NCT03761069

Study of PTC299 (Emvododstat) in Relapsed/Refractory Acute Leukemias

Phase 1B Study of PTC299 in Relapsed/Refractory Acute Leukemias

• Status: Terminated
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 33 (actual)
• Sponsor: PTC Therapeutics · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Leukemia, Myeloid, Acute
• AML

Interventions

Type	Name	Description
DRUG	PTC299	PTC299 will be administered per the treatment arm description

Timeline

• Start date: 2018-10-29
• Primary completion: 2021-12-28
• Completion: 2021-12-28
• First posted: 2018-12-03
• Last updated: 2022-02-02

Locations

17 sites across 1 country: United States

Regulatory

• FDA-regulated drug study