Contact SCGE
Your email
Message
Send
SCGE Consortium Home
About SCGE TCDC
Contact Us
License
Home
Gene Therapy Trial Browser
Clinical Trial Report
Gene Therapy Trial Report
Summary
Safety and Efficacy Study of rAAV.sFlt-1 in Patients With Exudative Age-Related Macular Degeneration
NCTID
NCT01494805
(View at clinicaltrials.gov)
Description
The study will involve approximately 40 subjects aged 55 or above who have exudative age-related macular degeneration (wet AMD). Patients will be randomized to receive one of two doses of rAAV.sFlt-1 or assigned to the control group.
(Show More)
Development Status
Inactive
Indication
Neovascular (Wet) Age-Related Macular Degeneration (nAMD)
Disease Ontology Term
DOID:10871
Compound Name
RAAV.sFlt-1
Compound Description
rAAV.CMV.sFlt-1
Sponsor
Lions Eye Institute, Perth, Western Australia
Funder Type
Other
Recruitment Status
Completed
Enrollment Count
40
Results Posted
Not Available
Therapy Information
Target Gene/Variant
Soluble VEGFR1
Therapy Type
Gene transfer
Therapy Route
In-vivo
Mechanism of Action
Genetic delivery of therapeutic protein
Route of Administration
Subretinal
Drug Product Type
Viral vector
Target Tissue/Cell
Delivery System
Viral transduction
Vector Type
AAV
Editor Type
Dose 1
1E10 vg (n=3)
Dose 2
1E11 vg (n=3)
Dose 3
1E11 vg (n=21)
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase1, Phase2
Submit Date
2011-12-14
Completion Date
2017-08
Last Update
2017-09-01
Participation Criteria
Eligible Age
>=55 Years
Standard Ages
Adult, Older adult
Sexes Eligible for Study
ALL
Locations
No.of Trial Sites
1
Locations
Australia
Regulatory Information
Has US IND
FDA Designations
Recent Updates
Product was well tolerated, unable to determine efficacy
Resources/Links
Clinical Publications
Gene therapy with recombinant adeno-associated vectors for neovascular age-related macular degeneration: 1 year follow-up of a phase 1 randomised clinical trial
Phase 2a Randomized Clinical Trial: Safety and Post Hoc Analysis of Subretinal rAAV.sFLT-1 for Wet Age-related Macular Degeneration
Three-Year Follow-Up of Phase 1 and 2a rAAV.sFLT-1 Subretinal Gene Therapy Trials for Exudative Age-Related Macular Degeneration
Preclinical Publications
Potential long-term inhibition of ocular neovascularisation by recombinant adeno-associated virus-mediated secretion gene therapy
Long-term evaluation of AAV-mediated sFlt-1 gene therapy for ocular neovascularization in mice and monkeys
rAAV.sFlt-1 gene therapy achieves lasting reversal of retinal neovascularization in the absence of a strong immune response to the viral vector
Preclinical safety evaluation of subretinal AAV2.sFlt-1 in non-human primates