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

Myotonic Dystrophy 1 Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02251457
• Other study ID #: IN-US-259-1605
• Secondary ID: IN-US-259-1605
• Status: Completed
• Phase: Phase 1
• Start date: August 2014
• Est. completion date: December 18, 2017

Study information

• Verified date: March 2019
• Source: Ohio State University
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 35
• Est. completion date: December 18, 2017
• Est. primary completion date: December 18, 2017
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 100 Years

Eligibility

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

Related Conditions & MeSH terms

• Myotonia
• Myotonia Congenita
• Myotonic Disorders
• Myotonic Dystrophy
• Myotonic Dystrophy 1
• Paramyotonia Congenita

Intervention

Drug:
• Ranolazine
Ranexa is FDA approved for chronic angina

Locations

Country	Name	City	State
United States	The Ohio State University Wexner Medical Center	Columbus	Ohio

Sponsors (2)

Lead Sponsor	Collaborator
Ohio State University	Gilead Sciences

Country where clinical trial is conducted

United States, 

References & Publications (1)

Novak KR, Norman J, Mitchell JR, Pinter MJ, Rich MM. Sodium channel slow inactivation as a therapeutic target for myotonia congenita. Ann Neurol. 2015 Feb;77(2):320-32. doi: 10.1002/ana.24331. Epub 2015 Jan 9. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Questionnaires: Short Form Health Survey (SF-36) and Individualized Neuromuscular Quality of Life Questionnaire (INQoL)	quality of life measurements for overall health and neuromuscular disease	1 month
Primary	Muscle tasks	The subject is observed and timed while rising from an arm chair, walking 3 meters, turning, walking back, and sitting down again	1 month
Primary	Electromyography (EMG) Myotonia	To see if the electrical potentials produced by the muscle fibers change.	1 month
Secondary	Electrocardiogram (ECG)	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)	1 month