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Pediatric Pyruvate Kinase Deficiency research hub
2 indexed studies · 0 currently recruiting
- active not recruitingPHASE3
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
This study is for children aged 1 to 17 with a blood condition called Pyruvate Kinase Deficiency (PKD) who don't regularly need blood transfusions. It's testing a new medicine called mitapivat to see if it's safe and effective, compared to a dummy medicine.
United States · Canada · France - active not recruitingPHASE3
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
This study is looking at a new medicine called Mitapivat for children with a rare blood disorder called Pyruvate Kinase Deficiency (PKD). It aims to see if Mitapivat can help children who need regular blood transfusions, compared to a dummy medicine.
United States · Canada · Czechia