Ignite Creation Date:
2025-12-24 @ 4:57 PM
Ignite Modification Date:
2025-12-30 @ 8:00 PM
Study NCT ID:
NCT00872950
Status:
APPROVED_FOR_MARKETING
Last Update Posted:
2022-04-11
First Post:
2009-03-30
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
3,4-Diaminopyridine Use in Lambert-Eaton Myasthenic Syndrome(LEMS) and Congenital Myasthenic Syndromes (CMS)
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D015624', 'term': 'Lambert-Eaton Myasthenic Syndrome'}, {'id': 'D020294', 'term': 'Myasthenic Syndromes, Congenital'}], 'ancestors': [{'id': 'D009157', 'term': 'Myasthenia Gravis'}, {'id': 'D020361', 'term': 'Paraneoplastic Syndromes, Nervous System'}, {'id': 'D009423', 'term': 'Nervous System Neoplasms'}, {'id': 'D009371', 'term': 'Neoplasms by Site'}, {'id': 'D009369', 'term': 'Neoplasms'}, {'id': 'D010257', 'term': 'Paraneoplastic Syndromes'}, {'id': 'D020274', 'term': 'Autoimmune Diseases of the Nervous System'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D019636', 'term': 'Neurodegenerative Diseases'}, {'id': 'D020511', 'term': 'Neuromuscular Junction Diseases'}, {'id': 'D009468', 'term': 'Neuromuscular Diseases'}, {'id': 'D001327', 'term': 'Autoimmune Diseases'}, {'id': 'D007154', 'term': 'Immune System Diseases'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D000077770', 'term': 'Amifampridine'}], 'ancestors': [{'id': 'D015761', 'term': '4-Aminopyridine'}, {'id': 'D000631', 'term': 'Aminopyridines'}, {'id': 'D000588', 'term': 'Amines'}, {'id': 'D009930', 'term': 'Organic Chemicals'}, {'id': 'D011725', 'term': 'Pyridines'}, {'id': 'D006573', 'term': 'Heterocyclic Compounds, 1-Ring'}, {'id': 'D006571', 'term': 'Heterocyclic Compounds'}]}}, 'protocolSection': {'designModule': {'studyType': 'EXPANDED_ACCESS', 'expandedAccessTypes': {'treatment': True}}, 'statusModule': {'overallStatus': 'APPROVED_FOR_MARKETING', 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2022-03', 'lastUpdateSubmitDate': '2022-03-24', 'studyFirstSubmitDate': '2009-03-30', 'studyFirstSubmitQcDate': '2009-03-31', 'lastUpdatePostDateStruct': {'date': '2022-04-11', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2009-04-01', 'type': 'ESTIMATED'}}, 'conditionsModule': {'keywords': ['LEMS', 'CMS', '3,4 DIAMINOPYRIDINE', '3,4 DAP'], 'conditions': ['Lambert-Eaton Myasthenic Syndrome', 'Congenital Myasthenic Syndrome']}, 'referencesModule': {'seeAlsoLinks': [{'url': 'http://www.lahey.org', 'label': 'Lahey Hospital \\& Medical Center'}]}, 'descriptionModule': {'briefSummary': 'The purpose of this study is to determine the effectiveness and adverse effects of 3,4-diaminopyridine for the treatment of the Lambert-Eaton Myasthenic Syndrome (LEMS) and Congenital Myasthenic Syndromes (CMS).', 'detailedDescription': 'This is an open-label, non-randomized, non-comparative expanded access study. Up to 25 patients with clinically proven paraneoplastic or primary autoimmune LEMS per EMG and positive voltage-gated calcium channel antibody serology, OR patients with clinically proven CMS per electromyogram (EMG), biopsy or genetic testing who meet the selection criteria outlined in sections 3.1 and 3.2 will be enrolled in this study. Subjects will receive 3,4-diaminopyridine (3,4-DAP) starting with a low dose and titrating up per efficacy and patient tolerance to a maximum daily dose of 100mg . Treatment will continue until the subject experiences a treatment-limiting toxicity, voluntarily withdraws consent, transfers to another site, dies, becomes lost to follow-up, is no longer receiving clinical benefit from 3,4-DAP (in the opinion of the subject and/or the investigator), or the Jacobus Pharmaceuticals or the FDA discontinues the study.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* LEMS diagnosis OR Pre or Post Synaptic CMS diagnosis\n* 18 years or older\n* Females must have negative pregnancy test and be willing to practice an effective form of birth control\n* No prolonged QT syndrome as indicated by baseline EKG\n\nExclusion Criteria:\n\n* Known sensitivity to 3,4-DIAMINOPYRIDINE\n* History of seizures and/or severe asthma'}, 'identificationModule': {'nctId': 'NCT00872950', 'acronym': 'LEMS', 'briefTitle': '3,4-Diaminopyridine Use in Lambert-Eaton Myasthenic Syndrome(LEMS) and Congenital Myasthenic Syndromes (CMS)', 'organization': {'class': 'OTHER', 'fullName': 'Lahey Clinic'}, 'officialTitle': 'Open Label Trial Of 3,4-Diaminopyridine In Lambert-Eaton Myasthenic Syndrome (LEMS) and Congenital Myasthenic Syndromes (CMS)', 'orgStudyIdInfo': {'id': '2001-040'}}, 'armsInterventionsModule': {'interventions': [{'name': '3,4-DIAMINOPYRIDINE', 'type': 'DRUG', 'otherNames': ['3,4 DAP'], 'description': 'Treatment will begin with a low dose and will be increased as clinically needed as tolerated. The upper limit will be a total of 100mg/day.'}]}, 'contactsLocationsModule': {'locations': [{'zip': '01805', 'city': 'Burlington', 'state': 'Massachusetts', 'country': 'United States', 'facility': 'Lahey Hospital & Medical Center', 'geoPoint': {'lat': 42.50482, 'lon': -71.19561}}], 'overallOfficials': [{'name': 'Jayashri Srinivasan, MD,MCRP,PhD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Lahey Clinic'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Lahey Clinic', 'class': 'OTHER'}, 'responsibleParty': {'type': 'SPONSOR'}}}}