Mitapivat for Thalassemia

(ENERGIZE Trial)

Recruiting at 108 trial locations

Overseen By Medical Affairs

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Agios Pharmaceuticals, Inc.

Must not be taking: Luspatercept, Hematopoietic stimulants, others

Disqualifiers: Pregnancy, Sickle cell, Malignancy, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

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

Compensation: Varies

Number of Visits: Unknown

194 Participants Needed

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.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop all current medications, but if you are taking hydroxyurea, your dose must be stable for at least 16 weeks before joining. If you are on strong CYP3A4/5 inhibitors or inducers, or anabolic steroids, you must stop them for a certain period before starting the trial.

Research Team

Medical Affairs

Principal Investigator

Agios Pharmaceuticals, Inc.

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.

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.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Double-Blind Treatment

Participants receive either mitapivat or placebo, orally, twice daily for 24 weeks

24 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-Label Extension

Participants may opt into continuation of mitapivat treatment for up to 5 years

Up to 5 years

Treatment Details

Interventions

• Mitapivat
• Placebo Matching Mitapivat

Trial Overview 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.

Participant Groups

Group I: Mitapivat Experimental Treatment 1 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: Placebo Placebo Group 2 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.

Who Is Running the Clinical Trial?

Agios Pharmaceuticals, Inc.

Lead Sponsor

Trials

55

Recruited

4,200+