NCT03559699

A Study Evaluating the Efficacy and Safety of AG-348 in Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD)

An Open-Label Study To Evaluate the Efficacy and Safety of AG-348 in Regularly Transfused Adult Subjects With Pyruvate Kinase (PK) Deficiency

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 27 (actual)
• Sponsor: Agios Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

Study AG348-C-007 was a multicenter study designed to evaluate the efficacy and safety of treatment with AG-348 in a minimum of 20, with up to 40, participants with pyruvate kinase (PK) deficiency, who were regularly receiving blood transfusions. The study was composed of two parts. During Part 1, Dose Optimization Period, participants started on a dose of 5 mg AG-348 administered twice daily. Over the course of Part 1 each participant's dose of AG-348 was sequentially increased to 20 mg twice a day, followed by 50 mg twice a day depending on their tolerance. During Part 2, Fixed-Dose Period, participants received AG-348 at their optimized dose from Part 1.

Conditions

• Pyruvate Kinase Deficiency
• Anemia, Hemolytic

Interventions

Type	Name	Description
DRUG	AG-348	Part 1 (Dose Optimization Period): Participants began by receiving 5 mg orally, BID. Each participant's dose of AG-348 was sequentially increased to 20 mg BID followed by 50 mg BID depending on their response to AG-348 and their tolerance. Part 2 (Fixed Dose Period): Optimized dose determined in Part 1.

Timeline

• Start date: 2018-06-26
• Primary completion: 2020-11-12
• Completion: 2020-11-12
• First posted: 2018-06-18
• Last updated: 2022-01-04
• Results posted: 2021-12-10

Locations

19 sites across 9 countries: United States, Canada, Denmark, France, Ireland, Italy, Netherlands, Thailand, United Kingdom

Regulatory

• FDA-regulated drug study