Last updated on August 2019

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


Brief description of study

Study AG348-C-007 is a multicenter study designed to evaluate the efficacy and safety of treatment with AG-348 in a minimum of 20, with up to 40, subjects with pyruvate kinase deficiency (PKD), who are regularly receiving blood transfusions. The study is comprised of two parts. During the Part 1 Dose Optimization Period of the study, all participants will start on a dose of 5 mg AG-348 administered twice daily. Over the course of Part 1 each participant's dose will be optimized individually, up to a maximum dose of 50 milligrams (mg), twice daily. During the Part 2 Fixed-Dose Period, participants will receive AG-348 at their optimized dose from Part 1.

Clinical Study Identifier: NCT03559699

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Stanford University

Palo Alto, CA United States
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Emory University

Atlanta, GA United States
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H pital de la Timone

Marseille, Cedex 5, France
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Addenbrooke's Hospital

Cambridge, United Kingdom
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Manchester Royal Infirmary

Manchester, United Kingdom
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