Age-related Maculopathies, Age-related Maculopathy, Eye Diseases, Macular Degeneration, Maculopathies,Age-related, Maculopathy,Age-related, Retinal Degeneration, Retinal Neovascularization
Conditions
Keywords
Age-related Macular Degeneration, AAV, AMD, Wet AMD, Neovascular AMD, Gene Therapy, Ocular, Ocular Gene Therapy, Eye diseases, Macular Degeneration, Retinal Degeneration, Retinal Neovascularization, Wet Macular Degeneration, Retinal Diseases
Brief summary
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.
Detailed description
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.
Interventions
BIOLOGICALrAAV.sFlt-1
1 x 10\^10 vector genomes (vg) rAAV.sFlt-1, delivered by subretinal injection
OTHERControl (ranibizumab alone)
Patients will not receive rAAV.sFlt-1, but will be eligible for retreatment with ranibizumab (Lucentis).
Sponsors
Adverum Biotechnologies, Inc.
Lions Eye Institute, Perth, Western Australia
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
SINGLE (Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
55 Years to No maximum
Healthy volunteers
No
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;
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| No sign of unresolved ophthalmic complications, toxicity or systemic complications as measured by laboratory tests from 1 month post injection | Primary endpoint at 1 month | 1. Ocular examination: * Ocular inflammation * Intraocular pressure * Visual acuity * Retinal bleeding 2. Abnormal laboratory data |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Maintenance or improvement of vision without the necessity of ranibizumab re-injections | Up to 3 years | 1. Best-corrected visual acuity 2. CNV lesion 3. Foveal thickness |
Countries
Australia
Outcome results
None listed