A Sequential Two-Stage Dose Escalation Study to Evaluate the Safety and Efficacy of Ruxolitinib for the Treatment of Chronic Myelomonocytic Leukemia (CMML)
This is a phase 1/2, two-stage, sequential cohort dose escalation study. If dose escalation
is completed as planned, no more than 53 subjects are expected to enroll onto this study at
a rate of approximately 3 subjects every month. For the Phase 2 study the Simon's optimal
two-stage design will be employed to test the null hypothesis that response rate (RR) equals
to 10% versus the alternative that RR equals to 30%.
Demographic and clinical variables for the study patients will be summarized using
descriptive statistics (mean, standard deviation, median, inter-quartile range, range, and
frequency counts and percentages). Safety and efficacy data will be analyzed overall as well
as separately for each dose cohort when appropriate.
Interventional
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
The maximum tolerated dose (MTD) of ruxolitinib for the treatment of Myelomonocytic Leukemia (CMML)
Phase I - The MTD is defined as the highest dose where less than 33% of subjects experience a drug related predefined dose limited toxicity (DLT).
17 weeks
Yes
Eric Padron, M.D.
Principal Investigator
H. Lee Moffitt Cancer Center and Research Institute
United States: Food and Drug Administration
MCC-17259
NCT01776723
February 2013
September 2017
Name | Location |
---|---|
H. Lee Moffitt Cancer Center and Research Institute | Tampa, Florida 33612 |