for people ages 16 years and up (full criteria)
at San Francisco, California and other locations
study started
estimated completion



The purpose of this study is to determine if emixustat hydrochloride reduces the rate of progression of macular atrophy compared to placebo in subjects with Stargardt disease.

Official Title

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


Stargardt Disease STGD ABCA4 Macular Degeneration Emixustat


You can join if…

Open to people ages 16 years and up

  • A clinical diagnosis of macular atrophy secondary to Stargardt disease (STGD)
  • Macular atrophy measured to fall within a defined size range
  • Two mutations of the ABCA4 gene. If only one mutation, a typical STGD appearance of the retina.
  • Visual acuity in the study eye of at least 20/320

You CAN'T join if...

  • Macular atrophy secondary to a disease other than STGD
  • Mutations of genes, other than ABCA4, that are associated with retinal degeneration
  • Surgery in the study eye in the past 3 months
  • Prior participation in a gene therapy or stem cell clinical trial for STGD
  • Recent participation in a clinical trial for STGD evaluating a complement inhibitor or vitamin A derivative
  • Use of certain medications in the past 4 weeks that might interfere with emixustat
  • An abnormal electrocardiogram (ECG)
  • Certain abnormalities on laboratory blood testing
  • Female subjects who are pregnant or nursing


  • UCSF Dept. of Ophthalmology accepting new patients
    San Francisco California 94143-0730 United States
  • Retina-Vitreous Associates Medical Group accepting new patients
    Beverly Hills California 90211 United States


accepting new patients
Start Date
Completion Date
Acucela Inc.
Phase 3
Study Type
Last Updated