Phase I Study of the Combination of 5-azacytidine With Valproic Acid and All-trans Retinoic Acid in Patients With High Risk Myelodysplastic Syndrome and Acute Myelogenous Leukemia
Participants receive 5-aza as an injection under the skin once a day for 7 days. This will
be repeated every 3-8 weeks depending on blood counts and how well bone marrow is
recovering. This is defined as 1 treatment cycle. Also during each cycle, participant will
take VPA by mouth for 7 days and ATRA by mouth for 5 days. VPA will be given on the same
days as 5-aza. ATRA will start on Day 3.
In the Phase I portion of the study, the dose of VPA will be increased in each new group of
participants until the highest safe dose is found. A minimum of 3 participants and a maximum
of 10 will be treated at each dose level.
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Maximal tolerated dose (MTD) of valproic acid (VPA) in combination with 5-azacytidine (5-aza) and all-trans retinoic acid (ATRA)
MTD defined as the dose level below where either 0 dose limiting toxicities (DLTs) out of the first 3 participants, or 1 DLT in the first 3 participants, and 0 DLTs in following additional 3 participants of a cohort. The MTD designation will apply to cycle 1 (28 day cycle). Routine blood tests (about 1-2 teaspoons each time) 2-3 times a week.
28 day cycle
Guillermo Garcia-Manero, MD
M.D. Anderson Cancer Center
United States: Institutional Review Board
2004-0799 Phase I
|The University of Texas M.D. Anderson Cancer Center||Houston, Texas|