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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT02251457
Recruitment Status : Completed
First Posted : September 29, 2014
Last Update Posted : March 5, 2019
Gilead Sciences
Information provided by (Responsible Party):
William Arnold, Ohio State University

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.

Condition or disease Intervention/treatment Phase
Myotonia Congenita Paramyotonia Congenita Myotonic Dystrophy 1 Drug: Ranolazine Phase 1

Detailed Description:
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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 35 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Open Label Trial of Ranolazine in Myotonia Congenita, Paramyotonia Congenita, & Myotonic Dystrophy Type 1
Study Start Date : August 2014
Actual Primary Completion Date : December 18, 2017
Actual Study Completion Date : December 18, 2017

Arm Intervention/treatment
Experimental: ranolazine
ranolazine 500mg, twice daily for two weeks; 1000mg twice daily for 2 weeks
Drug: Ranolazine
Ranexa is FDA approved for chronic angina
Other Name: Ranexa®

Primary Outcome Measures :
  1. 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

  2. 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

  3. Electromyography (EMG) Myotonia [ Time Frame: 1 month ]
    To see if the electrical potentials produced by the muscle fibers change.

Secondary Outcome Measures :
  1. 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)

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   18 Years to 100 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Diagnosis of myotonia congenital, paramyotonia congenital or Myotonic Dystrophy Type 1 established by genetic testing in the subject or in a first-degree relative.
  • Clinically evident myotonia

Exclusion Criteria:

  • Contraindications to ranolazine use:

    • for fungus infection: ketoconazole (Nizoral), itraconazole (Sporanox, Onmel)
    • for infection: clarithromycin (Biaxin)
    • for depression: nefazodone
    • for HIV: nelfinavir (Viracept), ritonavir (Norvir), lopinavir and ritonavir (Kaletra), indinavir (Crixivan), saquinavir (Invirase).
    • for tuberculosis (TB): rifampin (Rifadin), rifabutin (Mycobutin), rifapentine (Priftin)
    • for seizures: phenobarbital, phenytoin (Phenytek, Dilantin, Dilantin-125), carbamazepine (Tegretol)
    • the herbal supplement St. John's wort

      • you have scarring (cirrhosis) of your liver
  • Concurrent use of mexiletine, lacosamide, acetazolamide, phenytoin, quinine, procainamide, Saint John wort or tocainide. Patients who were previously treated with these medications may participate. They need to be off of the medication for at least a week prior to enrollment.
  • QTc >470 ms for men and >480 ms for women.
  • Women who are pregnant or breastfeeding
  • Direct family history of sudden cardiac death

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT02251457

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United States, Ohio
The Ohio State University Wexner Medical Center
Columbus, Ohio, United States, 43221
Sponsors and Collaborators
Ohio State University
Gilead Sciences
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Principal Investigator: William D Arnold, MD Ohio State University
Publications automatically indexed to this study by Identifier (NCT Number):
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Responsible Party: William Arnold, Associate Professor, Ohio State University Identifier: NCT02251457    
Other Study ID Numbers: IN-US-259-1605
IN-US-259-1605 ( Other Grant/Funding Number: Gilead Sciences )
First Posted: September 29, 2014    Key Record Dates
Last Update Posted: March 5, 2019
Last Verified: March 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided
Keywords provided by William Arnold, Ohio State University:
Myotonia Congenita
Ohio State
Open Label
Paramyotonia Congenita
Myotonic Dystrophy 1
Additional relevant MeSH terms:
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Myotonic Dystrophy
Myotonia Congenita
Myotonic Disorders
Muscular Dystrophies
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Genetic Diseases, Inborn
Neuromuscular Manifestations
Neurologic Manifestations
Sodium Channel Blockers
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action