NCT02476916

A Study of AG-348 in Adult Participants With Pyruvate Kinase (PK) Deficiency

A Phase 2, Open Label, Randomized, Dose Ranging, Safety, Efficacy, Pharmacokinetic and Pharmacodynamic Study of AG-348 in Adult Patients With Pyruvate Kinase Deficiency

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 52 (actual)
• Sponsor: Agios Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

Study AG348-C-003 is a multicenter study designed to evaluate the safety and efficacy of different dose levels of AG-348 (mitapivat) in participants with PK deficiency.

Detailed description

This is a Phase 2, open label, two arm, multicenter, randomized, dose-ranging study during which adult participants with PK deficiency will receive multiple doses of AG-348 for up to 24 weeks (Core Period); eligible participants may enter an Extension Period to receive AG-348 for up to 8 additional years. Data will be reviewed on a regular basis and study design, dose and schedule will be adapted based on these reviews. The study will evaluate the safety and tolerability of multiple doses of AG-348, pharmacokinetic and pharmacodynamic (PD) profile of AG-348 and early indicators of clinical efficacy.

Conditions

• Pyruvate Kinase Deficiency

Interventions

Type	Name	Description
DRUG	AG-348	Participants with PK deficiency were randomized to either receive AG-348, 50 or 300 mg, as initial doses, BID for the Core Period (Week 24). At the Week 24 visit, Core Period participants who had safely tolerated AG-348 and demonstrated clinical activity in response to AG-348 were potentially eligible to immediately roll over to the Extension Period for continued treatment. Participants were assigned to initial doses, however, over the course of the core period were treated across a range of doses due to treatment emergent adverse events (AEs) and hemoglobin (Hb) levels exceeding mid-point of sex-adjusted ranges.

Timeline

• Start date: 2015-06-26
• Primary completion: 2017-05-08
• Completion: 2025-04-02
• First posted: 2015-06-22
• Last updated: 2025-05-02
• Results posted: 2020-06-11

Locations

13 sites across 6 countries: United States, Canada, France, Italy, Netherlands, United Kingdom

Regulatory

• FDA-regulated drug study