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Recruiting Phase 3 Interventional

A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With α- or β-Non-Transfusion-Dependent Thalassemia

ClinicalTrials.gov ID: NCT07517133

Public ClinicalTrials.gov record NCT07517133. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.

ClinicalTrials.gov public records Last synced Sep 1, 2026, 7:42 PM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Official title

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-Non-Transfusion-Dependent Thalassemia

Brief summary

Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.

The primary objective of this study is to compare the effect of mitapivat versus placebo on anemia in pediatric participants with alpha- or beta-non-transfusion-dependent thalassemia.

Study identification

NCT ID
NCT07517133
Recruitment status
Recruiting
Study type
Interventional
Phase
Phase 3
Lead sponsor
Agios Pharmaceuticals, Inc.
Industry
Enrollment
45 participants

Conditions and interventions

Eligibility (public fields only)

Age range
1 Year to 17 Years
Sex
All
Healthy volunteers
Healthy volunteers not accepted

This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.

Study timeline

Start date
Aug 31, 2026
Primary completion
Feb 28, 2029
Completion
Feb 29, 2032
Last update posted
Aug 26, 2026

2026 – 2032

United States locations

U.S. sites
4
U.S. states
4
U.S. cities
4
Facility City State ZIP Site status
Stanford Medicine Palo Alto California 94304 Recruiting
Weill Cornell Medical College/ New York Presbyterian New York New York 10065 Recruiting
Children's Hospital of Philadelphia Philadelphia Pennsylvania 19104-4318 Recruiting
University of Texas Southwestern Medical Center Dallas Texas 75390 Recruiting

Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.

About this trial record page

What this page shows
Public field values for ClinicalTrials.gov record NCT07517133, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
What this page does not do
No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
Where the data comes from
Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
Last refresh
Last update posted Aug 26, 2026 · Synced Sep 1, 2026

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Open the official record

The complete protocol, eligibility criteria, and contact information for NCT07517133 live on ClinicalTrials.gov.

View official ClinicalTrials.gov record →