A Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Regularly Transfused, Followed by a 5-Year Extension Period - Join Clinical Trial NCTNCT05144256

How to Join This Clinical Trial - NCTNCT05144256

Learn how to participate in this PHASE3 trial studying an investigational therapy for Anemia, Hemolytic, Anemia, Hemolytic, Congenital Nonspherocytic, Pyruvate Metabolism, Inborn Errors. Current status: ACTIVE_NOT_RECRUITING.

Am I Eligible for This Clinical Trial?

This clinical research study is looking for participants with Anemia, Hemolytic, Anemia, Hemolytic, Congenital Nonspherocytic, Pyruvate Metabolism, Inborn Errors. Eligibility requirements include specific age ranges, health status, and medical history. Contact the study team to learn if you qualify.

Condition Being Studied: Anemia, Hemolytic, Anemia, Hemolytic, Congenital Nonspherocytic, Pyruvate Metabolism, Inborn Errors
Treatment Being Tested: Investigational treatment
Study Phase: PHASE3 - Large-scale efficacy study
Enrollment Status: ACTIVE_NOT_RECRUITING
Study Identifier: NCTNCT05144256 - ClinicalTrials.gov Identifier
Sponsored By: Study sponsor

What to Expect as a Participant

Participating in this clinical trial involves:

Initial screening to determine eligibility
Regular study visits and health assessments
Receiving the study treatment or placebo
Medical monitoring and follow-up care
Contributing to medical research that may help others
Potential access to new treatments before they're widely available

Clinical Trial Benefits and Compensation

Participants in this clinical research study may receive:

Close medical monitoring by healthcare professionals
Access to potential new treatments
Compensation for time and travel (varies by study)
No-cost study-related medical care
The opportunity to help advance medical knowledge