RecruitingTransfusion-dependent Alpha-ThalassemiaTransfusion-dependent Beta-Thalassemia
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)
Eligible age
1–17 yrs
Accepts
All genders
Locations
3 states
Healthy volunteers
No
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About this study
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.
Sponsor: Agios Pharmaceuticals, Inc.
You may qualify if…
- ✓ Written informed consent/assent from the participant (or their legally authorized representative, parent(s), or legal guardian) must be obtained before any study-related procedures are conducted and participants must be willing to comply with all study procedures for the duration of the study.
- ✓ Aged 1 to \<18 years and weighing at least 7 kilograms (kg) at the time of providing informed consent/assent.
- ✓ Documented diagnosis of thalassemia (β-thalassemia with or without α-globin gene mutations, hemoglobin E (HbE)/β-thalassemia, or α-thalassemia/hemoglobin H (HbH) disease) based on Hemoglobin (Hb) electrophoresis, Hb high-performance liquid chromatography, and/or DNA analysis from the participant's medical record. If this information is not available from the participant's medical record, the test(s) can be performed by a local laboratory during the Screening Period. If a local laboratory is unable to perform the test(s), results from the comprehensive α- and β-globin genotyping performed by the study central laboratory can be used.
- ✓ Transfusion dependent, defined as 6 to 20 transfusion episodes (also referred to as "transfusion events") and a ≤6-week transfusion-free period during the 24-week period before randomization.
- ✓ If taking hydroxyurea, the hydroxyurea dose must be stable for ≥16 weeks before randomization.
- ✓ Female participants who have attained menarche must be abstinent of sexual activities that may induce pregnancy as part of their usual lifestyle, or agree to use 2 forms of contraception, 1 of which must be considered highly effective, from the time of informed consent/assent, throughout the study, and for 28 days after the last dose of study drug. The second form of contraception can include an acceptable barrier method.
You may not qualify if…
- ✕ Pregnant or breastfeeding.
- ✕ Documented history of homozygous or heterozygous hemoglobin S (HbS) or hemoglobin C (HbC).
- ✕ Prior exposure to gene therapy or prior bone marrow or stem cell transplantation, including any prior exposure to myeloablative chemotherapy.
- ✕ Any conditions other than thalassemia expected to affect sexual maturation.
- ✕ Currently receiving treatment with luspatercept; the last dose must have been administered ≥36 weeks before randomization.
- ✕ Currently receiving treatment with hematopoietic stimulating agents; the last dose must have been administered ≥36 weeks before randomization.
- ✕ History of malignancy (active or treated) ≤5 years before providing informed consent/assent, except for nonmelanomatous skin cancer in situ, cervical carcinoma in situ, or breast carcinoma in situ.
- ✕ History of active and/or uncontrolled cardiac or pulmonary disease or clinically relevant QT prolongation within 6 months before providing informed consent/assent.
Where it's recruiting
Source: ClinicalTrials.gov · NCT07506863 · last updated 2026-08-27