Mitapivat for Thalassemia
(ENERGIZE-T Trial)

Recruiting in Palo Alto (17 mi)

+112 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?The primary purpose of this study is to compare the effect of mitapivat versus placebo on transfusion burden in participants with transfusion-dependent alpha- or beta-thalassemia (TDT).

Eligibility Criteria

This trial is for people with transfusion-dependent alpha- or beta-thalassemia who need regular blood transfusions. They must have a confirmed diagnosis, stable treatment if using hydroxyurea, and agree to use effective contraception if applicable. Excluded are those with certain medical conditions, recent treatments that could interfere, or risks that might affect study results.

Inclusion Criteria

I have needed 6 to 20 blood transfusions in the last 6 months without going more than 6 weeks without one.

Women of childbearing potential (WOCBP) must be abstinent of sexual activities that may induce pregnancy as part of their usual lifestyle or agree to use two forms of contraception, one of which must be considered highly effective, from the time of providing informed consent, throughout the study, and for 28 days after the last dose of study drug. The second form of contraception can be an acceptable barrier method

I have been diagnosed with thalassemia based on DNA analysis.

+2 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).

Pregnant, breastfeeding, or parturient

My kidney function is reduced, with an eGFR less than 45 mL/min/1.73m^2.

+18 more

Participant Groups

The trial is testing the effectiveness of Mitapivat versus a placebo in reducing the need for blood transfusions in patients with thalassemia. Participants will be randomly assigned to receive either Mitapivat or a placebo to compare outcomes between the two groups.

2Treatment groups

Experimental Treatment

Placebo Group

Group I: MitapivatExperimental Treatment1 Intervention

Double-Blind Period: Participants will receive mitapivat 100 milligrams (mg) orally, twice daily (BID) for 48 weeks. Open-label Extension Period: Participants who do not discontinue study drug may choose to continue to receive mitapivat for up to an additional 5 years after the Double-blind Period.

Group II: PlaceboPlacebo Group2 Interventions

Double-Blind Period: Participants will receive placebo matching mitapivat orally, BID for 48 weeks. Open-label Extension Period: Participants who do not discontinue study drug may choose to receive mitapivat for up to an additional 5 years after the Double-blind Period.