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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
Drug Product Name RAAV.sFlt-1
Drug Product 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
Therapeutic Modality Gene transfer
Therapy Route in vivo
Mechanism of Action Genetic delivery of therapeutic protein
Route of Administration Subretinal
Drug Product Type Viral vector
Gene Delivery System Type Viral transduction
Vector Type AAV
Dose 1 1E10 vg (n=3)
Dose 2 1E11 vg (n=24)

Study Record Dates


Current Phase 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