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Clinical Trial NCT05175105

AG348-C-023: A Phase 3, Multicenter, Randomized, Double-blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Subjects With Pyruvate Kinase Deficiency Who Are Not Regularly Transfused, Followed by a 5-Year Open-label Extension Period

  • ClinicalTrials.gov ID: NCT05175105
  • Recruiting participants (Starts Sep. 1, 2022)
  • Not accepting healthy volunteers
  • UTSW Principal Investigator: KATHRYN ELYSE DICKERSON

summary

This is a Phase 3, multicenter, randomized, double-blind, placebo-controlled study designed to evaluate the efficacy and safety of mitapivat versus placebo in pediatric subjects with PK deficiency who are not regularly transfused.

objective

To determine the efficacy of treatment with mitapivat compared with placebo, as assessed by the increase in Hb concentrations in pediatric subjects with PK deficiency who are not regularly transfused, followed by a 5 year open label extension period to evaluate long term safety and efficacy.

Participate

If you are interested in this clinical trial, please contact Laurie Rodgers-Augsutyniak on the Children’s Health Research Team.Call 214-456-6095Email