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A Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Not Regularly Transfused, Followed by a 5-Year Extension Period
Condition: Pediatric Pyruvate Kinase Deficiency · Pediatric Hemolytic Anemia · Sponsor: Agios Pharmaceuticals, Inc.
About this study
Study ACTIVATE-Kids (AG348-C-023) will evaluate the efficacy and safety of orally administered mitapivat as compared with placebo in pediatric participants with pyruvate kinase deficiency (PKD) who are not 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). Participants will be dosed by age and weight during a double-blind period consisting of an 8-week dose titration period followed by a 12-week fixed-dose period. Participants who complete the double-blind period will be eligible to receive mitapivat for up to 5 years in the open-label extension (OLE) period.
This description comes directly from the study's public registry record.
Talk to the study team
No contact information is available for this study as per the public registry record.
Always discuss trial participation with your own doctor first.
Locations (19)
| Stanford Medicine | Palo Alto, California, United States | — |
| Children's Hospital Colorado | Aurora, Colorado, United States | — |
| Children's Healthcare of Atlanta - Emory | Atlanta, Georgia, United States | — |
| UChicago Medicine | Chicago, Illinois, United States | — |
| Boston Children's Hospital | Boston, Massachusetts, United States | — |
| Children's Hospital of Michigan | Detroit, Michigan, United States | — |
| Duke University Medical Center | Durham, North Carolina, United States | — |
| Children's Hospital of Philadelphia | Philadelphia, Pennsylvania, United States | — |
| St Jude's Children's Research Hospital | Memphis, Tennessee, United States | — |
| UT Southwestern Medical Center | Dallas, Texas, United States | — |
| Texas Children's Hospital | Houston, Texas, United States | — |
| Centre hospitalier Universitaire de Sainte-Justine | Montreal, Quebec, Canada | — |
+ 7 more locations — full list on the registry record.
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Source record: clinicaltrials.gov/study/NCT05175105