Phase I Dose Escalation Safety Study of RetinoStat in Advanced Age-Related Macular Degeneration (AMD)

Study Overview
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Brief Summary
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The purpose of this first in man study is to examine the safety of an experimental gene transfer agent, RetinoStat, designed to treat neovascular age-related macular degeneration.

Detailed Description
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There are two parts to the study. A dose-escalation phase looking at three doses of RetinoStat starting with the lowest dose, three patients will be recruited at each dose level. The escalation phase will be followed by a dose confirmation phase where the highest dose that is safe and well tolerated will be examined in 9 patients.

Official Title
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A Phase I Dose Escalation Safety Study of Subretinally Injected RetinoStat, a Lentiviral Vector Expressing Endostatin and Angiostatin, in Patients With Advanced Neovascular Age-Related Macular Degeneration

Conditions
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Age Related Macular Degeneration

Intervention / Treatment
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* Drug: Subretinally injected RetinoStat


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Clinical diagnosis of AMD with active CNV that shows evidence of leakage. BCVA less than or equal to 20/200 in the study eye for dose escalation phase. BCVA less than or equal to 20/80 in the study eye for maximum tolerated dose phase. Exclusion Criteria: Significant ocular abnormalities that prevent retinal assessment. Treatment with steroids within three months of screening. Treatment with anti-VEGF therapy to either eye within one month of screening. Clinically significant intercurrent illnesses, laboratory, ECG or chest XRay abnormalities.

Ages Eligible for Study
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Minimum Age: 50 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Subretinally Injected RetinoStat<br>Subretinally injected RetinoStat | Drug: Subretinally injected RetinoStat<br>* Single subretinal injections, with increasing doses. 9 patients with 3 patients at each dose followed, by 12 patients at maximum tolerated dose.<br>* Other names: OXB-201;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| The incidence of adverse events | The number and percentage of patients with treatment emergent adverse events. | 24 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change from baseline in subretinal and intraretinal fluid as measured by OCT | The change from baseline in the amount of subretinal and intraretinal fluid measured by Optical Coherence tomography | 24 weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Age Related Macular Degeneration