Now Available for Public Comment: Notice of Proposed Rulemaking (NPRM) for FDAAA 801 and NIH Draft Reporting Policy for NIH-Funded Trials

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)

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Celgene Corporation
ClinicalTrials.gov Identifier:
NCT01178281
First received: July 15, 2010
Last updated: January 31, 2014
Last verified: January 2014
  Purpose

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)


Condition Intervention Phase
Primary Myelofibrosis
Drug: Pomalidomide 0.5 mg
Drug: Placebo
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, 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

Resource links provided by NLM:


Further study details as provided by Celgene Corporation:

Primary Outcome Measures:
  • Percentage of Participants Who Achieved Red Blood Cell (RBC) Transfusion Independence [ Time Frame: 168 days ] [ Designated as safety issue: No ]
    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


Secondary Outcome Measures:
  • Duration of RBC-transfusion Independence [ Time Frame: Up to 2.5 years ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    Number of days from randomization to achieving RBC transfusion independence as assessed every 28 days.

  • Healthcare Resource Utilization [ Time Frame: Every 28 days ] [ Designated as safety issue: No ]

    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: Yes ]
    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 ] [ Designated as safety issue: No ]
    The time from randomization to the death or to the latest date when participants are known to be alive.


Enrollment: 252
Study Start Date: September 2010
Estimated Study Completion Date: August 2017
Primary Completion Date: January 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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.
Drug: Placebo
Placebo Comparator to active drug

Detailed 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.

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01178281

  Show 85 Study Locations
Sponsors and Collaborators
Celgene Corporation
Investigators
Study Director: Robert P Gale, MD, Ph.D. Celgene Corporation
  More Information

No publications provided by Celgene Corporation

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Celgene Corporation
ClinicalTrials.gov Identifier: NCT01178281     History of Changes
Other Study ID Numbers: CC-4047-MF-002, 2010-018965-42
Study First Received: July 15, 2010
Results First Received: January 31, 2014
Last Updated: January 31, 2014
Health Authority: United States: Food and Drug Administration
Canada: Health Canada
Austria: Agency for Health and Food Safety
Belgium: Federal Agency for Medicinal Products and Health Products
China: Food and Drug Administration
France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
Germany: Federal Institute for Drugs and Medical Devices
Italy: The Italian Medicines Agency
Japan: Pharmaceuticals and Medical Devices Agency
Netherlands: Medicines Evaluation Board (MEB)
Poland: Office for Registration of Medicinal Products, Medical Devices and Biocidal Products
Russia: Ministry of Health of the Russian Federation
Spain: Spanish Agency of Medicines
Sweden: Medical Products Agency
United Kingdom: Medicines and Healthcare Products Regulatory Agency
Australia: Department of Health and Ageing Therapeutic Goods Administration

Keywords provided by Celgene Corporation:
Myelofibrosis
Post-polycythemia vera myelofibrosis
Post-essential thrombocythemia myelofibrosis
RBC-transfusion-dependence
Primary Myelofibrosis
RBC-Transfusion-Dependence

Additional relevant MeSH terms:
Myeloproliferative Disorders
Primary Myelofibrosis
Bone Marrow Diseases
Hematologic Diseases
Pomalidomide
Thalidomide
Angiogenesis Inhibitors
Angiogenesis Modulating Agents
Anti-Bacterial Agents
Anti-Infective Agents
Antineoplastic Agents
Growth Inhibitors
Growth Substances
Immunologic Factors
Immunosuppressive Agents
Leprostatic Agents
Pharmacologic Actions
Physiological Effects of Drugs
Therapeutic Uses

ClinicalTrials.gov processed this record on November 20, 2014