First in Human Study to Evaluate the Safety and Tolerability of GEM103 in Geographic Atrophy Secondary to Dry Age Related Macular Degeneration

Summary

The study is designed to identify the maximum tolerated dose (MTD) for intravitreal (IVT) administration of GEM103 in subjects with geographic atrophy (GA) secondary to dry AMD. Safety and tolerability of a single dose of GEM103 will be assessed based on the occurrence of dose-limiting toxicities (DLTs). Each subject will be followed for safety, pharmacokinetic (PK), clinical, and biomarker evaluations. Three escalating dose cohorts are planned.

Timeline

Start

2019-12-19

Primary completion

2020-10-21

Completion

2020-10-21

Publications

• Khanani AM, Maturi RK, Bagheri N, Bakall B, Boyer DS, Couvillion SS, Dhoot DS, Holekamp NM, Jamal KN, Marcus DM, Pieramici D, Aziz AA, Patki KC, Bridges WZ Jr, Barone SB. A Phase I, Single Ascending Dose Study of GEM103 (Recombinant Human Complement Factor H) in Patients with Geographic Atrophy. Ophthalmol Sci. 2022 Apr 11;2(2):100154. doi: 10.1016/j.xops.2022.100154. eCollection 2022 Jun.

Drugs

Indications