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Clinical Trial Report
Gene Therapy Trial Report
Summary
Clinical Trial of Gene Therapy for Leber Congenital Amaurosis Caused by RPE65 Mutations
NCTID
NCT00821340
(View at clinicaltrials.gov)
Description
The purpose of this clinical trial is to examine the safety of gene therapy for Lebers Congenital Amaurosis (LCA) caused by RPE65 mutations using a recombinant adeno-associated virus serotype 2 (rAAV2) vector carrying the human RPE65 (hRPE65) gene. Recently, three independent short-term gene therapy studies in humans with LCA due to RPE65 mutations were published, suggesting that subretinal delivery of rAAV virus carrying the RPE65 gene is safe. As a secondary outcome, improvement in visual function was observed in seven of the first nine treated patients. The proposed study is a similar open label, Phase I clinical trial of uniocular subretinal rAAV2-hRPE65 administration to individuals with RPE65-associated retinal disease. Two cohorts of three subjects each and one cohort of four subjects will be included in this trial. Cohort 1 and 2 will consist of individuals 18 years of age and older and Cohorts 3 will consist of individuals 8 years of age and older. In cohort 2, a larger volume of vector will be administered. Enrollment in Cohort 3 will begin only after confirming the safety of rAAV2-hRPE65 administration in the older group of participants.
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Development Status
Inactive
Indication
Leber Congenital Amaurosis-Type 2
Disease Ontology Term
DOID:0110016
Compound Name
RAAV2-hRPE65
Sponsor
Hadassah Medical Organization
Funder Type
Other
Recruitment Status
Completed
Enrollment Count
3
Results Posted
Not Available
Therapy Information
Target Gene/Variant
RPE65
Therapy Type
Gene transfer
Therapy Route
In-vivo
Mechanism of Action
Functional gene replacement
Route of Administration
Subretinal
Drug Product Type
Viral vector
Target Tissue/Cell
Delivery System
Viral transduction
Vector Type
AAV2
Editor Type
Dose 1
Undisclosed
Dose 2
Dose 3
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase1
Submit Date
2009-01-12
Completion Date
2017-01-01
Last Update
2018-04-10
Participation Criteria
Eligible Age
>=8 Years
Standard Ages
Child, Adult, Older adult
Sexes Eligible for Study
ALL
Locations
No.of Trial Sites
1
Locations
Israel
Regulatory Information
Has US IND
False
FDA Designations
Recent Updates
Enrolled 3/10 planned patients
Resources/Links
Clinical Publications
Molecular anthropology meets genetic medicine to treat blindness in the North African Jewish population: human gene therapy initiated in Israel