Last updated on July 2019

A Study to Determine the Efficacy Safety Pharmacokinetics and Pharmacodynamics of AG-348 in Adult Participants With Non-transfusion-dependent Thalassemia


Brief description of study

Study AG348-C-010 is a multicenter study to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of treatment with AG-348 in adult participants with non-transfusion-dependent thalassemia (NTDT). This study will include a 24-week core period followed by a 2-year extension period for eligible participants. Approximately 17 participants with NTDT will be enrolled. The initial dose of AG-348 will be 50 milligrams (mg) twice daily (BID) with one potential dose-level increase to 100 mg BID, at the Week 6 visit based on the participant's safety and hemoglobin (Hb) concentrations.

Clinical Study Identifier: NCT03692052

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