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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
Conditions
Interventions
Mitapivat
Mitapivat-matching placebo
Locations
22
United States
Phoenix Children's Hospital
Phoenix, Arizona, United States
Stanford Medicine
Palo Alto, California, United States
Children's Healthcare of Atlanta - Emory
Atlanta, Georgia, United States
Boston Children's Hospital
Boston, Massachusetts, United States
Cure 4 the Kids Foundation, A Division of Roseman University of Health Sciences
Las Vegas, Nevada, United States
Weill Cornell Medical College
New York, New York, United States
Start Date
June 8, 2022
Primary Completion Date
May 3, 2024
Completion Date
June 1, 2029
Last Updated
April 24, 2026
Lead Sponsor
Agios Pharmaceuticals, Inc.
Data Source & Attribution
This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.
Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.
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