Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA)
- Conditions
- Leber Congenital Amaurosis
- Registration Number
- NCT02781480
- Lead Sponsor
- MeiraGTx UK II Ltd
- Brief Summary
A clinical trial of AAV2/5 vector for patients with Defects in RPE65
- Detailed Description
A dose escalation and dose expansion (Phase I/II) trial of adults and children with retinal dystrophy associated with defects in RPE65. ATIMP will be administered to one eye only in a single sub-retinal procedure
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 15
- Aged 3 years or older
- Early-onset severe retinal dystrophy consistent with RPE65 deficiency
Key
- Females who are pregnant or breastfeeding
- Have participated in another research study involving an investigational therapy for ocular disease within the last 6 months.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SEQUENTIAL
- Primary Outcome Measures
Name Time Method Overall Safety of Adeno-Associated Virus Vector (AAV-OPTIRPE65) - Number of Participants With a Safety Event 6 months Safety was defined as an advanced therapy investigational medicinal product (ATIMP) related:
* Reduction in visual acuity by 15 Early Treatment Diabetic Retinopathy Study (ETDRS) letters or more.
* Severe unresponsive inflammation.
* Infective endophthalmitis.
* Ocular malignancy.
* Grade III or above non-ocular suspected unexpected serious adverse reaction (SUSAR).
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (2)
Kellogg Eye Centre, University of Michigan
🇺🇸Ann Arbor, Michigan, United States
Moorfields Eye Hospital NHS Foundation Trust
🇬🇧London, United Kingdom
Kellogg Eye Centre, University of Michigan🇺🇸Ann Arbor, Michigan, United States