Mitapivat for Thalassemia
(ENERGIZE-T Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
The primary objective of this study was to compare the effect of mitapivat versus placebo on transfusion burden in participants with α- or β-transfusion-dependent thalassemia.
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. Some medications, like strong CYP3A4/5 inhibitors or inducers, must be stopped for a certain period before joining.
Is mitapivat safe for humans?
What data supports the effectiveness of the drug Mitapivat for Thalassemia?
Research shows that Mitapivat, a drug that activates pyruvate kinase (an enzyme important for energy production in red blood cells), has been effective in increasing hemoglobin levels in adults with non-transfusion-dependent α-thalassemia or β-thalassemia, suggesting it could help manage these conditions.12356
Who Is on the Research Team?
Medical Affairs
Principal Investigator
Agios Pharmaceuticals, Inc.
Are You a Good Fit for This Trial?
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
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Double-Blind Treatment
Participants receive either mitapivat or placebo orally, twice daily for 48 weeks
Follow-up
Participants are monitored for safety and effectiveness after treatment
Open-label Extension
Participants may opt into continuation of mitapivat treatment for up to 5 years
What Are the Treatments Tested in This Trial?
Interventions
- Mitapivat
- Placebo Matching Mitapivat
Find a Clinic Near You
Who Is Running the Clinical Trial?
Agios Pharmaceuticals, Inc.
Lead Sponsor