Phase 3
N=194
Safety and Efficacy of Emixustat in Stargardt Disease
Stargardt Disease
Bottom Line
View on ClinicalTrials.gov: NCT03772665 ↗Enrolled (actual)
194
Serious AEs
5.7%
Results posted
Aug 2023
Primary outcome: Primary: 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)
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Emixustat (Drug); Placebo (Drug)
- Age
- Pediatric, Adult, Older Adult · 16+ yrs
- Sex
- All
- Sponsor
- Kubota Vision Inc.
- Primary completion
- Jun 2022
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 | — |
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
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
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.