Ignite Creation Date:
2025-12-24 @ 4:36 PM
Ignite Modification Date:
2025-12-25 @ 2:26 PM
Study NCT ID:
NCT03579966
Status:
TERMINATED
Last Update Posted:
2024-05-07
First Post:
2018-06-05
Is NOT Gene Therapy:
False
Has Adverse Events:
True
Brief Title:
Long Term Safety Study of Amifampridine Phosphate in MuSK-MG (Muscle Specific Tyrosine Kinase Myasthenia Gravis)
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'submissionTracking': {'firstMcpInfo': {'postDateStruct': {'date': '2024-04-10', 'type': 'ACTUAL'}}}}, 'conditionBrowseModule': {'meshes': [{'id': 'D009157', 'term': 'Myasthenia Gravis'}], 'ancestors': [{'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'}]}, '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'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'gingenito@catalystpharma.com', 'phone': '3054203200', 'title': 'Gary Ingenito, MD, Chief Medical Officer', 'organization': 'Catalyst Pharmaceuticals, Inc.'}, 'certainAgreement': {'piSponsorEmployee': False, 'restrictiveAgreement': False}, 'limitationsAndCaveats': {'description': 'The study was terminated due to lack of measure appropriate data. The analysis of the MG-ADL score was removed in Statistical Analysis Plan (SAP), dated 21FEB2023.'}}, 'adverseEventsModule': {'timeFrame': 'Study Duration 39 months', 'description': 'Serious classification based on the FDA regulatory definition of a serious AE.', 'eventGroups': [{'id': 'EG000', 'title': 'Serious Adverse Event', 'description': 'Amifampridine Phosphate: tablets equivalent to 10mg amifampridine, titrated to an efficacious and tolerable dose, 3 to 4 times a day.', 'otherNumAtRisk': 63, 'deathsNumAtRisk': 63, 'otherNumAffected': 58, 'seriousNumAtRisk': 63, 'deathsNumAffected': 0, 'seriousNumAffected': 14}], 'otherEvents': [{'term': 'Cataract', 'stats': [{'groupId': 'EG000', 'numAtRisk': 63, 'numEvents': 4, 'numAffected': 4}], 'organSystem': 'Ear and labyrinth disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Diplopia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 63, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Ear and labyrinth disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Vitreous Floaters', 'stats': [{'groupId': 'EG000', 'numAtRisk': 63, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Abdominal Pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 63, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Abdominal Pain Upper', 'stats': [{'groupId': 'EG000', 'numAtRisk': 63, 'numEvents': 5, 'numAffected': 5}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Diarrhea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 63, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Hypoasthesia Oral', 'stats': [{'groupId': 'EG000', 'numAtRisk': 63, 'numEvents': 4, 'numAffected': 4}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 63, 'numEvents': 4, 'numAffected': 4}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Paresthesia Oral', 'stats': [{'groupId': 'EG000', 'numAtRisk': 63, 'numEvents': 15, 'numAffected': 15}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Vomiting', 'notes': 'Does not include SAE', 'stats': [{'groupId': 'EG000', 'numAtRisk': 63, 'numEvents': 4, 'numAffected': 4}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Asthenia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 63, 'numEvents': 4, 'numAffected': 4}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Fatigue', 'stats': [{'groupId': 'EG000', 'numAtRisk': 63, 'numEvents': 6, 'numAffected': 6}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 63, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Bronchitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 63, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Corona Virus Infection', 'notes': 'Does not include the SAEs', 'stats': [{'groupId': 'EG000', 'numAtRisk': 63, 'numEvents': 4, 'numAffected': 4}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 63, 'numEvents': 4, 'numAffected': 4}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Onychomycosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 63, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Pneumonia', 'notes': 'Does not Include the SAEs', 'stats': [{'groupId': 'EG000', 'numAtRisk': 63, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Sinusitus', 'stats': [{'groupId': 'EG000', 'numAtRisk': 63, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Upper Respiratory Infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 63, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Urinary Tract Infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 63, 'numEvents': 6, 'numAffected': 6}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Contusion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 63, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Hypokalemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 63, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Arthralgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 63, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Back Pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 63, 'numEvents': 4, 'numAffected': 4}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Muscle Spasms', 'stats': [{'groupId': 'EG000', 'numAtRisk': 63, 'numEvents': 4, 'numAffected': 4}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Muscular Weakness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 63, 'numEvents': 4, 'numAffected': 4}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Myalgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 63, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Pain in extremity', 'stats': [{'groupId': 'EG000', 'numAtRisk': 63, 'numEvents': 4, 'numAffected': 4}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Dizziness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 63, 'numEvents': 4, 'numAffected': 4}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 63, 'numEvents': 7, 'numAffected': 7}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Hypoasthesia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 63, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Myasthenia Gravis', 'notes': 'Does not include SAEs', 'stats': [{'groupId': 'EG000', 'numAtRisk': 63, 'numEvents': 6, 'numAffected': 6}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Paraesthesia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 63, 'numEvents': 14, 'numAffected': 14}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Depression', 'stats': [{'groupId': 'EG000', 'numAtRisk': 63, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Insomnia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 63, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 63, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Dyspnea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 63, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Oropharyngeal Pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 63, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Alopecia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 63, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Rash', 'stats': [{'groupId': 'EG000', 'numAtRisk': 63, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}], 'seriousEvents': [{'term': 'Aortic Valve Stenosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 63, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Dysphagia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 63, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'small intestinal obstruction', 'stats': [{'groupId': 'EG000', 'numAtRisk': 63, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 63, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'corona virus infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 63, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Pneumonia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 63, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Staphylococcal infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 63, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'cervical spine stenosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 63, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Bone Neoplasm', 'stats': [{'groupId': 'EG000', 'numAtRisk': 63, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Neoplasms benign, malignant and unspecified (incl cysts and polyps)', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Papillary Thyroid Cancer', 'stats': [{'groupId': 'EG000', 'numAtRisk': 63, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Neoplasms benign, malignant and unspecified (incl cysts and polyps)', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Myasthenia Gravis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 63, 'numEvents': 4, 'numAffected': 4}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Myasthenia Gravis Crisis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 63, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'abortion Spontaneous', 'stats': [{'groupId': 'EG000', 'numAtRisk': 63, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Pregnancy, puerperium and perinatal conditions', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}], 'frequencyThreshold': '2'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Number of Participants With Treatment-Emergent Adverse Events (Safety and Tolerability)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '63', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Treatment Emergent Adverse Events', 'description': 'Amifampridine Phosphate: tablets equivalent to 10mg amifampridine, titrated to an efficacious and tolerable dose, 3 to 4 times a day.'}], 'classes': [{'categories': [{'measurements': [{'value': '58', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'over 39 months', 'description': 'Evaluate the long-term safety and tolerability of amifampridine phosphate through the number of patients with treatment-emergent adverse events mapped to the Medical Dictionary for Regulatory Activities (MedDRA) SOCs and PTs. \\[ Time Frame: over 39 months \\] Descriptive statistics will be used to summarize study data.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Amifampridine Phosphate', 'description': 'tablets equivalent to 10mg amifampridine, 3 to 4 times per day. Initially, the daily amifampridine doses were to be the doses that were determined at the end of the Run-in Period from the MSK-002 study. The usual range was from 30 to 80 mg total daily dose, given in three (3) or four (4) doses, with no single dose \\> 20 mg\n\nAmifampridine Phosphate: tablets equivalent to 10mg amifampridine, titrated to an efficacious and tolerable dose, 3 to 4 times a day'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '63'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '3'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '60'}]}], 'dropWithdraws': [{'type': 'Adverse Event', 'reasons': [{'groupId': 'FG000', 'numSubjects': '6'}]}, {'type': 'Lost to Follow-up', 'reasons': [{'groupId': 'FG000', 'numSubjects': '4'}]}, {'type': 'Physician Decision', 'reasons': [{'groupId': 'FG000', 'numSubjects': '10'}]}, {'type': 'Pregnancy', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}]}, {'type': 'Withdrawal by Subject', 'reasons': [{'groupId': 'FG000', 'numSubjects': '16'}]}, {'type': 'Study terminated by Sponsor', 'reasons': [{'groupId': 'FG000', 'numSubjects': '22'}]}, {'type': 'Protocol Violation', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}]}]}], 'recruitmentDetails': 'extension study to MSK-002'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '63', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'Amifampridine Phosphate', 'description': 'tablets equivalent to 10mg amifampridine, 3 to 4 times per day. Initially, the daily amifampridine doses were to be the doses that were determined at the end of the Run-in Period from the MSK-002 study. The usual range was from 30 to 80 mg total daily dose, given in three (3) or four (4) doses, with no single dose \\> 20 mg\n\nAmifampridine Phosphate: tablets equivalent to 10mg amifampridine, titrated to an efficacious and tolerable dose, 3 to 4 times a day'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '63', 'groupId': 'BG000'}]}], 'categories': [{'title': '<=18 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '53', 'groupId': 'BG000'}]}, {'title': '>=65 years', 'measurements': [{'value': '10', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Age, Continuous', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '63', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '53', 'spread': '14.08', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '63', 'groupId': 'BG000'}]}], 'categories': [{'title': 'Female', 'measurements': [{'value': '47', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '16', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race (NIH/OMB)', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '63', 'groupId': 'BG000'}]}], 'categories': [{'title': 'American Indian or Alaska Native', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Asian', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Black or African American', 'measurements': [{'value': '4', 'groupId': 'BG000'}]}, {'title': 'White', 'measurements': [{'value': '59', 'groupId': 'BG000'}]}, {'title': 'More than one race', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'United States', 'denoms': [{'units': 'Participants', 'counts': [{'value': '63', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '36', 'groupId': 'BG000'}]}]}, {'title': 'Italy', 'denoms': [{'units': 'Participants', 'counts': [{'value': '63', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '27', 'groupId': 'BG000'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}, {'title': 'Weight', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '59', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '85.9', 'spread': '24.6', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'Kg', 'dispersionType': 'STANDARD_DEVIATION', 'populationDescription': 'Baseline weight not recorded for 4 participants.'}]}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2017-11-12', 'size': 624867, 'label': 'Study Protocol: Original Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_000.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2024-03-07T14:54', 'hasProtocol': True}, {'date': '2020-02-07', 'size': 696022, 'label': 'Study Protocol: Amendment 1', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_001.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2024-03-07T14:57', 'hasProtocol': True}, {'date': '2022-10-25', 'size': 326224, 'label': 'Statistical Analysis Plan: Version 1 PBRS', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_002.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2024-03-07T14:59', 'hasProtocol': False}, {'date': '2021-10-15', 'size': 605150, 'label': 'Statistical Analysis Plan: Version 1 StatKing', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_003.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2024-03-07T15:14', 'hasProtocol': False}, {'date': '2023-02-21', 'size': 329746, 'label': 'Statistical Analysis Plan: version 2.0 PBRS', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_004.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2024-03-07T15:16', 'hasProtocol': False}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 63}}, 'statusModule': {'whyStopped': 'Lack of measure appropriate data (MG-ADL).', 'overallStatus': 'TERMINATED', 'startDateStruct': {'date': '2018-06-11', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2024-04', 'completionDateStruct': {'date': '2022-08-05', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2024-04-15', 'studyFirstSubmitDate': '2018-06-05', 'resultsFirstSubmitDate': '2024-03-12', 'studyFirstSubmitQcDate': '2018-07-05', 'lastUpdatePostDateStruct': {'date': '2024-05-07', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2024-04-15', 'studyFirstPostDateStruct': {'date': '2018-07-09', 'type': 'ACTUAL'}, 'resultsFirstPostDateStruct': {'date': '2024-05-07', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2022-08-05', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of Participants With Treatment-Emergent Adverse Events (Safety and Tolerability)', 'timeFrame': 'over 39 months', 'description': 'Evaluate the long-term safety and tolerability of amifampridine phosphate through the number of patients with treatment-emergent adverse events mapped to the Medical Dictionary for Regulatory Activities (MedDRA) SOCs and PTs. \\[ Time Frame: over 39 months \\] Descriptive statistics will be used to summarize study data.'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Amifampridine Phosphate Amifampridine', '3,4-Diaminopyridine Phosphate', '3,4-Diaminopyridine', '3,4-DAP', 'Firdapse®', 'Myasthenia Gravis', 'MuSK', 'AChR'], 'conditions': ['Myasthenia Gravis, MuSK', 'AChR Myasthenia Gravis']}, 'descriptionModule': {'briefSummary': 'Evaluate the long-term safety of amifampridine phosphate in patients with MuSK antibody positive and AChR antibody positive myasthenia gravis.', 'detailedDescription': 'The Primary objective :\n\nTo characterize the long-term safety and tolerability of amifampridine phosphate in patients with MG\n\nThe Secondary Objective:\n\nTo assess the clinical efficacy of amifampridine phosphate over time in patients with MG based on change in Myasthenia Gravis Activities of Daily Living Score (MG-ADL)\n\nThis was a long-term extension study for subjects who participated in Protocol MSK-002 where the efficacy and safety of amifampridine was evaluated in subjects diagnosed with MuSK MG or AChR-MG. The optimal dose and schedule for amifampridine from the end of the Run-in Period from Study MSK-002 was initially used for each patient. The Investigator could adjust the dose of amifampridine during the course of the trial, in order to optimize neuromuscular benefit for the patient. Clinic visits for safety assessment and for evaluation of MG-ADL were made at Months 3, 6, 9, 12, 15, 21, 27, 33 and 39. Additional visits could occur at the discretion of the Investigator.\n\nThe Original protocol (12 Nov 2017) included analysis of MG-ADL change over time, however, Statistical Analysis Plan (SAP), dated 21 Feb 2023, removed the analysis of MG-ADL score change over time.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria\n\n1. Participated in the MSK-002 study\n2. Willing and able to provide written informed consent after the nature of the study has been explained and before the start of any research-related procedures.\n3. Female patients of childbearing potential must have a negative pregnancy test (urine human chorionic gonadotropin \\[HCG\\] at the end of MSK-002 study); and must practice an effective, reliable contraceptive regimen during the study and for up to 30 days following discontinuation of treatment.\n4. Ability to participate in the study based on overall health of the patient and disease prognosis, as applicable, in the opinion of the Investigator; and able to comply with all requirements of the protocol, including completion of study questionnaires.\n\nIndividuals who met any of the exclusion criteria in the original protocol or those listed below are not eligible to participate in the study:\n\n1. Epilepsy and currently on medication.\n2. Clinically significant abnormalities in 12 lead ECG, in the opinion of the Investigator.\n3. Breastfeeding or pregnant at Screening or planning to become pregnant at any time during the study.\n4. Intolerable amifampridine-related side effects\n5. Treatment with an investigational drug (other than amifampridine) or device while participating in this study.\n6. Any medical condition that, in the opinion of the Investigator, might interfere with the patient's participation in the study, poses an added risk for the patient, or confound the assessment of the patient."}, 'identificationModule': {'nctId': 'NCT03579966', 'briefTitle': 'Long Term Safety Study of Amifampridine Phosphate in MuSK-MG (Muscle Specific Tyrosine Kinase Myasthenia Gravis)', 'organization': {'class': 'INDUSTRY', 'fullName': 'Catalyst Pharmaceuticals, Inc.'}, 'officialTitle': 'Long Term Safety Study of Amifampridine Phosphate in Patients With MuSK Antibody Positive and AChR (Acetylcholine Receptor) Antibody Positive Myasthenia Gravis', 'orgStudyIdInfo': {'id': 'MSK-003'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'amifampridine phosphate', 'description': 'tablets equivalent to 10mg amifampridine, 3 to 4 times per day. Initially, the daily amifampridine doses were to be the doses that were determined at the end of the Run-in Period from the MSK-002 study. The usual range was from 30 to 80 mg total daily dose, given in three (3) or four (4) doses, with no single dose \\> 20 mg', 'interventionNames': ['Drug: Amifampridine Phosphate']}], 'interventions': [{'name': 'Amifampridine Phosphate', 'type': 'DRUG', 'description': 'tablets equivalent to 10mg amifampridine, titrated to an efficacious and tolerable dose, 3 to 4 times a day', 'armGroupLabels': ['amifampridine phosphate']}]}, 'contactsLocationsModule': {'locations': [{'zip': '44195', 'city': 'Cleveland', 'state': 'Ohio', 'country': 'United States', 'facility': 'Cleveland Clinic', 'geoPoint': {'lat': 41.4995, 'lon': -81.69541}}, {'zip': '19104', 'city': 'Philadelphia', 'state': 'Pennsylvania', 'country': 'United States', 'facility': 'Univerity of Pennsylvania', 'geoPoint': {'lat': 39.95238, 'lon': -75.16362}}, {'zip': '11-201332', 'city': 'Milan', 'country': 'Italy', 'facility': 'Department of Neuroimmunology and Neuromuscular Diseases Carlo Besta Neurological Institute Via Celoria', 'geoPoint': {'lat': 45.46427, 'lon': 9.18951}}], 'overallOfficials': [{'name': 'Renato Mantegazza, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Carlo Besta Neurological Institute'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Catalyst Pharmaceuticals, Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}