Leber Congenital Amaurosis 10
6
1
2
1
Key Insights
Highlights
Success Rate
50% trial completion
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 50/100
16.7%
1 terminated out of 6 trials
50.0%
-36.5% vs benchmark
17%
1 trials in Phase 3/4
100%
1 of 1 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 1 completed trials
Clinical Trials (6)
Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)
Extension Study to Study PQ-110-001 (NCT03140969)
Single Ascending Dose Study in Participants With LCA10
Natural History Study of CEP290-Related Retinal Degeneration
An Open-label, Dose Escalation and Double-masked, Randomized, Controlled Trial Evaluating Safety and Tolerability of Sepofarsen in Children (<8 Years of Age) With LCA10 Caused by Mutations in the CEP290 Gene.
A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE)