Phase-3 Double-Blind, Placebo-Controlled Study of Pomalidomide in Persons With Myeloproliferative-Neoplasm-Associated Myelofibrosis and RBC-Transfusion-Dependence Myelofibrosis and RBC-Transfusion-Dependence (RESUME)
|Study Design:||Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Participant, Investigator
Primary Purpose: Treatment
|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|
- Percentage of Participants Who Achieved Red Blood Cell (RBC) Transfusion Independence [ Time Frame: 168 days ]Defined as the absence of intravenous RBC transfusions for any consecutive "rolling" 84 day interval (i.e. days 1 to 84, days 2 to 85 etc) before Day 169 visit. A responder is: a. One who received at least two RBC transfusions on/after the first dose of study drug, and at least one ≥ 84 days between two consecutive RBC-transfusions; b. One who received at least one RBC transfusion after the first dose of study drug and no RBC transfusion at the first study drug day, and the time interval between the first dose of study drug and the RBC transfusion date is ≥ 84 days; c. One who received at least one RBC transfusion on/after the first dose of study drug and the time interval between the last RBC transfusion and the last transfusion assessment date is ≥ 84 days; d. One who did not receive any RBC transfusions on and after the first dose of study drug, and the time interval between the first dose of study drug and the date of the last transfusion assessment is ≥ 84 days
- Duration of RBC-transfusion Independence [ Time Frame: Up to 2.5 years ]Number of days from randomization to achieving RBC transfusion independence as assessed every 28 days.
- Time to Becoming RBC-transfusion-independent [ Time Frame: Up to 2.5 years ]Number of days from randomization to achieving RBC transfusion independence as assessed every 28 days.
- Healthcare Resource Utilization [ Time Frame: Every 28 days ]
Characterization of medical resource utilization among participants treated with pomalidomide as compared to subjects receiving placebo treatment.
Information on the length of each hospitalization and other major outpatient resource use will be collected at designated study visits, including major diagnostic procedures and other interventions such as those required for transfusions or for treatment-related adverse events. Additionally, information on major categories of concomitant medications (eg., use of G-CSF, intravenous antibiotics, anti-virals, iron chelation) will be obtained.
- Euro QOL 5 Dimension Questionaire [ Time Frame: Up to treatment discontinuation ]The EQ-5D is a standardized instrument that measures health outcomes for a wide range of health conditions.
- FACT-Anemia Quality of Life Questionaire [ Time Frame: Up to treatment discontinuation ]The FACT-An questionnaire is a cancer-specific questionnaire measuring the four general domains of quality-of-life and an additional anemia questionnaire.
- Frequency of Adverse Events [ Time Frame: Up to 2.5 years ]An Adverse Event (AE) is any noxious, unintended or untoward medical occurrence that may appear or worsen in a participant during the course of a study.
- Overall Survival [ Time Frame: Up to 2.5 years ]The time from randomization to the death or to the latest date when participants are known to be alive.
|Study Start Date:||September 2010|
|Estimated Study Completion Date:||May 2018|
|Primary Completion Date:||January 2013 (Final data collection date for primary outcome measure)|
Experimental: Pomalidomide 0.5 mg
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.
Drug: Pomalidomide 0.5 mg
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.
Other Name: CC-4047; Pomalyst; Imnovid
Placebo Comparator: Placebo
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 Comparator to active drug
Please refer to this study by its ClinicalTrials.gov identifier: NCT01178281
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|Study Director:||Robert P Gale, MD, Ph.D.||Celgene Corporation|