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20 Participants Needed

AG-348 for Thalassemia

Recruiting at 3 trial locations

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Agios Pharmaceuticals, Inc.

Must not be taking: CYP3A4 inhibitors, P-gp inhibitors

Disqualifiers: Hb S or C thalassemia, Pregnancy, others

No Placebo GroupAll trial participants will receive the active study treatment (no placebo)

Prior Safety DataThis treatment has passed at least one previous human trial

Trial Summary

Will I have to stop taking my current medications?

You may need to stop taking certain medications before joining the trial. Specifically, if you are on medications that strongly affect certain liver enzymes (CYP3A4) or P-glycoprotein, or if you are taking digoxin, you must stop them at least 5 days before starting the study drug. Also, if you are on chronic anticoagulant therapy, anabolic steroids, or hematopoietic stimulating agents, you need to be on a stable dose for a specific period before starting the trial.

How does the drug AG-348 differ from other treatments for thalassemia?

AG-348 is unique because it targets the underlying cause of thalassemia by enhancing the activity of pyruvate kinase, an enzyme important for red blood cell energy production, which is different from traditional treatments like blood transfusions and iron chelation that manage symptoms rather than the root cause.¹ ² ³ ⁴ ⁵

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.

Research Team

Medical Affairs

Principal Investigator

Agios Pharmaceuticals, Inc.

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.

See 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.

See 12 more

Timeline

Screening

Participants are screened for eligibility to participate in the trial

Up to 4 weeks

Core Treatment

Participants receive AG-348 50 mg BID, with a potential increase to 100 mg BID based on safety and hemoglobin levels

24 weeks

Weekly visits for dose adjustment and monitoring

Extension

Eligible participants continue to receive AG-348 for long-term evaluation

Up to 10 years

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

AG-348

Trial Overview 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.

Participant Groups

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.

Find a Clinic Near You

Who Is Running the Clinical Trial?

Agios Pharmaceuticals, Inc.

Lead Sponsor

Trials

Recruited

4,200+

Findings from Research

In a study of 220 transfusion-dependent beta-thalassemia patients, those treated with the oral iron chelator deferasirox (Exjade®) showed significantly higher compliance and convenience compared to those receiving the standard treatment, deferoxamine (DFO).

While both treatments had comparable efficacy, nearly 45% of patients on DFO reported irritation and pain at the injection site, highlighting Exjade® as a potentially preferable option for iron chelation therapy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Compliance and satisfaction with deferasirox (Exjade®) compared with deferoxamine in patients with transfusion-dependent beta-thalassemia.Haghpanah, S., Zarei, T., Zahedi, Z., et al.[2022]