Pomalidomide for Myelofibrosis Patients

Study Description

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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

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Study Design

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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

Arms and Interventions

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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.

Outcome Measures

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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.

Eligibility Criteria

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Ages Eligible for Study:	18 Years and older (Adult, Senior)
Sexes Eligible for Study:	All
Accepts Healthy Volunteers:	No

Criteria

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

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Locations

United States, Texas
University of Texas MD Anderson Cancer Center
Houston, Texas, United States, 77030

Sponsors and Collaborators

M.D. Anderson Cancer Center

Celgene

Investigators

Principal Investigator:	Srdan Verstovsek, MD	M.D. Anderson Cancer Center

More Information

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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Additional Information:

University of Texas MD Anderson Cancer Center Website 

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Daver N, Shastri A, Kadia T, Newberry K, Pemmaraju N, Jabbour E, Zhou L, Pierce S, Cortes J, Kantarjian H, Verstovsek S. Phase II study of pomalidomide in combination with prednisone in patients with myelofibrosis and significant anemia. Leuk Res. 2014 Sep;38(9):1126-9. doi: 10.1016/j.leukres.2014.06.015. Epub 2014 Jul 6.

Daver N, Shastri A, Kadia T, Quintas-Cardama A, Jabbour E, Konopleva M, O'Brien S, Pierce S, Zhou L, Cortes J, Kantarjian H, Verstovsek S. Modest activity of pomalidomide in patients with myelofibrosis and significant anemia. Leuk Res. 2013 Nov;37(11):1440-4. doi: 10.1016/j.leukres.2013.07.007. Epub 2013 Jul 25.

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
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