A Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Not Regularly Transfused, Followed by a 5-Year Extension Period

Study ACTIVATE-Kids (AG348-C-023) will evaluate the efficacy and safety of orally administered mitapivat as compared with placebo in pediatric participants with pyruvate kinase deficiency (PKD) who are not regularly receiving blood transfusions. Participants will be randomized 2:1 to receive either mitapivat or matching placebo. Randomization will be stratified by age (1 to \< 6 years, 6 to \< 12 years, 12 to \< 18 years). Participants will be dosed by age and weight during a double-blind period consisting of an 8-week dose titration period followed by a 12-week fixed-dose period. Participants who complete the double-blind period will be eligible to receive mitapivat for up to 5 years in the open-label extension (OLE) period.

NCT Number	NCT05175105
Lead Sponsor	Agios Pharmaceuticals, Inc.
Conditions	Pediatric Pyruvate Kinase Deficiency, Pediatric Hemolytic Anemia
Enrollment	30 participants
Start Date	2022-06-06
Primary Completion	2024-12-13 (estimated)
Study Completion	2030-01 (estimated)
Updated on ClinicalTrials.gov	2026-07-30