A Phase I/II Trial of Pomalidomide and Dexamethasone in Subjects With Previously-Treated AL Amyloidosis

Sponsor
Boston Medical Center (Other)
Overall Status
Completed
CT.gov ID
NCT01570387
Collaborator
Celgene Corporation (Industry)
27
1
4
82
0.3

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

Study Type:
Interventional
Actual Enrollment :
27 participants
Allocation:
Non-Randomized
Intervention Model:
Single Group Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
A Phase I/II Trial of Pomalidomide and Dexamethasone in Subjects With Previously-Treated AL Amyloidosis
Actual Study Start Date :
Jun 1, 2012
Actual Primary Completion Date :
Nov 1, 2018
Actual Study Completion Date :
Apr 1, 2019

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:
  • pomalyst
  • imnovist
  • Drug: Dexamethasone
    10-20 mg on days 1, 8, 15, and 22
    Other Names:
  • Dexamethasone Acetate
  • 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:
  • pomalyst
  • imnovist
  • Drug: Dexamethasone
    10-20 mg on days 1, 8, 15, and 22
    Other Names:
  • Dexamethasone Acetate
  • 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:
  • pomalyst
  • imnovist
  • Drug: Dexamethasone
    10-20 mg on days 1, 8, 15, and 22
    Other Names:
  • Dexamethasone Acetate
  • 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:
  • pomalyst
  • imnovist
  • Drug: Dexamethasone
    10-20 mg on days 1, 8, 15, and 22
    Other Names:
  • Dexamethasone Acetate
  • Outcome Measures

    Primary Outcome Measures

    1. 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

    2. 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.

    3. 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

    1. 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

    Ages Eligible for Study:
    18 Years and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    Inclusion Criteria:
    1. Understand and voluntarily sign informed consent form.

    2. ≥18yrs old

    3. Able to adhere to the study visit schedule and other protocol requirements.

    4. Biopsy proven tissue amyloid deposits or positive fat aspirate

    5. Proof of AL type (a or b)

    6. Measurable plasma cell dyscrasia (a or b and c of the following required):

    7. Monoclonal protein in the serum or urine by immunofixation electrophoresis

    8. Plasmacytosis of bone marrow (<30% plasma cells) with monoclonal staining for kappa or lambda light-chain isotype

    9. dFLC of 50mg/L (dFLC=difference in involved and uninvolved serum free light-chain levels)

    10. 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

    11. 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

    12. Eastern Cooperative Group (ECOG) performance status ≤2 at study entry

    13. Lab test results within these ranges:

    1. 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
    1. 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.

    2. 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.

    3. 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:
    1. Secondary or familial amyloidosis

    2. Multiple myeloma (≥30% plasma cells in a bone marrow biopsy specimen or lytic bone lesions)

    3. Cytotoxic chemo or radiation therapy ≤4 weeks of study entry or following baseline evaluation

    4. Symptomatic cardiac arrhythmias or O2-dependent restrictive cardiomyopathy

    5. Dialysis-dependent

    6. Untreated or uncontrolled infections.

    7. Serious medical conditions, laboratory abnormality, or psychiatric illness that would prevent the subject from signing the informed consent form.

    8. Pregnant or breast feeding females (lactating females must agree not to breast feed while taking pomalidomide).

    9. 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.

    10. Use of any other experimental drug or therapy within 28 days of baseline.

    11. Known intolerance to steroids.

    12. Known hypersensitivity to thalidomide or lenalidomide

    13. The development of erythema nodosum if characterized by a desquamating rash while taking thalidomide or similar drugs.

    14. Concurrent use of other anti-cancer agents or treatments.

    15. 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.
    Responsible Party:
    Boston Medical Center
    ClinicalTrials.gov Identifier:
    NCT01570387
    Other Study ID Numbers:
    • H-31082
    • PO-AMYL-PI-0024
    First Posted:
    Apr 4, 2012
    Last Update Posted:
    Sep 22, 2020
    Last Verified:
    Sep 1, 2020
    Individual Participant Data (IPD) Sharing Statement:
    No
    Plan to Share IPD:
    No
    Studies a U.S. FDA-regulated Drug Product:
    Yes
    Studies a U.S. FDA-regulated Device Product:
    No
    Additional relevant MeSH terms:

    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

    1. Primary Outcome
    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%
    2. Primary Outcome
    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%
    3. Primary Outcome
    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%
    4. Secondary Outcome
    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

    [Not Specified]

    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
    Email vaishali.sanchorawala@bmc.org
    Responsible Party:
    Boston Medical Center
    ClinicalTrials.gov Identifier:
    NCT01570387
    Other Study ID Numbers:
    • H-31082
    • PO-AMYL-PI-0024
    First Posted:
    Apr 4, 2012
    Last Update Posted:
    Sep 22, 2020
    Last Verified:
    Sep 1, 2020