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.
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Bushby K, Finkel R, Birnkrant DJ, Case LE, Clemens PR, Cripe L et al (2010) Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management. Lancet Neurol 9:77–93
Leung DG, Wagner KR (2013) Therapeutic advances in muscular dystrophy. Ann Neurol 74:404–411
Mendell JR, Rodino-Klapac LR, Sahenk Z, Roush K, Bird L, Lowes LP et al (2013) Eteplirsen for the treatment of Duchenne muscular dystrophy. Ann Neurol 74:637–647
Voit T, Topaloglu H, Straub V, Muntoni F, Deconinck N, Campion G et al (2014) Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised, placebo-controlled phase 2 study. Lancet Neurol 13:987–996
Bushby K, Finkel R, Wong B, Barohn R, Campbell C, Comi GP et al (2014) Ataluren treatment of patients with nonsense mutation dystrophinopathy. Muscle Nerve 50:477–487
Rodger S, Lochmüller H, Tassoni A, Gramsch K, König K, Bushby K et al (2013) The TREAT-NMD care and trial site registry: an online registry to facilitate clinical research for neuromuscular diseases. Orphanet J Rare Dis 8:171
Sejersen T (2012) Standards of care in neuromuscular disorders. Neuromuscul Disord 22:872
TREAT-NMD Neuromuscular Network website. http://www.treat-nmd.eu/. Accessed 1 May 2015
The Cooperative International Neuromuscular Research Group website. http://www.cinrgresearch.org/. Accessed 1 May 2015
Australasian Neuromuscular Network website. http://www.ann.org.au/. Accessed 1 May 2015
Saito T, Tatara K (2012) Database of wards for patients with muscular dystrophy in Japan. In: Hedge M, Ankala A (eds) Muscular dystrophy. Intech, Rijeka, pp 247–260
Muscular Dystrophy Clinical Trial Network website. http://www.mdctn.jp/. Accessed 1 May 2015
Nakamura H, Kimura E, Mori-Yoshimura M, Komaki H, Matsuda Y, Goto K et al (2013) Characteristics of Japanese Duchenne and Becker muscular dystrophy patients in a novel Japanese national registry of muscular dystrophy (Remudy). Orphanet J Rare Dis 8:60
Acknowledgments
The author is deeply grateful to the member institutes of the MDCTN, namely, the National Hospital Organization (NHO) Asahikawa Medical Center, NHO Yakumo Hospital, NHO Aomori Hospital, NHO Sendai-Nishitaga National Hospital, NHO Akita National Hospital, NHO Higashisaitama Hospital, NHO Shimoshizu National Hospital, Tokyo Women’s Medical University (Department of Pediatrics, Department of Neurology, and Institute of Medical Genetics), National Center of Neurology and Psychiatry, Kitasato University (Department of Orthopedic Surgery), NHO Hakone Hospital, NHO Niigata National Hospital, NHO Iou Hospital, Muscular Dystrophy Network in Nagano Prefecture (representative: Shinshu University), Shinano Iryofukushi Center, Gifu University (Department of Pediatrics), NHO Nagara Medical Center, Nagoya City University (Department of Neonatology and Pediatrics), NHO Suzuka National Hospital, Shiga Medical Center for Children, NHO Utano Hospital, Kyoto City Hospital, NHO Toneyama National Hospital, Osaka University (Department of Neurology), Kobe University (Department of Pediatrics), Hyogo College of Medicine (Department of Pediatrics), Tottori University (Division of Child Neurology), NHO Tokushima National Hospital, Bunyuukai Hara Hospital, NHO Omuta Hospital, Kumamoto University (Department of Pediatrics), and NHO Kumamoto Saishunso National Hospital, as of April 2015. The author is also sincerely grateful to the members of the MDCTN steering committee and secretariat office, especially Dr. Hirofumi Komaki, Dr. En Kimura, Dr. Reiko Shimizu, Dr. Eri Takeshita, Ms. Akemi Tamaura, Ms. Maki Ohata, Ms. Mikiko Shigemori, and Dr. Shin’ichi Takeda (NCNP, Japan). The MDCTN has been supported by Intramural Research Grants (23–6, 26–6) for Neurological and Psychiatric Disorders of NCNP.
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Ogata, K. (2016). Muscular Dystrophy Clinical Trial Network in Japan. In: Takeda, S., Miyagoe-Suzuki, Y., Mori-Yoshimura, M. (eds) Translational Research in Muscular Dystrophy. Springer, Tokyo. https://doi.org/10.1007/978-4-431-55678-7_12
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DOI: https://doi.org/10.1007/978-4-431-55678-7_12
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