[Clinical Tests Testing New Therapies for Stargardt Disease].

Abstract

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.

KEY WORDS:

Stargardt disease, clinical trial, ABCA4, mutation.

• Authors: B Kousal, Ľ Ďuďáková, L Hlavatá, P Lišková
• Journal: Ceska a slovenska oftalmologie : casopis Ceske oftalmologicke spolecnosti a Slovenske oftalmologicke spolecnosti (Cesk Slov Oftalmol) Vol. 72 Issue 1 Pg. 293-7 (Feb 2016) ISSN: 1211-9059 [Print] Czech Republic
• Vernacular Title: Klinické zkoušky testující nové terapie pro Stargardtovu chorobu.
• PMID: 27041285 (Publication Type: Journal Article, Review)

Chemical References

• ABCA4 protein, human
• ATP-Binding Cassette Transporters

Topics

• ATP-Binding Cassette Transporters (genetics)
• Cell- and Tissue-Based Therapy
• Clinical Trials as Topic
• DNA Mutational Analysis
• Female
• Genetic Therapy
• Genotype
• Humans
• Macular Degeneration (congenital, diagnosis, genetics, therapy)
• Male
• Mutation
• Retinal Pigment Epithelium (pathology)
• Stargardt Disease
• Stem Cell Transplantation