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

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Medical Conditions
  • Retinal Degeneration
Primary Contact Details
Unfortunately contact details are not available for this trial.
Recruitment Status
Completed
Trial source and source ID number
NCT00643747

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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 shortly if the information you need is not here or, if named, 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.; 18441371
Result Reports
Check availability of results 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 shortly if the information you need is not here or, if named, 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 shortly if the information you need is not here or, if named, 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 shortly if the information you need is not here or, if named, 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 added to source

20 Mar 2008

Date updated in source

04 Dec 2015