CC-4047 in Treating Patients With Myelofibrosis
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.
Chronic Myeloproliferative Disorders
Other: questionnaire administration
Procedure: quality-of-life assessment
|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®|
- 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 ]
- 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 ]
|Study Start Date:||May 2008|
|Estimated Study Completion Date:||May 2013|
|Primary Completion Date:||July 2010 (Final data collection date for primary outcome measure)|
- 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])
- 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.
|United States, Minnesota|
|Rochester, Minnesota, United States, 55905|
|Principal Investigator:||Ruben A. Mesa, MD||Mayo Clinic|
|Study Chair:||Ayalew Tefferi, MD||Mayo Clinic|