Safety Study of RPE65 Gene Therapy to Treat Leber Congenital Amaurosis

This study is ongoing, but not recruiting participants.
Sponsor:
Collaborators:
Moorfields Eye Hospital NHS Foundation Trust
Targeted Genetics Corporation
Information provided by (Responsible Party):
University College, London
ClinicalTrials.gov Identifier:
NCT00643747
First received: March 20, 2008
Last updated: December 13, 2013
Last verified: December 2013
  Purpose

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.


Condition Intervention Phase
Retinal Degeneration
Biological: tgAAG76 (rAAV 2/2.hRPE65p.hRPE65)
Phase 1
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: 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

Resource links provided by NLM:


Further study details as provided by University College, London:

Primary Outcome Measures:
  • intraocular inflammation [ Time Frame: at intervals up to 12 months ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • visual function [ Time Frame: intervals up to 12 months ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 12
Study Start Date: January 2007
Estimated Primary Completion Date: March 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: A
Injection of vector
Biological: tgAAG76 (rAAV 2/2.hRPE65p.hRPE65)
Single subretinal injection of vector suspension; up to 3x10e12 vector particles
Other Name: rAAV 2/2.hRPE65p.hRPE65

Detailed Description:

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.

  Eligibility

Ages Eligible for Study:   5 Years to 30 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00643747

Locations
United Kingdom
Moorfields Eye Hospital NHS Foundation Trust
London, United Kingdom, EC1V 2PD
Sponsors and Collaborators
University College, London
Moorfields Eye Hospital NHS Foundation Trust
Targeted Genetics Corporation
Investigators
Study Director: Robin R Ali, PhD University College, London
  More Information

Publications:
Responsible Party: University College, London
ClinicalTrials.gov Identifier: NCT00643747     History of Changes
Other Study ID Numbers: ALIR1015
Study First Received: March 20, 2008
Last Updated: December 13, 2013
Health Authority: United Kingdom: Medicines and Healthcare Products Regulatory Agency

Keywords provided by University College, London:
retinal dystrophy
Leber congenital amaurosis
RPE65
gene therapy

Additional relevant MeSH terms:
Retinal Degeneration
Leber Congenital Amaurosis
Blindness
Retinal Diseases
Eye Diseases
Eye Diseases, Hereditary
Vision Disorders
Sensation Disorders
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms

ClinicalTrials.gov processed this record on April 22, 2014