Pomalidomide for Myelofibrosis Patients

NCT00946270 | Completed Phase 2 Results FDA Drug

• 2 other identifiers: 2007-0199NCI-2012-00360
• Study Type: interventional
• Target: 70
• Locations: 1 country
• Sites: 1

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.

Conditions

Polycythemia Vera; Thrombocythemia

Keywords

CC-4047; Pomalidomide; polycythemia vera; essential thrombocythemia myelofibrosis

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Best Overall Response

  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 clinical improvement (CI) as determined by International Working Group Criteria over the first 6 cycles of study treatment. International Working Group (IWG) consensus criteria for treatment response in myelofibrosis - Clinical improvement (CI) in anemia 1/ A minimum 20g/L increase in hemoglobin level or 2. becoming transfusion independent for at least 8 week duration.

  6 months

Study Arms (3)

Group 3 CC-4047 + Prednisone

EXPERIMENTAL

CC-4047 0.5 mg orally daily starting on day 1 through 28. 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; Drug: Prednisone

Group 1 CC-4047

EXPERIMENTAL

CC-4047 3.0 mg orally daily starting on day 1 through 21.

Drug: CC-4047

Group 2 CC-4047

EXPERIMENTAL

CC-4047 0.5 mg orally daily starting on day 1 through 28.

Drug: CC-4047

Interventions

CC-4047 DRUG

0.5 mg capsules daily by mouth day 1 through day 28.

Also known as: Pomalidomide

Group 2 CC-4047; Group 3 CC-4047 + Prednisone

Prednisone DRUG

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.

Group 3 CC-4047 + Prednisone

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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 International Working Group (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: ·Alanine transaminase (ALT) (SGOT) and Aspartate aminotransferase (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
• Eastern Cooperative Oncology Group (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).

You may not qualify if:

• 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

Sponsors & Collaborators

• M.D. Anderson Cancer Center: lead
• Celgene: collaborator

Study Sites (1)

University of Texas MD Anderson Cancer Center

Houston, Texas, 77030, United States

Location

Related Publications (2)

• 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.

  PMID: 25047979 DERIVED

• 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.

  PMID: 23890523 DERIVED

Related Links

• University of Texas MD Anderson Cancer Center Website

MeSH Terms

Conditions

Polycythemia Vera; Thrombocytosis

Interventions

pomalidomide; Prednisone

Condition Hierarchy (Ancestors)

Bone Marrow Neoplasms; Hematologic Neoplasms; Neoplasms by Site; Neoplasms; Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases; Myeloproliferative Disorders; Blood Platelet Disorders

Intervention Hierarchy (Ancestors)

Pregnadienediols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds

Results Point of Contact

• Title: Dr. Srdan Verstovsek, MD./Professor
• Organization: The University of Texas MD Anderson Cancer Center

sverstov@mdanderson.org

713-745-3429

Study Officials

• Srdan Verstovsek, MD

  M.D. Anderson Cancer Center

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: The first 21 participants received pomalidomide 3.0 mg/day on a 21 day schedule. Due to poor tolerance the study was suspended and amended to treat 20 participants with pomalidomide 0.5 mg/day continuously for 28 days. The study was amended to treat an additional 29 participants with pomalidomide 0.5 mg/day for 28 days plus prednisone.

Study Record Dates

• First Submitted: July 23, 2009
• First Posted: July 24, 2009
• Study Start: July 22, 2009
• Primary Completion: May 22, 2018
• Study Completion: May 22, 2018
• Last Updated: June 10, 2019
• Results First Posted: June 10, 2019

Record last verified: 2019-06

Locations

US (1)