Ignite Creation Date:
2025-12-24 @ 6:47 PM
Ignite Modification Date:
2026-01-02 @ 12:35 PM
Study NCT ID:
NCT02251457
Status:
COMPLETED
Last Update Posted:
2019-03-05
First Post:
2014-09-25
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Study of Ranolazine in Myotonia Congenita, Paramyotonia Congenita and Myotonic Dystrophy Type 1
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D009224', 'term': 'Myotonia Congenita'}, {'id': 'D020967', 'term': 'Myotonic Disorders'}, {'id': 'D009223', 'term': 'Myotonic Dystrophy'}], 'ancestors': [{'id': 'D009135', 'term': 'Muscular Diseases'}, {'id': 'D009140', 'term': 'Musculoskeletal Diseases'}, {'id': 'D020271', 'term': 'Heredodegenerative Disorders, Nervous System'}, {'id': 'D019636', 'term': 'Neurodegenerative Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D009468', 'term': 'Neuromuscular Diseases'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D009136', 'term': 'Muscular Dystrophies'}, {'id': 'D020966', 'term': 'Muscular Disorders, Atrophic'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D000069458', 'term': 'Ranolazine'}], 'ancestors': [{'id': 'D000083', 'term': 'Acetanilides'}, {'id': 'D000813', 'term': 'Anilides'}, {'id': 'D000577', 'term': 'Amides'}, {'id': 'D009930', 'term': 'Organic Chemicals'}, {'id': 'D000814', 'term': 'Aniline Compounds'}, {'id': 'D000588', 'term': 'Amines'}, {'id': 'D010879', 'term': 'Piperazines'}, {'id': 'D006573', 'term': 'Heterocyclic Compounds, 1-Ring'}, {'id': 'D006571', 'term': 'Heterocyclic Compounds'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 35}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2014-08'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2019-03', 'completionDateStruct': {'date': '2017-12-18', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2019-03-01', 'studyFirstSubmitDate': '2014-09-25', 'studyFirstSubmitQcDate': '2014-09-26', 'lastUpdatePostDateStruct': {'date': '2019-03-05', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2014-09-29', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2017-12-18', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Questionnaires: Short Form Health Survey (SF-36) and Individualized Neuromuscular Quality of Life Questionnaire (INQoL)', 'timeFrame': '1 month', 'description': 'quality of life measurements for overall health and neuromuscular disease'}, {'measure': 'Muscle tasks', 'timeFrame': '1 month', 'description': 'The subject is observed and timed while rising from an arm chair, walking 3 meters, turning, walking back, and sitting down again'}, {'measure': 'Electromyography (EMG) Myotonia', 'timeFrame': '1 month', 'description': 'To see if the electrical potentials produced by the muscle fibers change.'}], 'secondaryOutcomes': [{'measure': 'Electrocardiogram (ECG)', 'timeFrame': '1 month', '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)"}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'keywords': ['Myotonia Congenita', 'ranolazine', 'Ohio State', 'Open Label', 'Paramyotonia Congenita', 'Myotonic Dystrophy 1'], 'conditions': ['Myotonia Congenita', 'Paramyotonia Congenita', 'Myotonic Dystrophy 1']}, 'referencesModule': {'references': [{'pmid': '25515836', 'type': 'BACKGROUND', '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.'}, {'pmid': '40197813', 'type': 'DERIVED', 'citation': 'Spillane J, Trip J, Drost G, Faber CG, Hanna MG, Nevitt SJ, Vivekanandam V. Drug treatment for myotonia. Cochrane Database Syst Rev. 2025 Apr 8;4(4):CD004762. doi: 10.1002/14651858.CD004762.pub3.'}, {'pmid': '30390395', 'type': 'DERIVED', '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.'}]}, 'descriptionModule': {'briefSummary': '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.', 'detailedDescription': '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.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '100 Years', 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* Diagnosis of myotonia congenital, paramyotonia congenital or Myotonic Dystrophy Type 1 established by genetic testing in the subject or in a first-degree relative.\n* Clinically evident myotonia\n\nExclusion Criteria:\n\n* Contraindications to ranolazine use:\n\n * for fungus infection: ketoconazole (Nizoral), itraconazole (Sporanox, Onmel)\n * for infection: clarithromycin (Biaxin)\n * for depression: nefazodone\n * for HIV: nelfinavir (Viracept), ritonavir (Norvir), lopinavir and ritonavir (Kaletra), indinavir (Crixivan), saquinavir (Invirase).\n * for tuberculosis (TB): rifampin (Rifadin), rifabutin (Mycobutin), rifapentine (Priftin)\n * for seizures: phenobarbital, phenytoin (Phenytek, Dilantin, Dilantin-125), carbamazepine (Tegretol)\n * the herbal supplement St. John's wort\n\n * you have scarring (cirrhosis) of your liver\n* 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.\n* QTc \\>470 ms for men and \\>480 ms for women.\n* Women who are pregnant or breastfeeding\n* Direct family history of sudden cardiac death"}, 'identificationModule': {'nctId': 'NCT02251457', 'briefTitle': 'Study of Ranolazine in Myotonia Congenita, Paramyotonia Congenita and Myotonic Dystrophy Type 1', 'organization': {'class': 'OTHER', 'fullName': 'Ohio State University'}, 'officialTitle': 'Open Label Trial of Ranolazine in Myotonia Congenita, Paramyotonia Congenita, & Myotonic Dystrophy Type 1', 'orgStudyIdInfo': {'id': 'IN-US-259-1605'}, 'secondaryIdInfos': [{'id': 'IN-US-259-1605', 'type': 'OTHER_GRANT', 'domain': 'Gilead Sciences'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'ranolazine', 'description': 'ranolazine 500mg, twice daily for two weeks; 1000mg twice daily for 2 weeks', 'interventionNames': ['Drug: Ranolazine']}], 'interventions': [{'name': 'Ranolazine', 'type': 'DRUG', 'otherNames': ['Ranexa®'], 'description': 'Ranexa is FDA approved for chronic angina', 'armGroupLabels': ['ranolazine']}]}, 'contactsLocationsModule': {'locations': [{'zip': '43221', 'city': 'Columbus', 'state': 'Ohio', 'country': 'United States', 'facility': 'The Ohio State University Wexner Medical Center', 'geoPoint': {'lat': 39.96118, 'lon': -82.99879}}], 'overallOfficials': [{'name': 'William D Arnold, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Ohio State University'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'UNDECIDED'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Ohio State University', 'class': 'OTHER'}, 'collaborators': [{'name': 'Gilead Sciences', 'class': 'INDUSTRY'}], 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'Associate Professor', 'investigatorFullName': 'William Arnold', 'investigatorAffiliation': 'Ohio State University'}}}}