Leber Congenital Amaurosis Clinical Trial
— OPTIRPE65Official title:
An Open-label, Multi-centre, Phase I/II Dose Escalation Trial of an Adeno Associated Virus Vector for Gene Therapy of Adults And Children With Retinal Dystrophy Associated With Defects in RPE65 (LCA)
| Verified date | June 2021 |
| Source | MeiraGTx UK II Ltd |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | |
| Study type | Interventional |
A clinical trial of AAV2/5 vector for patients with Defects in RPE65
| Status | Completed |
| Enrollment | 15 |
| Est. completion date | December 2018 |
| Est. primary completion date | December 2018 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | 3 Years and older |
| Eligibility | 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. |
| Country | Name | City | State |
|---|---|---|---|
| United Kingdom | Moorfields Eye Hospital NHS Foundation Trust | London | |
| United States | Kellogg Eye Centre, University of Michigan | Ann Arbor | Michigan |
| Lead Sponsor | Collaborator |
|---|---|
| MeiraGTx UK II Ltd |
United States, United Kingdom,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | Overall Safety of Adeno-Associated Virus Vector (AAV-OPTIRPE65) - Number of Participants With a Safety Event | 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). |
6 months |
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