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Abstract Details

Is a Personalized N-of-1 Trial Feasible to Test Shakuyakukanzoto (SKT), a Kampo (Japanese Herbal Medicine) for ALS Patients with Muscle Cramps?
Neuromuscular and Clinical Neurophysiology (EMG)
P7 - Poster Session 7 (5:30 PM-6:30 PM)
1-001
This study aims to develop and examine a personalized N-of-1 clinical trial to test the potential benefits of SKT on muscle cramps in patients with ALS. 
Muscle cramps are the most frequent, bothersome symptoms experienced by patients with ALS that often reduce their quality of life. There have been no medications specifically approved for the treatment of cramps in ALS within Western countries. SKT has been widely prescribed by Japanese doctors in Japan to control muscle cramps of diverse origins; therefore, we would like to introduce SKT to Western medicine. We plan to evaluate whether SKT has any potential benefits in individual patients with ALS to determine if a large-scale randomized control trial is warranted.
In this personalized N-of-1 trial, each participant will be randomized to treatment or placebo in six two-week periods using a crossover design. The MCS is a novel muscle cramp scale (Mitsumoto et al., 2019) that will be scored daily and used as the primary outcome measure. We will use mixed effects models to estimate the average treatment effects of SKT across participants. There will be provisions in place for stopping the medication in the case of adverse events. The study will include microbiome analyses, which will provide critical information on the role of intestinal flora in herbal absorption. We will also use plasma metabolomic analyses to investigate underlying mechanisms of SKT in the treatment of muscle cramps in ALS. 
A preliminary sample size calculation based on MCS indicates that a series of 23 N-of-1 trials will provide adequate power to detect an average treatment effect (a 4-point difference on the MCS).  
We will examine the potential benefits of SKT in ALS in terms of individual effects as well as average effects across all participants. (Tsumura Inc. supports this study). 
Authors/Disclosures
Hiroshi Mitsumoto, MD, FÂ鶹´«Ã½Ó³»­ (Neurological Institute (NI-9))
PRESENTER 		Dr. Mitsumoto has nothing to disclose.
Ken Cheung Ken Cheung has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Tsumura.
No disclosure on file
Bjorn E. Oskarsson, MD, FÂ鶹´«Ã½Ó³»­ (Mayo Clinic) Dr. Oskarsson has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Amylyx. The institution of Dr. Oskarsson has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Biogen. Dr. Oskarsson has received personal compensation in the range of $500-$4,999 for serving as a Consultant for AnnJi. The institution of Dr. Oskarsson has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Mitsubishi. Dr. Oskarsson has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Tsumura. The institution of Dr. Oskarsson has received personal compensation in the range of $500-$4,999 for serving as a Consultant for MediciNova. The institution of Dr. Oskarsson has received research support from Biogen. The institution of Dr. Oskarsson has received research support from Medicinova. The institution of Dr. Oskarsson has received research support from Cytokinetics. The institution of Dr. Oskarsson has received research support from Calico. The institution of Dr. Oskarsson has received research support from Mitsubishi. The institution of Dr. Oskarsson has received research support from Tsumura. The institution of Dr. Oskarsson has received research support from Sanofi. The institution of Dr. Oskarsson has received research support from AZTherapeutics. The institution of Dr. Oskarsson has received research support from Orion. The institution of Dr. Oskarsson has received research support from Esaii.