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Completed

NCT Number: NCT02251457

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

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.

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Key information

Conditions

Myotonia Congenita Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Muscular Diseases Muscular Disorders, Atrophic Muscular Dystrophies Musculoskeletal Diseases Myotonic Disorders Myotonic Dystrophy Myotonic Dystrophy 1 Nervous System Diseases Neurodegenerative Diseases Neuromuscular Diseases Paramyotonia Congenita

Age range

18 year–100 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

The Ohio State University Wexner Medical Center

Columbus, Ohio, 43221, United States

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Ranolazine

Drug

Ranexa is FDA approved for chronic angina

Other names: Ranexa®

Primary outcomes

  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 outcomes

  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)

Sponsors and collaborators

Lead sponsor

Ohio State University

Other

Collaborators

  • Gilead Sciences

Registry information

Official study title

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

Important dates

Study start
2014
Primary completion
2017
Study completion
2017
First posted
Sep 29, 2014
Registry last updated
Mar 5, 2019

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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