AG-348 for Pyruvate Kinase Deficiency

Recruiting at 12 trial locations

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Agios Pharmaceuticals, Inc.

Must be taking: Folic acid

Must not be taking: CYP3A4 inhibitors, CYP3A4 inducers

Disqualifiers: Blood disorders, Heart failure, Diabetes, others

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

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 24 weeks

52 Participants Needed

Trial Summary

What is the purpose of this trial?

Study AG348-C-003 is a multicenter study designed to evaluate the safety and efficacy of different dose levels of AG-348 (mitapivat) in participants with PK deficiency.

Eligibility Criteria

Adults with Pyruvate Kinase (PK) Deficiency who are not pregnant or breastfeeding, have a hemoglobin level below set thresholds, and haven't had recent blood transfusions can join. They must be in stable health, not on certain medications, and agree to use contraception. Those with psychiatric disorders or other conditions that could affect study participation are excluded.

Inclusion Criteria

You have been diagnosed with pyruvate kinase deficiency through a specific test.

My hemoglobin level is low, below 12 g/dL if I'm male, or below 11 g/dL if I'm female.

I had my spleen removed over 6 months ago and my vaccinations are current.

Exclusion Criteria

Any other medical or psychological condition deemed by the Investigator to be likely to interfere with a patient's ability to participate in the study

You have a mental health condition that might make it hard for you to take part in the study.

Your blood has high levels of bilirubin for reasons other than a specific type of genetic condition or breakdown of red blood cells.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive multiple doses of AG-348 for up to 24 weeks

24 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Extension

Eligible participants may enter an Extension Period to receive AG-348 for up to 8 additional years

Up to 8 years

Treatment Details

Interventions

• AG-348

Trial Overview The trial is testing the safety and effectiveness of AG-348 (mitapivat) at various doses for treating PK deficiency. It's a multicenter study where participants receive different amounts of this medication to see how well it works and what side effects occur.

Participant Groups

Group I: AG-348 50 mg BID Experimental Treatment 1 Intervention

Participants with PK deficiency received AG-348, 50 milligrams (mg), as initial dose, twice daily (BID) for the Core Period (Week 24).

Group II: AG-348 300 mg BID Experimental Treatment 1 Intervention

Participants with PK deficiency received AG-348, 300 mg, as initial dose, BID for the Core Period (Week 24).

Who Is Running the Clinical Trial?

Agios Pharmaceuticals, Inc.

Lead Sponsor

Trials

55

Recruited

4,200+