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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

35 participants

Study Classification

INTERVENTIONAL

Study Start Date

2014-08-31

Study Completion Date

2017-12-18

Brief Summary

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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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Detailed Description

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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.

Conditions

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Myotonia Congenita Paramyotonia Congenita Myotonic Dystrophy 1

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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ranolazine

ranolazine 500mg, twice daily for two weeks; 1000mg twice daily for 2 weeks

Group Type EXPERIMENTAL

Ranolazine

Intervention Type DRUG

Ranexa is FDA approved for chronic angina

Interventions

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Ranolazine

Ranexa is FDA approved for chronic angina

Intervention Type DRUG

Other Intervention Names

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Ranexa®

Eligibility Criteria

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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

Minimum Eligible Age

18 Years

Maximum Eligible Age

100 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No