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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


Phase 3
18 Years
N/A
Open (Enrolling)
Both
Primary Myelofibrosis

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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 CenterNew York, New York  10021
UCLA School of MedicineLos Angeles, California  900121973
Mayo ClinicRochester, Minnesota  55905
University of Michigan Comprehensive Cancer CenterAnn Arbor, Michigan  48109-0752
Weill Medical College of Cornell UniversityNew York, New York  10021
University of Illinois at ChicagoChicago, Illinois  60612
University of Florida Shands Cancer CenterGainesville, Florida  32610-0232
Mayo ClinicJacksonville, Florida  32224
University of UtahSalt Lake City, Utah  
Thomas Jefferson UniversityPhiladelphia, Pennsylvania  19107-6541
Mayo ClinicScottsdale, Arizona  
Fred Hutchinson Cancer CenterSeattle, Washington  98109
Avera Hematology and TransplantSioux Falls, South Dakota  57105
Mount Sinai School of Medicine Brookdale University HospitalBrooklyn, New York  11212
Ruttenberg Treatment CenterNew York, New York  10029
Medicine Taussig Cancer InstituteCleveland, Ohio  44195