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

NCT05175105 · clinicaltrials.gov ↗

PHASE3

Phase

ACTIVE_NOT_RECRUITING

Status

30

Enrollment

INDUSTRY

Sponsor class

Conditions

Pediatric Pyruvate Kinase Deficiency
Pediatric Hemolytic Anemia

Interventions

DRUG: Mitapivat
DRUG: Mitapivat-matching placebo

Sponsor

Agios Pharmaceuticals, Inc.