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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
Condition: Pediatric Pyruvate Kinase Deficiency · Pediatric Hemolytic Anemia · Sponsor: Agios Pharmaceuticals, Inc.
About this study
ACTIVATE-KidsT (AG348-C-022) is a multicenter study designed to evaluate the efficacy and safety of treatment with mitapivat compared with placebo in pediatric participants with pyruvate kinase deficiency (PK deficiency) who are regularly receiving blood transfusions. Participants will be randomized 2:1 to receive either mitapivat or matching placebo. Randomization will be stratified by age (1 to \< 6 years, 6 to \< 12 years, 12 to \< 18 years) and splenectomy status. Participants will be dosed by age and weight during a double-blind period consisting of an 8-week dose titration period followed by a 24-week fixed-dose period. Participants who complete the double-blind period will be eligible to receive mitapivat in the open-label extension (OLE) period.
This description comes directly from the study's public registry record.
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No contact information is available for this study as per the public registry record.
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Locations (22)
| Phoenix Children's Hospital | Phoenix, Arizona, United States | — |
| Stanford Medicine | Palo Alto, California, United States | — |
| Children's Healthcare of Atlanta - Emory | Atlanta, Georgia, United States | — |
| Boston Children's Hospital | Boston, Massachusetts, United States | — |
| Cure 4 the Kids Foundation, A Division of Roseman University of Health Sciences | Las Vegas, Nevada, United States | — |
| Weill Cornell Medical College | New York, New York, United States | — |
| Children's Hospital of Philadelphia | Philadelphia, Pennsylvania, United States | — |
| UT Southwestern Medical Center | Dallas, Texas, United States | — |
| Texas Children's Hospital | Houston, Texas, United States | — |
| Comprehensive Hemophilia Care Clinic at CHEO (Children's Hospital Eastern Ontario) | Ottawa, Ontario, Canada | — |
| Fakultní Nemocnice Olomouc | Olomouc, Czechia | — |
| Aarhus University Hospital | Aarhus, Central Jutland, Denmark | — |
+ 10 more locations — full list on the registry record.
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Source record: clinicaltrials.gov/study/NCT05144256