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
Post-approval studies (Phase 4)Enrolling By InvitationNCT05777993What this trial is testingProvide Continued Access to Mitapivat for Participants Who Previously Completed an Agios-Sponsored Mitapivat StudyWho this might be right forPyruvate Kinase DeficiencyAnemia, Hemolytic Agios Pharmaceuticals, Inc. 6
Not applicableActive Not RecruitingNCT03481738What this trial is testingPyruvate Kinase Deficiency Global Longitudinal RegistryWho this might be right forPyruvate Kinase Deficiency Agios Pharmaceuticals, Inc. 500
Not applicableStudy completedNCT02053480What this trial is testingPyruvate Kinase Deficiency Natural History StudyWho this might be right forPyruvate Kinase DeficiencyCongenital Non-Spherocytic Hemolytic Anemia Boston Children's Hospital 254
Large-scale testing (Phase 3)Study completedNCT03559699What this trial is testingA Study Evaluating the Efficacy and Safety of AG-348 in Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD)Who this might be right forPyruvate Kinase DeficiencyAnemia, Hemolytic Agios Pharmaceuticals, Inc. 27
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)Study completedNCT03548220What this trial is testingEvaluate Efficacy and Safety of AG-348 in Not Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD)Who this might be right forPyruvate Kinase DeficiencyAnemia, Hemolytic Agios Pharmaceuticals, Inc. 80
Not applicableStudy completedNCT04995315What this trial is testingPyruvate Kinase Deficiency Global Longitudinal Registry Substudy of Protocol AG348-C-008Who this might be right forPyruvate Kinase Deficiency Agios Pharmaceuticals, Inc. 13
Not applicableActive Not RecruitingNCT04902833What this trial is testingAcquired Pyruvate Kinase Deficiency In Clonal Myeloid NeoplasmsWho this might be right forPyruvate Kinase DeficiencyPyruvate Kinase Deficiency AnemiaHereditary Hemolytic Anemia+8 more Massachusetts General Hospital 100
Not applicableEnded earlyNCT04964323What this trial is testingPyruvate Kinase (PK) Deficiency Global Longitudinal Registry: Patient-Reported Outcomes (PRO)Who this might be right forPyruvate Kinase DeficiencyPyruvate Kinase Deficiency AnemiaAnemia+1 more Agios Pharmaceuticals, Inc. 15
Not applicableLooking for participantsNCT06213402What this trial is testingRADeep Multicenter European Epidemiological Platform for Patients Diagnosed With Rare Anemia Disorders (RADs)Who this might be right forSickle Cell DiseaseThalassemiaHemolytic; Anemia, Hereditary, Due to Enzyme Disorder+6 more Hospital Universitari Vall d'Hebron Research Institute 32,564
Testing effectiveness (Phase 2)Study completedNCT02476916What this trial is testingAG-348 in Adult Participants With Pyruvate Kinase (PK) DeficiencyWho this might be right forPyruvate Kinase Deficiency Agios Pharmaceuticals, Inc. 52
Not applicableStudy completedNCT03866590What this trial is testingPyruvate Kinase Deficiency Epidemiological Study (PIECE)Who this might be right forPyruvate Kinase Deficiency CENTOGENE GmbH Rostock 75
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