Safety Study of RPE65 Gene Therapy to Treat Leber Congenital Amaurosis | Not Recruiting
Safety Study of RPE65 Gene Therapy to Treat Leber Congenital Amaurosis

Trial Source

There is no location for this trial

Location data is sourced from multiple external providers and UKCTG is not responsible for and cannot guarantee the accuracy of data.

Health Conditions
  • Retinal Degeneration
Unfortunately contact details are not available for this trial.
Primary Contact Details
Not Recruiting
Recruitment Status
Primary Trial ID Number
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.
Research Details
  • 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.
Phase 1/Phase 2
Study Design
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Study Type
Biological : tgAAG76 (rAAV 2/2.hRPE65p.hRPE65)

Study Arm Groups : A

Intervention Type
See Interventions above
Primary Outcome Measures
  • intraocular inflammation; at intervals up to 12 months
Secondary Outcome Measures
  • visual function; intervals up to 12 months
Bainbridge JW, Smith AJ, Barker SS, Robbie S, Henderson R, Balaggan K, Viswanathan A, Holder GE, Stockman A, Tyler N, Petersen-Jones S, Bhattacharya SS, Thrasher AJ, Fitzke FW, Carter BJ, Rubin GS, Moore AT, Ali RR. Effect of gene therapy on visual function in Leber's congenital amaurosis. N Engl J Med. 2008 May 22;358(21):2231-9. doi: 10.1056/NEJMoa0802268. Epub 2008 Apr 27.; 18441371
Result Reports
This is available on the website
Age Range
5 Years - 30 Years
Who Can Participate
Number of Participants
Participant Inclusion 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
Participant Exclusion Criteria
This is in the inclusion criteria above
Trial Location(s)
For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician.
Trial Contact(s)
Primary Trial Contact
Sorry, this information is not available
Other Trial Contacts
Sorry, this information is not available
Countries Recruiting
United Kingdom
Scientific 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
EudraCT Number
Not available for this trial
  • Moorfields Eye Hospital NHS Foundation Trust
  • Targeted Genetics Corporation
Other Study ID Numbers
University College, London
Key Dates

Recruitment Start Date

Jan 2007

Recruitment End Date

Mar 2014

Trial Start Date
Date Not Available
Trial End Date
Date Not Available
Date Assigned

20 Mar 2008

Last Updated

13 Dec 2013