ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 1 of 1 for:    Study of Pomalidomide in Persons with Myelofibrosis
Previous Study | Return to List | Next Study

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)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT01178281
Recruitment Status : Active, not recruiting
First Posted : August 10, 2010
Results First Posted : March 14, 2014
Last Update Posted : March 29, 2018
Sponsor:
Information provided by (Responsible Party):
Celgene

July 15, 2010
August 10, 2010
January 31, 2014
March 14, 2014
March 29, 2018
September 8, 2010
January 1, 2013   (Final data collection date for primary outcome measure)
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
Proportion of subjects achieving RBC-transfusion-independence [ Time Frame: 6 months ]
Complete list of historical versions of study NCT01178281 on ClinicalTrials.gov Archive Site
  • 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.
  • 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
Not Provided
Not Provided
 
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
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
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)
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.
Interventional
Phase 3
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Primary Myelofibrosis
  • 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
  • Drug: Placebo
    Placebo Comparator to active drug
  • 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.
    Intervention: Drug: Pomalidomide 0.5 mg
  • 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.
    Intervention: Drug: Placebo

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Active, not recruiting
252
210
May 31, 2018
January 1, 2013   (Final data collection date for primary outcome measure)

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
Sexes Eligible for Study: All
18 Years and older   (Adult, Older Adult)
No
Contact information is only displayed when the study is recruiting subjects
Australia,   Austria,   Belgium,   Canada,   China,   France,   Germany,   Italy,   Japan,   Netherlands,   Poland,   Russian Federation,   Spain,   Sweden,   United Kingdom,   United States
 
 
NCT01178281
CC-4047-MF-002
2010-018965-42 ( EudraCT Number )
Yes
Not Provided
Not Provided
Celgene
Celgene
Not Provided
Study Director: Robert P Gale, MD, Ph.D. Celgene Corporation
Celgene
March 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP