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Gene Therapy Trial Browser
Clinical Trial Report
Gene Therapy Trial Report
Summary
Clinical Gene Therapy Protocol for the Treatment of Retinal Dystrophy Caused by Defects in RPE65
NCTID
NCT01496040
(View at clinicaltrials.gov)
Description
The purpose of the study is to assess the safety and efficacy of the active substance rAAV-2/4.hRPE65 in patients with Leber Congenital Amaurosis or Congenital severe early-onset retinal degeneration associated with RPE65 mutation.
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Development Status
Inactive
Indication
Leber Congenital Amaurosis
Disease Ontology Term
DOID:0110016
Compound Name
RAAV2/4.hRPE65
Sponsor
Nantes University Hospital
Funder Type
Other
Recruitment Status
Completed
Enrollment Count
9
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
AAV2/4
Editor Type
Dose 1
1.22E10 -2E10 vg
Dose 2
3.27E10-4.8E10 vg
Dose 3
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase1, Phase2
Submit Date
2011-11-24
Completion Date
2014-08
Last Update
2015-10-07
Participation Criteria
Eligible Age
6 Years - 50 Years
Standard Ages
Child, Adult
Sexes Eligible for Study
ALL
Locations
No.of Trial Sites
1
Locations
France
Regulatory Information
Has US IND
FDA Designations
Recent Updates
Clinical study demonstrated safety, inconsistent findings on long-term efficacy
Resources/Links
Clinical Publications
Safety and Long-Term Efficacy of AAV4 Gene Therapy in Patients with RPE65 Leber Congenital Amaurosis
Preclinical Publications
Recombinant adeno-associated virus serotype 4 mediates unique and exclusive long-term transduction of retinal pigmented epithelium in rat, dog, and nonhuman primate after subretinal delivery
Restoration of vision in RPE65-deficient Briard dogs using an AAV serotype 4 vector that specifically targets the retinal pigmented epithelium
Gene therapeutic prospects in early onset of severe retinal dystrophy: restoration of vision in RPE65 Briard dogs using an AAV serotype 4 vector that specifically targets the retinal pigmented epithelium
(Abstract 207) Evaluation of the Immune Responses in the Treatment of RPE65 Deficient Dogs by Subretinal Injection of a Recombinant AAV Serotype 4 - ASGCT 2011