Condition
Aspartylglucosaminuria
Total Trials
6
Recruiting
0
Active
1
Completed
2
Success Rate
50.0%-37% vs avg
Key Insights
Highlights
Success Rate
50% trial completion
Clinical Risk Assessment
Based on trial outcomes
High Risk
Score: 75/100
Termination Rate
33.3%
2 terminated out of 6 trials
Success Rate
50.0%
-36.5% vs benchmark
Late-Stage Pipeline
0%
0 trials in Phase 3/4
Results Transparency
100%
2 of 2 completed with results
Key Signals
2 with results50% success
Data Visualizations
Phase Distribution
4Total
P 1 (1)
P 2 (3)
Trial Status
Completed2
Terminated2
Active Not Recruiting1
Not Yet Recruiting1
Trial Success Rate
50.0%
Benchmark: 86.5%
Based on 2 completed trials
Clinical Trials (6)
Showing 6 of 6 trials
NCT07530796Phase 1Not Yet RecruitingPrimary
Safety and Efficacy of scAAV9/AGA Gene Therapy in Participants With Aspartylglucosaminuria (AGU)
NCT02171104Phase 2Active Not Recruiting
MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
NCT01891422CompletedPrimary
Longitudinal Studies of the Glycoproteinoses
NCT03853876TerminatedPrimary
A Natural History Study of Aspartylglucosaminuria
NCT01043640Phase 2Completed
Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders
NCT00668564Phase 2Terminated
Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism
Showing all 6 trials