/6 (66.7%) | 6 | 3/12 (25%) | 4 |
Injury, poisoning and procedural complications | ||||||||
bruising | 0/6 (0%) | 0 | 0/3 (0%) | 0 | 2/6 (33.3%) | 2 | 1/12 (8.3%) | 2 |
Investigations | ||||||||
White blood cell count decreased | 3/6 (50%) | 3 | 0/3 (0%) | 0 | 1/6 (16.7%) | 4 | 10/12 (83.3%) | 15 |
platelet count decreased | 2/6 (33.3%) | 5 | 0/3 (0%) | 0 | 2/6 (33.3%) | 2 | 4/12 (33.3%) | 9 |
neutrophil count decreased | 1/6 (16.7%) | 1 | 1/3 (33.3%) | 1 | 6/6 (100%) | 9 | 9/12 (75%) | 16 |
lymphocyte count decreased | 0/6 (0%) | 0 | 0/3 (0%) | 0 | 5/6 (83.3%) | 18 | 5/12 (41.7%) | 9 |
leukocyte count decreased | 0/6 (0%) | 0 | 1/3 (33.3%) | 1 | 4/6 (66.7%) | 5 | 1/12 (8.3%) | 1 |
creatinine elevated | 4/6 (66.7%) | 8 | 1/3 (33.3%) | 1 | 5/6 (83.3%) | 8 | 5/12 (41.7%) | 10 |
Blood bilirubin increased | 0/6 (0%) | 0 | 0/3 (0%) | 0 | 1/6 (16.7%) | 1 | 3/12 (25%) | 5 |
anemia | 5/6 (83.3%) | 8 | 1/3 (33.3%) | 2 | 4/6 (66.7%) | 15 | 6/12 (50%) | 8 |
alkaline phosphatase increased | 1/6 (16.7%) | 1 | 1/3 (33.3%) | 1 | 2/6 (33.3%) | 2 | 3/12 (25%) | 5 |
hypokalemia | 1/6 (16.7%) | 2 | 0/3 (0%) | 0 | 2/6 (33.3%) | 2 | 0/12 (0%) | 0 |
aspartate aminotransferase increased | 5/6 (83.3%) | 5 | 0/3 (0%) | 0 | 2/6 (33.3%) | 2 | 4/12 (33.3%) | 4 |
Metabolism and nutrition disorders | ||||||||
hyperuricemia | 0/6 (0%) | 0 | 1/3 (33.3%) | 1 | 3/6 (50%) | 12 | 5/12 (41.7%) | 6 |
hypertriglyceridemia | 0/6 (0%) | 0 | 1/3 (33.3%) | 1 | 2/6 (33.3%) | 4 | 2/12 (16.7%) | 2 |
hypophosphatemia | 1/6 (16.7%) | 1 | 0/3 (0%) | 0 | 2/6 (33.3%) | 4 | 3/12 (25%) | 9 |
hyponatremia | 3/6 (50%) | 9 | 0/3 (0%) | 0 | 2/6 (33.3%) | 2 | 4/12 (33.3%) | 4 |
hypoalbuminemia | 2/6 (33.3%) | 3 | 1/3 (33.3%) | 1 | 5/6 (83.3%) | 7 | 2/12 (16.7%) | 3 |
hyperkalemia | 3/6 (50%) | 6 | 0/3 (0%) | 0 | 3/6 (50%) | 4 | 1/12 (8.3%) | 1 |
hyperglycemia | 0/6 (0%) | 0 | 1/3 (33.3%) | 1 | 6/6 (100%) | 12 | 2/12 (16.7%) | 4 |
anorexia | 1/6 (16.7%) | 1 | 1/3 (33.3%) | 1 | 2/6 (33.3%) | 2 | 3/12 (25%) | 3 |
Musculoskeletal and connective tissue disorders | ||||||||
back pain | 1/6 (16.7%) | 1 | 1/3 (33.3%) | 1 | 3/6 (50%) | 3 | 0/12 (0%) | 0 |
pain-extremity | 1/6 (16.7%) | 1 | 2/3 (66.7%) | 2 | 2/6 (33.3%) | 2 | 2/12 (16.7%) | 2 |
arthralgia | 0/6 (0%) | 0 | 1/3 (33.3%) | 1 | 0/6 (0%) | 0 | 0/12 (0%) | 0 |
muscle disorder - cramping | 1/6 (16.7%) | 1 | 1/3 (33.3%) | 1 | 4/6 (66.7%) | 4 | 3/12 (25%) | 3 |
Nervous system disorders | ||||||||
tremor | 0/6 (0%) | 0 | 2/3 (66.7%) | 2 | 1/6 (16.7%) | 1 | 4/12 (33.3%) | 5 |
peripheral sensory neuropathy | 0/6 (0%) | 0 | 1/3 (33.3%) | 1 | 1/6 (16.7%) | 1 | 1/12 (8.3%) | 1 |
paresthesia | 1/6 (16.7%) | 1 | 1/3 (33.3%) | 1 | 1/6 (16.7%) | 2 | 2/12 (16.7%) | 3 |
lightheadedness | 1/6 (16.7%) | 1 | 2/3 (66.7%) | 2 | 1/6 (16.7%) | 1 | 2/12 (16.7%) | 2 |
dysgeusia | 0/6 (0%) | 0 | 0/3 (0%) | 0 | 0/6 (0%) | 0 | 5/12 (41.7%) | 5 |
dizziness | 0/6 (0%) | 0 | 1/3 (33.3%) | 1 | 2/6 (33.3%) | 3 | 2/12 (16.7%) | 4 |
headache | 1/6 (16.7%) | 1 | 0/3 (0%) | 0 | 0/6 (0%) | 0 | 2/12 (16.7%) | 2 |
Psychiatric disorders | ||||||||
mood alteration | 3/6 (50%) | 3 | 0/3 (0%) | 0 | 2/6 (33.3%) | 2 | 2/12 (16.7%) | 2 |
insomnia | 1/6 (16.7%) | 1 | 0/3 (0%) | 0 | 2/6 (33.3%) | 2 | 4/12 (33.3%) | 5 |
anxiety | 1/6 (16.7%) | 1 | 0/3 (0%) | 0 | 1/6 (16.7%) | 1 | 0/12 (0%) | 0 |
Renal and urinary disorders | ||||||||
urinary urgency | 0/6 (0%) | 0 | 0/3 (0%) | 0 | 0/6 (0%) | 0 | 1/12 (8.3%) | 1 |
urinary incontinence | 1/6 (16.7%) | 1 | 1/3 (33.3%) | 1 | 0/6 (0%) | 0 | 2/12 (16.7%) | 2 |
urinary frequency | 4/6 (66.7%) | 5 | 0/3 (0%) | 0 | 4/6 (66.7%) | 5 | 0/12 (0%) | 0 |
urinary hesitancy | 1/6 (16.7%) | 2 | 0/3 (0%) | 0 | 1/6 (16.7%) | 1 | 1/12 (8.3%) | 2 |
chronic kidney disease | 2/6 (33.3%) | 4 | 0/3 (0%) | 0 | 2/6 (33.3%) | 5 | 1/12 (8.3%) | 1 |
hematuria | 0/6 (0%) | 0 | 1/3 (33.3%) | 2 | 2/6 (33.3%) | 2 | 0/12 (0%) | 0 |
Respiratory, thoracic and mediastinal disorders | ||||||||
wheezing | 0/6 (0%) | 0 | 1/3 (33.3%) | 1 | 2/6 (33.3%) | 2 | 0/12 (0%) | 0 |
sore throat | 1/6 (16.7%) | 1 | 0/3 (0%) | 0 | 1/6 (16.7%) | 1 | 1/12 (8.3%) | 1 |
pneumonia | 1/6 (16.7%) | 1 | 0/3 (0%) | 0 | 1/6 (16.7%) | 1 | 1/12 (8.3%) | 1 |
nasal congestion | 0/6 (0%) | 0 | 0/3 (0%) | 0 | 0/6 (0%) | 0 | 2/12 (16.7%) | 2 |
hoarseness | 2/6 (33.3%) | 2 | 3/3 (100%) | 3 | 2/6 (33.3%) | 3 | 4/12 (33.3%) | 4 |
epistaxis | 2/6 (33.3%) | 2 | 0/3 (0%) | 0 | 0/6 (0%) | 0 | 2/12 (16.7%) | 2 |
dyspnea | 1/6 (16.7%) | 1 | 2/3 (66.7%) | 2 | 5/6 (83.3%) | 10 | 6/12 (50%) | 6 |
cough | 1/6 (16.7%) | 1 | 2/3 (66.7%) | 2 | 2/6 (33.3%) | 4 | 4/12 (33.3%) | 6 |
Skin and subcutaneous tissue disorders | ||||||||
rash | 0/6 (0%) | 0 | 1/3 (33.3%) | 1 | 3/6 (50%) | 3 | 5/12 (41.7%) | 7 |
pruritis | 1/6 (16.7%) | 1 | 1/3 (33.3%) | 1 | 0/6 (0%) | 0 | 3/12 (25%) | 3 |
infection - skin | 0/6 (0%) | 0 | 1/3 (33.3%) | 1 | 1/6 (16.7%) | 1 | 0/12 (0%) | 0 |
dry skin | 2/6 (33.3%) | 2 | 1/3 (33.3%) | 1 | 2/6 (33.3%) | 3 | 0/12 (0%) | 0 |
Vascular disorders | ||||||||
hypotension | 1/6 (16.7%) | 1 | 0/3 (0%) | 0 | 1/6 (16.7%) | 1 | 1/12 (8.3%) | 1 |
hypertension | 1/6 (16.7%) | 1 | 0/3 (0%) | 0 | 1/6 (16.7%) | 1 | 0/12 (0%) | 0 |
Limitations/Caveats
More Information
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Name/Title | Vaishali Sanchorawala |
---|---|
Organization | Boston Medical Center |
Phone | 617-638-7017 |
vaishali.sanchorawala@bmc.org |
- H-31082
- PO-AMYL-PI-0024