Completed

An Open-label Dose Escalation Study of an Adeno-associated Virus Vector (AAV2/2-hRPE65p-hRPE65) for Gene Therapy of Severe Early-onset Retinal Degeneration

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What is being tested

tgAAG76 (rAAV 2/2.hRPE65p.hRPE65)

Biological
Who is being recruted

Eye Diseases+3

+ Retinal Degeneration

+ Retinal Diseases

From 5 to 30 Years
+11 Eligibility Criteria
See all eligibility criteria
How is the trial designed

Treatment Study

Phase 1 & 2
Interventional
Study Start: January 2007
See protocol details

Summary

Principal SponsorUniversity College, London
Last updated: January 27, 2026
Sourced from a government-validated database.Claim as a partner

Study start date: January 1, 2007

Actual date on which the first participant was enrolled.

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.

Official TitleAn Open-label Dose Escalation Study of an Adeno-associated Virus Vector (AAV2/2-hRPE65p-hRPE65) for Gene Therapy of Severe Early-onset Retinal Degeneration
NCT00643747
Principal SponsorUniversity College, London
Last updated: January 27, 2026
Sourced from a government-validated database.Claim as a partner

Protocol

This section provides details of the study plan, including how the study is designed and what the study is measuring.
Design Details

12 patients to be enrolled

Total number of participants that the clinical trial aims to recruit.

Treatment Study

These studies test new ways to treat a disease, condition, or health issue. The goal is to see if a new drug, therapy, or approach works better or has fewer side effects than existing options.

Eligibility

Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.
Conditions
Criteria

Any sex

Biological sex of participants that are eligible to enroll.

From 5 to 30 Years

Range of ages for which participants are eligible to join.

Healthy volunteers not allowed

If individuals who are healthy and do not have the condition being studied can participate.

Conditions

Pathology

Eye DiseasesRetinal DegenerationRetinal DiseasesEye Diseases, HereditaryLeber Congenital AmaurosisRetinal Dystrophies

Criteria

1 inclusion criteria required to participate
Clinical diagnosis of severe early-onset retinal dystrophy confirmed missense mutation(s) in RPE65
10 exclusion criteria prevent from participating
Visual acuity in the study eye better than 6/36 Snellen
Hypertension
Diabetes mellitus
Tuberculosis
Show More Criteria

Study Plan

Find out more about all the medication administered in this study, their detailed description and what they involve.
Treatment Groups
Study Objectives

One single intervention group is designated in this study

This study does not include a placebo group 

Treatment Groups

Group I

Experimental
Injection of vector

Study Objectives

Primary Objectives

Secondary Objectives

Study Centers

These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.

This study has 1 location

Suspended

Moorfields Eye Hospital NHS Foundation Trust

London, United KingdomOpen Moorfields Eye Hospital NHS Foundation Trust in Google Maps
CompletedOne Study Center
An Open-label Dose Escalation Study of an Adeno-associated Virus Vector (AAV2/2-hRPE65p-hRPE65) for Gene Therapy of Severe Early-onset Retinal Degeneration | PatLynk