Phase 3 Completed N=194 Randomized Quadruple-blind Treatment

Safety and Efficacy of Emixustat in Stargardt Disease

Stargardt Disease

Source: ClinicalTrials.gov NCT03772665 ↗

Enrolled (actual)

194

Serious AEs

5.7%

Results posted

Aug 2023

Primary outcomePrimary: Mean Rate of Change in Total Area of Macular Atrophy, as Measured by Fundus Autofluorescence (FAF) — 1.280; 1.309 Rate of change from BL (mm^2/yr)

◆ Published Evidence

No publication linked

No peer-reviewed publication reporting this trial's results has been linked yet. This can indicate results are unpublished — a known publication-bias signal. We re-check periodically.

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

Outcome Measures

Outcome	Result	p-value
PRIMARY Mean Rate of Change in Total Area of Macular Atrophy, as Measured by Fundus Autofluorescence (FAF)	1.280; 1.309	—

Eligibility 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

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT03772665). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.