Clinical Trial

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

Active, Not Recruiting Phase 2
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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 includes a core period (up to 24 weeks) followed by an extension period (up to 10 years) for eligible participants. 20 participants with NTDT were enrolled. The initial dose of AG-348 was 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.
Protocol Amendment History 65 amendments
This ClinicalTrials.gov record has been amended 65 times since 2018-09-28; most recent amendment 2026-03-27.
Status change: Recruiting → Active, Not Recruiting 2020-03-10
Status change: Not Yet Recruiting → Recruiting 2018-12-12
Trial Details
NCT Number NCT03692052
Lead Sponsor Agios Pharmaceuticals, Inc.
Conditions Thalassemia
Enrollment 20 participants
Start Date 2019-03-20
Primary Completion 2020-08-20 (estimated)
Study Completion 2030-09-30 (estimated)
Updated on ClinicalTrials.gov 2026-03-30