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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 · Status: NOT_YET_RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 54

Last updated 2026-04-08

No results posted yet for this study

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

DRUG

Mitapivat Matched Placebo

Tablets or Granules

DRUG

Mitapivat

Tablets or Granules

Sponsors & Collaborators

  • Agios Pharmaceuticals, Inc.

    lead INDUSTRY

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
1 Year
Max Age
17 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-09-30
Primary Completion
2029-06-30
Completion
2032-06-30
FDA Drug
Yes

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07506863 on ClinicalTrials.gov