Overview

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

Status:
Active, not recruiting
Trial end date:
2030-09-30
Target enrollment:
Participant gender:
Summary
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 core period (up to 24 weeks) followed by an extension period (up to 10 years) 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.
Phase:
Phase 2
Details
Lead Sponsor:
Agios Pharmaceuticals, Inc.