Study of PTC299 (Emvododstat) in Relapsed/Refractory Acute Leukemias
Status:
Recruiting
Trial end date:
2022-05-31
Target enrollment:
Participant gender:
Summary
This is an open-label, non-randomized, Phase 1b study to evaluate the safety,
pharmacokinetics (PK) profiles, and preliminary evidence of antitumor activity of PTC299 and
the metabolite, O-desmethyl PTC299, in participants with relapsed/refractory acute myeloid
leukemia (AML) who have exhausted standard available therapies known to provide clinical
benefit. The study is designed as a series of cohort-based dose escalations. For each cohort,
a minimum of 3 evaluable participants with PK and safety data will be assessed. Additional
participants will be recruited if additional PK data are needed to assess mean exposure based
on the observed variability.