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

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: Open Label Trial of Ranolazine in Myotonia Congenita, Paramyotonia Congenita, & Myotonic Dystrophy Type 1

Brief 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.

Detailed Summary: Recent advances in the understanding of myotonia congenita have identified potential areas that could possibly respond to treatment in a drug study. The drug ranolazine (trade name Ranexa) is a FDA-approved medication to treat chest pain in patients with heart disease. Ranolazine has been studied in mice with myotonia congenita. The data from this animal model suggest that ranolazine may improve the symptoms and signs of myotonia. All individuals that participate will be placed on active drug. The investigators want to see if this drug is safe to take without causing too many side effects for people with myotonia congenita, paramyotonia congenital and myotonic dystrophy type 1. Participants will go to The Ohio State University for study visits. Participants will take ranolazine for four weeks. Participants can expect a total of 4 study visits and 2 phone calls over the 5 week period.
Sponsor: Ohio State University

Current Primary Outcome:

  • Questionnaires: Short Form Health Survey (SF-36) and Individualized Neuromuscular Quality of Life Questionnaire (INQoL) [ Time Frame: 1 month ]
    quality of life measurements for overall health and neuromuscular disease
  • Muscle tasks [ Time Frame: 1 month ]
    The subject is observed and timed while rising from an arm chair, walking 3 meters, turning, walking back, and sitting down again
  • Electromyography (EMG) Myotonia [ Time Frame: 1 month ]
    To see if the electrical potentials produced by the muscle fibers change.


Original Primary Outcome: Same as current

Current Secondary Outcome: Electrocardiogram (ECG) [ Time Frame: 1 month ]

to measure heart function and observe QT interval (a measure of the time between the start of the Q wave and the end of the T wave in the heart's electrical cycle)


Original Secondary Outcome: Same as current

Information By: Ohio State University

Dates:
Date Received: September 25, 2014
Date Started: August 2014
Date Completion: December 2018
Last Updated: March 18, 2017
Last Verified: March 2017