Safety and Efficacy of Emixustat in Stargardt Disease

• Status: Active, not recruiting
• Trial end date: 2022-06-01
• Target enrollment: 0
• Participant gender: All

Summary

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.

Funding Source -- FDA OOPD

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Acucela Inc.; Kubota Vision Inc.

Criteria

Inclusion Criteria:

• 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

Exclusion Criteria:

• 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