PEPN2312: A Phase 1 study of GRN163L (Imetelstat, IND# 170891, NSC# 754228) in combination with fludarabine and cytarabine for patients with acute myeloid leukemia that is in second or greater relapse or that is refractory to relapse therapy; myelodysplastic syndrome or juvenile myelomonocytic leukemia in first or greater relapse or is refractory to relapse therapy
PEPN2312
What is the goal of the study?
This is a phase I study of imetelstat, a topoisomerase inhibitor, in combination with fludarabine and cytarabine chemotherapy for 2nd or greater relapsed and refractory AML, as well as MDS and JMML that is in 1st or greater relapse or refractory. Telomerase can be highly expressed in some cancers, including AML. Preclinical studies have demonstrated that targeting this with telomerase inhibitors can be an effective strategy. Imetelstat has been used in adult MDS and has been found to have a higher rate of transfusion independence compared to placebo. It has been evaluated in pediatric patients at higher doses than DL1 here. Imetelstat will be given on days 1 and 8 of the regimen with FLA givens days 2-6 and disease response after 28 days. Patients may receive up to 2 cycles of this therapy.
Who can participate in the study?
Please contact the study team listed below to learn more.