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®
- 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
- To determine the safety of this drug in these patients. (Phase II [open to accrual as
- 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
- 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.
Masking: Open Label, Primary Purpose: Treatment
Rate and frequency of dose-limiting toxicity as measured by NCI CTCAE v3.0
Ruben A. Mesa, MD
United States: Food and Drug Administration
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