Ignite Creation Date:
2025-12-25 @ 4:02 AM
Ignite Modification Date:
2026-03-03 @ 6:45 PM
Study NCT ID:
NCT02561702
Status:
COMPLETED
Last Update Posted:
2018-03-26
First Post:
2015-09-24
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Mexiletine for Muscle Cramps in Charcot Marie Tooth Disease
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D002607', 'term': 'Charcot-Marie-Tooth Disease'}], 'ancestors': [{'id': 'D015417', 'term': 'Hereditary Sensory and Motor Neuropathy'}, {'id': 'D009421', 'term': 'Nervous System Malformations'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D020271', 'term': 'Heredodegenerative Disorders, Nervous System'}, {'id': 'D019636', 'term': 'Neurodegenerative Diseases'}, {'id': 'D011115', 'term': 'Polyneuropathies'}, {'id': 'D010523', 'term': 'Peripheral Nervous System Diseases'}, {'id': 'D009468', 'term': 'Neuromuscular Diseases'}, {'id': 'D000013', 'term': 'Congenital Abnormalities'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D008801', 'term': 'Mexiletine'}], 'ancestors': [{'id': 'D011437', 'term': 'Propylamines'}, {'id': 'D000588', 'term': 'Amines'}, {'id': 'D009930', 'term': 'Organic Chemicals'}, {'id': 'D010647', 'term': 'Phenyl Ethers'}, {'id': 'D010636', 'term': 'Phenols'}, {'id': 'D001555', 'term': 'Benzene Derivatives'}, {'id': 'D006841', 'term': 'Hydrocarbons, Aromatic'}, {'id': 'D006844', 'term': 'Hydrocarbons, Cyclic'}, {'id': 'D006838', 'term': 'Hydrocarbons'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'david_herrmann@urmc.rochester.edu', 'phone': '585-275-4568', 'title': 'David Herrmann', 'organization': 'University of Rochester'}, 'certainAgreement': {'piSponsorEmployee': True}}, 'adverseEventsModule': {'eventGroups': [{'id': 'EG000', 'title': 'Mexiletine', 'description': 'Participants will receive 150 mg of mexiletine by mouth 3 times daily for 5-7 days followed by 7 day washout period and 5-7 days of placebo (240 mg of lactose powder) taken by mouth 3 times daily\n\nPlacebo: 240 mg lactose powder in a size 3 capsule taken by mouth 3 times daily for 5-7 days\n\nMexiletine: 150 mg Mexiletine taken by mouth in capsule form 3 times daily for 5-7 days', 'otherNumAtRisk': 4, 'deathsNumAtRisk': 4, 'otherNumAffected': 0, 'seriousNumAtRisk': 4, 'deathsNumAffected': 0, 'seriousNumAffected': 0}, {'id': 'EG001', 'title': 'Placebo', 'description': 'Participants will receive placebo (240 mg of lactose powder) taken by mouth 3 times daily for 5-7 days followed by 7 day washout period and 5-7 days of 150 mg of mexiletine by mouth 3 times daily\n\nPlacebo: 240 mg lactose powder in a size 3 capsule taken by mouth 3 times daily for 5-7 days\n\nMexiletine: 150 mg Mexiletine taken by mouth in capsule form 3 times daily for 5-7 days', 'otherNumAtRisk': 4, 'deathsNumAtRisk': 4, 'otherNumAffected': 0, 'seriousNumAtRisk': 4, 'deathsNumAffected': 0, 'seriousNumAffected': 0}], 'frequencyThreshold': '0'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Number of Participants With a Decrease in Cramp Duration', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Mexiletine', 'description': 'Participants will receive 150 mg of mexiletine by mouth 3 times daily for 5-7 days followed by 7 day washout period and 5-7 days of placebo (240 mg of lactose powder) taken by mouth 3 times daily\n\nPlacebo: 240 mg lactose powder in a size 3 capsule taken by mouth 3 times daily for 5-7 days\n\nMexiletine: 150 mg Mexiletine taken by mouth in capsule form 3 times daily for 5-7 days'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Participants will receive placebo (240 mg of lactose powder) taken by mouth 3 times daily for 5-7 days followed by 7 day washout period and 5-7 days of 150 mg of mexiletine by mouth 3 times daily\n\nPlacebo: 240 mg lactose powder in a size 3 capsule taken by mouth 3 times daily for 5-7 days\n\nMexiletine: 150 mg Mexiletine taken by mouth in capsule form 3 times daily for 5-7 days'}], 'classes': [{'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}], 'paramType': 'NUMBER', 'timeFrame': '120 minutes', 'description': 'Participant will be evaluated for muscle cramps 120 minutes following a single dose of oral mexiletine. The Clinical Evaluator applied pressure to provoke hamstring cramps bilaterally, one at a time 2 hours after dose. The cramp duration in seconds of the right hamstring was used.', 'unitOfMeasure': 'participants', 'reportingStatus': 'POSTED'}, {'type': 'PRIMARY', 'title': 'Number of Participants With a Decrease in Cramp Intensity', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Mexiletine', 'description': 'Participants will receive 150 mg of mexiletine by mouth 3 times daily for 5-7 days followed by 7 day washout period and 5-7 days of placebo (240 mg of lactose powder) taken by mouth 3 times daily\n\nPlacebo: 240 mg lactose powder in a size 3 capsule taken by mouth 3 times daily for 5-7 days\n\nMexiletine: 150 mg Mexiletine taken by mouth in capsule form 3 times daily for 5-7 days'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Participants will receive placebo (240 mg of lactose powder) taken by mouth 3 times daily for 5-7 days followed by 7 day washout period and 5-7 days of 150 mg of mexiletine by mouth 3 times daily\n\nPlacebo: 240 mg lactose powder in a size 3 capsule taken by mouth 3 times daily for 5-7 days\n\nMexiletine: 150 mg Mexiletine taken by mouth in capsule form 3 times daily for 5-7 days'}], 'classes': [{'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}]}]}], 'paramType': 'NUMBER', 'timeFrame': '120 minutes', 'description': 'Participant will be evaluated for muscle cramps 120 minutes following a single dose of oral mexiletine. The Clinical Evaluator applied pressure to provoke hamstring cramps bilaterally, one at a time 2 hours after dose. The cramp intensity of the right hamstring was reported by the subject on a scale of 1-10 with 1 being weak and 10 being severe.', 'unitOfMeasure': 'participants', 'reportingStatus': 'POSTED'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Mexiletine First/Placebo Second', 'description': 'Participants will receive 150 mg of mexiletine by mouth 3 times daily for 5-7 days followed by 7 day washout period and 5-7 days of placebo (240 mg of lactose powder) taken by mouth 3 times daily\n\nPlacebo: 240 mg lactose powder in a size 3 capsule taken by mouth 3 times daily for 5-7 days\n\nMexiletine: 150 mg Mexiletine taken by mouth in capsule form 3 times daily for 5-7 days'}, {'id': 'FG001', 'title': 'Placebo First/Mexiletine Second', 'description': 'Participants will receive placebo (240 mg of lactose powder) taken by mouth 3 times daily for 5-7 days followed by 7 day washout period and 5-7 days of 150 mg of mexiletine by mouth 3 times daily\n\nPlacebo: 240 mg lactose powder in a size 3 capsule taken by mouth 3 times daily for 5-7 days\n\nMexiletine: 150 mg Mexiletine taken by mouth in capsule form 3 times daily for 5-7 days'}], 'periods': [{'title': 'First Intervention', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '2'}, {'groupId': 'FG001', 'numSubjects': '2'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '2'}, {'groupId': 'FG001', 'numSubjects': '2'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}]}]}, {'title': 'Washout', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '2'}, {'groupId': 'FG001', 'numSubjects': '2'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '2'}, {'groupId': 'FG001', 'numSubjects': '2'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}]}]}, {'title': 'Second Intervention', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '2'}, {'groupId': 'FG001', 'numSubjects': '2'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '2'}, {'groupId': 'FG001', 'numSubjects': '2'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}]}]}]}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'All Participants'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '3', 'groupId': 'BG000'}]}, {'title': '>=65 years', 'measurements': [{'value': '1', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '2', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '2', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'United States', 'categories': [{'measurements': [{'value': '4', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'CROSSOVER'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 4}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2015-09'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2018-02', 'completionDateStruct': {'date': '2016-12-31', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2018-02-23', 'studyFirstSubmitDate': '2015-09-24', 'resultsFirstSubmitDate': '2018-01-25', 'studyFirstSubmitQcDate': '2015-09-25', 'lastUpdatePostDateStruct': {'date': '2018-03-26', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2018-02-23', 'studyFirstPostDateStruct': {'date': '2015-09-28', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2018-03-26', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2016-12-31', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of Participants With a Decrease in Cramp Duration', 'timeFrame': '120 minutes', 'description': 'Participant will be evaluated for muscle cramps 120 minutes following a single dose of oral mexiletine. The Clinical Evaluator applied pressure to provoke hamstring cramps bilaterally, one at a time 2 hours after dose. The cramp duration in seconds of the right hamstring was used.'}, {'measure': 'Number of Participants With a Decrease in Cramp Intensity', 'timeFrame': '120 minutes', 'description': 'Participant will be evaluated for muscle cramps 120 minutes following a single dose of oral mexiletine. The Clinical Evaluator applied pressure to provoke hamstring cramps bilaterally, one at a time 2 hours after dose. The cramp intensity of the right hamstring was reported by the subject on a scale of 1-10 with 1 being weak and 10 being severe.'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'conditions': ['Charcot Marie Tooth Disease']}, 'descriptionModule': {'briefSummary': 'Charcot Marie Tooth Disease is a family of inherited peripheral neuropathies, with over 70 causative genes identified to date.1-4 Muscle cramps are frequent in CMT, affecting up to 85% of patients with some subtypes of CMT. These cramps impact quality of life and have been identified as an important therapeutic target for clinical trials in CMT.1-4 There is no FDA approved treatment for muscle cramps.5 Mexiletine is a sodium channel blocker approved for treatment of arrhythmias. As a sodium channel blocker, mexiletine offers the promise of effective therapy for muscle cramps.', 'detailedDescription': 'This study will provide data on the short term efficacy of oral mexiletine in helping to prevent muscle cramps in adults with CMT. The study will also assess the short-term safety and tolerability of low dose mexiletine in adults with CMT.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* The subject has known CMT, previously diagnosed by a neurologist with subspeciality expertise in neuromuscular disorders/peripheral neuropathy. The diagnosis of CMT relies on a combination of clinical history (including family history), neurological examination electrophysiological features, and prior genetic testing results.\n\n * The subject is at least 18 years old, and has signed the Informed Consent Form.\n * The subject is ambulatory (cane, walker, orthoses allowed).\n * The subject has experienced muscle cramps and has known provokable hamstring or calf muscle cramps on MVC.\n * The subject has a reliable method of birth control (if a female subject of child bearing potential). Reliable birth control is defined as Hormonal contraception Intrauterine contraception/device Any two barrier methods (combination of male or female condom with diaphragm sponge or cervical cap) together with spermicidal foam/gel/film/cream/suppository True abstinence\n\nExclusion Criteria:\n\n* The subject has a known neuropathy from another source (e.g., diabetes, drug induced, alcohol, etc.).\n\n * The subject has an untreated medical disorder known to predispose to muscle cramps\n * The subject is pregnant or nursing, has a known mexiletine allergy or has taken mexiletine in the past 12 weeks.\n * The subject is participating in another therapeutic trial.\n * The subject has second or 3rd degree heart block, atrial flutter/fibrillation, ventricular arrhythmias, or is receiving treatment of a cardiac arrhythmia.\n * The subject is currently taking another agent for muscle cramps or a muscle relaxant (e.g., benzodiazepine, baclofen, tizanidine, soma, meprobromate).\n * The subject is on another sodium channel blocker or medication that precludes administration of mexiletine.\n * The subject has known diabetes mellitus, liver or kidney disease requiring ongoing treatment, untreated thyroid dysfunction, symptomatic cardiomyopathy, or symptomatic coronary artery disease.\n * The subject, in the opinion of the investigator, is unsuitable for enrollment for any other reason'}, 'identificationModule': {'nctId': 'NCT02561702', 'briefTitle': 'Mexiletine for Muscle Cramps in Charcot Marie Tooth Disease', 'organization': {'class': 'OTHER', 'fullName': 'University of Rochester'}, 'officialTitle': 'Mexiletine for Muscle Cramps in Charcot Marie Tooth Disease', 'orgStudyIdInfo': {'id': 'RSRB00057653'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Mexiletine first/placebo second', 'description': 'Participants will receive 150 mg of mexiletine by mouth 3 times daily for 5-7 days followed by 7 day washout period and 5-7 days of placebo (240 mg of lactose powder) taken by mouth 3 times daily', 'interventionNames': ['Other: Placebo', 'Drug: Mexiletine']}, {'type': 'EXPERIMENTAL', 'label': 'placebo first/mexiletine second', 'description': 'Participants will receive placebo (240 mg of lactose powder) taken by mouth 3 times daily for 5-7 days followed by 7 day washout period and 5-7 days of 150 mg of mexiletine by mouth 3 times daily', 'interventionNames': ['Other: Placebo', 'Drug: Mexiletine']}], 'interventions': [{'name': 'Placebo', 'type': 'OTHER', 'otherNames': ['lactose powder'], 'description': '240 mg lactose powder in a size 3 capsule taken by mouth 3 times daily for 5-7 days', 'armGroupLabels': ['Mexiletine first/placebo second', 'placebo first/mexiletine second']}, {'name': 'Mexiletine', 'type': 'DRUG', 'description': '150 mg Mexiletine taken by mouth in capsule form 3 times daily for 5-7 days', 'armGroupLabels': ['Mexiletine first/placebo second', 'placebo first/mexiletine second']}]}, 'contactsLocationsModule': {'locations': [{'zip': '14642', 'city': 'Rochester', 'state': 'New York', 'country': 'United States', 'facility': 'University of Rochester', 'geoPoint': {'lat': 43.15478, 'lon': -77.61556}}], 'overallOfficials': [{'name': 'David Herrmann, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'University of Rochester'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'University of Rochester', 'class': 'OTHER'}, 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'Professor', 'investigatorFullName': 'David Herrmann', 'investigatorAffiliation': 'University of Rochester'}}}}