Pomalidomide for Myelofibrosis Patients

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. Identifier: NCT00946270
Recruitment Status : Active, not recruiting
First Posted : July 24, 2009
Last Update Posted : January 10, 2018
Information provided by (Responsible Party):
M.D. Anderson Cancer Center

Brief Summary:
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 or disease Intervention/treatment Phase
Polycythemia Vera Thrombocythemia Drug: CC-4047 Drug: Prednisone Phase 2

  Show Detailed Description

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 70 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase II, Prospective, Open Label Study (PO-MMM-PI-0011) 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)
Actual Study Start Date : July 2009
Estimated Primary Completion Date : July 2019
Estimated Study Completion Date : July 2019

Arm Intervention/treatment
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.

Primary Outcome Measures :
  1. Best Overall Response [ Time Frame: 6 months ]
    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.

Information from the National Library of Medicine

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, Learn About Clinical Studies.

Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): 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

Additional Information:
Publications automatically indexed to this study by Identifier (NCT Number):
Responsible Party: M.D. Anderson Cancer Center Identifier: NCT00946270     History of Changes
Other Study ID Numbers: 2007-0199
NCI-2012-00360 ( Registry Identifier: NCI CTRP )
First Posted: July 24, 2009    Key Record Dates
Last Update Posted: January 10, 2018
Last Verified: January 2018

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

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

Additional relevant MeSH terms:
Polycythemia Vera
Primary Myelofibrosis
Thrombocythemia, Essential
Myeloproliferative Disorders
Bone Marrow Diseases
Hematologic Diseases
Bone Marrow Neoplasms
Hematologic Neoplasms
Neoplasms by Site
Blood Platelet Disorders
Blood Coagulation Disorders
Hemorrhagic Disorders
Anti-Inflammatory Agents
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs
Antineoplastic Agents, Hormonal
Antineoplastic Agents
Immunologic Factors
Angiogenesis Inhibitors
Angiogenesis Modulating Agents
Growth Substances
Growth Inhibitors