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

Conditions

• Non-Transfusion-dependent Alpha-Thalassemia
• Non-Transfusion-dependent Beta-Thalassemia

Eligibility Criteria

Sex

ALL

Age

1 Year - 17 Years

Healthy Volunteers

Not accepted

Interventions

• Mitapivat — DRUG
  Tablets or Granules
• Placebo Matching Mitapivat — DRUG
  Tablets or Granules

Study Details

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.

Key Dates

First listed

Apr 8, 2026

Start date

Sep 30, 2026

Status verified

Jul 2026

Primary completion

Mar 31, 2029

Completion

Mar 31, 2032

Study Design

Enrollment

45 participants (estimated)

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Arms

• Experimental: Mitapivat
  Participants will receive oral mitapivat twice daily (BID), with the dose determined by age and weight, for 24 weeks during the double blind (DB) period. Enrollment is conducted sequentially by age cohort, beginning with the oldest cohort. Cohort 1: 12 to \<18 years Cohort 2: 6 to \<12 years Cohort 3: 1 to \<6 years Participants who complete the DB period may continue receiving mitapivat in the open label extension (OLE) period for up to 144 weeks.
• Placebo Comparator: Placebo
  Participants will receive oral placebo matching mitapivat, administered BID, with dosing based on age and weight, for 24 weeks during the DB period. Enrollment is conducted sequentially by age cohort, beginning with the oldest cohort. Cohort 1: 12 to \<18 years Cohort 2: 6 to \<12 years Cohort 3: 1 to \<6 years Participants who complete the DB period may transition to receive mitapivat in the OLE period for up to 144 weeks.

Primary Outcome Measure

Percentage of Participants Who Achieved a Hemoglobin (Hb) Response [ Time Frame: Baseline, Week 12 through Week 24 ]

Central Contacts

• Agios Medical Affairs
  833-228-8474
  [email protected]

Locations (1)

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