The MDS study is designed as a protocol amendment expanding the ongoing Phase II trial of sapacitabine in elderly patients with acute myeloid leukemia (AML) to include a cohort of patients with myelodysplastic syndromes (MDS).
Cyclacel is currently enrolling patients in an open-label, multicenter, randomized Phase II trial of oral sapacitabine in elderly patients with AML who are previously untreated or in first relapse.
Based on encouraging safety and efficacy data from the elderly AML patients, Cyclacel has amended the protocol to include a cohort of MDS patients who have been previously treated with hypomethylating agents. Following the protocol amendment the trial will enroll a total of approximately 120 patients in two separate strata, AML and MDS, with approximately 60 patients in each stratum.
As with the original Phase II study in elderly patients with AML, the primary objective of the MDS stratum is to evaluate the one-year survival rate of three dosing schedules of sapacitabine. The study uses a selection design with the objective of identifying a dosing schedule which produces a better one year survival rate for each stratum in the event that all three dosing schedules are active.
Spiro Rombotis, president and CEO of Cyclacel, said: “Expanding our ongoing AML study to include an MDS stratum is a rapid and cost effective strategy to obtain Phase II data in an important second indication. We believe that this study design enhances the commercial prospects of sapacitabine as it expands the drug’s potential market.”