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

Sponsor
Agios Pharmaceuticals, Inc.
Study ID
NCT07517133
Phase
PHASE3
Status
Not Yet Recruiting

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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

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

Start date
Sep 30, 2026
Status verified
May 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