Phase 1B Study of PTC299 in Relapsed/Refractory Acute Leukemias
What is the purpose of this trial?
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.
- Trial withPTC Therapeutics, Inc.
- Start Date08/24/2021
- End Date05/29/2020
- Last Updated08/25/2021
- Study HIC#2000024928