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

• Status: Completed
• Trial end date: 2017-12-18
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Ohio State University
• Collaborator: Gilead Sciences
• Treatments: Ranolazine

Criteria

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