A Phase 3, Multicenter, Randomized, Double-blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Subjects With Pyruvate Kinase Deficiency Who Are Regularly Transfused, Followed by a 5-Year Open-label Extension Period

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTIS

Registry ID

CTIS2024-515024-37-00

Acronym

AG348-C-022

Enrollment

Registered

2024-08-12

Start date

2022-05-27

Completion date

Unknown

Last updated

2025-11-19

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Pyruvate Kinase Deficiency

Brief summary

Transfusion reduction response (TRR), defined as achievement of a ≥33% reduction in the total red blood cell (RBC) transfusion volume from Week 9 through Week 32 of the Double-blind Period normalized by weight and actual study drug duration compared with the historical transfusion volume standardized by weight and to 24 weeks

Interventions

DRUGMITAPIVAT

DRUGPlacebo for MITAPIVAT

Eligibility

Sex/Gender

All

Age

0 Years to 17 Years

Design outcomes

Primary

Measure	Time frame
Transfusion reduction response (TRR), defined as achievement of a ≥33% reduction in the total red blood cell (RBC) transfusion volume from Week 9 through Week 32 of the Double-blind Period normalized by weight and actual study drug duration compared with the historical transfusion volume standardized by weight and to 24 weeks	—

Countries

Czechia, Denmark, Netherlands, Spain

Outcome results

None listed

Source: EU CTIS · Data processed: Feb 4, 2026