A Phase 2 Study of Suberoylanilide Hydroxamic Acid (SAHA) in Acute Myeloid Leukemia (AML)
I. Determine the toxicity and the proportion of complete remissions associated with two
different treatment schedules of vorinostat (SAHA) in patients with acute myeloid leukemia.
I. Determine the toxic effects of SAHA in this study population. II. Examine for preliminary
evidence of re-expression of silenced genes in leukemic blasts in response to SAHA.
OUTLINE: This is a multicenter, randomized study. Patients are stratified according to
disease status (relapsed vs untreated). Patients are randomized to 1 of 2 treatment arms.
ARM A: Patients receive oral vorinostat (SAHA) once a day on days 1-21. In both arms,
treatment repeats every 21 days for up to 17 courses in the absence of disease progression
or unacceptable toxicity.
ARM B: Patients receive oral SAHA three times a day on days 1-14. In both arms, treatment
repeats every 21 days for up to 17 courses in the absence of disease progression or
After completion of study treatment, patients are followed periodically for up to 2 years.
PROJECTED ACCRUAL: A total of 44 patients will be accrued for this study.
Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
Confirmed Complete Response (CR) Rate
The confirmed complete response rate was estimated by the number of participants with CR divided by the total number of evaluable participants. According to the International Working Group (IWG) Criteria for response in AML, to be considered a CR, the following must be met for at least 4 weeks: ANC > 1500/mL, platelets > 100000/mL, no circulating blasts, bone marrow cellularity >20% (biopsy), trilineage maturation, < 5% bone marrow blasts, no auer rods and no extramedullary disease.
Up to 2 years
United States: Food and Drug Administration
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