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Mitapivat in pediatric pyruvate kinase deficiency patients who are not regularly transfused
2021-003333-11Phase 3Active
- Sponsor
- Agios Pharmaceuticals
- Start
- Dec 2021
- Modality
- Small Molecules
- Countries
- 1
01
Overview
Phase 3, randomised, double-blind trial in pyruvate kinase deficiency (PKD) comparing Mitapivat with Placebo (2 arms). Primary endpoint: Hemoglobin response. Started Dec 2021; status: active.
02
Drugs
mitapivat
03
Conditions
pyruvate kinase deficiency (PKD)
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