Ignite Creation Date:
2025-12-25 @ 2:06 AM
Ignite Modification Date:
2026-01-07 @ 1:24 AM
Study NCT ID:
NCT03819660
Status:
TERMINATED
Last Update Posted:
2023-11-30
First Post:
2019-01-23
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Long Term Safety of Amifampridine Phosphate in Spinal Muscular Atrophy 3
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'submissionTracking': {'firstMcpInfo': {'postDateStruct': {'date': '2023-10-25', 'type': 'ACTUAL'}}}}, 'conditionBrowseModule': {'meshes': [{'id': 'D014897', 'term': 'Spinal Muscular Atrophies of Childhood'}], 'ancestors': [{'id': 'D009134', 'term': 'Muscular Atrophy, Spinal'}, {'id': 'D013118', 'term': 'Spinal Cord Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D020271', 'term': 'Heredodegenerative Disorders, Nervous System'}, {'id': 'D019636', 'term': 'Neurodegenerative Diseases'}, {'id': 'D016472', 'term': 'Motor Neuron Disease'}, {'id': 'D009468', 'term': 'Neuromuscular Diseases'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D000077770', 'term': 'Amifampridine'}, {'id': 'D013607', 'term': 'Tablets'}], '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'}, {'id': 'D004304', 'term': 'Dosage Forms'}, {'id': 'D004364', 'term': 'Pharmaceutical Preparations'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'gingenito@catalystpharma.com', 'phone': '3054203200', 'title': 'Gary Ingenito', 'organization': 'Catalyst Pharmaceuticals, Inc.'}, 'certainAgreement': {'piSponsorEmployee': False, 'restrictiveAgreement': False}, 'limitationsAndCaveats': {'description': 'The results of efficacy are limited to the assessment of quality of life using the INQoL. The premature interruption of the study determined that few patients had available data of INQoL from Month 6 onwards.'}}, 'adverseEventsModule': {'timeFrame': '27 months', 'description': 'The reporting period for SAEs began after obtaining informed consent and continued through 4 weeks after the last visit. A treatment emergent adverse event (TEAE) was defined as any AE that started at or after the first dose of study drug for the long-term study.', 'eventGroups': [{'id': 'EG000', 'title': 'Amifampridine Phosphate', 'description': 'The dose of amifampridine was based on optimal neuromuscular benefit determined from the Run-in Period from SMA-001 or could be modified as the discretion of the Investigator. The maximum single dose was 20 mg. The dose range for patients 6 to 16 years of age was 15 to 60 mg, and for those 17 years and older the range was from 30 to 80 mg daily.\n\nAmifampridine Phosphate 10 MG Oral Tablet: Oral tablets', 'otherNumAtRisk': 13, 'deathsNumAtRisk': 13, 'otherNumAffected': 5, 'seriousNumAtRisk': 13, 'deathsNumAffected': 0, 'seriousNumAffected': 1}], 'otherEvents': [{'term': 'Paraesthesia oral', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.0)'}, {'term': 'ABDOMINAL PAIN UPPER', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.0)'}, {'term': 'HYPERPYREXIA', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.0)'}, {'term': 'FEELING HOT', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.0)'}, {'term': 'INFLUENZA LIKE ILLNESS', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.0)'}, {'term': 'MALAISE', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.0)'}, {'term': 'FALL', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.0)'}, {'term': 'POST LUMBAR PUNCTURE SYNDROME', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.0)'}, {'term': 'HYPOKALAEMIA', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.0)'}, {'term': 'Paraesthesia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.0)'}, {'term': 'HEADACHE', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.0)'}, {'term': 'TREMOR', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.0)'}, {'term': 'RASH', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.0)'}], 'seriousEvents': [{'term': 'Intellectual Disability', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (23.0)'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Long-term Safety and Tolerability of Amifampridine', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Amifampridine Phosphate', 'description': 'The dose of amifampridine was based on optimal neuromuscular benefit determined from the Run-in Period from SMA-001 or could be modified as the discretion of the Investigator. The maximum single dose was 20 mg. The dose range for patients 6 to 16 years of age was 15 to 60 mg, and for those 17 years and older the range was from 30 to 80 mg daily.\n\nAmifampridine Phosphate 10 MG Oral Tablet: Oral tablets'}], 'classes': [{'categories': [{'measurements': [{'value': '5', 'groupId': 'OG000'}]}]}], 'analyses': [{'groupIds': ['OG000'], 'paramType': 'percentage', 'ciNumSides': 'TWO_SIDED', 'paramValue': '38.5', 'nonInferiorityType': 'OTHER'}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': '18 months', 'description': 'Number of subjects with treatment emergent adverse events (TEAE).', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'TEAE = any AE started at or after the first dose of drug for the current Long-Term Study.'}, {'type': 'SECONDARY', 'title': 'To Assess the Clinical Efficacy of Amifampridine Phosphate Over Time in Patients With SMA Type 3 Based on Changes in Quality of Life (QoL).', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Amifampridine Phosphate', 'description': 'The dose of amifampridine was based on optimal neuromuscular benefit determined from the Run-in Period from SMA-001 or could be modified as the discretion of the Investigator. The maximum single dose was 20 mg. The dose range for patients 6 to 16 years of age was 15 to 60 mg, and for those 17 years and older the range was from 30 to 80 mg daily.\n\nAmifampridine Phosphate 10 MG Oral Tablet: Oral tablets'}], 'classes': [{'title': 'screening', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '33.737', 'spread': '17.677', 'groupId': 'OG000'}]}]}, {'title': 'End of Study', 'denoms': [{'units': 'Participants', 'counts': [{'value': '09', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '39.012', 'spread': '14.337', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Screening to end of study.', 'description': 'Quality of life (QoL): the Individualized Quality of Life for neuromuscular disease (INQoL) or the Pediatric Quality of Life (PEDSQLTM) was used for adult or pediatric patients, respectively. Since there were no pediatric patients, none of the patients completed the PEDSQL.\n\nThe INQol evaluated weakness, pain, fatigue, double vision, muscle locking, droopy eyelids, swallowing difficulties, activities, social relationships, emotions and body image. Each of these areas were measured in four categories as follows: 1- Incidence (0= No, 1 =Yes), 2-Severity (0= None to 7 = extreme), 3- Impact - (0= None to 6 = extreme) and 4-Importance (0= None to 6 = extreme). The numbers were summative and are input to the QOL calculation, which is a percentage of severity on a scale of 0-100. The mean value was taken across this population. The higher scores were a worse outcome.', 'unitOfMeasure': 'score on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': '11 patients completed the screening Quality of Life Score and only 9 patients completed the EOS Quality of Life Score .'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Amifampridine Phosphate', 'description': 'The dose of amifampridine was based on optimal neuromuscular benefit determined from the Run-in Period from SMA-001 or could be modified as the discretion of the Investigator. The maximum single dose was 20 mg. The dose range for patients 6 to 16 years of age was 15 to 60 mg, and for those 17 years and older the range was from 30 to 80 mg daily.\n\nAmifampridine Phosphate 10 MG Oral Tablet: Oral tablets'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '13'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '13'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}]}]}]}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'Amifampridine Phosphate', 'description': 'The dose of amifampridine was based on optimal neuromuscular benefit determined from the Run-in Period from SMA-001 or could be modified as the discretion of the Investigator. The maximum single dose was 20 mg. The dose range for patients 6 to 16 years of age was 15 to 60 mg, and for those 17 years and older the range was from 30 to 80 mg daily.\n\nAmifampridine Phosphate 10 MG Oral Tablet: Oral tablets'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '13', 'groupId': 'BG000'}]}, {'title': '>=65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'description': 'Male or female between the ages of 6 and 50 years and who had completed the SMA-001. This is part of the eligibility criteria.', 'unitOfMeasure': 'Participants'}, {'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '34.8', 'spread': '11.3', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '5', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '8', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race (NIH/OMB)', 'classes': [{'categories': [{'title': 'American Indian or Alaska Native', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Asian', 'measurements': [{'value': '1', 'groupId': 'BG000'}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Black or African American', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'White', 'measurements': [{'value': '12', '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': 'Italy', 'categories': [{'measurements': [{'value': '6', 'groupId': 'BG000'}]}]}, {'title': 'Serbia', 'categories': [{'measurements': [{'value': '7', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Height', 'classes': [{'categories': [{'measurements': [{'value': '171.9', 'groupId': 'BG000', 'lowerLimit': '163', 'upperLimit': '187'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'CM', 'dispersionType': 'FULL_RANGE'}, {'title': 'Weight', 'classes': [{'categories': [{'measurements': [{'value': '67.1', 'groupId': 'BG000', 'lowerLimit': '53', 'upperLimit': '86'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'Kg', 'dispersionType': 'FULL_RANGE'}, {'title': 'BMI', 'classes': [{'categories': [{'measurements': [{'value': '22.7', 'groupId': 'BG000', 'lowerLimit': '16.7', 'upperLimit': '28.4'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'kg/m^2', 'dispersionType': 'FULL_RANGE'}]}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2018-07-24', 'size': 26344701, 'label': 'Study Protocol: original protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_000.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2023-08-31T16:03', 'hasProtocol': True}, {'date': '2019-04-12', 'size': 660322, 'label': 'Study Protocol: Amendment 1', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_001.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2023-08-31T16:11', 'hasProtocol': True}, {'date': '2020-06-29', 'size': 1024490, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_002.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2023-09-30T10:16', 'hasProtocol': False}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP', 'interventionModelDescription': 'Open-label, extension study , long term safety and tolerability'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 13}}, 'statusModule': {'whyStopped': 'Development of indication not being pursued', 'overallStatus': 'TERMINATED', 'startDateStruct': {'date': '2019-03-07', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2023-11', 'completionDateStruct': {'date': '2021-09-13', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2023-11-29', 'studyFirstSubmitDate': '2019-01-23', 'resultsFirstSubmitDate': '2023-09-01', 'studyFirstSubmitQcDate': '2019-01-24', 'lastUpdatePostDateStruct': {'date': '2023-11-30', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2023-11-29', 'studyFirstPostDateStruct': {'date': '2019-01-28', 'type': 'ACTUAL'}, 'resultsFirstPostDateStruct': {'date': '2023-11-30', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2021-09-13', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Long-term Safety and Tolerability of Amifampridine', 'timeFrame': '18 months', 'description': 'Number of subjects with treatment emergent adverse events (TEAE).'}], 'secondaryOutcomes': [{'measure': 'To Assess the Clinical Efficacy of Amifampridine Phosphate Over Time in Patients With SMA Type 3 Based on Changes in Quality of Life (QoL).', 'timeFrame': 'Screening to end of study.', 'description': 'Quality of life (QoL): the Individualized Quality of Life for neuromuscular disease (INQoL) or the Pediatric Quality of Life (PEDSQLTM) was used for adult or pediatric patients, respectively. Since there were no pediatric patients, none of the patients completed the PEDSQL.\n\nThe INQol evaluated weakness, pain, fatigue, double vision, muscle locking, droopy eyelids, swallowing difficulties, activities, social relationships, emotions and body image. Each of these areas were measured in four categories as follows: 1- Incidence (0= No, 1 =Yes), 2-Severity (0= None to 7 = extreme), 3- Impact - (0= None to 6 = extreme) and 4-Importance (0= None to 6 = extreme). The numbers were summative and are input to the QOL calculation, which is a percentage of severity on a scale of 0-100. The mean value was taken across this population. The higher scores were a worse outcome.'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Type 3'], 'conditions': ['Spinal Muscular Atrophy Type 3']}, 'descriptionModule': {'briefSummary': 'A long term safety study of amifampridine phosphate in patients with spinal muscular atrophy (SMA) Type 3.', 'detailedDescription': 'This open-label outpatient extension study was designed to evaluate the long-term safety and tolerability of amifampridine in patients diagnosed with SMA Type 3. In addition, the evaluation of the effects of amifampridine on the QoL was performed. The study was planned to enroll those patients who had completed the SMA-001 study and after all final evaluations for that study had been completed, or those who demonstrated benefit after completing the dose titration period but failed to meet the randomization criteria on Day 0 of SMA-001.\n\nThe duration of participation for each patient was expected to be at least 12 months as patients could continue in the study until amifampridine was approved by Regulatory Agencies or the clinical development of amifampridine was terminated for this indication. In addition to amifampridine, patients continued to receive previous concomitant medications, as needed.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '50 Years', 'minimumAge': '6 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\nIndividuals eligible to participate in this study must meet all the following inclusion criteria:\n\n1. Participated in the SMA-001 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 SMA-001 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\nExclusion Criteria:\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. Uncontrolled asthma.\n3. Concomitant use with sultopride.\n4. Concomitant use with medicinal products with a narrow therapeutic window.\n5. Concomitant use with medicinal products with a known to cause QTc prolongation.\n6. Clinically significant abnormalities in 12 lead ECG, in the opinion of the Investigator.\n7. Subjects with congenital QT syndromes.\n8. Breastfeeding or pregnant at Screening or planning to become pregnant at any time during the study.\n9. Intolerable amifampridine-related side effects\n10. Treatment with an investigational drug (other than amifampridine) or device while participating in this study.\n11. 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.\n12. History of drug allergy to any pyridine-containing substances or any amifampridine excipient(s)."}, 'identificationModule': {'nctId': 'NCT03819660', 'acronym': 'SMA3', 'briefTitle': 'Long Term Safety of Amifampridine Phosphate in Spinal Muscular Atrophy 3', 'organization': {'class': 'INDUSTRY', 'fullName': 'Catalyst Pharmaceuticals, Inc.'}, 'officialTitle': 'Long Term Safety Study of Amifampridine Phosphate in Ambulatory Patients With Spinal Muscular Atrophy (SMA) Type 3', 'orgStudyIdInfo': {'id': 'SMA-002'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'amifampridine phosphate', 'description': 'The dose of amifampridine was based on optimal neuromuscular benefit determined from the Run-in Period from SMA-001 or could be modified as the discretion of the Investigator. The maximum single dose was 20 mg. The dose range for patients 6 to 16 years of age was 15 to 60 mg, and for those 17 years and older the range was from 30 to 80 mg daily.', 'interventionNames': ['Drug: Amifampridine Phosphate 10 MG Oral Tablet']}], 'interventions': [{'name': 'Amifampridine Phosphate 10 MG Oral Tablet', 'type': 'DRUG', 'description': 'Oral tablets', 'armGroupLabels': ['amifampridine phosphate']}]}, 'contactsLocationsModule': {'locations': [{'zip': '20133', 'city': 'Milan', 'state': 'Lombardy', 'country': 'Italy', 'facility': 'Neurological Institute Carlo Besta', 'geoPoint': {'lat': 45.46427, 'lon': 9.18951}}], 'overallOfficials': [{'name': 'Lorenzo Maggi, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Carlo Besta Institute, Milan, Italy'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Catalyst Pharmaceuticals, Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}