Summary

A consortium of eight partners prepared and started a phase II proof of concept clinical study with Remofuscin® in adult patients with Stargardt’s disease. Active ingredient of the drug is Soraprazan, which gave the name of this EU financed H2020 project. For the clinical study, the consortium has meanwhile used the acronym STARTT (STArgardt Remofuscin® Treatment Trial).

Lipofuscin is a fluorescent waste material accumulated in the retinal pigment epithelium (RPE), which is a mono cell layer supplying the retina. Accumulation of lipofuscin is associated with various negative effects for the RPE and photoreceptors, and may ultimately lead to retinal degeneration. Excessive lipofuscin accumulation in Stargardt patients is caused by a genetic defect and is considered to play a major role in the pathogenesis. Treatment with Soraprazan, the active ingredient of Remofuscin®, has been shown to result in removal of lipofuscin from RPE cells in various animal and cell models.

Within our clinical study 87 adult patients with Stargardt disease are treated. These patients match the inclusion criteria listed in the clinical study protocol, the most important of which include

• age over 18
• onset of disease before age 45
• confirmation of Stargardt disease by genetic testing
• presence of lipofuscin accumulation above a certain level as assessed by autofluorescence measurement.

In a randomisation pattern of 2:1, patients receive treatment or placebo. Primary endpoint of the study is the level of autofluorescence. Secondary endpoints include functional assessments (visual acuity, retinal sensitivity) and morphological assessments from OCT imaging.

More information about the trial is available in the clinical trials register at www.clinicaltrialsregister.eu/ctr-search/trial/2018-001496-20/NL (EudraCT Number 2018-001496-20).

More information on the EC funding and on the funding scheme can be found on 'CORDIS EU research results'.