G. Garcia-Manero, S. Luger, P. Venugopal
Nov 19, 2010
Citations
0
Influential Citations
4
Citations
Quality indicators
Journal
Blood
Abstract
Abstract 1857 This study is registered with ClinicalTrials.gov, number NCT00590187. Background: Sapacitabine is an orally administered nucleoside analogue with a unique ability to cause irreparable single-strand DNA breaks and induce G2 cell cycle arrest. We are conducting a multi-center, randomized phase 2 study to evaluate 3 dose regimens of this drug in older patients with MDS refractory to hypomethylating agents. The primary endpoint is 1-year survival. Secondary endpoints include the rate of CR, CRp, PR, CRi or hematological improvement (HI) and their corresponding durations. The study uses a selection design to identify a dose regimen which produces a better 1-year survival rate in the event that all three dose regimens are active. The planned sample size consists of 60 patients, 20 patients in each arm. Methods: Eligible patients must be ≥60 years with intermediate-2 or higher risk MDS previously treated with hypomethylating agents, ECOG 0–2, and adequate renal and hepatic functions. Patients were randomized 1:1:1 to receive sapacitabine every 3 –4 weeks at 200 mg b.i.d . × 7 days (Arm A), 300 mg b.i.d . × 7 days (Arm B), or 400 mg b.i.d . × 3 days per week × 2 weeks (Arm C). The number of cycles is not limited. Results: As of July 2010, 61 patients were treated with mean follow-up of 230 days. Median age was 73 years and 77% of patients were 70 or older. Baseline bone marrow contained Conclusions: Sapacitabine appears to be safe across all 3 dose regimens with the highest overall response rates observed on the 7-day dose regimens. Results for the primary endpoint of 1-year survival will be presented at the meeting. Disclosures: Wetzler: Cyclacel: Research Funding. Claxton: Cyclacel: Research Funding. Foran: Cyclacel: Research Funding. Goldberg: Cyclacel: Research Funding; Cyclacel: Research Funding. Chiao: Cyclacel: Employment, Equity Ownership. Kantarjian: Cyclacel: Research Funding.