Eye Diseases, Hereditary
20
4
9
6
Key Insights
Highlights
Success Rate
67% trial completion
Clinical Risk Assessment
Based on trial outcomes
High Risk
Score: 59/100
15.0%
3 terminated out of 20 trials
66.7%
-19.8% vs benchmark
5%
1 trials in Phase 3/4
50%
3 of 6 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 6 completed trials
Clinical Trials (20)
Natural History of PRPF31 Mutation-Associated Retinal Dystrophy
Study to Evaluate Ultevursen in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene
Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)
Rate of Progression of PCDH15-Related Retinal Degeneration in Usher Syndrome 1F
A Repeat-Dose, Open-Label, Two Arm Safety and Efficacy Study of Two Doses of VP-001 Administered Intravitreally in Participants With Confirmed PRPF31 Mutation-Associated Retinal Dystrophy, Including Participants Previously Treated With VP001
Inherited Retinal Degenerative Disease Registry
Non-Interventional Long Term Follow-up Study of Participants Previously Enrolled in the RESTORE Study
Extension Study to Study PQ-110-001 (NCT03140969)
Study to Evaluate the Efficacy Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene With Early to Moderate Vision Loss (Celeste)
Study to Evaluate the Efficacy Safety and Tolerability of Ultevursen in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene (Sirius)
Long-Term Follow-Up Gene Therapy Study for Leber Congenital Amaurosis OPTIRPE65 (Retinal Dystrophy Associated With Defects in RPE65)
Efficacy and Safety of MCO-010 Optogenetic Therapy in Adults With Retinitis Pigmentosa [RESTORE]
Single Ascending Dose Study in Participants With LCA10
Natural History Study of CEP290-Related Retinal Degeneration
A Study to Evaluate the Safety and Tolerability of QR-1123 in Subjects With Autosomal Dominant Retinitis Pigmentosa Due to the P23H Mutation in the RHO Gene
Study to Evaluate Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene
A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE)
Doxycycline Treatment in Mild Thyroid-Associated Ophthalmopathy
Efficacy of Subantimicrobial Dose Doxycycline for Moderate to Severe and Active Graves' Orbitopathy
Fundus Changes in the Microphthalmy Eyes