Clinical Trial

A Study to Provide Access to Mitapivat for Participants Who Previously Participated in an Agios-Sponsored Mitapivat Study

Enrolling by Invitation Phase 4
View on ClinicalTrials.gov →
Summary
The primary objective of the study is to provide continued access to participants who are deriving clinical benefit from ongoing mitapivat treatment in an Agios-sponsored mitapivat study, in the opinion of the Investigator.
Protocol Amendment History 3 changes
critical Primary endpoint(s) modified 2026-08-13
notable Enrollment increased: 6 -> 59 participants 2026-08-13
notable Trial sites expanded: 4 -> 23 locations 2026-08-13
Trial Details
NCT Number NCT05777993
Lead Sponsor Agios Pharmaceuticals, Inc.
Conditions Pyruvate Kinase Deficiency, Anemia, Hemolytic, Sickle Cell Disease, Thalassemia
Enrollment 59 participants
Start Date 2023-07-06
Primary Completion 2029-08 (estimated)
Study Completion 2029-08 (estimated)
Updated on ClinicalTrials.gov 2026-08-12