Muscular Dystrophy Clinical Trial Network in Japan

Abstract

Muscular dystrophy is a rare disease, affecting less than 0.01 % of the population. The establishment of clinical trial networks between medical institutes reportedly helps to improve readiness for trials on rare diseases such as muscular dystrophy. Some networks for clinical research on neuromuscular diseases have already been established in other parts of the world. The Muscular Dystrophy Clinical Trial Network (MDCTN) was established in 2012 to promote and activate clinical research on neuromuscular diseases in Japan. This network was organized based on an existing study group for clinical myology funded by a Japanese national research grant and is a network of national hospitals with wards specifically for patients with progressive neuromuscular disorders. As of April 2015, 33 medical institutions had joined this network. Site registry queries have revealed that approximately 6000 patients with neuromuscular diseases visit the member hospitals annually. The work of the MDCTN includes sharing updates in health-care information, developing standardized means of evaluation through workshops, supporting clinical trials with feasibility surveys and patient recruitment, and conducting collaborative research. Working closely with patient registries is one of the keys to the functioning of this network. The MDCTN has collaborated with one patient registry, Remudy, to support remarkably rapid recruitment of subjects with Duchenne muscular dystrophy who meet criteria of specific genetic mutations and conditions for some clinical trials. The goal of MDCTN is to be a model for developing pharmaceuticals and medical devices for rare diseases in Japan.