A Phase I, Single Ascending Dose Study of GEM103 (Recombinant Human Complement Factor H) in Patients with Geographic Atrophy.

ABSTRACT:

Purpose

To establish the safety, tolerability, pharmacokinetics, and pharmacodynamics of an intravitreal injection of recombinant human complement factor H (CFH), GEM103, in individuals with genetically defined age-related macular degeneration (AMD) and geographic atrophy (GA).

Design

Phase I single ascending-dose, open-label clinical trial (ClinicalTrials.gov identifier, NCT04246866).

Participants

Twelve individuals 50 years of age or older with a confirmed diagnosis of foveal GA in the study eye.

Methods

Participants were assigned to the increasing dose cohorts and received 1 50-μl intravitreal injection of GEM103 at doses of 50 μg/eye, 100 μg/eye, 250 μg/eye, or 500 μg/eye; dose escalation was dependent on the occurrence of dose-limiting toxicities.

Main

SUBMITTER: Khanani AM 

PROVIDER: S-EPMC9559901 | biostudies-literature | 2022 Jun

REPOSITORIES: biostudies-literature