Pomalidomide for Myelofibrosis Patients
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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: NCT00946270 |
Recruitment Status
:
Active, not recruiting
First Posted
: July 24, 2009
Last Update Posted
: January 10, 2018
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Polycythemia Vera Thrombocythemia | Drug: CC-4047 Drug: Prednisone | Phase 2 |

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.
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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.
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- 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.

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Ages Eligible for Study: | 18 Years and older (Adult, Senior) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- 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.
- 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]).
- 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)
- 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)
- Subjects must be willing to receive transfusion of blood products
- ECOG performance status (PS) of 0, 1, or 2 at screening.
- Must be willing to adhere to the study visit schedule and other protocol requirements.
- 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
- 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.
- 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:
- Known positive status for HIV, hepatitis B carrier, or active hepatitis C infection.
- 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.
- 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.
- Pregnant or lactating females
- Prior use of CC-4047
- Currently enrolled on another clinical trial or receiving investigational agent

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 ClinicalTrials.gov identifier (NCT number): NCT00946270
United States, Texas | |
University of Texas MD Anderson Cancer Center | |
Houston, Texas, United States, 77030 |
Principal Investigator: | Srdan Verstovsek, MD | M.D. Anderson Cancer Center |
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 ( 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:
CC-4047 Pomalidomide polycythemia vera essential thrombocythemia myelofibrosis |
Additional relevant MeSH terms:
Primary Myelofibrosis Polycythemia Polycythemia Vera Thrombocytosis Thrombocythemia, Essential Myeloproliferative Disorders Bone Marrow Diseases Hematologic Diseases Bone Marrow Neoplasms Hematologic Neoplasms Neoplasms by Site Neoplasms Blood Platelet Disorders Blood Coagulation Disorders Hemorrhagic Disorders |
Prednisone Pomalidomide Thalidomide Anti-Inflammatory Agents Glucocorticoids Hormones 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 |