Safety and Efficacy of Emixustat in Stargardt Disease

Launched by KUBOTA VISION INC. · Dec 9, 2018

Keywords

ClinConnect Summary

Stargardt disease is a rare, inherited degenerative disease of the retina affecting approximately 1 in 8000 to 10 000 people and is the most common type of hereditary macular dystrophy. There are no approved treatments for STGD. This disease is characterized by an excessive accumulation of lipofuscin at the level of the retinal pigment epithelium (RPE). Lipofuscin is made of lipids, proteins, and toxic bis retinoids (such as N retinylidene N retinylethanolamine \[A2E\]). Accumulation of the toxic bis retinoids found in lipofuscin is thought to cause RPE cell dysfunction and eventual apoptos...

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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