Title
Safety and Efficacy Study of rAAV.sFlt-1 in Patients With Exudative Age-Related Macular Degeneration
A Phase I/II Controlled Dose-escalating Trial to Establish the Baseline Safety and Efficacy of a Single Subretinal Injection of rAAV.sFlt-1 Into Eyes of Patients With Exudative Age-related Macular Degeneration (AMD)
Phase
Phase 1/Phase 2Lead Sponsor
Lions Eye InstituteStudy Type
InterventionalStatus
Completed No Results PostedIndication/Condition
Maculopathies,Age-related Maculopathy,Age-related Retinal Degeneration Retinal Neovascularization Eye Diseases ...Intervention/Treatment
ranibizumab raav.sflt-1 ...Study Participants
40The 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.
A new treatment for exudative age-related macular degeneration (wet AMD) is being investigated. The purpose of this Phase I/II clinical research study is to examine the baseline safety and efficacy of an experimental study drug to treat a complication of the disease which leads to vision loss. The name of the study drug is rAAV.sFlt-1.
This experimental study uses a non-pathogenic virus to express a therapeutic protein within the eye. The therapeutic diminishes the growth of abnormal blood vessels under the retina. The duration of effect is thought to be long-term (years) following a single administration.
The clinical research study will look at the baseline safety and efficacy of a single injection of rAAV.sFlt-1 injected directly into the eye.
Approximately forty (40) subjects will participate in Australia. The primary endpoint of the study is at one month, with extended follow up for 3 years.
1 x 10^10 vector genomes (vg) rAAV.sFlt-1, delivered by subretinal injection
1 x 10^11 vector genomes (vg) rAAV.sFlt-1, delivered by subretinal injection
Patients will not receive rAAV.sFlt-1, but will be eligible for retreatment with ranibizumab (Lucentis).
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;