Ignite Creation Date:
2025-12-24 @ 7:56 PM
Ignite Modification Date:
2025-12-25 @ 5:31 PM
Study NCT ID:
NCT00442104
Status:
TERMINATED
Last Update Posted:
2024-05-28
First Post:
2007-02-27
Is NOT Gene Therapy:
False
Has Adverse Events:
True
Brief Title:
Open-label Extension to Protocol 1042-0500
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D013036', 'term': 'Spasms, Infantile'}], 'ancestors': [{'id': 'D004829', 'term': 'Epilepsy, Generalized'}, {'id': 'D004827', 'term': 'Epilepsy'}, {'id': 'D001927', 'term': 'Brain Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D000073376', 'term': 'Epileptic Syndromes'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C105051', 'term': 'ganaxolone'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'clinicaltrials@marinuspharma.com', 'phone': '484-801-4670', 'title': 'Marinus', 'organization': 'Marinus Pharmaceuticals, Inc.'}, 'certainAgreement': {'piSponsorEmployee': False, 'restrictiveAgreement': False}, 'limitationsAndCaveats': {'description': 'Seizure-free information is not available'}}, 'adverseEventsModule': {'timeFrame': 'Up to Week 32', 'description': 'AEs and SAEs were collected in ITT Population. No separate analysis was performed to report results by doses.', 'eventGroups': [{'id': 'EG000', 'title': 'Ganaxolone', 'description': 'Participants were administered a maximum dose of 54 mg/kg/day ganaxolone oral suspension. The investigator could adjust (increase or decrease) the dose of ganaxolone by 3 mg/kg t.i.d. (total daily dose adjustment = 9 mg/kg) until the optimal dose for efficacy and tolerability was achieved.', 'otherNumAtRisk': 54, 'deathsNumAtRisk': 54, 'otherNumAffected': 47, 'seriousNumAtRisk': 54, 'deathsNumAffected': 2, 'seriousNumAffected': 24}], 'otherEvents': [{'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 54, 'numAffected': 26}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (9.0)'}, {'term': 'Cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 54, 'numAffected': 19}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (9.0)'}, {'term': 'Upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 54, 'numAffected': 18}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (9.0)'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 54, 'numAffected': 15}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (9.0)'}, {'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 54, 'numAffected': 13}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (9.0)'}, {'term': 'Nasal congestion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 54, 'numAffected': 11}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (9.0)'}, {'term': 'Convulsion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 54, 'numAffected': 9}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (9.0)'}, {'term': 'Urinary tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 54, 'numAffected': 8}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (9.0)'}, {'term': 'Diarrhea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 54, 'numAffected': 8}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (9.0)'}, {'term': 'Constipation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 54, 'numAffected': 7}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (9.0)'}, {'term': 'Teething', 'stats': [{'groupId': 'EG000', 'numAtRisk': 54, 'numAffected': 7}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (9.0)'}, {'term': 'Somnolence', 'stats': [{'groupId': 'EG000', 'numAtRisk': 54, 'numAffected': 7}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (9.0)'}, {'term': 'Gastroesophageal reflux disease', 'stats': [{'groupId': 'EG000', 'numAtRisk': 54, 'numAffected': 6}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (9.0)'}, {'term': 'Irritability', 'stats': [{'groupId': 'EG000', 'numAtRisk': 54, 'numAffected': 6}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (9.0)'}, {'term': 'Infantile spasms', 'stats': [{'groupId': 'EG000', 'numAtRisk': 54, 'numAffected': 6}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (9.0)'}, {'term': 'Ear infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 54, 'numAffected': 5}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (9.0)'}, {'term': 'Influenza', 'stats': [{'groupId': 'EG000', 'numAtRisk': 54, 'numAffected': 5}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (9.0)'}, {'term': 'Rhinitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 54, 'numAffected': 5}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (9.0)'}, {'term': 'Dehydration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 54, 'numAffected': 5}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (9.0)'}, {'term': 'Tonic convulsion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 54, 'numAffected': 4}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (9.0)'}, {'term': 'Oral intake reduced', 'stats': [{'groupId': 'EG000', 'numAtRisk': 54, 'numAffected': 4}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (9.0)'}, {'term': 'Insomnia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 54, 'numAffected': 4}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (9.0)'}, {'term': 'Bronchitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 54, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (9.0)'}, {'term': 'Gastroenteritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 54, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (9.0)'}, {'term': 'Otitis media', 'stats': [{'groupId': 'EG000', 'numAtRisk': 54, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (9.0)'}, {'term': 'Pneumonia aspiration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 54, 'numAffected': 3}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (9.0)'}, {'term': 'Lethargy', 'stats': [{'groupId': 'EG000', 'numAtRisk': 54, 'numAffected': 3}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (9.0)'}, {'term': 'Metabolic acidosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 54, 'numAffected': 3}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (9.0)'}, {'term': 'Hemoglobin decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 54, 'numAffected': 3}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (9.0)'}, {'term': 'Anemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 54, 'numAffected': 3}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (9.0)'}], 'seriousEvents': [{'term': 'Infantile spasms', 'stats': [{'groupId': 'EG000', 'numAtRisk': 54, 'numAffected': 5}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (9.0)'}, {'term': 'Convulsion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 54, 'numAffected': 4}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (9.0)'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 54, 'numAffected': 4}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (9.0)'}, {'term': 'Pneumonia aspiration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 54, 'numAffected': 3}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (9.0)'}, {'term': 'Gastroesophageal reflux disease', 'stats': [{'groupId': 'EG000', 'numAtRisk': 54, 'numAffected': 3}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (9.0)'}, {'term': 'Epilepsy', 'stats': [{'groupId': 'EG000', 'numAtRisk': 54, 'numAffected': 2}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (9.0)'}, {'term': 'Gastroenteritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 54, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (9.0)'}, {'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 54, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (9.0)'}, {'term': 'Upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 54, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (9.0)'}, {'term': 'Cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 54, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (9.0)'}, {'term': 'Respiratory distress', 'stats': [{'groupId': 'EG000', 'numAtRisk': 54, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (9.0)'}, {'term': 'Failure to thrive', 'stats': [{'groupId': 'EG000', 'numAtRisk': 54, 'numAffected': 2}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (9.0)'}, {'term': 'Oral intake reduced', 'stats': [{'groupId': 'EG000', 'numAtRisk': 54, 'numAffected': 2}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (9.0)'}, {'term': 'Dehydration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 54, 'numAffected': 3}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (9.0)'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Percentage of Participants Who Were Free of Spasms', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ganaxolone', 'description': 'Participants were administered a maximum dose of 54 mg/kg/day ganaxolone oral suspension. The investigator could adjust (increase or decrease) the dose of ganaxolone by 3 mg/kg t.i.d. (total daily dose adjustment = 9 mg/kg) until the optimal dose for efficacy and tolerability was achieved.'}], 'classes': [{'categories': [{'measurements': [{'value': '100', 'groupId': 'OG000', 'lowerLimit': '59.0', 'upperLimit': '100.0'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Weeks 4 through Week 96', 'description': 'Clinical spasms were determined by video-electroencephalography (VEEG). The number of participants with spasm-free duration have been presented.', 'unitOfMeasure': 'percentage of participants', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'ITT Population included all participants who received at least 1 dose of open-label study medication. Only those participants with data available at the indicated time point were analyzed.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Frequency of Spasm Clusters', 'denoms': [{'units': 'Participants', 'counts': [{'value': '40', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ganaxolone', 'description': 'Participants were administered a maximum dose of 54 mg/kg/day ganaxolone oral suspension. The investigator could adjust (increase or decrease) the dose of ganaxolone by 3 mg/kg t.i.d. (total daily dose adjustment = 9 mg/kg) until the optimal dose for efficacy and tolerability was achieved.'}], 'classes': [{'title': 'Spasm Clusters - Week 4', 'denoms': [{'units': 'Participants', 'counts': [{'value': '40', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-2.9', 'spread': '7.94', 'groupId': 'OG000'}]}]}, {'title': 'Spasm Clusters - Week 8', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-4.1', 'spread': '8.35', 'groupId': 'OG000'}]}]}, {'title': 'Spasm Clusters - Week 14', 'denoms': [{'units': 'Participants', 'counts': [{'value': '31', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-4.1', 'spread': '9.12', 'groupId': 'OG000'}]}]}, {'title': 'Spasm Clusters - Week 20', 'denoms': [{'units': 'Participants', 'counts': [{'value': '30', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-5.8', 'spread': '8.58', 'groupId': 'OG000'}]}]}, {'title': 'Spasm Clusters - Week 26', 'denoms': [{'units': 'Participants', 'counts': [{'value': '27', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-5.6', 'spread': '7.98', 'groupId': 'OG000'}]}]}, {'title': 'Spasm Clusters - Week 32', 'denoms': [{'units': 'Participants', 'counts': [{'value': '23', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-5.3', 'spread': '9.96', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline and Week 4 through Week 32', 'description': 'Infantile spasms that come one after another in a cluster and lasts several minutes are called Spasm Clusters. Spasm clusters were determined by a 24-hour video-electroencephalography (vEEG). Change from Baseline was calculated as the post-Baseline value minus the Baseline value. Baseline was defined as the Day 0 assessment prior to ganaxolone dosing in Protocol 1042-0500.', 'unitOfMeasure': 'Spasm clusters per day', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'ITT Population. Only those participants with data available at the indicated time point were analyzed.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Frequency of Individual Spasm', 'denoms': [{'units': 'Participants', 'counts': [{'value': '39', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ganaxolone', 'description': 'Participants were administered a maximum dose of 54 mg/kg/day ganaxolone oral suspension. The investigator could adjust (increase or decrease) the dose of ganaxolone by 3 mg/kg t.i.d. (total daily dose adjustment = 9 mg/kg) until the optimal dose for efficacy and tolerability was achieved.'}], 'classes': [{'title': 'Individual Spasms - Week 4', 'denoms': [{'units': 'Participants', 'counts': [{'value': '39', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-6.8', 'spread': '40.70', 'groupId': 'OG000'}]}]}, {'title': 'Individual Spasms - Week 8', 'denoms': [{'units': 'Participants', 'counts': [{'value': '31', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-17.4', 'spread': '31.28', 'groupId': 'OG000'}]}]}, {'title': 'Individual Spasms - Week 14', 'denoms': [{'units': 'Participants', 'counts': [{'value': '30', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-20.8', 'spread': '31.21', 'groupId': 'OG000'}]}]}, {'title': 'Individual Spasms - Week 20', 'denoms': [{'units': 'Participants', 'counts': [{'value': '29', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-15.8', 'spread': '52.46', 'groupId': 'OG000'}]}]}, {'title': 'Individual Spasms - Week 26', 'denoms': [{'units': 'Participants', 'counts': [{'value': '26', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-19.9', 'spread': '39.27', 'groupId': 'OG000'}]}]}, {'title': 'Individual Spasms - Week 32', 'denoms': [{'units': 'Participants', 'counts': [{'value': '21', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-17.7', 'spread': '36.35', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline and Week 4 through Week 32', 'description': 'Individual Spasm are seizures that may last only a second or two and were determined by a 24-hour video-electroencephalography (vEEG). Change from Baseline was calculated as the post-Baseline value minus the Baseline value. Baseline was defined as the Day 0 assessment prior to ganaxolone dosing in Protocol 1042-0500.', 'unitOfMeasure': 'Individual spasms per day', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'ITT Population. Only those participants with data available at the indicated time point were analyzed.'}, {'type': 'SECONDARY', 'title': "Number of Participants With Change in Clinical Status on Caregiver's Global Assessment", 'denoms': [{'units': 'Participants', 'counts': [{'value': '45', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ganaxolone', 'description': 'Participants were administered a maximum dose of 54 mg/kg/day ganaxolone oral suspension. The investigator could adjust (increase or decrease) the dose of ganaxolone by 3 mg/kg t.i.d. (total daily dose adjustment = 9 mg/kg) until the optimal dose for efficacy and tolerability was achieved.'}], 'classes': [{'title': "Caregiver's- Week 4 - Marked Improvement", 'denoms': [{'units': 'Participants', 'counts': [{'value': '45', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '11', 'groupId': 'OG000'}]}]}, {'title': "Caregiver's - Week 4 - Moderate Improvement", 'denoms': [{'units': 'Participants', 'counts': [{'value': '45', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '11', 'groupId': 'OG000'}]}]}, {'title': "Caregiver's -Week 4 - Slight Improvement", 'denoms': [{'units': 'Participants', 'counts': [{'value': '45', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '16', 'groupId': 'OG000'}]}]}, {'title': "Caregiver's- Week 4 - No change from Baseline", 'denoms': [{'units': 'Participants', 'counts': [{'value': '45', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '6', 'groupId': 'OG000'}]}]}, {'title': "Caregiver's - Week 4 - Moderate Worsening", 'denoms': [{'units': 'Participants', 'counts': [{'value': '45', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': "Caregiver's - Week 4 - Slight Worsening", 'denoms': [{'units': 'Participants', 'counts': [{'value': '45', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': "Caregiver's - Week 4 - Marked Worsening", 'denoms': [{'units': 'Participants', 'counts': [{'value': '45', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': "Caregiver's - Week 8 - Marked Improvement", 'denoms': [{'units': 'Participants', 'counts': [{'value': '40', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '7', 'groupId': 'OG000'}]}]}, {'title': "Caregiver's- Week 8 - Moderate Improvement", 'denoms': [{'units': 'Participants', 'counts': [{'value': '40', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '12', 'groupId': 'OG000'}]}]}, {'title': "Caregiver's -Week 8 - Slight Improvement", 'denoms': [{'units': 'Participants', 'counts': [{'value': '40', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '18', 'groupId': 'OG000'}]}]}, {'title': "Caregiver's- Week 8 - No change from Baseline", 'denoms': [{'units': 'Participants', 'counts': [{'value': '40', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': "Caregiver's - Week 8 - Slight Worsening", 'denoms': [{'units': 'Participants', 'counts': [{'value': '40', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}, {'title': "Caregiver's - Week 8 - Moderate Worsening", 'denoms': [{'units': 'Participants', 'counts': [{'value': '40', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': "Caregiver's - Week 8 - Marked Worsening", 'denoms': [{'units': 'Participants', 'counts': [{'value': '40', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': "Caregiver's- Week 14 - Marked Improvement", 'denoms': [{'units': 'Participants', 'counts': [{'value': '38', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '8', 'groupId': 'OG000'}]}]}, {'title': "Caregiver's - Week 14 - Moderate Improvement", 'denoms': [{'units': 'Participants', 'counts': [{'value': '38', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '13', 'groupId': 'OG000'}]}]}, {'title': "Caregiver's -Week 14 - Slight Improvement", 'denoms': [{'units': 'Participants', 'counts': [{'value': '38', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '14', 'groupId': 'OG000'}]}]}, {'title': "Caregiver's - Week 14 - No change from Baseline", 'denoms': [{'units': 'Participants', 'counts': [{'value': '38', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}, {'title': "Caregiver's - Week 14 - Slight Worsening", 'denoms': [{'units': 'Participants', 'counts': [{'value': '38', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': "Caregiver's - Week 14 - Moderate Worsening", 'denoms': [{'units': 'Participants', 'counts': [{'value': '38', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': "Caregiver's - Week 14 - Marked Worsening", 'denoms': [{'units': 'Participants', 'counts': [{'value': '38', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': "Caregiver's - Week 20 - Marked Improvement", 'denoms': [{'units': 'Participants', 'counts': [{'value': '36', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '9', 'groupId': 'OG000'}]}]}, {'title': "Caregiver's - Week 20 - Moderate Improvement", 'denoms': [{'units': 'Participants', 'counts': [{'value': '36', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '13', 'groupId': 'OG000'}]}]}, {'title': "Caregiver's -Week 20 - Slight Improvement", 'denoms': [{'units': 'Participants', 'counts': [{'value': '36', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '10', 'groupId': 'OG000'}]}]}, {'title': "Caregiver's- Week 20 - No change from Baseline", 'denoms': [{'units': 'Participants', 'counts': [{'value': '36', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '3', 'groupId': 'OG000'}]}]}, {'title': "Caregiver's- Week 20 - Slight Worsening", 'denoms': [{'units': 'Participants', 'counts': [{'value': '36', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': "Caregiver's - Week 20 - Moderate Worsening", 'denoms': [{'units': 'Participants', 'counts': [{'value': '36', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': "Caregiver's - Week 20 - Marked Worsening", 'denoms': [{'units': 'Participants', 'counts': [{'value': '36', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': "Caregiver's- Week 26 - Marked Improvement", 'denoms': [{'units': 'Participants', 'counts': [{'value': '31', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '9', 'groupId': 'OG000'}]}]}, {'title': "Caregiver's- Week 26 - Moderate Improvement", 'denoms': [{'units': 'Participants', 'counts': [{'value': '31', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '12', 'groupId': 'OG000'}]}]}, {'title': "Caregiver's -Week 26 - Slight Improvement", 'denoms': [{'units': 'Participants', 'counts': [{'value': '31', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '7', 'groupId': 'OG000'}]}]}, {'title': "Caregiver's - Week 26 - No change from Baseline", 'denoms': [{'units': 'Participants', 'counts': [{'value': '31', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '3', 'groupId': 'OG000'}]}]}, {'title': "Caregiver's - Week 26 - Slight Worsening", 'denoms': [{'units': 'Participants', 'counts': [{'value': '31', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': "Caregiver's - Week 26 - Moderate Worsening", 'denoms': [{'units': 'Participants', 'counts': [{'value': '31', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': "Caregiver's - Week 26 - Marked Worsening", 'denoms': [{'units': 'Participants', 'counts': [{'value': '31', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': "Caregiver's - Week 32 - Marked Improvement", 'denoms': [{'units': 'Participants', 'counts': [{'value': '26', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '8', 'groupId': 'OG000'}]}]}, {'title': "Caregiver's - Week 32 - Moderate Improvement", 'denoms': [{'units': 'Participants', 'counts': [{'value': '26', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '7', 'groupId': 'OG000'}]}]}, {'title': "Caregiver's-Week 32 - Slight Improvement", 'denoms': [{'units': 'Participants', 'counts': [{'value': '26', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '8', 'groupId': 'OG000'}]}]}, {'title': "Caregiver's - Week 32 - No change from Baseline", 'denoms': [{'units': 'Participants', 'counts': [{'value': '26', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}, {'title': "Caregiver's - Week 32 - Slight Worsening", 'denoms': [{'units': 'Participants', 'counts': [{'value': '26', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': "Caregiver's - Week 32 - Moderate Worsening", 'denoms': [{'units': 'Participants', 'counts': [{'value': '26', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': "Caregiver's - Week 32 - Marked Worsening", 'denoms': [{'units': 'Participants', 'counts': [{'value': '26', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Week 4 through Week 32', 'description': "Caregiver global assessment of seizure severity and response to treatment rated the participants' based on 7 clinical factors: seizure frequency, duration, and intensity; adverse experiences; social, intellectual, and motor functioning. Using a 7-point scale (\\[1\\] marked improvement, \\[2\\] moderate improvement, \\[3\\] slight improvement, \\[4\\] no change from baseline, \\[5\\] slight worsening, \\[6\\] moderate worsening, or \\[7\\] marked worsening). Higher score indicated worse symptoms. The assessment compared the participants' current status to their condition prior to initiating study medication.", 'unitOfMeasure': 'participants', 'reportingStatus': 'POSTED', 'populationDescription': 'ITT population. Only those participants with data available at the indicated time point were analyzed.'}, {'type': 'SECONDARY', 'title': "Number of Participants With Change in Clinical Status on the Investigator's Global Assessment", 'denoms': [{'units': 'Participants', 'counts': [{'value': '45', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ganaxolone', 'description': 'Participants were administered a maximum dose of 54 mg/kg/day ganaxolone oral suspension. The investigator could adjust (increase or decrease) the dose of ganaxolone by 3 mg/kg t.i.d. (total daily dose adjustment = 9 mg/kg) until the optimal dose for efficacy and tolerability was achieved.'}], 'classes': [{'title': "Investigator's- Week 4 - Marked Improvement", 'denoms': [{'units': 'Participants', 'counts': [{'value': '45', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '7', 'groupId': 'OG000'}]}]}, {'title': "Investigator's - Week 4 - Moderate Improvement", 'denoms': [{'units': 'Participants', 'counts': [{'value': '45', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '15', 'groupId': 'OG000'}]}]}, {'title': "Investigator's -Week 4 - Slight Improvement", 'denoms': [{'units': 'Participants', 'counts': [{'value': '45', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '18', 'groupId': 'OG000'}]}]}, {'title': "Investigator's- Week 4 - No change from Baseline", 'denoms': [{'units': 'Participants', 'counts': [{'value': '45', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '4', 'groupId': 'OG000'}]}]}, {'title': "Investigator's - Week 4 - Moderate Worsening", 'denoms': [{'units': 'Participants', 'counts': [{'value': '45', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': "Investigator's - Week 4 - Slight Worsening", 'denoms': [{'units': 'Participants', 'counts': [{'value': '45', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': "Investigator's - Week 4 - Marked Worsening", 'denoms': [{'units': 'Participants', 'counts': [{'value': '45', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': "Investigator's - Week 8 - Marked Improvement", 'denoms': [{'units': 'Participants', 'counts': [{'value': '40', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '6', 'groupId': 'OG000'}]}]}, {'title': "Investigator's- Week 8 - Moderate Improvement", 'denoms': [{'units': 'Participants', 'counts': [{'value': '40', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '12', 'groupId': 'OG000'}]}]}, {'title': "Investigator's -Week 8 - Slight Improvement", 'denoms': [{'units': 'Participants', 'counts': [{'value': '40', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '19', 'groupId': 'OG000'}]}]}, {'title': "Investigator's- Week 8 - No change from Baseline", 'denoms': [{'units': 'Participants', 'counts': [{'value': '40', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '3', 'groupId': 'OG000'}]}]}, {'title': "Investigator's - Week 8 - Slight Worsening", 'denoms': [{'units': 'Participants', 'counts': [{'value': '40', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': "Investigator's - Week 8 - Moderate Worsening", 'denoms': [{'units': 'Participants', 'counts': [{'value': '40', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': "Investigator's - Week 8 - Marked Worsening", 'denoms': [{'units': 'Participants', 'counts': [{'value': '40', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': "Investigator's- Week 14 - Marked Improvement", 'denoms': [{'units': 'Participants', 'counts': [{'value': '38', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '6', 'groupId': 'OG000'}]}]}, {'title': "Investigator's - Week 14 - Moderate Improvement", 'denoms': [{'units': 'Participants', 'counts': [{'value': '38', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '16', 'groupId': 'OG000'}]}]}, {'title': "Investigator's -Week 14 - Slight Improvement", 'denoms': [{'units': 'Participants', 'counts': [{'value': '38', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '15', 'groupId': 'OG000'}]}]}, {'title': "Investigator's - Week 14 - No change from Baseline", 'denoms': [{'units': 'Participants', 'counts': [{'value': '38', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': "Investigator's - Week 14 - Slight Worsening", 'denoms': [{'units': 'Participants', 'counts': [{'value': '38', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': "Investigator's - Week 14 - Moderate Worsening", 'denoms': [{'units': 'Participants', 'counts': [{'value': '38', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': "Investigator's - Week 14 - Marked Worsening", 'denoms': [{'units': 'Participants', 'counts': [{'value': '38', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': "Investigator's - Week 20 - Marked Improvement", 'denoms': [{'units': 'Participants', 'counts': [{'value': '34', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '7', 'groupId': 'OG000'}]}]}, {'title': "Investigator's - Week 20 - Moderate Improvement", 'denoms': [{'units': 'Participants', 'counts': [{'value': '34', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '14', 'groupId': 'OG000'}]}]}, {'title': "Investigator's -Week 20 - Slight Improvement", 'denoms': [{'units': 'Participants', 'counts': [{'value': '34', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '11', 'groupId': 'OG000'}]}]}, {'title': "Investigator's- Week 20 - No change from Baseline", 'denoms': [{'units': 'Participants', 'counts': [{'value': '34', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': "Investigator's- Week 20 - Slight Worsening", 'denoms': [{'units': 'Participants', 'counts': [{'value': '34', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': "Investigator's - Week 20 - Moderate Worsening", 'denoms': [{'units': 'Participants', 'counts': [{'value': '34', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': "Investigator's - Week 20 - Marked Worsening", 'denoms': [{'units': 'Participants', 'counts': [{'value': '34', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': "Investigator's- Week 26 - Marked Improvement", 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '5', 'groupId': 'OG000'}]}]}, {'title': "Investigator's- Week 26 - Moderate Improvement", 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '14', 'groupId': 'OG000'}]}]}, {'title': "Investigator's -Week 26 - Slight Improvement", 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '9', 'groupId': 'OG000'}]}]}, {'title': "Investigator's - Week 26 - No change from Baseline", 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '4', 'groupId': 'OG000'}]}]}, {'title': "Investigator's - Week 26 - Slight Worsening", 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': "Investigator's - Week 26 - Moderate Worsening", 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': "Investigator's - Week 26 - Marked Worsening", 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': "Investigator's - Week 32 - Marked Improvement", 'denoms': [{'units': 'Participants', 'counts': [{'value': '27', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '6', 'groupId': 'OG000'}]}]}, {'title': "Investigator's - Week 32 - Moderate Improvement", 'denoms': [{'units': 'Participants', 'counts': [{'value': '27', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '9', 'groupId': 'OG000'}]}]}, {'title': "Investigator's -Week 32 - Slight Improvement", 'denoms': [{'units': 'Participants', 'counts': [{'value': '27', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '8', 'groupId': 'OG000'}]}]}, {'title': "Investigator's - Week 32 - No change from Baseline", 'denoms': [{'units': 'Participants', 'counts': [{'value': '27', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '3', 'groupId': 'OG000'}]}]}, {'title': "Investigator's - Week 32 - Slight Worsening", 'denoms': [{'units': 'Participants', 'counts': [{'value': '27', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': "Investigator's - Week 32 - Moderate Worsening", 'denoms': [{'units': 'Participants', 'counts': [{'value': '27', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': "Investigator's - Week 32 - Marked Worsening", 'denoms': [{'units': 'Participants', 'counts': [{'value': '27', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Week 4 through Week 32', 'description': "The investigators rated the participants' overall clinical status based on 7 clinical factors: seizure frequency, duration, and intensity; adverse experiences; social, intellectual, and motor functioning. Using a 7-point scale (\\[1\\] marked improvement, \\[2\\] moderate improvement, \\[3\\] slight improvement, \\[4\\] no change from baseline, \\[5\\] slight worsening, \\[6\\] moderate worsening, or \\[7\\] marked worsening). Higher score indicated worse symptoms. The investigators assessed the participants' status compared to their condition prior to initiating study medication.", 'unitOfMeasure': 'participants', 'reportingStatus': 'POSTED', 'populationDescription': 'ITT population. Only those participants with data available at the indicated time point were analyzed.'}, {'type': 'SECONDARY', 'title': 'Number of Participants With Spasm-free Durations', 'denoms': [{'units': 'Participants', 'counts': [{'value': '43', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ganaxolone', 'description': 'Participants were administered a maximum dose of 54 mg/kg/day ganaxolone oral suspension. The investigator could adjust (increase or decrease) the dose of ganaxolone by 3 mg/kg t.i.d. (total daily dose adjustment = 9 mg/kg) until the optimal dose for efficacy and tolerability was achieved.'}], 'classes': [{'title': 'Spasm Free at Week 4', 'denoms': [{'units': 'Participants', 'counts': [{'value': '43', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '19', 'groupId': 'OG000'}]}]}, {'title': 'Spasm Free at Week 8', 'denoms': [{'units': 'Participants', 'counts': [{'value': '37', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '18', 'groupId': 'OG000'}]}]}, {'title': 'Spasm Free at Week 14', 'denoms': [{'units': 'Participants', 'counts': [{'value': '36', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '18', 'groupId': 'OG000'}]}]}, {'title': 'Spasm Free at Week 20', 'denoms': [{'units': 'Participants', 'counts': [{'value': '34', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '18', 'groupId': 'OG000'}]}]}, {'title': 'Spasm Free at Week 26', 'denoms': [{'units': 'Participants', 'counts': [{'value': '30', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '17', 'groupId': 'OG000'}]}]}, {'title': 'Spasm Free at Week 32', 'denoms': [{'units': 'Participants', 'counts': [{'value': '26', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '13', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Week 4 through Week 32', 'description': 'Spasm-free duration is defined as total number of spasm-free days recorded in the spasm/seizure diary. Parents/Guardians or nursing staff maintained a spasm/seizure diary to record the number and type of seizures each day, including spasms, throughout the entire study. The number of participants with spasm-free duration of at least 24 hours have been presented.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'ITT Population. Only those participants with data available at the indicated time point were analyzed.'}, {'type': 'SECONDARY', 'title': 'Number of Participants With Absence of Spasms', 'denoms': [{'units': 'Participants', 'counts': [{'value': '43', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ganaxolone', 'description': 'Participants were administered a maximum dose of 54 mg/kg/day ganaxolone oral suspension. The investigator could adjust (increase or decrease) the dose of ganaxolone by 3 mg/kg t.i.d. (total daily dose adjustment = 9 mg/kg) until the optimal dose for efficacy and tolerability was achieved.'}], 'classes': [{'title': 'Achieved absence of spasms at Week 4', 'denoms': [{'units': 'Participants', 'counts': [{'value': '43', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}, {'title': 'Achieved absence of spasms at Week 8', 'denoms': [{'units': 'Participants', 'counts': [{'value': '37', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '3', 'groupId': 'OG000'}]}]}, {'title': 'Achieved absence of spasms at Week 14', 'denoms': [{'units': 'Participants', 'counts': [{'value': '36', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '3', 'groupId': 'OG000'}]}]}, {'title': 'Achieved absence of spasms at Week 20', 'denoms': [{'units': 'Participants', 'counts': [{'value': '34', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '4', 'groupId': 'OG000'}]}]}, {'title': 'Achieved absence of spasms at Week 26', 'denoms': [{'units': 'Participants', 'counts': [{'value': '30', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '5', 'groupId': 'OG000'}]}]}, {'title': 'Achieved absence of spasms at Week 32', 'denoms': [{'units': 'Participants', 'counts': [{'value': '26', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Week 4 through Week 32', 'description': 'Absence of spasms is defined as percentage of total spasm-free days during a visit period=100%. Parents/Guardians or nursing staff maintained a spasm/seizure diary to record the number and type of seizures each day, including spasms, throughout the entire study. The participants achieving absence of spasms have been presented.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'ITT Population. Only those participants with data available at the indicated time point were analyzed.'}, {'type': 'SECONDARY', 'title': 'Developmental Assessment Using Denver-II Developmental Test', 'denoms': [{'units': 'Participants', 'counts': [{'value': '34', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ganaxolone', 'description': 'Participants were administered a maximum dose of 54 mg/kg/day ganaxolone oral suspension. The investigator could adjust (increase or decrease) the dose of ganaxolone by 3 mg/kg t.i.d. (total daily dose adjustment = 9 mg/kg) until the optimal dose for efficacy and tolerability was achieved.'}], 'classes': [{'title': 'Week 8 - Personal Social', 'denoms': [{'units': 'Participants', 'counts': [{'value': '34', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.72', 'spread': '3.899', 'groupId': 'OG000'}]}]}, {'title': 'Week 8 - Fine Motor-Adaptive', 'denoms': [{'units': 'Participants', 'counts': [{'value': '34', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-1.56', 'spread': '3.131', 'groupId': 'OG000'}]}]}, {'title': 'Week 8 - Language', 'denoms': [{'units': 'Participants', 'counts': [{'value': '34', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-1.45', 'spread': '2.798', 'groupId': 'OG000'}]}]}, {'title': 'Week 8 - Gross Motor', 'denoms': [{'units': 'Participants', 'counts': [{'value': '34', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-1.05', 'spread': '2.285', 'groupId': 'OG000'}]}]}, {'title': 'Week 20- Personal Social', 'denoms': [{'units': 'Participants', 'counts': [{'value': '34', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-2.82', 'spread': '6.081', 'groupId': 'OG000'}]}]}, {'title': 'Week 20- Fine Motor-Adaptive', 'denoms': [{'units': 'Participants', 'counts': [{'value': '34', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-3.73', 'spread': '5.123', 'groupId': 'OG000'}]}]}, {'title': 'Week 20- Language', 'denoms': [{'units': 'Participants', 'counts': [{'value': '34', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-2.39', 'spread': '5.615', 'groupId': 'OG000'}]}]}, {'title': 'Week 20 - Gross Motor', 'denoms': [{'units': 'Participants', 'counts': [{'value': '34', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-2.69', 'spread': '3.584', 'groupId': 'OG000'}]}]}, {'title': 'Week 32-Personal Social', 'denoms': [{'units': 'Participants', 'counts': [{'value': '26', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-4.86', 'spread': '5.659', 'groupId': 'OG000'}]}]}, {'title': 'Week 32-Fine Motor-Adaptive', 'denoms': [{'units': 'Participants', 'counts': [{'value': '26', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-6.80', 'spread': '4.952', 'groupId': 'OG000'}]}]}, {'title': 'Week 32-Language', 'denoms': [{'units': 'Participants', 'counts': [{'value': '26', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-6.58', 'spread': '4.336', 'groupId': 'OG000'}]}]}, {'title': 'Week 32-Gross Motor', 'denoms': [{'units': 'Participants', 'counts': [{'value': '26', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-6.13', 'spread': '4.509', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Week 8 through Week 32', 'description': "Denver-II Developmental Test measures a child's development in several areas:Personal-Social,Fine Motor-Adaptive,Language,and Gross Motor,from birth to 6 years old.It consists of 125 items that are organized into subscales and scored as pass,fail,or refused.To evaluate a child's progress,test compares their performance to a normative sample of children of same age.For each item,age at which 90% of children in normative sample pass it is determined.Derived score for each subscale is sum of item scores and represents difference between child's chronological age and age at which 90% of children in normative sample pass the items in that subscale.A higher derived score on a subscale indicates better performance on items in that subscale relative to other children of same age who have taken the test.Among subscales,Personal-Social subscale ranges from -16 months to 24 months;others range from - 12 months to 24 months.All subscales have a population mean of 0 and a standard deviation of 3.", 'unitOfMeasure': 'Scores on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'ITT Population. Only those participants with data available at the indicated time point were analyzed.'}, {'type': 'SECONDARY', 'title': 'Percentage of Total Spasm-free Days and Cumulative Spasm-free Days as Determined From the Seizure Diary.', 'denoms': [{'units': 'Participants', 'counts': [{'value': '45', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ganaxolone', 'description': 'Participants were administered a maximum dose of 54 mg/kg/day ganaxolone oral suspension. The investigator could adjust (increase or decrease) the dose of ganaxolone by 3 mg/kg t.i.d. (total daily dose adjustment = 9 mg/kg) until the optimal dose for efficacy and tolerability was achieved.'}], 'classes': [{'title': 'Total Spasm-free days during Week 4 visit', 'denoms': [{'units': 'Participants', 'counts': [{'value': '43', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '11.10', 'spread': '23.657', 'groupId': 'OG000'}]}]}, {'title': 'Total Spasm-free days during Week 8 visit', 'denoms': [{'units': 'Participants', 'counts': [{'value': '37', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '18.74', 'spread': '32.654', 'groupId': 'OG000'}]}]}, {'title': 'Total Spasm-free days during Week 14 visit', 'denoms': [{'units': 'Participants', 'counts': [{'value': '36', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '20.74', 'spread': '34.817', 'groupId': 'OG000'}]}]}, {'title': 'Total Spasm-free days during Week 20 visit', 'denoms': [{'units': 'Participants', 'counts': [{'value': '34', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '28.79', 'spread': '40.163', 'groupId': 'OG000'}]}]}, {'title': 'Total Spasm-free days during Week 26 visit', 'denoms': [{'units': 'Participants', 'counts': [{'value': '30', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '28.73', 'spread': '39.711', 'groupId': 'OG000'}]}]}, {'title': 'Total Spasm-free days during week 32 visit', 'denoms': [{'units': 'Participants', 'counts': [{'value': '26', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '27.99', 'spread': '39.259', 'groupId': 'OG000'}]}]}, {'title': 'Cumulative Spasm-free days during Week 4 visit', 'denoms': [{'units': 'Participants', 'counts': [{'value': '45', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '10.86', 'spread': '20.374', 'groupId': 'OG000'}]}]}, {'title': 'Cumulative Spasm-free days during Week 8 visit', 'denoms': [{'units': 'Participants', 'counts': [{'value': '40', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '13.82', 'spread': '23.433', 'groupId': 'OG000'}]}]}, {'title': 'Cumulative Spasm-free days during Week 14 visit', 'denoms': [{'units': 'Participants', 'counts': [{'value': '38', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '15.70', 'spread': '25.769', 'groupId': 'OG000'}]}]}, {'title': 'Cumulative Spasm-free days during Week 20 visit', 'denoms': [{'units': 'Participants', 'counts': [{'value': '36', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '19.97', 'spread': '28.501', 'groupId': 'OG000'}]}]}, {'title': 'Cumulative Spasm-free days during Week 26 visit', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '22.07', 'spread': '31.105', 'groupId': 'OG000'}]}]}, {'title': 'Cumulative Spasm-free days during Week 32 visit', 'denoms': [{'units': 'Participants', 'counts': [{'value': '27', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '22.99', 'spread': '30.140', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Weeks 4 through Week 32', 'description': 'Percentage of total spasm-free days during a visit period is defined as total number of spasm free days as recorded in the Seizure Diary/ total number of days during that period, multiplied by 100.\n\nPercentage of cumulative total spasm-free days during a visit period is defined as sum of total number of spasm-free days during this period as recorded in the Seizure Diary/ total number of days in the treatment period, multiplied by 100.', 'unitOfMeasure': 'Percentage of Days', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'ITT Population. Only those participants with data available at the indicated time point were analyzed.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Ganaxolone', 'description': 'Participants were administered a maximum dose of 54 milligrams per kilogram (mg/kg)/day ganaxolone oral suspension. The investigator could adjust (increase or decrease) the dose of ganaxolone by 3 mg/kg three times a day (total daily dose adjustment = 9 mg/kg) until the optimal dose for efficacy and tolerability was achieved'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '54'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '7'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '47'}]}], 'dropWithdraws': [{'type': 'Adverse Event', 'reasons': [{'groupId': 'FG000', 'numSubjects': '5'}]}, {'type': 'Death', 'reasons': [{'groupId': 'FG000', 'numSubjects': '2'}]}, {'type': 'Lost to Follow-up', 'reasons': [{'groupId': 'FG000', 'numSubjects': '3'}]}, {'type': 'Withdrawal by Subject', 'reasons': [{'groupId': 'FG000', 'numSubjects': '5'}]}, {'type': 'Insufficient Clinical Response', 'reasons': [{'groupId': 'FG000', 'numSubjects': '10'}]}, {'type': 'Other', 'reasons': [{'groupId': 'FG000', 'numSubjects': '21'}]}, {'type': 'Discretion of the Investigator/Sponsor', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}]}]}], 'preAssignmentDetails': 'Participants who completed the previous double-blind controlled trial (Protocol 1042-0500, NCT00441896) were enrolled in this study. No separate analysis was performed to report results by doses.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '54', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'Ganaxolone', 'description': 'Participants were administered a maximum dose of 54 mg/kg/day ganaxolone oral suspension. The investigator could adjust (increase or decrease) the dose of ganaxolone by 3 mg/kg t.i.d. (total daily dose adjustment = 9 mg/kg) until the optimal dose for efficacy and tolerability was achieved.'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '13.4', 'spread': '6.38', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'months', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '32', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '22', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Ethnicity (NIH/OMB)', 'classes': [{'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '7', 'groupId': 'BG000'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '47', 'groupId': 'BG000'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', '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': '6', 'groupId': 'BG000'}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Black or African American', 'measurements': [{'value': '8', 'groupId': 'BG000'}]}, {'title': 'White', 'measurements': [{'value': '33', 'groupId': 'BG000'}]}, {'title': 'More than one race', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '7', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 54}}, 'statusModule': {'whyStopped': "Sponsor's decision", 'overallStatus': 'TERMINATED', 'startDateStruct': {'date': '2007-01', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2024-05', 'dispFirstSubmitDate': '2009-10-28', 'completionDateStruct': {'date': '2009-03', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2024-05-23', 'studyFirstSubmitDate': '2007-02-27', 'dispFirstSubmitQcDate': '2009-10-28', 'resultsFirstSubmitDate': '2022-05-16', 'studyFirstSubmitQcDate': '2007-02-28', 'dispFirstPostDateStruct': {'date': '2009-11-02', 'type': 'ESTIMATED'}, 'lastUpdatePostDateStruct': {'date': '2024-05-28', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2023-05-05', 'studyFirstPostDateStruct': {'date': '2007-03-01', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2023-06-02', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2009-03', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Percentage of Participants Who Were Free of Spasms', 'timeFrame': 'Weeks 4 through Week 96', 'description': 'Clinical spasms were determined by video-electroencephalography (VEEG). The number of participants with spasm-free duration have been presented.'}], 'secondaryOutcomes': [{'measure': 'Change From Baseline in Frequency of Spasm Clusters', 'timeFrame': 'Baseline and Week 4 through Week 32', 'description': 'Infantile spasms that come one after another in a cluster and lasts several minutes are called Spasm Clusters. Spasm clusters were determined by a 24-hour video-electroencephalography (vEEG). Change from Baseline was calculated as the post-Baseline value minus the Baseline value. Baseline was defined as the Day 0 assessment prior to ganaxolone dosing in Protocol 1042-0500.'}, {'measure': 'Change From Baseline in Frequency of Individual Spasm', 'timeFrame': 'Baseline and Week 4 through Week 32', 'description': 'Individual Spasm are seizures that may last only a second or two and were determined by a 24-hour video-electroencephalography (vEEG). Change from Baseline was calculated as the post-Baseline value minus the Baseline value. Baseline was defined as the Day 0 assessment prior to ganaxolone dosing in Protocol 1042-0500.'}, {'measure': "Number of Participants With Change in Clinical Status on Caregiver's Global Assessment", 'timeFrame': 'Week 4 through Week 32', 'description': "Caregiver global assessment of seizure severity and response to treatment rated the participants' based on 7 clinical factors: seizure frequency, duration, and intensity; adverse experiences; social, intellectual, and motor functioning. Using a 7-point scale (\\[1\\] marked improvement, \\[2\\] moderate improvement, \\[3\\] slight improvement, \\[4\\] no change from baseline, \\[5\\] slight worsening, \\[6\\] moderate worsening, or \\[7\\] marked worsening). Higher score indicated worse symptoms. The assessment compared the participants' current status to their condition prior to initiating study medication."}, {'measure': "Number of Participants With Change in Clinical Status on the Investigator's Global Assessment", 'timeFrame': 'Week 4 through Week 32', 'description': "The investigators rated the participants' overall clinical status based on 7 clinical factors: seizure frequency, duration, and intensity; adverse experiences; social, intellectual, and motor functioning. Using a 7-point scale (\\[1\\] marked improvement, \\[2\\] moderate improvement, \\[3\\] slight improvement, \\[4\\] no change from baseline, \\[5\\] slight worsening, \\[6\\] moderate worsening, or \\[7\\] marked worsening). Higher score indicated worse symptoms. The investigators assessed the participants' status compared to their condition prior to initiating study medication."}, {'measure': 'Number of Participants With Spasm-free Durations', 'timeFrame': 'Week 4 through Week 32', 'description': 'Spasm-free duration is defined as total number of spasm-free days recorded in the spasm/seizure diary. Parents/Guardians or nursing staff maintained a spasm/seizure diary to record the number and type of seizures each day, including spasms, throughout the entire study. The number of participants with spasm-free duration of at least 24 hours have been presented.'}, {'measure': 'Number of Participants With Absence of Spasms', 'timeFrame': 'Week 4 through Week 32', 'description': 'Absence of spasms is defined as percentage of total spasm-free days during a visit period=100%. Parents/Guardians or nursing staff maintained a spasm/seizure diary to record the number and type of seizures each day, including spasms, throughout the entire study. The participants achieving absence of spasms have been presented.'}, {'measure': 'Developmental Assessment Using Denver-II Developmental Test', 'timeFrame': 'Week 8 through Week 32', 'description': "Denver-II Developmental Test measures a child's development in several areas:Personal-Social,Fine Motor-Adaptive,Language,and Gross Motor,from birth to 6 years old.It consists of 125 items that are organized into subscales and scored as pass,fail,or refused.To evaluate a child's progress,test compares their performance to a normative sample of children of same age.For each item,age at which 90% of children in normative sample pass it is determined.Derived score for each subscale is sum of item scores and represents difference between child's chronological age and age at which 90% of children in normative sample pass the items in that subscale.A higher derived score on a subscale indicates better performance on items in that subscale relative to other children of same age who have taken the test.Among subscales,Personal-Social subscale ranges from -16 months to 24 months;others range from - 12 months to 24 months.All subscales have a population mean of 0 and a standard deviation of 3."}, {'measure': 'Percentage of Total Spasm-free Days and Cumulative Spasm-free Days as Determined From the Seizure Diary.', 'timeFrame': 'Weeks 4 through Week 32', 'description': 'Percentage of total spasm-free days during a visit period is defined as total number of spasm free days as recorded in the Seizure Diary/ total number of days during that period, multiplied by 100.\n\nPercentage of cumulative total spasm-free days during a visit period is defined as sum of total number of spasm-free days during this period as recorded in the Seizure Diary/ total number of days in the treatment period, multiplied by 100.'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'keywords': ['infantile spasms', 'anticonvulsant', 'pediatric epilepsy', 'West Syndrome', 'epileptic spasms'], 'conditions': ['Infantile Spasms']}, 'descriptionModule': {'briefSummary': 'To allow open-label extension to patients who have completed Protocol 1042-0500', 'detailedDescription': 'Patient should have completed all scheduled clinical study visits in the double blind, controlled trial (Protocol 1042-0500) and have been deemed eligible (had a response to treatment) by the Investigator. Male or female, with a diagnosis of IS with a video EEG (vEEG) recording confirming the diagnosis.\n\nThere will be a total of 14 visits over 99(+or-1)week. A 24-hr vEEG is only required if the subject has been spasm-free for more than 24-hrs.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD'], 'maximumAge': '24 Months', 'minimumAge': '4 Months', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Have completed all scheduled clinical study visits in the previous Protocol 1042 0500 and have been deemed eligible (no SAEs thought to be drug related and had a response to treatment) by the Investigator.\n* Be diagnosed with IS regardless of etiology. Diagnostic Criteria: Seizures consisting of single or repetitive short muscular contractions leading to flexion or extension. Spasms may be characterized as tonic or myoclonic contractions, may occur singly or in clusters, and typically occur bilaterally and symmetrically. The EEG pattern must be consistent with the diagnosis of IS (hypsarrhythmia, modified hypsarrhythmia, multifocal spike wave discharges, etc).\n* Have a 24 hour vEEG recording confirming the diagnosis of IS.\n* Have had a magnetic resonance imaging (MRI) performed to determine any possible causes of IS.\n* Have been previously treated with 3 AEDs or fewer.\n* Have a parent/guardian who is properly informed of the nature and potential risks and benefits of the clinical study, is willing and capable of complying with all clinical study procedures, and has given informed consent in writing prior to entering the clinical study.\n\nExclusion Criteria:\n\n* Current treatment with more than 2 concomitant AEDs.\n* Have an active CNS infection, demyelinating disease, degenerative neurological disease, or CNS disease deemed progressive (with the exception of tuberous sclerosis) as evaluated by brain MRI.\n* Have any disease or condition (medical or surgical) at Screening that might compromise the hematologic, cardiovascular, pulmonary, renal, GI, or hepatic systems; or other conditions that might interfere with the absorption, distribution, metabolism, or excretion of the investigational product, or would place the subject at increased risk.\n* Aspartate transaminase (AST), alanine transaminase (ALT), or total bilirubin greater than 4 times the upper limit of laboratory normal or any clinical laboratory value deemed clinically significant by the Investigator.\n* History of recurrent status epilepticus.\n* Have been exposed to any other investigational drug within 30 days prior to enrollment.'}, 'identificationModule': {'nctId': 'NCT00442104', 'briefTitle': 'Open-label Extension to Protocol 1042-0500', 'organization': {'class': 'INDUSTRY', 'fullName': 'Marinus Pharmaceuticals'}, 'officialTitle': 'An Open-label Clinical Study to Evaluate the Safety and Antiepileptic Activity of Ganaxolone in Treatment of Patients Diagnosed With Infantile Spasms.', 'orgStudyIdInfo': {'id': '1042-0501'}, 'secondaryIdInfos': [{'id': 'Amend 5 (ROW)'}, {'id': 'Amend 6 (US)'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'ganaxolone', 'interventionNames': ['Drug: Ganaxolone']}], 'interventions': [{'name': 'Ganaxolone', 'type': 'DRUG', 'armGroupLabels': ['ganaxolone']}]}, 'contactsLocationsModule': {'locations': [{'zip': '90027', 'city': 'Los Angeles', 'state': 'California', 'country': 'United States', 'facility': "Children's Hospital of Los Angeles", 'geoPoint': {'lat': 34.05223, 'lon': -118.24368}}, {'zip': '90095', 'city': 'Los Angeles', 'state': 'California', 'country': 'United States', 'facility': "Mattel Children's Hospital at UCLA", 'geoPoint': {'lat': 34.05223, 'lon': -118.24368}}, {'zip': '20010', 'city': 'Washington D.C.', 'state': 'District of Columbia', 'country': 'United States', 'facility': "Children's National Medical Center", 'geoPoint': {'lat': 38.89511, 'lon': -77.03637}}, {'zip': '33155', 'city': 'Miami', 'state': 'Florida', 'country': 'United States', 'facility': "Miami Children's Hospital, The Brain Institute", 'geoPoint': {'lat': 25.77427, 'lon': -80.19366}}, {'zip': '32504', 'city': 'Pensacola', 'state': 'Florida', 'country': 'United States', 'facility': 'Child Neurology Center of Nrothwest Florida, P.A.', 'geoPoint': {'lat': 30.42131, 'lon': -87.21691}}, {'zip': '60637', 'city': 'Chicago', 'state': 'Illinois', 'country': 'United States', 'facility': "University of Chicago Comer Children's Hospital", 'geoPoint': {'lat': 41.85003, 'lon': -87.65005}}, {'zip': '55102', 'city': 'Saint Paul', 'state': 'Minnesota', 'country': 'United States', 'facility': 'Minnesota Epilepsy Group, P.A.', 'geoPoint': {'lat': 44.94441, 'lon': -93.09327}}, {'zip': '10467', 'city': 'The Bronx', 'state': 'New York', 'country': 'United States', 'facility': 'Montefiore Medical Center- Albert Einstein College of Medicine', 'geoPoint': {'lat': 40.84985, 'lon': -73.86641}}, {'zip': '38105', 'city': 'Memphis', 'state': 'Tennessee', 'country': 'United States', 'facility': "Le Bonheur Children's Medical Center", 'geoPoint': {'lat': 35.14953, 'lon': -90.04898}}, {'zip': '75230', 'city': 'Dallas', 'state': 'Texas', 'country': 'United States', 'facility': 'Dallas Pediatric Neurology Associates', 'geoPoint': {'lat': 32.78306, 'lon': -96.80667}}, {'zip': '77030', 'city': 'Houston', 'state': 'Texas', 'country': 'United States', 'facility': "Texas Children's Hospital", 'geoPoint': {'lat': 29.76328, 'lon': -95.36327}}, {'zip': '23298', 'city': 'Richmond', 'state': 'Virginia', 'country': 'United States', 'facility': 'Virginia Commonwealth University Health Systems', 'geoPoint': {'lat': 37.55376, 'lon': -77.46026}}, {'zip': '98105', 'city': 'Seattle', 'state': 'Washington', 'country': 'United States', 'facility': "Children's Hospital and Regional Medical Center", 'geoPoint': {'lat': 47.60621, 'lon': -122.33207}}, {'zip': '53201', 'city': 'Milwaukee', 'state': 'Wisconsin', 'country': 'United States', 'facility': "Children's Hospital of Wisconsin", 'geoPoint': {'lat': 43.0389, 'lon': -87.90647}}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Marinus Pharmaceuticals', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}