Pomalidomide for Myelofibrosis Patients

This study is ongoing, but not recruiting participants.
Information provided by (Responsible Party):
M.D. Anderson Cancer Center
ClinicalTrials.gov Identifier:
First received: July 23, 2009
Last updated: April 13, 2016
Last verified: April 2016
The goal of this clinical research study is to learn if CC-4047 (now called pomalidomide) and prednisone can help to control MMM. The safety of this therapy will also be studied.

Condition Intervention Phase
Polycythemia Vera
Drug: CC-4047
Drug: Prednisone
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Phase II, Prospective, Open Label Study to Determine the Safety and Efficacy of Pomalidomide (CC-4047) in Subjects With Primary, Post Polycythemia Vera, or Post Essential Thrombocythemia Myelofibrosis (PMF; Post-PV MF, or Post-ET MF)

Resource links provided by NLM:

Further study details as provided by M.D. Anderson Cancer Center:

Primary Outcome Measures:
  • Best Overall Response [ Time Frame: 6 months ] [ Designated as safety issue: No ]
    Primary endpoint is best overall response. An evaluable subject classified as a treatment success for the primary endpoint if the subject's best overall response is complete remission (CR), partial remission (PR) or clinical improvement (CI) as determined by International Working Group Criteria over the first 6 cycles of study treatment.

Estimated Enrollment: 70
Study Start Date: July 2009
Estimated Primary Completion Date: August 2018 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: CC-4047
CC-4047 0.5 mg orally daily. Prednisone given during first 3 cycles of therapy. It will be dosed orally at the dose of 30 mg/day during cycle 1, 15 mg/day during cycle 2, and 15 mg every other day during cycle 3, and then it will be discontinued.
Drug: CC-4047
0.5 mg capsules daily by mouth day 1 through day 28.
Other Name: Pomalidomide
Drug: Prednisone
30 mg by mouth daily during cycle 1, 15 mg/day during cycle 2, and 15 mg every other day during cycle 3, and then it will be discontinued.

  Show Detailed Description


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

Inclusion Criteria:

  1. Must be >/= 18 years of age at the time of voluntarily signing an Institutional Review Board/Independent Ethics Committee (IRB/IEC) - approved informed consent form.
  2. Must be diagnosed with myelofibrosis requiring therapy including myelofibrosis with myeloid metaplasia (MMM), de novo presentation (i.e. agnogenic myeloid metaplasia [AMMM], and developing after an antecedent history of Polycythemia vera (i.e., post-polycythemic myeloid metaplasia [PPMM]), or essential Polycythemia (i.e., post thrombocythemic myeloid metaplasia [PTMM]).
  3. Screening total hemoglobin level < 10 g/dL or transfusion-dependent anemia defined as per IWG criteria (transfusion dependency defined by a history of a least 2 units of red blood cell transfusions in the last 28 days for hemoglobin < 8.5 g/dL that was not associated with overt bleeding)
  4. Must have adequate organ function as demonstrated by the following </= 14 days prior to starting study drug: ·ALT (SGOT) and AST (SGPT) </= 3 x upper limit of normal (ULN), [unless upon judgment of the treating physician, it is believed to be due to extramedullary hematopoiesis (EMH)] ·Total bilirubin < 3 x ULN or Direct Bilirubin < 2 x ULN ·Serum creatinine </= 2.5 mg/dL ·Absolute neutrophil count >/= 1,000/µL (>/=1.0 x 10^9/L) ·Platelet count >/= 50,000/µL (>/=50 x 10^9/L)
  5. Subjects must be willing to receive transfusion of blood products
  6. ECOG performance status (PS) of 0, 1, or 2 at screening.
  7. Must be willing to adhere to the study visit schedule and other protocol requirements.
  8. No active malignancies with the exception of basal cell or squamous cell carcinoma of the skin, or carcinoma (in situ) of the cervix or breast
  9. All study participants must be registered into the mandatory POMALYST REMS™ program, and be willing and able to comply with the requirements of the POMALYST REMS™ program.
  10. Females of reproductive potential (FCBP†) must adhere to the scheduled pregnancy testing as required in the POMALYST REMS™ program. Able to take aspirin (81 or 325 mg) daily as prophylactic anticoagulation (patients intolerant to ASA may use warfarin or low molecular weight heparin).

Exclusion Criteria:

  1. Known positive status for HIV, hepatitis B carrier, or active hepatitis C infection.
  2. The use of any growth factors, cytotoxic chemotherapeutic agents (e.g. hydroxyurea), corticosteroids, or experimental drug or therapy within 14 days of starting CC-4047 and/or lack of recovery from all toxicity from previous therapy to grade 1 or better.
  3. Any serious medical condition or psychiatric illness that would prevent, (as judged by the treating physician) the subject from signing the informed consent form or any condition, including the presence of laboratory abnormalities, which places the subject at unacceptable risk if he/she were to participate in the study or confounds the ability to interpret data from the study.
  4. Pregnant or lactating females
  5. Prior use of CC-4047
  6. Currently enrolled on another clinical trial or receiving investigational agent
  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: NCT00946270

United States, Texas
University of Texas MD Anderson Cancer Center
Houston, Texas, United States, 77030
Sponsors and Collaborators
M.D. Anderson Cancer Center
Principal Investigator: Srdan Verstovsek, MD M.D. Anderson Cancer Center
  More Information

Additional Information:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: M.D. Anderson Cancer Center
ClinicalTrials.gov Identifier: NCT00946270     History of Changes
Other Study ID Numbers: 2007-0199  NCI-2012-00360 
Study First Received: July 23, 2009
Last Updated: April 13, 2016
Health Authority: United States: Food and Drug Administration

Keywords provided by M.D. Anderson Cancer Center:
polycythemia vera
essential thrombocythemia myelofibrosis

Additional relevant MeSH terms:
Polycythemia Vera
Primary Myelofibrosis
Thrombocythemia, Essential
Blood Coagulation Disorders
Blood Platelet Disorders
Bone Marrow Diseases
Hematologic Diseases
Hemorrhagic Disorders
Myeloproliferative Disorders
Angiogenesis Inhibitors
Angiogenesis Modulating Agents
Antineoplastic Agents
Growth Inhibitors
Growth Substances
Immunologic Factors
Pharmacologic Actions
Physiological Effects of Drugs
Therapeutic Uses

ClinicalTrials.gov processed this record on May 01, 2016