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)

NCT07506863 · ENERGIZEKids-T Phase 3Recruiting

Lead sponsor
Agios Pharmaceuticals, Inc. (Industry)
Enrollment
54 (estimated)
Start date
24 Sep 2026
Primary completion
Jun 2029 (estimated)
Study completion
1 Jun 2032
Last update posted
29 Oct 2026

Design

Study type
Interventional
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel
Masking
Quadruple
Arms
2

Interventions

  • Mitapivat Matched Placebo Drug
  • Mitapivat Drug

Conditions and primary outcome

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

Primary outcome

Percentage of Participants Who Achieved Transfusion Reduction Response (TRR) Through Week 48 (Baseline, through Week 48)

Posted results on ClinicalTrials.gov

Results posted on ClinicalTrials.gov: No

Results from company and journal sources

No editor-confirmed results are on record for this study.

Publications

No PubMed citation lists NCT07506863.

CT-VCA trial quality

About CT-VCA

Loading CT-VCA scores…

Data integrity

Compare what the registry, company releases, FDA documents and publications say about this study.

Drugs on HealthTrade

Source

Source: ClinicalTrials.gov, U.S. National Library of Medicine. Listing a study does not mean NLM has evaluated it.

Nothing on this page is medical or investment advice.