Phase I Study of BAY 43-9006 (NSC 724772) in Patients With Acute Leukemias, Myelodysplastic Syndromes and Chronic Myeloid Leukemia in Blast Phase
I. Determine the maximum tolerated dose of sorafenib when administered in two different
schedules in patients with refractory or relapsed acute leukemia, myelodysplastic syndromes,
or blastic phase chronic myelogenous leukemia.
II. Determine the dose-limiting toxicity of this drug in these patients.
I. Determine the clinical activity of this drug in these patients. II. Determine the
biologic effect of this drug in these patients.
OUTLINE: This is a randomized, dose-escalation phase I study. Patients are randomized to 1
of 2 treatment arms.
Arm I: Patients receive oral sorafenib once or twice daily on days 1-5, 8-12, and 15-19.
Arm II: Patients receive oral sorafenib once or twice daily on days 1-14.
In both arms, treatment repeats every 21 days for up to 6 months in the absence of disease
progression or unacceptable toxicity. Patients achieving complete remission or partial
remission after 6 months may continue therapy at the discretion of the principal
In both arms, cohorts of 3-6 patients receive escalating doses of sorafenib until the
maximum tolerated dose (MTD) is determined. The MTD is defined as the dose preceding that at
which 2 of 3 or 2 of 6 patients experience dose-limiting toxicity. Up to 10 patients are
treated at the MTD.
Allocation: Randomized, Endpoint Classification: Safety Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
Maximum tolerated dose (MTD) assessed by Common Terminology Criteria for Adverse Events (CTCAE) version 3.0
M.D. Anderson Cancer Center
United States: Food and Drug Administration
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