Leukemia Clinical Trial
Official title:
Phase I, Dose De-Escalation to Minimal Effective Pharmacologic Dose Trial of Sodium Phenylbutyrate (PB, NSC 657802) in Combination With 5-Azacytidine (5-AZA, NSC 102816) in Patients With Myelodysplastic Syndromes (MDS) and Acute Myeloid Leukemia (AML)
RATIONALE: Azacitidine plus phenylbutyrate may help leukemia cells develop into normal white
blood cells.
PURPOSE: Phase I trial to study the effectiveness of combining azacitidine and
phenylbutyrate in treating patients who have acute myeloid leukemia or myelodysplastic
syndrome.
OBJECTIVES:
- Determine the safety and toxicity of azacitidine in combination with phenylbutyrate in
patients with recurrent, refractory, or untreated acute myeloid leukemia or
myelodysplastic syndrome.
- Determine the minimal effective pharmacologic dose of azacitidine required to
consistently inhibit DNA methyltransferase in this patient population.
- Obtain preliminary clinical and/or laboratory data suggesting potential therapeutic
activity of this combination regimen in these patients.
OUTLINE: This is a dose deescalation study of azacitidine.
Patients receive azacitidine subcutaneously daily on days 1-5 and 29-33 followed by
phenylbutyrate IV continuously on days 5-12 and 33-40. Treatment continues for at least 2
courses in the absence of disease progression. Patients with responsive disease may receive
an additional 2 months of therapy.
Cohorts of 3-6 patients receive deescalating doses of azacitidine until the minimal
effective pharmacologic dose (MEPD) is determined. The MEPD is defined as the dose above the
dose at which more than 1 of 6 patients do not meet the target enzyme inhibition of greater
than 90%.
Once the MEPD and toxicity have been established for a 5 day schedule, daily dose schedule
of azacitidine is increased to 10, 14, and 21 days, followed by phenylbutyrate for 7 days.
Courses are repeated every 28 days.
PROJECTED ACCRUAL: Approximately 32 patients will be accrued for this study within 2 years.
;
Primary Purpose: Treatment
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