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

Primary Purpose

Myotonia Congenita, Paramyotonia Congenita, Myotonic Dystrophy 1

Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
Ranolazine
Sponsored by
Ohio State University
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Myotonia Congenita focused on measuring Myotonia Congenita, ranolazine, Ohio State, Open Label, Paramyotonia Congenita, Myotonic Dystrophy 1

Eligibility Criteria

18 Years - 100 Years (Adult, Older Adult)All SexesDoes not accept 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

Sites / Locations

  • The Ohio State University Wexner Medical Center

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

ranolazine

Arm Description

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

Outcomes

Primary Outcome Measures

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
Muscle tasks
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
To see if the electrical potentials produced by the muscle fibers change.

Secondary Outcome Measures

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)

Full Information

First Posted
September 25, 2014
Last Updated
March 1, 2019
Sponsor
Ohio State University
Collaborators
Gilead Sciences
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1. Study Identification

Unique Protocol Identification Number
NCT02251457
Brief Title
Study of Ranolazine in Myotonia Congenita, Paramyotonia Congenita and Myotonic Dystrophy Type 1
Official Title
Open Label Trial of Ranolazine in Myotonia Congenita, Paramyotonia Congenita, & Myotonic Dystrophy Type 1
Study Type
Interventional

2. Study Status

Record Verification Date
March 2019
Overall Recruitment Status
Completed
Study Start Date
August 2014 (undefined)
Primary Completion Date
December 18, 2017 (Actual)
Study Completion Date
December 18, 2017 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Ohio State University
Collaborators
Gilead Sciences

4. Oversight

Data Monitoring Committee
No

5. 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.
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Myotonia Congenita, Paramyotonia Congenita, Myotonic Dystrophy 1
Keywords
Myotonia Congenita, ranolazine, Ohio State, Open Label, Paramyotonia Congenita, Myotonic Dystrophy 1

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
35 (Actual)

8. Arms, Groups, and Interventions

Arm Title
ranolazine
Arm Type
Experimental
Arm Description
ranolazine 500mg, twice daily for two weeks; 1000mg twice daily for 2 weeks
Intervention Type
Drug
Intervention Name(s)
Ranolazine
Other Intervention Name(s)
Ranexa®
Intervention Description
Ranexa is FDA approved for chronic angina
Primary Outcome Measure Information:
Title
Questionnaires: Short Form Health Survey (SF-36) and Individualized Neuromuscular Quality of Life Questionnaire (INQoL)
Description
quality of life measurements for overall health and neuromuscular disease
Time Frame
1 month
Title
Muscle tasks
Description
The subject is observed and timed while rising from an arm chair, walking 3 meters, turning, walking back, and sitting down again
Time Frame
1 month
Title
Electromyography (EMG) Myotonia
Description
To see if the electrical potentials produced by the muscle fibers change.
Time Frame
1 month
Secondary Outcome Measure Information:
Title
Electrocardiogram (ECG)
Description
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)
Time Frame
1 month

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
100 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
William D Arnold, MD
Organizational Affiliation
Ohio State University
Official's Role
Principal Investigator
Facility Information:
Facility Name
The Ohio State University Wexner Medical Center
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43221
Country
United States

12. IPD Sharing Statement

Plan to Share IPD
Undecided
Citations:
PubMed Identifier
25515836
Citation
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.
Results Reference
background
PubMed Identifier
30390395
Citation
Lorusso S, Kline D, Bartlett A, Freimer M, Agriesti J, Hawash AA, Rich MM, Kissel JT, David Arnold W. Open-label trial of ranolazine for the treatment of paramyotonia congenita. Muscle Nerve. 2019 Feb;59(2):240-243. doi: 10.1002/mus.26372. Epub 2018 Dec 21.
Results Reference
derived

Learn more about this trial

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

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