Clinical Trial: 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

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

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

Brief 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)

Detailed Summary: 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.
Sponsor: Celgene

Current Primary Outcome: 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


Original Primary Outcome: Proportion of subjects achieving RBC-transfusion-independence [ Time Frame: 6 months ]

Current Secondary Outcome:

  • 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.


Original Secondary Outcome:

  • Duration of RBC-transfusion Independence [ Time Frame: Up to 3.5 years ]
  • Time to becoming RBC-transfusion-independent - every 28 days [ Time Frame: 2 years ]
  • Healthcare resource utilization [ Time Frame: Every 28 days ]
  • EQ-5D Health Outcome Assessment [ Time Frame: Day 1, 85, 169 and treatment discontinuation ]
  • FACT-An Quality of Life (QoL) Assessment [ Time Frame: D1, D84 and every 84 days while on treatment and treatment discontinuation ]
  • Frequency of AE's [ Time Frame: up to 2 years ]
  • Survival - alive or dead [ Time Frame: 6 months, 2 years and 5 years after the last subject is randomized ]
    Survival - alive or dead at 6 months, 2 years and 5 years after the last subject is randomized


Information By: Celgene

Dates:
Date Received: July 15, 2010
Date Started: September 2010
Date Completion: May 2018
Last Updated: February 1, 2017
Last Verified: February 2017