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)

Conditions

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

Eligibility Criteria

Sex

ALL

Age

1 Year - 17 Years

Healthy Volunteers

Not accepted

Interventions

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

Study Details

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.

Key Dates

First listed

Apr 2, 2026

Start date

Sep 30, 2026

Status verified

Jul 2026

Primary completion

Jun 30, 2029

Completion

Jun 30, 2032

Study Design

Enrollment

54 participants (estimated)

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Arms

• Experimental: Mitapivat
  Participants will receive oral mitapivat twice daily (BID), with dose determined by age and weight, for 48 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 48 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 Transfusion Reduction Response (TRR) Through Week 48 [ Time Frame: Baseline, through Week 48 ]

Central Contacts

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

Locations (1)

Find similar trials in Dallas, TX

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