Trials / Not Yet Recruiting
Not Yet RecruitingNCT07506863
A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)
A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study Evaluating the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Subjects With α- or β- Transfusion-Dependent Thalassemia
- Status
- Not Yet Recruiting
- Phase
- Phase 3
- Study type
- Interventional
- Enrollment
- 54 (estimated)
- Sponsor
- Agios Pharmaceuticals, Inc. · Industry
- Sex
- All
- Age
- 1 Year – 17 Years
- Healthy volunteers
- Not accepted
Summary
The primary objective of this study is to compare the effect of mitapivat versus placebo on transfusion burden in pediatric participants with α- or β-transfusion-dependent thalassemia.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | Mitapivat Matched Placebo | Tablets or Granules |
| DRUG | Mitapivat | Tablets or Granules |
Timeline
- Start date
- 2026-09-01
- Primary completion
- 2029-06-01
- Completion
- 2032-06-01
- First posted
- 2026-04-02
- Last updated
- 2026-04-08
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT07506863. Inclusion in this directory is not an endorsement.