Study of Ranolazine in Myotonia Congenita, Paramyotonia Congenita and Myotonic Dystrophy Type 1

Summary

The purpose of this study is to gather preliminary data to determine if ranolazine is a safe and effective treatment for the symptoms of myotonia congenital, paramyotonia congenita, and myotonic dystrophy type 1. The duration of the study is 5 weeks.

Timeline

Start

2014-08

Primary completion

2017-12-18

Completion

2017-12-18

Publications

• Background Novak KR, Norman J, Mitchell JR, Pinter MJ, Rich MM. Sodium channel slow inactivation as a therapeutic target for myotonia congenita. Ann Neurol. 2015 Feb;77(2):320-32. doi: 10.1002/ana.24331. Epub 2015 Jan 9.
• Spillane J, Trip J, Drost G, Faber CG, Hanna MG, Nevitt SJ, Vivekanandam V. Drug treatment for myotonia. Cochrane Database Syst Rev. 2025 Apr 8;4(4):CD004762. doi: 10.1002/14651858.CD004762.pub3.
• Lorusso S, Kline D, Bartlett A, Freimer M, Agriesti J, Hawash AA, Rich MM, Kissel JT, David Arnold W. Open-label trial of ranolazine for the treatment of paramyotonia congenita. Muscle Nerve. 2019 Feb;59(2):240-243. doi: 10.1002/mus.26372. Epub 2018 Dec 21.

Drugs

Indications