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)
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
- 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)
Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Investigator), Primary Purpose: Treatment
Proportion of subjects achieving RBC-transfusion-independence
Robert P Gale, MD, Ph.D.
United States: Food and Drug Administration
|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|