Safety Study of RPE65 Gene Therapy to Treat Leber Congenital Amaurosis | Completed
Safety Study of RPE65 Gene Therapy ... | Completed
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.

Medical Conditions
  • Retinal Degeneration
Unfortunately contact details are not available for this trial.
Primary Contact Details
Completed
Recruitment Status
NCT00643747
Primary Trial ID Number
This information is designed to help you decide whether this trial is of interest. In some cases it is provided as a link to more detailed patient information, or it may still be awaited from the organisation running the trial. Please look again in a few days if the information you need is not here, or contact the researcher named above.
Summary
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
Phase 1/Phase 2
Study Design
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Study Type
Interventional
Intervention
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
Publication(s)
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 Clinicaltrials.gov website
This information is designed to help you decide whether this trial is of interest. In some cases it is provided as a link to more detailed patient information, or it may still be awaited from the organisation running the trial. Please look again in a few days if the information you need is not here, or contact the researcher named above.
Gender
Both
Age Range
5 Years - 30 Years
Who Can Participate
Patients
Number of Participants
Sorry, this information is not available
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
This information is designed to help you decide whether this trial is of interest. In some cases it is provided as a link to more detailed patient information, or it may still be awaited from the organisation running the trial. Please look again in a few days if the information you need is not here, or contact the researcher named above.
Trial Location(s)
London
EC1V 2PD
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
This information is designed to help you decide whether this trial is of interest. In some cases it is provided as a link to more detailed patient information, or it may still be awaited from the organisation running the trial. Please look again in a few days if the information you need is not here, or contact the researcher named above.
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
Funder(s)
  • Moorfields Eye Hospital NHS Foundation Trust
  • Targeted Genetics Corporation
Other Study ID Numbers
06/061
Sponsor(s)
University College, London
Key Dates

Recruitment Start Date

Jan 2007

Recruitment End Date

Dec 2014

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

20 Mar 2008

Last Updated

04 Dec 2015

We need your help to advance medical research

You can help accelerate the discovery of cures of medical conditions by signing up and creating a profile. By doing so you can register your interest in clinical trials and researchers will be able to get in touch about trials that are suitable for you.