A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)
Summary
The primary objective of this study was to compare the effect of mitapivat versus placebo on transfusion burden in participants with α- or β-transfusion-dependent thalassemia.
Timeline
- Start
- 2021-11-30
- Primary completion
- 2024-04-11
- Completion
- 2029-06
Outcome
Met primary endpoint
registry analysis (superiority test); Mitapivat vs Placebo; p = 0.0003; Common Risk Difference on Response Rate 17.6 (95% CI 8.0 to 27.2); Mantel Haenszel NCT04770779 ↗
release “the global Phase 3 ENERGIZE-T study of mitapivat in adults with transfusion-dependent (TD) alpha- or beta-thalassemia achieved its primary endpoint of transfusion reduction response.” agios.com ↗
release “ENERGIZE-T Study Achieved Primary and All Key Secondary Endpoints in Adult Patients with Transfusion-Dependent Alpha- or Beta-Thalassemia” agios.com ↗
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Mitapivat | Small molecule | 100 mg | Oral |