Designing a clinical trial to evaluate the safety and efficacy of oral soraprazan in Stargardt Disease

Hoyng, Carel, Lotery, Andrew, Stingl, Katarina, Boon, Camiel J.F., Parodi, Maurizio, Dhooge, Patty, Peters, Tobias, Klein, Wolfgang, Fsadni, Mario G., Müller, Hans and Jungmann, Oliver (2019) Designing a clinical trial to evaluate the safety and efficacy of oral soraprazan in Stargardt Disease. Investigative Ophthalmology & Visual Science, 60 (9), 5704.

Abstract

Purpose: 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.
Methods: 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

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Contributors

Author: Andrew Lotery

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