Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA)

MeiraGTx UK II Ltd15 enrolled

Overview

A clinical trial of AAV2/5 vector for patients with Defects in RPE65

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

Study Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Leber Congenital Amaurosis

Interventions

AAV RPE65BIOLOGICAL

Comparison of different dosages of AAV RPE65

Eligibility

Sex: ALLMin age: 3 Years

Medical Language ↔ Plain English

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.

Locations (2)

Kellogg Eye Centre, University of Michigan

Ann Arbor, Michigan, United States

Moorfields Eye Hospital NHS Foundation Trust

London, United Kingdom

Outcomes

Primary Outcomes

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).

Time frame: 6 months

Data from ClinicalTrials.gov

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