Safety Study of RPE65 Gene Therapy to Treat Leber Congenital Amaurosis

University College, London12 enrolled

Overview

The purpose of the study is to determine whether gene therapy is safe and effective for the treatment of severe childhood blindness caused by mutations in RPE65.

The main objective of the proposed trial is to determine the safety and efficacy subretinal administration of a recombinant adeno-associated viral vector (rAAV 2/2.hRPE65p.hRPE65) at three different dosage levels in individuals with autosomal recessive severe early-onset retinal degeneration due to mutations in RPE65. We have a comprehensive clinical monitoring plan to investigate the safety and efficacy of vector delivery.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Retinal Degeneration

Interventions

tgAAG76 (rAAV 2/2.hRPE65p.hRPE65)BIOLOGICAL

Single subretinal injection of vector suspension; up to 3x10e12 vector particles

Eligibility

Sex: ALLMin age: 5 YearsMax age: 30 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Clinical diagnosis of severe early-onset retinal dystrophy confirmed missense mutation(s) in RPE65 Exclusion Criteria: * Visual acuity in the study eye better than 6/36 Snellen * Hypertension * Diabetes mellitus * Tuberculosis * Renal impairment * Immunocompromise * Osteoporosis * Gastric ulceration * Severe affective disorder) * Pregnancy or lactation

Locations (1)

Moorfields Eye Hospital NHS Foundation Trust

London, United Kingdom

Outcomes

Primary Outcomes

intraocular inflammation

Time frame: at intervals up to 12 months

Secondary Outcomes

visual function

Time frame: intervals up to 12 months

Data from ClinicalTrials.gov

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