CC-4047 in Treating Patients With Myelofibrosis

This study is ongoing, but not recruiting participants.
Sponsor:
Collaborator:
Information provided by:
Mayo Clinic
ClinicalTrials.gov Identifier:
NCT00669578
First received: April 29, 2008
Last updated: June 25, 2012
Last verified: June 2012
  Purpose

RATIONALE: Biological therapies, such as CC-4047, may stimulate the immune system in different ways and stop cancer cells from growing. CC-4047 may also stop the growth of cancer cells by blocking blood flow to the cancer.

PURPOSE: This phase I/II trial is studying the side effects and best dose of CC-4047 and to see how well it works in treating patients with myelofibrosis.


Condition Intervention Phase
Chronic Myeloproliferative Disorders
Secondary Myelofibrosis
Drug: pomalidomide
Other: questionnaire administration
Procedure: quality-of-life assessment
Phase 1
Phase 2

Study Type: Interventional
Study Design: Masking: Open Label
Primary Purpose: Treatment
Official Title: A Phase I/II, Prospective, Open-Label Study to Determine the Safety and Efficacy of CC-4047 in Patients With Primary, Post Polycythemia Vera, or Post Essential Thrombocythemia Myelofibrosis®

Resource links provided by NLM:


Further study details as provided by Mayo Clinic:

Primary Outcome Measures:
  • Rate and frequency of dose-limiting toxicity as measured by NCI CTCAE v3.0 [ Designated as safety issue: Yes ]
  • Best overall response over the first 6 courses of treatment [ Designated as safety issue: No ]
  • Safety as measured by NCI CTCAE v3.0 [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Duration of response [ Designated as safety issue: No ]
  • Time to response [ Designated as safety issue: No ]
  • Best overall response over the first 12 courses of treatment [ Designated as safety issue: No ]
  • Cytogenetic response in patients with a baseline abnormality [ Designated as safety issue: No ]
  • Molecular response (JAK2V617F mutation burden) in peripheral blood in mutation positive patients with granulocytosis [ Designated as safety issue: No ]
  • Bone marrow fibrosis [ Designated as safety issue: No ]
  • Quality of life [ Designated as safety issue: No ]

Estimated Enrollment: 77
Study Start Date: May 2008
Estimated Study Completion Date: May 2013
Primary Completion Date: July 2010 (Final data collection date for primary outcome measure)
Detailed Description:

OBJECTIVES:

Primary

  • To determine the maximum tolerated dose of CC-4047 in patients with primary myelofibrosis and post polycythemia vera or post essential thrombocythemia myelofibrosis. (Phase I [closed to accrual as of 12/3/2008])
  • To determine the effect of this drug in these patients. (Phase II [open to accrual as of 1/7/2009])
  • To determine the safety of this drug in these patients. (Phase II [open to accrual as of 1/7/2009])

Secondary

  • To further evaluate the nature and quality of responses to CC-4047.

OUTLINE: This is a phase I dose-escalation study followed by a phase II study.

  • Phase I (closed to accrual as of 12/3/2008): Patients receive oral CC-4047 on days 1-21. Treatment repeats every 28 days for up to 12 courses in the absence of disease progression or unacceptable toxicity. After 12 courses, patients with responding disease may continue to receive CC-4047 in the absence of disease progression or unacceptable toxicity.
  • Phase II (open to accrual as of 1/7/2009): Patients receive oral CC-4047 at the maximum tolerated dose determined in phase I.

Patients complete quality of life questionnaires at baseline, every 28 days for the first 3 courses, and then every 3 courses (every 84 days) thereafter.

After completion of study treatment, patients are followed at 28 days and then every 6 months for up to 3 years.

  Eligibility

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

DISEASE CHARACTERISTICS:

  • Diagnosis of primary and post essential thrombocythemia (ET) or post polycythemia vera (PV) myelofibrosis requiring therapy

    • De novo presentation (i.e., agnogenic myeloid metaplasia AND post ET or post PV myelofibrosis)
    • Developed after an antecedent history of PV (i.e., post polycythemic myeloid metaplasia) or essential polycythemia (i.e., post thrombocythemic myeloid metaplasia)
  • Total hemoglobin < 10 g/dL OR transfusion dependent anemia (defined by a history of ≥ 2 units of RBC transfusions within the past 28 days for hemoglobin < 8.5 g/dL that was not associated with overt bleeding) OR marked splenomegaly (e.g., ≥ 10 cm below costal margin)

PATIENT CHARACTERISTICS:

  • ECOG performance status 0-2
  • ANC ≥ 500/μL
  • Platelet count ≥ 20,000/μL
  • AST and ALT ≤ 3 times upper limit of normal (ULN) (≤ 5 times ULN if attributed to hepatic extramedullary hematopoiesis)
  • Total bilirubin ≤ 3 times ULN OR direct bilirubin ≤ 2 times ULN
  • Serum creatinine ≤ 2.0 mg/dL
  • Not pregnant or nursing
  • Negative pregnancy test
  • Fertile patients must use effective double-method contraception for ≥ 28 days before, during, and for ≥ 28 days after completion of study treatment
  • Agrees to abstain from donating blood, semen, or sperm during and for ≥ 28 days after completion of study treatment
  • Willing to undergo transfusion of blood products (if applicable)
  • Able to complete questionnaire(s) alone or with assistance
  • No known HIV positivity, hepatitis B carrier, or active hepatitis C infection
  • No serious medical condition, psychiatric illness, or any other condition, including the presence of laboratory abnormalities, that (as judged by the treating physician) would preclude giving informed consent or participating in the study or confound the ability to interpret data from the study
  • No other active malignancies, except basal cell or squamous cell carcinoma of the skin or carcinoma in situ of the cervix or breast
  • No active deep vein thrombosis or pulmonary embolism that has not been therapeutically anticoagulated

PRIOR CONCURRENT THERAPY:

  • Recovered from all prior therapy
  • No prior CC-4047
  • More than 28 days since prior growth factors, cytotoxic chemotherapeutic agents (e.g., hydroxyurea or anagrelide), corticosteroids, or experimental drugs or therapies
  • No other concurrent experimental drugs or therapies or cytotoxic chemotherapeutic agents (e.g., hydroxyurea or anagrelide) for myelofibrosis
  • No concurrent growth factors (including erythropoietin) for myelofibrosis, except G-CSF or pegfilgrastim
  • No concurrent chronic use (i.e., > 2 weeks) of more than physiologic doses of corticosteroids (dose equivalent to > 10 mg/day of prednisone)
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00669578

Locations
United States, Minnesota
Mayo Clinic
Rochester, Minnesota, United States, 55905
Sponsors and Collaborators
Mayo Clinic
Investigators
Principal Investigator: Ruben A. Mesa, MD Mayo Clinic
Study Chair: Ayalew Tefferi, MD Mayo Clinic
  More Information

Additional Information:
No publications provided

Responsible Party: Ruben A. Mesa, M.D., Mayo Clinic Cancer Center
ClinicalTrials.gov Identifier: NCT00669578     History of Changes
Other Study ID Numbers: MC078B, P30CA015083, MC078B, NCI-2009-01331, 07-005317, PO-MMM-PI-0007
Study First Received: April 29, 2008
Last Updated: June 25, 2012
Health Authority: United States: Food and Drug Administration

Keywords provided by Mayo Clinic:
secondary myelofibrosis
polycythemia vera
essential thrombocythemia
primary myelofibrosis

Additional relevant MeSH terms:
Primary Myelofibrosis
Myeloproliferative Disorders
Polycythemia
Polycythemia Vera
Thrombocythemia, Essential
Thrombocytosis
Bone Marrow Diseases
Hematologic Diseases
Blood Coagulation Disorders
Blood Platelet Disorders
Hemorrhagic Disorders

ClinicalTrials.gov processed this record on April 16, 2014