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Mitapivat in Pediatric Pyruvate Kinase Deficiency (Not Regularly Transfused)
2024-515025-28-00Phase 3Authorised
- Sponsor
- Agios Pharmaceuticals
- Start
- Jul 2024
- Modality
- Small Molecules
- Countries
- 8
01
Overview
Phase 3, randomised, double-blind trial in pyruvate kinase deficiency (PKD) comparing Mitapivat with Placebo (2 arms). Primary endpoint: Hemoglobin response. Planned start Jul 2024; status: authorised.
02
Drugs
mitapivat
03
Conditions
pyruvate kinase deficiency (PKD)
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