Pyruvate Kinase Deficiency Clinical Trials

22 clinical trials for Pyruvate Kinase Deficiency across international registries, including 5 currently recruiting participants. Compare status, phase, eligibility criteria and locations.

• A Study to Provide Access to Mitapivat for Participants Who Previously Participated in an Agios-Sponsored Mitapivat Study — RECRUITING · PHASE4
• A Phase 3, Multicenter, Randomized, Double-blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Subjects With Pyruvate Kinase Deficiency Who Are Not Regularly Transfused, Followed by a 5-Year Open-label Extension Period — RECRUITING · PHASE3
• A ROLLOVER STUDY TO PROVIDE CONTINUED ACCESS TO MITAPIVAT FOR PARTICIPANTS WHO PREVIOUSLY COMPLETED AN AGIOS-SPONSORED MITAPIVAT STUDY — RECRUITING
• A PHASE 3, MULTICENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED STUDY TO EVALUATE THE EFFICACY AND SAFETY OF MITAPIVAT IN PEDIATRIC SUBJECTS WITH PYRUVATE KINASE DEFICIENCY WHO ARE REGULARLY TRANSFUSED, FOLLOWED BY A 5-YEAR OPEN-LABEL EXTENSION PERIOD — RECRUITING · PHASE3
• A PHASE 3, MULTICENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED STUDY TO EVALUATE THE EFFICACY AND SAFETY OF MITAPIVAT IN PEDIATRIC SUBJECTS WITH PYRUVATE KINASE DEFICIENCY WHO ARE NOT REGULARLY TRANSFUSED, FOLLOWED BY A 5-YEAR OPEN-LABEL EXTENSION PERIOD — RECRUITING · PHASE3
• Pyruvate Kinase Deficiency Global Longitudinal Registry — ACTIVE_NOT_RECRUITING
• High-Dose Vitamin C in G6PDA and Pyruvate Kinase Deficiency: A Safety Study — NOT_YET_RECRUITING · PHASE1
• Acquired Pyruvate Kinase Deficiency In Clonal Myeloid Neoplasms — COMPLETED
• A Study of AG-348 in Adult Participants With Pyruvate Kinase (PK) Deficiency — COMPLETED · PHASE2
• Clinical Trial to Evaluate the Efficacy of Gene Therapy for Pyruvate Kinase Deficiency — SUSPENDED · PHASE2
• A Phase 3, Multicenter, Randomized, Double-blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Subjects With Pyruvate Kinase Deficiency Who Are Regularly Transfused, Followed by a 5-Year Open-label Extension Period — TERMINATED · PHASE3
• Extension Study of AG-348 in Adult Participants With Pyruvate Kinase Deficiency Previously Enrolled in AG-348-006 or AG348-C-007 — COMPLETED · PHASE3
• Gene Therapy for Pyruvate Kinase Deficiency (PKD) — COMPLETED · PHASE1
• Pyruvate Kinase Deficiency Global Longitudinal Registry Substudy of Protocol AG348-C-008 — COMPLETED
• Pyruvate Kinase (PK) Deficiency Global Longitudinal Registry: Patient-Reported Outcomes (PRO) — TERMINATED
• Pyruvate Kinase Deficiency Epidemiological Study (PIECE) — COMPLETED
• A Study to Evaluate Efficacy and Safety of AG-348 in Not Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD) — COMPLETED · PHASE3
• A Study Evaluating the Efficacy and Safety of AG-348 in Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD) — COMPLETED · PHASE3
• Pyruvate Kinase Deficiency Natural History Study — COMPLETED
• AN OPEN-LABEL, MULTICENTER, EXTENSION STUDY OF AG 348 IN ADULT SUBJECTS WITH PYRUVATE KINASE DEFICIENCY PREVIOUSLY ENROLLED IN AG-348 STUDIES — COMPLETED
• A PHASE 3, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED STUDY TO EVALUATE THE EFFICACY AND SAFETY OF AG-348 IN NOT REGULARLY TRANSFUSED ADULT SUBJECTS WITH PYRUVATE KINASE DEFICIENCY — COMPLETED · PHASE3
• AN OPEN-LABEL STUDY TO EVALUATE THE EFFICACY AND SAFETY OF AG-348 IN REGULARLY TRANSFUSED ADULT SUBJECTS WITH PYRUVATE KINASE (PK) DEFICIENCY — COMPLETED

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