Pyruvate Kinase Deficiency
14
0
1
9
Key Insights
Highlights
Success Rate
90% trial completion (above average)
Clinical Risk Assessment
Based on trial outcomes
Low Risk
Score: 17/100
7.1%
1 terminated out of 14 trials
90.0%
+3.4% vs benchmark
29%
4 trials in Phase 3/4
44%
4 of 9 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.6%
Based on 9 completed trials
Clinical Trials (14)
Pyruvate Kinase Deficiency Global Longitudinal Registry
A Study to Provide Continued Access to Mitapivat for Participants Who Previously Completed an Agios-Sponsored Mitapivat Study
High-Dose Vitamin C in G6PDA and Pyruvate Kinase Deficiency: A Safety Study
Acquired Pyruvate Kinase Deficiency In Clonal Myeloid Neoplasms
A Study of AG-348 in Adult Participants With Pyruvate Kinase (PK) Deficiency
Clinical Trial to Evaluate the Efficacy of Gene Therapy for Pyruvate Kinase Deficiency
Extension Study of AG-348 in Adult Participants With Pyruvate Kinase Deficiency Previously Enrolled in AG-348-006 or AG348-C-007
Gene Therapy for Pyruvate Kinase Deficiency (PKD)
Pyruvate Kinase Deficiency Global Longitudinal Registry Substudy of Protocol AG348-C-008
Pyruvate Kinase (PK) Deficiency Global Longitudinal Registry: Patient-Reported Outcomes (PRO)
Pyruvate Kinase Deficiency Epidemiological Study (PIECE)
A Study to Evaluate Efficacy and Safety of AG-348 in Not Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD)
A Study Evaluating the Efficacy and Safety of AG-348 in Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD)
Pyruvate Kinase Deficiency Natural History Study