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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
A Study to Provide Access to Mitapivat for Participants Who Previously Participated in an Agios-Sponsored Mitapivat StudyCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, anemia, familial hemolytic anemia, hematologic disorder, hemolytic anemia, hereditary disease, inherited hemoglobinopathy, pyruvate kinase deficiency of red cells, sickle cell disease, thalassemiaEnrolling by invitationPhase 42026-09-29NCT05777993
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 PeriodHemic and Lymphatic Diseases, Nutritional and Metabolic Diseases, anemia, hematologic disorder, metabolic disease, pyruvate kinase deficiency of red cellsActive, not recruitingPhase 32026-09-28NCT05144256
Pyruvate Kinase Deficiency Global Longitudinal RegistryCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, anemia, congenital nonspherocytic hemolytic anemia, familial hemolytic anemia, hematologic disorder, hemolytic anemia, hereditary disease, pyruvate kinase deficiency of red cellsActive, not recruitingNot stated2026-09-28NCT03481738
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 PeriodHemic and Lymphatic Diseases, Nutritional and Metabolic Diseases, anemia, hematologic disorder, metabolic disease, pyruvate kinase deficiency of red cellsActive, not recruitingPhase 32026-08-27NCT05175105
High-Dose Vitamin C in G6PDA and Pyruvate Kinase Deficiency: A Safety StudyCongenital, Hereditary, and Neonatal Diseases and Abnormalities, G6PD deficiency, Hemic and Lymphatic Diseases, Nutritional and Metabolic Diseases, anemia, familial hemolytic anemia, hematologic disorder, hemolytic anemia, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, metabolic disease, pyruvate kinase deficiency of red cellsNot yet recruitingPhase 12026-05-28NCT07612345
RADeep Multicenter European Epidemiological Platform for Patients Diagnosed With Rare Anemia Disorders (RADs)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, Hemolysis, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, alpha thalassemia spectrum, anemia, beta thalassemia, bone marrow disorder, congenital dyserythropoietic anemia, cystinosis, familial hemolytic anemia, hematologic disorder, hemolytic anemia, hereditary anemia, hereditary disease, inborn errors of metabolism, inherited aplastic anemia, inherited hemoglobinopathy, iron metabolism disease, lysosomal storage disease, metabolic disease, myelodysplastic syndrome, pyruvate kinase deficiency of red cells, sickle cell disease, sideroblastic anemia, thalassemiaRecruitingNot stated2024-01-19NCT06213402