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

Sponsor

Ohio State University (Other)

Overall Status

Completed

CT.gov ID

NCT02251457

Collaborator

Gilead Sciences (Industry)

Enrollment

Location

Arm

40.6

Duration (Months)

0.9

Patients Per Site Per Month

Study Details

Study Description

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.

Study Design

Study Type:

Interventional

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 :

Aug 1, 2014

Actual Primary Completion Date :

Dec 18, 2017

Actual Study Completion Date :

Dec 18, 2017

Arms and Interventions

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 Names: Ranexa®

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 Names:

Ranexa®

Outcome Measures

Primary Outcome Measures

Questionnaires: Short Form Health Survey (SF-36) and Individualized Neuromuscular Quality of Life Questionnaire (INQoL) [1 month]
quality of life measurements for overall health and neuromuscular disease
Muscle tasks [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 [1 month]
To see if the electrical potentials produced by the muscle fibers change.

Secondary Outcome Measures

Electrocardiogram (ECG) [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)

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years to 100 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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