NCT04770753

A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT)

A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study Evaluating the Efficacy and Safety of Mitapivat in Subjects With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (ENERGIZE)

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 194 (actual)
• Sponsor: Agios Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The primary purpose of this study was to compare the effect of mitapivat versus placebo on hemolytic anemia in participants with alpha- or beta-non-transfusion dependent thalassemia (NTDT).

Detailed description

The mitapivat group included 130 participants whereas the placebo group had 64 participants.

Conditions

• Non-Transfusion-dependent Alpha-Thalassemia
• Non-Transfusion-dependent Beta-Thalassemia

Interventions

Type	Name	Description
DRUG	Placebo Matching Mitapivat	Tablets
DRUG	Mitapivat	Tablets

Timeline

• Start date: 2021-12-20
• Primary completion: 2023-11-13
• Completion: 2028-12-01
• First posted: 2021-02-25
• Last updated: 2026-03-30
• Results posted: 2025-01-24

Locations

68 sites across 18 countries: United States, Brazil, Bulgaria, Canada, Denmark, France, Greece, Italy, Lebanon, Malaysia, Netherlands, Saudi Arabia, Spain, Taiwan, Thailand, Turkey (Türkiye), United Arab Emirates, United Kingdom

Regulatory

• FDA-regulated drug study