What is mitapivat studying for pediatric thalassemia?

Mitapivat is being studied to see if it can reduce the need for blood transfusions in children with transfusion-dependent alpha- or beta-thalassemia. This research aims to evaluate the drug's effects on transfusion burden and overall safety in this young population. The study is sponsored by Agios Pharmaceuticals, Inc.

Who may be eligible for this transfusion-dependent thalassemia clinical trial?

Children aged 1 to under 18 years with a confirmed diagnosis of alpha- or beta-thalassemia who require regular blood transfusions may be eligible. Participants must meet specific transfusion frequency and health criteria and be willing to follow study procedures. Please review eligibility criteria for more details.

Is there any cost to participate in this transfusion-dependent thalassemia clinical trial?

Study-related care, including the study drug or placebo and all trial visits, is provided at no cost. However, personal travel expenses may vary and are not guaranteed to be covered. Participants should plan accordingly for any travel needs.

Actively Recruiting

Phase 3

Age: 1Year - 17Years

All Genders

ID07506863

Phase 3 Study of Mitapivat in Children With Transfusion-Dependent Alpha- or Beta-Thalassemia to Assess Effectiveness and Safety

Led by Agios Pharmaceuticals, Inc. · Updated on 2026-08-03

Participants Needed

Research Sites

156 weeks

Total Duration

AI-Summary

What this Trial Is About

This clinical trial focuses on pediatric participants aged 1 to under 18 years with transfusion-dependent alpha- or beta-thalassemia. The study aims to compare the effect of the drug mitapivat against a placebo on reducing the burden of blood transfusions in these young patients. It is a Phase 3, randomized, double-blind study designed to evaluate efficacy, pharmacokinetics, and safety in this population. Participants are assigned to one of two groups one receives oral mitapivat twice daily, with doses adjusted by age and weight, and the other receives a matching placebo twice daily. Enrollment occurs sequentially by age cohorts 12 to under 18 years, 6 to under 12 years, and 1 to under 6 years. The treatment period lasts 48 weeks during the double-blind phase. Participants who complete this phase may continue receiving mitapivat in an open-label extension for up to 144 additional weeks. Throughout the study, participants will undergo assessments including tracking transfusion reductions, serum iron and related blood markers, hormone levels and sexual maturation in older children, growth measurements, bone density, and safety monitoring of adverse events. Pharmacokinetic sampling of mitapivat levels also occurs. The trial continues through 2032, with regular visits and evaluations to monitor treatment effects and participant health over time.

CONDITIONS

Brief Title

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)

Who Can Participate

Age: 1Year - 17Years

All Genders

Eligibility Criteria

You may qualify if you...

Written informed consent/assent provided by participant or guardian before study procedures
Aged 1 to under 18 years and weighing at least 7 kilograms at consent
Documented diagnosis of transfusion-dependent alpha- or beta-thalassemia
History of 6 to 20 transfusion episodes and no transfusions for 6 weeks within 24 weeks before randomization
Stable hydroxyurea dose for 16 weeks if currently taking
Female participants who have begun menstruation must agree to abstain from pregnancy-risk sexual activity or use two forms of contraception during study and for 28 days after last dose

You will not qualify if you...

Pregnant or breastfeeding
History of hemoglobin S or hemoglobin C
Prior gene therapy or bone marrow/stem cell transplantation
Conditions affecting sexual maturation other than thalassemia
Current treatment with luspatercept or hematopoietic stimulating agents within 36 weeks
History of malignancy within 5 years except certain skin or cervical cancers
Active or uncontrolled heart or lung disease, or relevant QT prolongation within 6 months
Liver disease including cirrhosis, symptomatic gallbladder disease, or drug-induced hepatitis
Renal dysfunction with eGFR below 60 mL/min/1.73 m2
High triglycerides over 215 mg/dL
Active infection requiring systemic antimicrobial therapy at consent
Active hepatitis B, hepatitis C, or HIV infection
Major surgery within 6 months or planned during study
Recent participation in other clinical trials involving investigational treatments
Use of strong CYP3A4/5 inhibitors or inducers without required washout
Use of anabolic steroids without required washout; stable testosterone therapy allowed
Allergy or contraindication to mitapivat or its excipients
Any condition or therapy posing unacceptable risk or confounding data interpretation
Institutionalization by regulatory or court order, or conditions causing undue influence

Research Team

Agios Medical Affairs

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