Mitapivat for Pyruvate Kinase Deficiency
(ACTIVATE-KidsT Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial is testing mitapivat, a medication that may help children with a blood disorder called pyruvate kinase deficiency. These children often need frequent blood transfusions. Mitapivat aims to improve the function of their red blood cells, potentially reducing the need for these transfusions.
Will I have to stop taking my current medications?
The trial requires that you stop taking certain medications before participating. Specifically, you must stop taking strong inhibitors or inducers of CYP3A4, anabolic steroids, and any pyruvate kinase activators for a specified period before joining the study. You must also continue taking folic acid supplements during the study.
Is Mitapivat safe for humans?
What makes the drug Mitapivat unique for treating pyruvate kinase deficiency?
What data supports the effectiveness of the drug Mitapivat for treating pyruvate kinase deficiency?
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 kids with Pyruvate Kinase Deficiency who need regular blood transfusions. They must be between 1-18 years old, not pregnant or breastfeeding, and without serious heart, liver, or kidney issues. Participants should have a history of receiving multiple transfusions and agree to use contraception if applicable.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Dose Titration
Participants undergo an 8-week dose titration period to optimize dosing based on age and weight
Fixed-dose Treatment
Participants receive a fixed dose of mitapivat or placebo for 24 weeks
Open-label Extension
Participants who complete the double-blind period may continue receiving mitapivat in an open-label extension period
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Mitapivat
- Mitapivat-matching placebo
Find a Clinic Near You
Who Is Running the Clinical Trial?
Agios Pharmaceuticals, Inc.
Lead Sponsor