Overview

This trial is active, not recruiting.

Condition primary myelofibrosis
Treatments pomalidomide 0.5 mg, placebo
Phase phase 3
Sponsor Celgene Corporation
Start date September 2010
End date January 2013
Trial size 252 participants
Trial identifier NCT01178281, 2010-018965-42, CC-4047-MF-002

Summary

The objective of this study is to determine whether pomalidomide is safe and effective in reversing red blood cell (RBC)-transfusion-dependence in persons with myeloproliferative neoplasm (MPN)-associated myelofibrosis. (Global Study) and to describe the frequency of anemia response to pomalidomide in Chinese participants with MPN-associated myelofibrosis and severe anemia not receiving REC-transfusions (China Extension Study only)

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification efficacy study
Intervention model parallel assignment
Masking double blind (subject, investigator)
Primary purpose treatment
Arm
(Experimental)
Pomalidomide 0.5 mg capsule taken by mouth once daily. Participants may take capsules for at least 168 days unless there are unacceptable side effects, progression of MPN-associated myelofibrosis or recurrence of RBC-transfusion-dependence.
pomalidomide 0.5 mg CC-4047; Pomalyst; Imnovid
Pomalidomide 0.5 mg capsule taken by mouth once daily. Immunomodulatory agent with demonstrated efficacy in the treatment of subjects with RBC-transfusion-dependence associated with MNP-associated myelofibrosis.
(Placebo Comparator)
One placebo capsule taken by mouth once daily. Participants may take capsules for at least 168 days unless there are unacceptable side effects, progression of MPN-associated myelofibrosis or recurrence of RBC-transfusion-dependence.
placebo
Placebo Comparator to active drug

Primary Outcomes

Measure
Percentage of Participants Who Achieved Red Blood Cell (RBC) Transfusion Independence
time frame: 168 days

Secondary Outcomes

Measure
Duration of RBC-transfusion Independence
time frame: Up to 2.5 years
Time to Becoming RBC-transfusion-independent
time frame: Up to 2.5 years
Healthcare Resource Utilization
time frame: Every 28 days
Euro QOL 5 Dimension Questionaire
time frame: Up to treatment discontinuation
FACT-Anemia Quality of Life Questionaire
time frame: Up to treatment discontinuation
Frequency of Adverse Events
time frame: Up to 2.5 years
Overall Survival
time frame: Up to 2.5 years

Eligibility Criteria

Male or female participants at least 18 years old.

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 <0.5x10^9 /L - Platelets <25 x 10^9 /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

Additional Information

Official title 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
Description Study sites in China will also participate in a China-specific, single-arm, open label extension of the current study. Participants will have myeloproliferative neoplasm (MPN)-associated myelofibrosis and severe anemia and not be receiving red blood cell (RBC)-transfusions. Eligible participants will receive pomalidomide (0.5 mg/day) and will be evaluated on a schedule parallel to that of the global study.
Trial information was received from ClinicalTrials.gov and was last updated in March 2015.
Information provided to ClinicalTrials.gov by Celgene Corporation.