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Study of PTC299 in Relapsed/Refractory Acute Leukemias

Research Question:
What is 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?

Basic Study Information

Purpose:
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.

Location: University of Rochester Medical Center
Study Reference #: ILEU18153

Lead Researcher (Principal Investigator)

Lead Researcher:  Michael Becker, MD

Study Contact Information

Study Coordinator: Haley Misch
Phone: (585) 275-9485
Email: Haley_Misch@URMC.Rochester.edu

Additional Study Details

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