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
NCT05144256
Site status not listed — check the official record
- Conditions
- Pediatric Pyruvate Kinase Deficiency, Pediatric Hemolytic Anemia
- Interventions
- Mitapivat, Mitapivat-matching placebo
- Drug
- Lead sponsor
- Agios Pharmaceuticals, Inc.
- Industry
- Eligibility
- 1 Year to 17 Years
- Enrollment
- 49 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2029
- U.S. locations
- 9
- States / cities
- Phoenix, Arizona • Palo Alto, California • Atlanta, Georgia + 6 more