VEGFR2 x SRC

Primary objectives:
To determine the maximum tolerated dose (MTD) of SAR103168 and to characterize the dose limiting toxicities (DLTs) in the proposed dose regimen
To evaluate the pharmacokinetic (PK) profile of SAR103168
Secondary objectives:
To characterize the global safety profile of SAR103168
To evaluate preliminary anti-leukemia activity
To investigate the potential induction effect on CYP3A4 and persistence of this effect by using oral midazolam as a probe substrate in patients enrolled into the expanded cohort at the MTD
To determine the metabolic pathways of SAR103168 and identify the chemical structures of metabolites
To determine the potential impact of SAR103168 on the QTc interval in patients enrolled at the MTD

Wet age-related macular degeneration (AMD) is caused by the formation and growth of abnormal blood vessels (angiogenesis) in the retina. The new blood vessels have fragile walls and can leak fluid into the retina. The build-up of fluid (edema) under the macula can distort vision or cause vision loss. TG100801 is a topical (eye drop) therapy that has been shown to inhibit ocular angiogenesis, vascular leak, and inflammation in laboratory studies. The primary purpose of this pilot study is to evaluate the ability of topical administration of TG100801 to reduce the amount of fluid in the retina in patients with AMD following 30 days of treatment. An additional objective is to evaluate the safety of TG100801 in patients with AMD.

The formation of new blood vessels (angiogenesis), blood vessel leakage, and inflammation contribute to the progression of the eye disease, age-related macular degeneration (AMD), which is the leading cause of irreversible, severe loss of vision in people 55 years of age and older in the developed world. TG100801 is a new drug that inhibits ocular angiogenesis, vascular leak, and inflammation in laboratory studies, and may have great utility in the treatment of diseases such as AMD.
The purpose of this study is to assess the safety, ocular tolerability, and blood pharmacokinetics of TG100801 at escalating doses in healthy volunteers.