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

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

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.

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.

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.