Phase 2
N=15
Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA)
Leber Congenital Amaurosis
Bottom Line
View on ClinicalTrials.gov: NCT02781480 ↗Enrolled (actual)
15
Serious AEs
20.0%
Results posted
Apr 2021
Primary outcome: Primary: Overall Safety of Adeno-Associated Virus Vector (AAV-OPTIRPE65) - Number of Participants With a Safety Event — 0; 0; 1; 0 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- AAV RPE65 (Biological)
- Age
- Pediatric, Adult, Older Adult · 3+ yrs
- Sex
- All
- Sponsor
- MeiraGTx UK II Ltd
- Primary completion
- Dec 2018
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 | — |
Summary
A clinical trial of AAV2/5 vector for patients with Defects in RPE65
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.