Casey Eye Institute

Acucela 4429-301 Emixust for Stargardt Disease

A Phase 3 Multicenter, Randomized, Double-Masked Study Comparing the Efficacy and Safety of Emixustat Hydrochloride with Placebo for the Treatment of Macular Atrophy Secondary to Stargardt Disease

The purpose of this study is to learn about a new drug that may help patients with Stargardt Macular Degeneration. This study is experimental. It is meant to investigate the safety and effectiveness of an experimental drug (called “emixustat hydrochloride”) in slowing the progression of Stargardt Macular Degeneration compared to a placebo in subjects with Stargardt disease. Emixustat has been given to over 650 people in previous studies, including healthy subjects and subjects with retina diseases. If you participate in this study, you will be randomized to either the study drug or a placebo, which you will take each night for 2 years.

Eligibility Criteria:

  • Age: 16 years or older
  • Clinical diagnosis of macular atrophy secondary to Stargardt disease in one or both eyes
  • At least 1 pathogenic mutation of the ABCA4 gene
  • Vision must be 20/320 or better
  • Good general health

This study will occur at the Casey Eye Institute in Portland, OR. Participation in this study will last a little over two years (27 months). There are 10 required in-person visits during the course of this study, and 3 telephone visits (interviews). The in-person visit will involve vision functions tests, dilated eye exams, and blood draws.

For more information, please call 503-494-0020 or email at ordc@ohsu.edu

Principal Investigator:

Mark Pennesi, MD, PhD

Casey Eye Institute

3375 SW Terwilliger Blvd.

Portland, OR 97239

eIRB#: 19458

Return to the Gene Therapy Center