# 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** · PHASE3 · NOT_YET_RECRUITING · sponsor: **Agios Pharmaceuticals, Inc.** · enrollment: 54 (estimated)

## Conditions studied

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

## Interventions

- **DRUG:** Mitapivat Matched Placebo
- **DRUG:** Mitapivat

## Key facts

- **NCT ID:** NCT07506863
- **Lead sponsor:** Agios Pharmaceuticals, Inc.
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE3
- **Study type:** INTERVENTIONAL
- **Status:** NOT_YET_RECRUITING
- **Start date:** 2026-09
- **Primary completion:** 2029-06
- **Final completion:** 2032-06
- **Target enrollment:** 54 (ESTIMATED)
- **Last updated:** 2026-04-08


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT07506863

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT07506863, "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)". Retrieved via AI Analytics 2026-06-02 from https://api.ai-analytics.org/clinical/NCT07506863. Licensed CC0.

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*[Clinical trials dataset](/datasets/clinical-trials) · CC0 1.0*