Pyruvate Kinase Deficiency
13
0
2
8
Key Insights
Highlights
Success Rate
89% trial completion (above average)
Clinical Risk Assessment
Based on trial outcomes
Low Risk
Score: 17/100
7.7%
1 terminated out of 13 trials
88.9%
+2.4% vs benchmark
31%
4 trials in Phase 3/4
50%
4 of 8 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 8 completed trials
Clinical Trials (13)
A Study of AG-348 in Adult Participants With Pyruvate Kinase (PK) Deficiency
Pyruvate Kinase Deficiency Global Longitudinal Registry
Clinical Trial to Evaluate the Efficacy of Gene Therapy for Pyruvate Kinase Deficiency
A Study to Provide Continued Access to Mitapivat for Participants Who Previously Completed an Agios-Sponsored Mitapivat Study
Acquired Pyruvate Kinase Deficiency In Clonal Myeloid Neoplasms
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