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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
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 (ACTUAL)
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
Delivery System Viral transduction
Vector Type AAV
Dose 1 1E10 vg (n=3)
Dose 2 1E11 vg (n=24)

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
Eligibility Criteria
Inclusion Criteria: * Age greater than or equal to 55 years; * Subfoveal CNV secondary to AMD and with best corrected visual acuity of 3/60 - 6/9 with 6/60 or better in the other eye; * Fluorescein angiogram of the study eye must show evidence of a leaking subfoveal choroidal neovascular lesion, or CNV currently under active management with anti-VEGF therapy; * Must be a candidate for anti-VEGF intravitreal injections; * No previous retinal treatment of photodynamic therapy or laser; * Able to provide informed consent; * Able to comply with protocol requirements, including follow-up visits. Exclusion Criteria: * Liver enzymes \> 2 X upper limit of normal; * Any prior treatment for AMD in the study / control eye, excluding anti-VEGF injections; * Extensive sub-foveal scarring, extensive geographic atrophy, or thick subretinal blood in the study eye as determined by the investigator; * Significant retinal disease other than sub-foveal CNV AMD;
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

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