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Results for “Pediatric Pyruvate Kinase Deficiency”

3 trials

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Showing 3 of 3 results

Early research (Phase 1)Study completedNCT04105166
What this trial is testing

Gene Therapy for Pyruvate Kinase Deficiency (PKD)

Who this might be right for
Pyruvate Kinase Deficiency
Rocket Pharmaceuticals Inc. 4
Large-scale testing (Phase 3)Active Not RecruitingNCT05175105
What this trial is testing

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

Who this might be right for
Pediatric Pyruvate Kinase DeficiencyPediatric Hemolytic Anemia
Agios Pharmaceuticals, Inc. 30
Large-scale testing (Phase 3)Active Not RecruitingNCT05144256
What this trial is testing

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

Who this might be right for
Pediatric Pyruvate Kinase DeficiencyPediatric Hemolytic Anemia
Agios Pharmaceuticals, Inc. 49