Early research (Phase 1)Study completedNCT04105166What this trial is testingGene Therapy for Pyruvate Kinase Deficiency (PKD)Who this might be right forPyruvate Kinase Deficiency Rocket Pharmaceuticals Inc. 4
Large-scale testing (Phase 3)Active Not RecruitingNCT05175105What this trial is testingEvaluate 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 PeriodWho this might be right forPediatric Pyruvate Kinase DeficiencyPediatric Hemolytic Anemia Agios Pharmaceuticals, Inc. 30
Large-scale testing (Phase 3)Active Not RecruitingNCT05144256What this trial is testingEvaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Regularly Transfused, Followed by a 5-Year Extension PeriodWho this might be right forPediatric Pyruvate Kinase DeficiencyPediatric Hemolytic Anemia Agios Pharmaceuticals, Inc. 49