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Mitapivat versus placebo in regularly transfused children with pyruvate kinase deficiency
NL-OMON60495Phase 3Completed
- Sponsor
- Agios Pharmaceuticals
- Start
- Apr 2023
- 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: Transfusion reduction response. Started Apr 2023; status: completed.
02
Drugs
mitapivat
03
Conditions
pyruvate kinase deficiency (PKD)
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