A Phase-3, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Compare Efficacy and Safety of Pomalidomide in Subjects With Myeloproliferative Neoplasm-Associated Myelofibrosis and Red Blood Cell Transfusion Dependence
18 Years
N/A
Both
Primary Myelofibrosis
Trial Information
A Phase-3, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Compare Efficacy and Safety of Pomalidomide in Subjects With Myeloproliferative Neoplasm-Associated Myelofibrosis and Red Blood Cell Transfusion Dependence
Study sites in China will also participate in a China-specific, single-arm, open label
extension of the current study. Subjects will have myeloproliferative neoplasm
(MPN)-associated myelofibrosis and severe anemia and not be receiving red blood cell
(RBC)-transfusions. Eligible subjects will receive pomalidomide (0.5 mg/day) and will be
evaluated on a schedule parallel to that of the global study.
- China Extension Study Description: Multicenter,single-arm, open-label study to
describe safety and efficacy of pomalidomide in subjects with MPN-associated
myelofibrosis and severe anemia, not receiving RBC-transfusions.
- China Extension Study Primary Outcome: Describe the frequency of anemia response to
pomalidomide in Chinese subjects with MPN-associated myelofibrosis and severe anemia,
not receiving RBC-transfusions.
- China Extension Study Secondary Outcomes:
1. Duration of anemia response (hemoglobin increase ≥15 g/L)
2. Time to anemia response (beginning of a hemoglobin increase ≥15 g/L)
3. Survival
4. Frequency of adverse events (AEs)
- China Extension Estimated Enrollment: 50-75 Subjects
- China Extension Enrollment Interval: approximately 9 months to begin after closure of
enrollment into the global study.
- China Extension Duration: 5 years after last subject enrolled
- China Extension Analysis: Approximately 6 months after enrollment of the last subject.
- China Extension Inclusion Criteria (Same as for global study except for items 3 and 4
noted below):
- 3).Severe Anemia: ≥2 hemoglobin levels ≤80 g/L for ≥84 days immediately before the
day of enrollment (anticipated first day of treatment). No RBC-transfusion within
6 months prior to enrollment.
- 4).Hemoglobin ≤80 g/L at enrollment.
- China Extension Exclusion Criteria (Same as for global study)
Inclusion Criteria:
- Age ≥18 years
- Myeloproliferative-neoplasm (MPN)-associated myelofibrosis
- RBC-transfusion-dependence
- Bone marrow biopsy within 6 months
- Inappropriate to receive blood cell or bone marrow allotransplant, erythropoietin and
androgenic steroids
- Eastern Cooperative Oncology Group (ECOG) performance score ≤2.
- Agree to follow pregnancy precautions as required by the protocol.
- Agree to receive counseling related to teratogenic and other risks of pomalidomide
- Agree not to donate blood or semen
Exclusion Criteria:
- Prior blood cell or bone marrow allotransplant.
- Use of drugs to treat MPN-associated myelofibrosis ≤30 - 42 days before starting
study drug.
- Treatment with erythropoietin or androgenic steroids ≤84 days before starting study
drug.
- Anemia due to reasons other than MPN-associated myelofibrosis.
- Pregnant or lactating females.
- More than 10% blasts by bone marrow examination or more than 10% blasts in blood in
consecutive measurements spanning at least 8 weeks
- Prior history of malignancies,other than the disease being studied, unless the
subject has been free of the malignancy for ≥5 years. Following exceptions:
- Carcinoma in situ of the cervix
- Carcinoma in situ of the breast
- Incidental histologic finding of prostate cancer (T 1a or T 1b using TNM [tumor,
nodes, metastasis] clinical staging system)
- Human Immunodeficiency Virus infection (HIV-infection), active hepatitis B virus
(HBV) or active hepatitis C virus (HCV) infection.
- Prior treatment with pomalidomide.
- Allergic reaction or rash after treatment with thalidomide or lenalidomide
- Any of the following laboratory abnormalities:
- Neutrophils <0.5x10e9 /L
- Platelets <25 x 10e9 /L
- Estimated glomerular filtration rate (kidney function) <30 mL/min/1.73m^2
- Aspartate aminotransferase (AST) and Alanine transaminase (ALT) >3.0 x upper
limit of normal
- Total bilirubin ≥4 x Upper Limit of Normal (ULN);
- Uncontrolled hyperthyroidism or hypothyroidism.
- Deep venous thrombosis (DVT) or pulmonary embolus (PE) <6 months before starting
study drug
- Clinically-important heart disease within the past 6 months
Type of Study:
Interventional
Study Design:
Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Investigator), Primary Purpose: Treatment
Outcome Measure:
Proportion of subjects achieving RBC-transfusion-independence
Outcome Time Frame:
6 months
Safety Issue:
No
Principal Investigator
Robert P Gale, MD, Ph.D.
Investigator Role:
Study Director
Investigator Affiliation:
Celgene Corporation
Authority:
United States: Food and Drug Administration
Study ID:
CC-4047-MF-002
NCT ID:
NCT01178281
Start Date:
August 2010
Completion Date:
March 2017
Related Keywords:
- Primary Myelofibrosis
- Myelofibrosis
- Post-polycythemia vera myelofibrosis
- Post-essential thrombocythemia myelofibrosis
- RBC-transfusion-dependence
- Primary Myelofibrosis
- RBC-Transfusion-Dependence
- Primary Myelofibrosis
- Myeloproliferative Disorders
Name | Location |
|---|---|
| Memorial Sloan Kettering Cancer Center | New York, New York 10021 |
| UCLA School of Medicine | Los Angeles, California 900121973 |
| Mayo Clinic | Rochester, Minnesota 55905 |
| University of Michigan Comprehensive Cancer Center | Ann Arbor, Michigan 48109-0752 |
| Weill Medical College of Cornell University | New York, New York 10021 |
| University of Illinois at Chicago | Chicago, Illinois 60612 |
| University of Florida Shands Cancer Center | Gainesville, Florida 32610-0232 |
| Mayo Clinic | Jacksonville, Florida 32224 |
| University of Utah | Salt Lake City, Utah |
| Thomas Jefferson University | Philadelphia, Pennsylvania 19107-6541 |
| Mayo Clinic | Scottsdale, Arizona |
| Fred Hutchinson Cancer Center | Seattle, Washington 98109 |
| Avera Hematology and Transplant | Sioux Falls, South Dakota 57105 |
| Mount Sinai School of Medicine Brookdale University Hospital | Brooklyn, New York 11212 |
| Ruttenberg Treatment Center | New York, New York 10029 |
| Medicine Taussig Cancer Institute | Cleveland, Ohio 44195 |
