Treatment of geographic atrophy with subconjunctival sirolimus: results of a phase I/II clinical trial.

ABSTRACT:

Purpose

To investigate the safety and effects of subconjunctival sirolimus, an mTOR inhibitor and immunosuppressive agent, for the treatment of geographic atrophy (GA).

Methods

The study was a single-center, open-label phase II trial, enrolling 11 participants with bilateral GA; eight participants completed 24 months of follow-up. Sirolimus (440 μg) was administered every 3 months as a subconjunctival injection in only one randomly assigned eye in each participant for 24 months. Fellow eyes served as untreated controls. The primary efficacy outcome measure was the change in the total GA area at 24 months. Secondary outcomes included changes in visual acuity, macular sensitivity, central retinal thickness, and total drusen area.

Results

The study drug was well tolerate

SUBMITTER: Wong WT 

PROVIDER: S-EPMC3638660 | biostudies-literature | 2013 Apr

REPOSITORIES: biostudies-literature