Study Protocol for a Multicenter, Open-Label, Single-Arm Study of Tranilast for Cardiomyopathy of Muscular Dystrophy

  • MATSUMURA TSUYOSHI
    Department of Neurology, National Hospital Organization Osaka Toneyama Medical Center
  • HASHIMOTO HIROYA
    Clinical Research Center
  • SEKIMIZU MASAHIRO
    Clinical Research Center Department of Pediatrics, National Hospital Organization Nagoya Medical Center
  • SAITO AKIKO M.
    Clinical Research Center
  • IWATA YUKO
    Department of Clinical Research and Development, National Cerebral and Cardiovascular Center
  • ASAKURA MASANORI
    Department of Internal Medicine, Cardiovascular Division, Hyogo College of Medicine
  • KIMURA KOICHI
    Department of General Medicine, The Institute of Medical Science, The University of Tokyo
  • TAMURA TAKUHISA
    Department of Neurology, National Hospital Organization Higashisaitama National Hospital
  • FUNATO MICHINORI
    Department of Pediatrics, Nagara Medical Center
  • SEGAWA KAZUHIKO
    Department of Cardiology, National Center Hospital, National Center of Neurology and Psychiatry
  • OGATA KATSUHISA
    Department of Neurology, National Hospital Organization Higashisaitama National Hospital
  • NAKAJIMA TAKASHI
    Department of Clinical research, Department of Neurology, Niigata National Hospital

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Abstract

<p>Summary: Duchenne (DMD) and other forms of muscular dystrophy (MD) are collectively rare and affect approx imately 20 per 100,000 people. The on-going development of exon skipping and other novel therapies for DMD is expected to lead to improvements in motor function prognosis. However, improvements in motor dysfunction with these novel therapies are associated with the risk of increase in cardiac burden. Development of therapies to improve cardiac function, therefore, is an urgent issue. This single-arm, open-label, multicenter study will include 20 patients with MD aged 13 years or older. Tranilast, a transient receptor potential cation channel subfamily V member 2 (TRPV2) inhibitor, will be administered orally for a period of 28 weeks at a dose of 300 mg/day divided into three daily doses. If consent to continue administration is obtained at 28 weeks, the drug will be administered for an additional 116 weeks. The primary outcome will be the change in brain natriuretic peptide (BNP) at 6 months after the start of administration compared to baseline. Tranilast is an anti-allergy agent that was developed in Japan. It has been used in a large number of clinical cases, including pediatric cases, and has been shown to be safe. We expect this study to provide basic data for developing new treatment method in cardiomyopathy/skeletal myopathy using TRPV2 inhibitors. Moreover, such therapies may also be effective in treating general heart failure without MD. Therefore, if the effectiveness of TRPV2 inhibitors could be confirmed in this study, great social and economic benefits could be achieved.</p>

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