Periodic Reporting for period 1 - Soraprazan (Soraprazan - a new regenerative therapy for Stargardt's disease)
Reporting period: 2017-12-01 to 2019-11-30
Participating partners include six clinical sites (Eberhard Karls Universität Tübingen, Radboud University Medical Center, Nijmegen, Ospedale San Raffaele, Milano, Leiden University Medical Center, University of Southampton, and Universitätsklinikum Bonn), the international clinical research organization Smerud Medical Research, Oslo, and Katairo assuming the role of the sponsor by regulatory definition. The project is coordinated by the Eberhard Karls Universität Tübingen.
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 of the RPE in various animal and cell models.
The protocol foresees to treat 90 adult patients with Stargardt disease matching the inclusion criteria. The most important of which include age over 18, onset of disease before age 45, confirmation of Stargardt disease by genetic testing, and 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.
Project start was in December 2017. The project is scheduled to finish in November 2021.
More information about the trial is available in the clinical trials register at EudraCT Number 2018-001496-20.
- the manufacture of the active ingredient under GMP
- the manufacture of the pharmaceutical form (tablets) under GMP
- the testing of stability of active ingredient and pharmaceutical form
- the supply of the trial-sites with packaged and labelled test material
- the drafting of the clinical trial protocol
- the seeking of regulatory advice for the draft protocol
- the adjustment of the protocol following regulatory advice
- the submission of the study for regulatory approval in the four countries of the clinical study
- the setup of the data base
- the setup and maintenance of a pharmacovigilance system
- the initiation of the study in three of four countries and five of six trial sites
- the screening of over 50 patients
- the recruitment of over 45 patients
- the treatment and testing of all recruited patients for up to 8 months
- the information of patients via press releases, information of patient organizations, and setup and supply of newsletters
- the information of key opinion leaders and the pharmaceutical industry in ophthalmic congresses by direct contact and publications
- the coordination of the project