NCT05144256

A Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Regularly Transfused, Followed by a 5-Year Extension Period

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 Regularly Transfused, Followed by a 5-Year Open-label Extension Period

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 49 (actual)
• Sponsor: Agios Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 1 Year – 17 Years
• Healthy volunteers: Not accepted

Summary

ACTIVATE-KidsT (AG348-C-022) is a multicenter study designed to evaluate the efficacy and safety of treatment with mitapivat compared with placebo in pediatric participants with pyruvate kinase deficiency (PK deficiency) who are 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) and splenectomy status. Participants will be dosed by age and weight during a double-blind period consisting of an 8-week dose titration period followed by a 24-week fixed-dose period. Participants who complete the double-blind period will be eligible to receive mitapivat in the open-label extension (OLE) period.

Conditions

• Pediatric Pyruvate Kinase Deficiency
• Pediatric Hemolytic Anemia

Interventions

Type	Name	Description
DRUG	Mitapivat	Tablets or granules
DRUG	Mitapivat-matching placebo	Tablets or granules

Timeline

• Start date: 2022-06-08
• Primary completion: 2024-05-03
• Completion: 2029-06-01
• First posted: 2021-12-03
• Last updated: 2026-03-30

Locations

22 sites across 10 countries: United States, Canada, Czechia, Denmark, Germany, Netherlands, Spain, Switzerland, Turkey (Türkiye), United Kingdom

Regulatory

• FDA-regulated drug study