Global Rare Disease Network Research
Academic Archives
Mecasin treatment in patients with amyotrophic lateral sclerosis: study protocol for a randomized controlled trial |
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Abstract
Background: Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease that causes paralysis of limb,
swallowing, and breathing muscles. Riluzole, the Food and Drug Administration-approved drug for ALS, provides
minimal benefit, prolonging patient life by only 2–3 months. Previous studies have found a neuro-protective and
anti-neuroinflammatory effect of Mecasin, with retrospective studies providing suggestive evidence for a beneficial
effect of Mecasin. The aim of this study was to develop a protocol to determine the proper dosage of Mecasin.
Methods: This is a phase II-A, multi-center, randomized study with three arms. Thirty-six patients with ALS will be
randomly assigned to one of three groups, each receiving the standard treatment with 100 mg of riluzole in
addition to one of 1.6 g of Mecasin, 2.4 g of Mecasin, or a placebo. The Primary outcome is the Korean version of
the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised result after 12 weeks of treatment. Secondary
outcomes include results of the Short Form Health Survey-8, Medical Research Council Scale, Visual Analogue
Scale for Pain, Hamilton Rating Scale for Depression, Fatigue Severity Scale, Patient Global Impression of Change,
pulmonary function test, forced expiratory volume in 1 s and its ratio to forced vital capacity, creatine kinase,
and body weight. The frequencies of total adverse events and serious adverse events will be described and
documented. The trial protocol has been approved by the Institutional Review Board of the Wonkwang University
Gwangju and Sanbon Hospital (2016–5-4 and 2016–34-01, respectively). An Investigational New Drug status (30731)
was granted by the Korea Food and Drug Administration. |