Overview

A Study to Evaluate Efficacy and Safety of AG-348 in Not Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD)

Status:
Completed
Trial end date:
2020-10-09
Target enrollment:
Participant gender:
Summary
Study AG348-C-006 will evaluate the efficacy and safety of orally administered AG-348 as compared with placebo in participants with pyruvate kinase deficiency (PKD), who are not regularly receiving blood transfusions. Participants will be randomized 1:1 to receive either AG-348 or matching placebo. The study is comprised of two parts. During the Part 1 Dose Optimization Period of the study, participants will start on a dose of 5 mg AG-348 administered twice daily. Over the course of Part 1, each participant's dose of AG-348 may be sequentially increased to 20 mg twice a day, followed by 50 mg twice a day depending on their tolerance. During the Part 2 Fixed-Dose Period, participants will receive AG-348 at their optimized dose from Part 1.
Phase:
Phase 3
Details
Lead Sponsor:
Agios Pharmaceuticals, Inc.