AG-348 for Thalassemia

Recruiting in Palo Alto (17 mi)

+3 other locations

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: Agios Pharmaceuticals, Inc.

No Placebo Group

Prior Safety Data

Trial Summary

What is the purpose of this trial?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.

Eligibility Criteria

Adults with non-transfusion-dependent thalassemia (NTDT) who haven't had more than 5 blood transfusions in the last 24 weeks and none in the past 8 weeks. Participants must have a documented history of thalassemia, adequate organ function, and agree to use two forms of contraception if applicable. Exclusions include prior treatments like gene therapy or certain drugs, recent major surgery, or serious allergies.

Inclusion Criteria

Informed consent

My average hemoglobin level is 10 g/dL or less, based on two tests.

I haven't needed more than 5 blood transfusions in the last 6 months and none in the last 2 months.

+6 more

Exclusion Criteria

Currently pregnant or breastfeeding

Exposure to any investigational drug, device, or procedure within 3 months prior to the first day of study drug

I am scheduled for a spleen removal soon or had one within the last year.

+12 more

Participant Groups

The trial is testing AG-348's effectiveness for NTDT over up to 10 years. Starting with a dose of 50 mg twice daily, which may increase to 100 mg based on safety and hemoglobin levels after six weeks. The study aims to understand how well AG-348 works and its effects on the body.

1Treatment groups

Experimental Treatment

Group I: AG-348Experimental Treatment1 Intervention

Participants with alpha or beta thalassemia received AG-348 50 mg twice daily (BID), orally up to Week 6. Following Week 6, depending on the participants' safety and hemoglobin (Hb) concentrations, they could undergo one potential dose-level increase from 50 to 100 mg BID. After completion of the Core Period of 24 weeks, participants were eligible to continue to receive AG-348 in the Extension Period which is up to 10 years.