Condition
Zellweger Syndrome
Total Trials
6
Recruiting
1
Active
2
Completed
3
Success Rate
75.0%-12% vs avg
Key Insights
Highlights
Success Rate
75% trial completion
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 37/100
Termination Rate
16.7%
1 terminated out of 6 trials
Success Rate
75.0%
-11.5% vs benchmark
Late-Stage Pipeline
17%
1 trials in Phase 3/4
Results Transparency
67%
2 of 3 completed with results
Key Signals
2 with results75% success
Data Visualizations
Phase Distribution
4Total
Not Applicable (1)
P 2 (2)
P 3 (1)
Trial Status
Completed3
Terminated1
Recruiting1
Active Not Recruiting1
Trial Success Rate
75.0%
Benchmark: 86.5%
Based on 3 completed trials
Clinical Trials (6)
Showing 6 of 6 trials
NCT02171104Phase 2Active Not Recruiting
MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
NCT02699190Completed
LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies
NCT03047369Recruiting
The Myelin Disorders Biorepository Project
NCT00007020Phase 3Completed
Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid
NCT03856866Phase 2CompletedPrimary
Hydroxychloroquine Administration for Reduction of Pexophagy
NCT00004442Not ApplicableTerminated
Study of Bile Acids in Patients With Peroxisomal Disorders
Showing all 6 trials