2016 - 1 Issue

Original article

Active Clinical Trials Testing New Therapies for Stargardt Disease

Bohdan Kousal
Ľubica Ďuďáková ORCID – Lucie Hlavatá

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Published in Czech and Slovak Ophtalmology: 2016 - 1

Published on website: 30.01.2016

Keywords: Stargardt disease, clinical trial, ABCA4, mutation

Summary

Purpose: To provide information on currently ongoing clinical trials for Stargardt disease.

Methods: We have searched the clinical trial register (www.clinicaltrials.gov) for the keyword „Stargardt“ and list active ongoing studies.

Results: There are currently eight registered clinical trials enrolling patients with Stargardt disease; all in phase I or II aiming at four mechanisms of action: inhibition of the production of vitamin A toxic dimers, gene therapy restoring wild type transcription of the ABCA4 gene, neuroprotection preventing retinal cells from oxidative damage, and replacement of the damaged retinal pigment epithelium using stem cell therapy. The basic prerequisite for enrolment in the vast majority of clinical trials is confirmation of the clinical diagnosis by mutational analysis.

Conclusion: The wide variety of therapies that are registered as clinical trials for Stargardt disease significantly raises the possibility that effective treatments will be available in the
near future for this currently incurable condition and that molecular genetic testing should be increasingly considered.

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