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Carel C B Hoyng, Andrew Lotery, Katarina Stingl, Camiel Boon, Maurizio Parodi, Patty Dhooge, Tobias Peters, Wolfgang Klein, Mario G Fsadni, Hans Müller, Oliver Jungmann; Designing a clinical trial to evaluate the safety and efficacy of oral soraprazan in Stargardt Disease. Invest. Ophthalmol. Vis. Sci. 2019;60(9):5704.
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To date there are no treatments available for Stargardt disease (STGD). The investigational drug soraprazan has demonstrated (in pre-clinical studies) the ability to remove lipofuscin, one of the hallmarks in STGD pathogenesis, from Retinal Pigment Epithelium (RPE) cells. Soraprazan was originally designed to treat gastro-esophageal reflux disease and successfully completed phase I and phase II clinical trials, where safety and tolerability was already demonstrated. The aim is to start a phase II trial to evaluate the efficacy of soraprazan in reducing the amount of lipofuscin in RPE cells of subjects with STGD.
An international consortium was formed consisting of 5 investigator sites (2 in Netherlands and 1 in each of Germany, UK, and Italy), a Contract Research Organisation, and a start-up company as a sponsor (Katairo). A draft protocol and a business plan were fully costed and timelines defined. An application was filed with Horizon 2020.
The application was successful and a grant of €6m was received in December 2017. In 11 months, new batches of Soraprazan have been manufactured and the consortium finalized the protocol. The outcome is a randomized (2:1) proof of concept, double-masked, placebo-controlled, two arm, multicenter trial to evaluate the efficacy and safety of soraprazan (20 mg orally once a day) in subjects with STGD by measuring the reduction of the amount of lipofuscin in the RPE cells of subjects with quantitative autofluorescence (qAF). In total 90 subjects are planned. Additional investigations included microperimetry, OCT, and BCVA. Clinical trial applications and Ethics Committee filing have been filed in all relevant countries (EudraCT No. 2018-001496-20, Sponsored by Katairo GmbH, supported by a grant from the European Union Horizon 2020 program) for patient enrollment in 2019.
This experience demonstrated that by forming a multi-disciplinary consortium involving industry and clinical experts, clinicians can secure adequate funding to effectively pursue projects of interest. Soraprazan is potentially a new therapy for STGD which removes lipofuscin from RPE cells, thereby preventing or slowing disease progression. Patient enrollment will be completed (90 subjects) in 2019.
This abstract was presented at the 2019 ARVO Annual Meeting, held in Vancouver, Canada, April 28 - May 2, 2019.
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