Phase 2
Completed N=15
Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA)
Leber Congenital Amaurosis
Source: ClinicalTrials.gov NCT02781480 ↗
Enrolled (actual)
15
Serious AEs
20.0%
Results posted
Apr 2021
Primary outcomePrimary: Overall Safety of Adeno-Associated Virus Vector (AAV-OPTIRPE65) - Number of Participants With a Safety Event — 0; 0; 1; 0 Participants
Summary
A clinical trial of AAV2/5 vector for patients with Defects in RPE65
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Overall Safety of Adeno-Associated Virus Vector (AAV-OPTIRPE65) - Number of Participants With a Safety Event |
0; 0; 1; 0; 0; 1 | — |
Eligibility Criteria
Key Inclusion Criteria
- Aged 3 years or older
- Early-onset severe retinal dystrophy consistent with RPE65 deficiency
Key Exclusion Criteria
- Females who are pregnant or breastfeeding
- Have participated in another research study involving an investigational therapy for ocular disease within the last 6 months.
Data sourced from ClinicalTrials.gov (NCT02781480). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.