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



Status:Completed
Conditions:Neurology
Therapuetic Areas:Neurology
Healthy:No
Age Range:18 - 100
Updated:3/7/2019
Start Date:August 2014
End Date:December 18, 2017

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Open Label Trial of Ranolazine in Myotonia Congenita, Paramyotonia Congenita, & 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.

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.

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
We found this trial at
1
site
410 W 10th Ave
Columbus, Ohio 43210
(614) 293-8652
Principal Investigator: William D. Arnold, MD
Phone: 614-366-9050
The Ohio State University, Wexner Medical Center Located in Columbus, The Ohio State University Wexner...
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mi
from
Columbus, OH
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