Minnelide in Adult Patients With Relapsed or Refractory Acute Myeloid Leukemia
Status:
Withdrawn
Trial end date:
2022-04-01
Target enrollment:
Participant gender:
Summary
Minnelide, a water-soluble disodium salt variant of triptolide, is a diterpenoid heat shock
protein 70 (HSP70) inhibitor. Studies using AML cell lines, primary patient samples, and
mouse transplant models demonstrate that Minnelide has potent cell killing effects. Minnelide
has already been developed for human use and given to patients in a phase I trial for
gastrointestinal (GI) cancers. Given the clinical safety profile and preliminary activity
described in human GI cancers, the low-nanomolar anti-leukemic potency of triptolide in
vitro, and that minnelide doses predicted to be significantly below the maximum tolerated
dose (MTD) in human GI cancers decreased leukemia burden in animal models, the investigators
propose a phase I trial in acute myeloid leukemia (AML).