Trial intelligence / Profile preview

Mitapivat in Pediatric Subjects With Pyruvate Kinase Deficiency

NCT05144256Phase 3

Key facts

Status: Active, not recruiting
Start: Jun 2022
Primary completion: May 2024
Countries: 10
Sponsor: Agios Pharmaceuticals
Modality: Small Molecules

Drugs: mitapivat
Conditions: pyruvate kinase deficiency (PKD)

Overview

Phase 3, randomised, double-blind trial in pyruvate kinase deficiency (PKD) comparing Mitapivat with Placebo (2 arms). Primary endpoint: Transfusion Reduction Response. Started Jun 2022; status: active, not recruiting.

Design

2 arms

Study design: RandomizedDouble-blind

Mitapivat
Participants receive Mitapivat orally twice daily, optimized through dose titration, followed by a fixed-dose period, and then continue Mitapivat in the OLE period.
Placebo
Participants receive matched placebo orally twice daily, optimized through dose titration, followed by a fixed-dose period, and then transition to Mitapivat in the OLE period.

Who it enrols

Pediatric patients aged 1 to <18 years with confirmed pyruvate kinase deficiency, receiving regular transfusions, and on folic acid supplementation.

Endpoints

Primary

Transfusion Reduction ResponseWeek 9 to Week 32

Secondary

Transfusion-free ResponseWeek 9 to Week 32
Number of Transfusion EpisodesWeek 9 to Week 32
Total Transfusion VolumeWeek 9 to Week 32
Normal Hemoglobin ResponseWeek 9 to Week 32
Estradiol ConcentrationBaseline up to Week 298
Estrone ConcentrationBaseline up to Week 298
Total Testosterone ConcentrationBaseline up to Week 298
Free Testosterone ConcentrationBaseline up to Week 298

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