Mitapivat for Thalassemia
(ENERGIZE Trial)

Recruiting in Palo Alto (17 mi)

+108 other locations

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Waitlist Available

Sponsor: Agios Pharmaceuticals, Inc.

Pivotal Trial (Near Approval)

Prior Safety Data

Trial Summary

What is the purpose of this trial?This trial is testing mitapivat, a drug that may help improve anemia by making red blood cells healthier and last longer. It targets patients with a specific type of thalassemia who don't need regular blood transfusions but still suffer from anemia.

Eligibility Criteria

This trial is for individuals with alpha- or beta-non-transfusion dependent thalassemia (NTDT) who have stable hemoglobin levels ≤10.0 g/dL and haven't had more than 5 blood transfusions in the past 24 weeks. Participants must not be pregnant, breastfeeding, or have certain other health conditions like uncontrolled heart disease, severe kidney issues, or active infections.

Inclusion Criteria

I have not needed more than 5 blood transfusions in the last 6 months and none in the last 2 months.

I have been diagnosed with thalassemia based on specific blood tests or DNA analysis.

My hydroxyurea dose has been the same for at least 16 weeks.

+3 more

Exclusion Criteria

Participants with any condition(s) that could create undue influence (including but not limited to incarceration, involuntary psychiatric confinement, and financial or familial affiliation with the Investigator or Sponsor)

Current enrollment or past participation (≤12 weeks before administration of the first dose of study drug or a timeframe equivalent to 5 half-lives of the investigational study drug, whichever is longer) in any other clinical study involving an investigational treatment or device

I do not have any health conditions that my doctor thinks would make this study unsafe for me.

+18 more

Participant Groups

The study is testing Mitapivat against a placebo to see if it helps improve anemia in people with NTDT. Patients will either receive the actual drug or a placebo that has no therapeutic effect to compare outcomes between the two groups.

2Treatment groups

Experimental Treatment

Placebo Group

Group I: MitapivatExperimental Treatment1 Intervention

Mitapivat 100 milligrams (mg), orally, twice daily (BID) for 24 weeks in double blind (DB) period and for up to 5 years in open label extension (OLE) period.

Group II: PlaceboPlacebo Group2 Interventions

Placebo matching mitapivat, orally, BID for 24 weeks in double blind period followed by Mitapivat 100 mg, orally, BID for up to 5 years in open label extension period.