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

Conditions

Leukemia, other

Phase I

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 with
    PTC Therapeutics, Inc.
  • Start Date
    08/24/2021
  • End Date
    05/29/2020

I'm interested in volunteering

If you would prefer to contact a member of the Help us Discover team about this trial and other similar trials, please email helpusdiscover@yale.edu or call 877.978.8343

  • Last Updated
    08/25/2021
  • Study HIC
    #2000024928