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Safety Study of RPE65 Gene Therapy to Treat Leber Congenital Amaurosis

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT00643747
First Posted: March 26, 2008
Last Update Posted: December 7, 2015
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Collaborators:
Moorfields Eye Hospital NHS Foundation Trust
Targeted Genetics Corporation
Information provided by (Responsible Party):
University College, London
  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: Intervention Model: Single Group Assignment
Masking: None (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 ]

Secondary Outcome Measures:
  • visual function [ Time Frame: intervals up to 12 months ]

Enrollment: 12
Study Start Date: January 2007
Study Completion Date: December 2014
Primary Completion Date: December 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

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   5 Years to 30 Years   (Child, Adult)
Sexes Eligible for Study:   All
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
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): 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:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: University College, London
ClinicalTrials.gov Identifier: NCT00643747     History of Changes
Other Study ID Numbers: 06/061
First Submitted: March 20, 2008
First Posted: March 26, 2008
Last Update Posted: December 7, 2015
Last Verified: December 2013

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

Additional relevant MeSH terms:
Retinal Degeneration
Leber Congenital Amaurosis
Retinal Diseases
Eye Diseases
Eye Diseases, Hereditary