Ignite Creation Date:
2025-12-25 @ 12:14 AM
Ignite Modification Date:
2026-01-31 @ 6:58 AM
Study NCT ID:
NCT02300558
Status:
TERMINATED
Last Update Posted:
2018-01-12
First Post:
2014-11-21
Is Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Effect of Eleclazine on Shortening of the QT Interval, Safety, and Tolerability in Adults With Long QT Syndrome Type 3
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'removedCountries': ['Finland', 'Spain']}, 'conditionBrowseModule': {'meshes': [{'id': 'C537034', 'term': 'Long QT syndrome type 3'}, {'id': 'D008133', 'term': 'Long QT Syndrome'}, {'id': 'D016757', 'term': 'Death, Sudden, Cardiac'}], 'ancestors': [{'id': 'D001145', 'term': 'Arrhythmias, Cardiac'}, {'id': 'D006331', 'term': 'Heart Diseases'}, {'id': 'D002318', 'term': 'Cardiovascular Diseases'}, {'id': 'D000075224', 'term': 'Cardiac Conduction System Disease'}, {'id': 'D006330', 'term': 'Heart Defects, Congenital'}, {'id': 'D018376', 'term': 'Cardiovascular Abnormalities'}, {'id': 'D000013', 'term': 'Congenital Abnormalities'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D010335', 'term': 'Pathologic Processes'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}, {'id': 'D006323', 'term': 'Heart Arrest'}, {'id': 'D003645', 'term': 'Death, Sudden'}, {'id': 'D003643', 'term': 'Death'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C000624281', 'term': 'eleclazine'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'ClinicalTrialDisclosures@gilead.com', 'title': 'Clinical Trial Disclosures', 'organization': 'Gilead Sciences'}, 'certainAgreement': {'otherDetails': 'After conclusion of the study and without prior written approval from Gilead, investigators in this study may communicate, orally present, or publish in scientific journals or other media only after the following conditions have been met:\n\n* The results of the study in their entirety have been publicly disclosed by or with the consent of Gilead in an abstract, manuscript, or presentation form; or\n* The study has been completed at all study sites for at least 2 years', 'restrictionType': 'OTHER', 'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': 'Day 1 to the last dose date plus 30 days (median exposure to eleclazine: 396 days)', 'description': 'Safety Analysis Set', 'eventGroups': [{'id': 'EG000', 'title': 'Placebo', 'description': 'Single oral loading dose of placebo to match eleclazine loading dose (8 x 6 mg placebo to match tablets) on Day 1', 'otherNumAtRisk': 41, 'deathsNumAtRisk': 41, 'otherNumAffected': 5, 'seriousNumAtRisk': 41, 'deathsNumAffected': 0, 'seriousNumAffected': 0}, {'id': 'EG001', 'title': 'Eleclazine Loading Dose (LD) 48 mg', 'description': 'Eleclazine 48 mg ( 8 x 6 mg tablets) administered orally on Day 2', 'otherNumAtRisk': 41, 'deathsNumAtRisk': 41, 'otherNumAffected': 5, 'seriousNumAtRisk': 41, 'deathsNumAffected': 0, 'seriousNumAffected': 0}, {'id': 'EG002', 'title': 'Eleclazine Maintenance Dose (MD) 3 mg', 'description': 'Eleclazine 3 mg (1 x 3 mg tablet) administered once daily from Day 3 to the Week 12 Visit', 'otherNumAtRisk': 41, 'deathsNumAtRisk': 41, 'otherNumAffected': 17, 'seriousNumAtRisk': 41, 'deathsNumAffected': 0, 'seriousNumAffected': 1}, {'id': 'EG003', 'title': 'Eleclazine MD 6 mg', 'description': 'Eleclazine 6 mg (1 x 6 mg tablet) administered orally from the day after the Week 12 Visit through Week 24 and open-label extension.', 'otherNumAtRisk': 41, 'deathsNumAtRisk': 41, 'otherNumAffected': 14, 'seriousNumAtRisk': 41, 'deathsNumAffected': 0, 'seriousNumAffected': 0}, {'id': 'EG004', 'title': 'All Eleclazine', 'description': '* Loading Dose: Eleclazine 48 mg ( 8 x 6 mg tablets) administered on Day 2 and 3 mg (1 x 3 mg tablet) once daily from Day 3 to the Week 12 Visit\n* Maintenance dose: Eleclazine 6 mg (1 x 6 mg tablet) from the day after the Week 12 Visit through Week 24 and open-label extension.\n\nAdverse events in this reporting group include those that occurred any time during the study by participants while receiving loading dose or maintenance dose of eleclazine.', 'otherNumAtRisk': 41, 'deathsNumAtRisk': 41, 'otherNumAffected': 24, 'seriousNumAtRisk': 41, 'deathsNumAffected': 0, 'seriousNumAffected': 1}], 'otherEvents': [{'term': 'Vertigo', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 41, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 41, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 41, 'numAffected': 2}, {'groupId': 'EG004', 'numAtRisk': 41, 'numAffected': 3}], 'organSystem': 'Ear and labyrinth disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.1'}, {'term': 'Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 41, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 41, 'numAffected': 3}, {'groupId': 'EG003', 'numAtRisk': 41, 'numAffected': 1}, {'groupId': 'EG004', 'numAtRisk': 41, 'numAffected': 4}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.1'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 41, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 41, 'numAffected': 3}, {'groupId': 'EG003', 'numAtRisk': 41, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 41, 'numAffected': 4}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.1'}, {'term': 'Asthenia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 41, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 41, 'numAffected': 2}, {'groupId': 'EG003', 'numAtRisk': 41, 'numAffected': 1}, {'groupId': 'EG004', 'numAtRisk': 41, 'numAffected': 3}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.1'}, {'term': 'Fatigue', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 41, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 41, 'numAffected': 3}, {'groupId': 'EG003', 'numAtRisk': 41, 'numAffected': 2}, {'groupId': 'EG004', 'numAtRisk': 41, 'numAffected': 5}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.1'}, {'term': 'Influenza', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 41, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 41, 'numAffected': 3}, {'groupId': 'EG003', 'numAtRisk': 41, 'numAffected': 3}, {'groupId': 'EG004', 'numAtRisk': 41, 'numAffected': 6}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.1'}, {'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 41, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 41, 'numAffected': 2}, {'groupId': 'EG003', 'numAtRisk': 41, 'numAffected': 2}, {'groupId': 'EG004', 'numAtRisk': 41, 'numAffected': 4}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.1'}, {'term': 'Weight increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 41, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 41, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 41, 'numAffected': 2}, {'groupId': 'EG004', 'numAtRisk': 41, 'numAffected': 3}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.1'}, {'term': 'Arthralgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 41, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 41, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 41, 'numAffected': 2}, {'groupId': 'EG004', 'numAtRisk': 41, 'numAffected': 2}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.1'}, {'term': 'Back pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 41, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 41, 'numAffected': 3}, {'groupId': 'EG003', 'numAtRisk': 41, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 41, 'numAffected': 3}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.1'}, {'term': 'Dizziness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 41, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 41, 'numAffected': 2}, {'groupId': 'EG003', 'numAtRisk': 41, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 41, 'numAffected': 4}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.1'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 41, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 41, 'numAffected': 2}, {'groupId': 'EG003', 'numAtRisk': 41, 'numAffected': 4}, {'groupId': 'EG004', 'numAtRisk': 41, 'numAffected': 6}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.1'}], 'seriousEvents': [{'term': 'Nephrolithiasis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 41, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 41, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 41, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 41, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 41, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.1'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Change From Baseline in Mean Daytime QT Interval in Lead V5 Corrected for Heart Rate Using the Fridericia Formula (QTcF) Interval to Week 24 (Based on Standard 12-lead ECG Data)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '35', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Eleclazine', 'description': '* Single-Blind Treatment Period: Single oral loading dose of placebo to match eleclazine loading dose (8 x 6 mg placebo to match tablets) on Day 1; eleclazine 48 mg ( 8 x 6 mg tablets) on Day 2; followed by eleclazine 3 mg (1 x 3 mg tablet) once daily from Day 3 to the Week 12 Visit; then once daily maintenance dose of eleclazine 6 mg (1 x 6 mg tablet) from the day after the Week 12 Visit through Week 24\n* OLE: Eleclazine 6 mg or 3 mg tablets orally once daily'}], 'classes': [{'categories': [{'measurements': [{'value': '-8.5', 'spread': '18.03', 'groupId': 'OG000'}]}]}], 'analyses': [{'pValue': '0.0087', 'groupIds': ['OG000'], 'groupDescription': 'Based on a 2-sided, paired t-test ( α = 0.05), and a standard deviation (SD) of 30 msec, a sample size of 40 participants provided greater than 95% power assuming a difference in mean daytime QTcF interval (AUC0-6/6) as measured by standard 12-lead ECG between baseline and Week 24 of 20 msec. The null hypothesis was that the mean difference between the baseline and Week 24 mean daytime QTcF interval was zero.', 'statisticalMethod': 'paired t- test', 'nonInferiorityType': 'OTHER'}], 'paramType': 'MEAN', 'timeFrame': 'Baseline; Week 24', 'description': "* Baseline was the Day 1 value.\n* QTcF is corrected QT interval using Fridericia's formula. QTcF = QT/cube root (RR), where RR is in seconds.\n* AUC0-6 for QTcF was calculated using the trapezoidal rule, mean of triplicate values, and actual time (latest of triplicate times).\n* Mean daytime QTcF (AUC0-6/6) was computed by dividing AUC0-6 by the time from dosing to the 6 hour postdose time point.", 'unitOfMeasure': 'msec', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Participants in Full Analysis Set (FAS) with available data were analyzed. FAS was defined as all enrolled participants who have confirmed LQT3 genotype, do not have confirmed LQT1 or LQT2 mutations, received at least 1 dose of active eleclazine, and have both a baseline and at least 1 postbaseline mean daytime QTcF interval (standard 12-lead).'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Mean Daytime QTcF Interval (AUC0-6/6) to Week 12 (Lead V5; Standard 12-lead ECG)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '40', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Eleclazine', 'description': '* Single-Blind Treatment Period: Single oral loading dose of placebo to match eleclazine loading dose (8 x 6 mg placebo to match tablets) on Day 1; eleclazine 48 mg ( 8 x 6 mg tablets) on Day 2; followed by eleclazine 3 mg (1 x 3 mg tablet) once daily from Day 3 to the Week 12 Visit; then once daily maintenance dose of eleclazine 6 mg (1 x 6 mg tablet) from the day after the Week 12 Visit through Week 24\n* OLE: Eleclazine 6 mg or 3 mg tablets orally once daily'}], 'classes': [{'categories': [{'measurements': [{'value': '2.2', 'groupId': 'OG000', 'lowerLimit': '-4.7', 'upperLimit': '9.2'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline; Week 12', 'description': "* Baseline was the Day 1 value.\n* QTcF is corrected QT interval using Fridericia's formula. QTcF = QT/cube root (RR), where RR is in seconds.\n* AUC0-6 for QTcF was calculated using the trapezoidal rule, mean of triplicate values, and actual time (latest of triplicate times) .\n* Mean daytime QTcF (AUC0-6/6) was computed by dividing AUC0-6 by the time from dosing to the 6 hour postdose time point.", 'unitOfMeasure': 'msec', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'Participants in the Full Analysis Set with available data were analyzed.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Mean Daily (Daytime and Nocturnal) QTcF Interval to Week 24 (Lead V5; Holter)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '30', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Eleclazine', 'description': '* Single-Blind Treatment Period: Single oral loading dose of placebo to match eleclazine loading dose (8 x 6 mg placebo to match tablets) on Day 1; eleclazine 48 mg ( 8 x 6 mg tablets) on Day 2; followed by eleclazine 3 mg (1 x 3 mg tablet) once daily from Day 3 to the Week 12 Visit; then once daily maintenance dose of eleclazine 6 mg (1 x 6 mg tablet) from the day after the Week 12 Visit through Week 24\n* OLE: Eleclazine 6 mg or 3 mg tablets orally once daily'}], 'classes': [{'categories': [{'measurements': [{'value': '-1.8', 'groupId': 'OG000', 'lowerLimit': '-13.6', 'upperLimit': '10.0'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline; Week 24', 'description': "* Baseline was the Day 1 value.\n* QTcF is corrected QT interval using Fridericia's formula. QTcF = QT/cube root (RR), where RR is in seconds.\n* Mean daytime QTcF (AUC0-6/6) was computed by dividing AUC0-6 by the time from the first nonmissing nominal time point to the last nonmissing nominal time point, from predose to 6 hours postdose. Mean nocturnal QTcF (AUC0-6/6) was computed by dividing AUC0-6 by the time from the first nonmissing nominal time point to the last nonmissing nominal time point, from midnight to 6:00 AM. Daily was computed as the average of daytime (AUC0-6/6) and nocturnal (AUC0-6/6), with both values required to compute the average.", 'unitOfMeasure': 'msec', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'Participants in the Full Analysis Set with available data were analyzed.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Mean Nocturnal QTcF Interval to Week 24 (Lead V5; Holter)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '31', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Eleclazine', 'description': '* Single-Blind Treatment Period: Single oral loading dose of placebo to match eleclazine loading dose (8 x 6 mg placebo to match tablets) on Day 1; eleclazine 48 mg ( 8 x 6 mg tablets) on Day 2; followed by eleclazine 3 mg (1 x 3 mg tablet) once daily from Day 3 to the Week 12 Visit; then once daily maintenance dose of eleclazine 6 mg (1 x 6 mg tablet) from the day after the Week 12 Visit through Week 24\n* OLE: Eleclazine 6 mg or 3 mg tablets orally once daily'}], 'classes': [{'categories': [{'measurements': [{'value': '-3.0', 'groupId': 'OG000', 'lowerLimit': '-17.1', 'upperLimit': '11.0'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline; Week 24', 'description': "* Baseline was the Day 1 value.\n* QTcF is corrected QT interval using Fridericia's formula. QTcF = QT/cube root (RR), where RR is in seconds.\n* Mean nocturnal QTcF (AUC0-6/6) was computed by dividing AUC0-6 by the time from the first nonmissing nominal time point to the last nonmissing nominal time point, from midnight to 6:00 AM.", 'unitOfMeasure': 'msec', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'Participants in the the Full Analysis Set with available data were analyzed.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Eleclazine', 'description': '* Single-Blind Treatment Period: Single oral loading dose of placebo to match eleclazine loading dose (8 x 6 mg placebo to match tablets) on Day 1; eleclazine 48 mg ( 8 x 6 mg tablets) on Day 2; followed by eleclazine 3 mg (1 x 3 mg tablet) once daily from Day 3 to the Week 12 Visit; then once daily maintenance dose of eleclazine 6 mg (1 x 6 mg tablet) from the day after the Week 12 Visit through Week 24\n* Open-label Extension (OLE): Eleclazine 6 mg or 3 mg tablets orally once daily'}], 'periods': [{'title': 'Single Blind Phase (24 Weeks)', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '41'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '35'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '6'}]}], 'dropWithdraws': [{'type': 'Study Terminated by Sponsor', 'reasons': [{'groupId': 'FG000', 'numSubjects': '6'}]}]}, {'title': 'Open-Label (OL) Extension Phase', 'milestones': [{'type': 'STARTED', 'comment': '3 participants completed the single-blind phase (24 weeks) but did not continue to OL Extension.', 'achievements': [{'groupId': 'FG000', 'numSubjects': '32'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '32'}]}], 'dropWithdraws': [{'type': 'Lack of Efficacy', 'reasons': [{'groupId': 'FG000', 'numSubjects': '2'}]}, {'type': 'Withdrew Consent', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}]}, {'type': 'Study Terminated by Sponsor', 'reasons': [{'groupId': 'FG000', 'numSubjects': '29'}]}]}], 'recruitmentDetails': 'Participants were enrolled at study sites in North America, Europe, and Asia. The first participant was screened on 17 December 2014. The last study visit occurred on 15 February 2017.', 'preAssignmentDetails': '54 participants were screened.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '41', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'Eleclazine', 'description': '* Single-Blind Treatment Period: Single oral loading dose of placebo to match eleclazine loading dose (8 x 6 mg placebo to match tablets) on Day 1; eleclazine 48 mg ( 8 x 6 mg tablets) on Day 2; followed by eleclazine 3 mg (1 x 3 mg tablet) once daily from Day 3 to the Week 12 Visit; then once daily maintenance dose of eleclazine 6 mg (1 x 6 mg tablet) from the day after the Week 12 Visit through Week 24\n* OLE: Eleclazine 6 mg or 3 mg tablets orally once daily'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '46', 'spread': '12.5', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'Years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '24', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '17', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race/Ethnicity, Customized', 'classes': [{'categories': [{'title': 'American Indian or Alaska Native', 'measurements': [{'value': '1', 'groupId': 'BG000'}]}, {'title': 'Asian', 'measurements': [{'value': '1', 'groupId': 'BG000'}]}, {'title': 'Black or African American', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'White', 'measurements': [{'value': '36', 'groupId': 'BG000'}]}, {'title': 'Not Permitted', 'measurements': [{'value': '1', 'groupId': 'BG000'}]}, {'title': 'Other', 'measurements': [{'value': '2', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race/Ethnicity, Customized', 'classes': [{'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '40', 'groupId': 'BG000'}]}, {'title': 'Not Permitted', 'measurements': [{'value': '1', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'Canada', 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}]}]}, {'title': 'Netherlands', 'categories': [{'measurements': [{'value': '3', 'groupId': 'BG000'}]}]}, {'title': 'United States', 'categories': [{'measurements': [{'value': '11', 'groupId': 'BG000'}]}]}, {'title': 'Italy', 'categories': [{'measurements': [{'value': '12', 'groupId': 'BG000'}]}]}, {'title': 'United Kingdom', 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}]}]}, {'title': 'Israel', 'categories': [{'measurements': [{'value': '3', 'groupId': 'BG000'}]}]}, {'title': 'France', 'categories': [{'measurements': [{'value': '9', 'groupId': 'BG000'}]}]}, {'title': 'Germany', 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Mean Daytime QTcF Lead V5 (Standard 12-lead ECG)', 'classes': [{'categories': [{'measurements': [{'value': '507.5', 'spread': '38.11', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'description': '* QTcF refers to QT interval corrected for heart rate using the Fridericia formula. QTcF = QT/cube root (RR), where RR is in seconds.\n* AUC0-6 for QTcF was calculated using the trapezoidal rule, mean of triplicate values, and actual time (latest of triplicate times).\n* Mean daytime QTcF (AUC0-6/6) was computed by dividing AUC0-6 by the time from dosing to the 6 hour postdose time point.', 'unitOfMeasure': 'msec', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Mean Daily QTcF in Lead V5 (Holter)', 'classes': [{'categories': [{'measurements': [{'value': '514.7', 'spread': '45.76', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'description': '* AUC (0-6) was calculated using the trapezoidal rule, mean of triplicate vales, and nominal time.\n* Mean daytime QTcF (AUC 0-6/6) was computed by dividing AUC(0-6) by the time from the first nonmissing time\\^ to the last nonmissing time\\^, from predose to 6 hours postdose.\n* Mean nocturnal QTcF (AUC 0-6/6) was computed by dividing AUC(0-6) by the time from the first nonmissing time\\^ to the last nonmissing time\\^, from midnight to 6:00 AM.\n* Daily was computed as the average of daytime AUC(0-6/6) and nocturnal AUC (0-6/6), when both values are not missing.\n* \\^ = nominal time point', 'unitOfMeasure': 'msec', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Mean Nocturnal QTcF in Lead V5 (Holter)', 'classes': [{'categories': [{'measurements': [{'value': '530.6', 'spread': '52.25', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'description': 'AUC(0-6) for QTcF was calculated using the trapezoidal rule, mean of triplicate values, and nominal time.\n\nMean nocturnal QTcF (AUC 0-6/6) was computed by dividing AUC(0-6) by the time from the first nonmissing nominal time point to the last nonmissing nominal time point, from midnight to 6:00 AM.', 'unitOfMeasure': 'msec', 'dispersionType': 'STANDARD_DEVIATION'}], 'populationDescription': 'Safety Analysis Set: participants who received at least 1 dose of study drug (either active or placebo).'}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'SINGLE', 'whoMasked': ['PARTICIPANT']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 41}}, 'statusModule': {'overallStatus': 'TERMINATED', 'startDateStruct': {'date': '2014-12-17', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2017-12', 'completionDateStruct': {'date': '2017-02-15', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2017-12-12', 'studyFirstSubmitDate': '2014-11-21', 'resultsFirstSubmitDate': '2017-12-12', 'studyFirstSubmitQcDate': '2014-11-21', 'lastUpdatePostDateStruct': {'date': '2018-01-12', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2017-12-12', 'studyFirstPostDateStruct': {'date': '2014-11-25', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2018-01-12', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2016-12-12', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Change From Baseline in Mean Daytime QT Interval in Lead V5 Corrected for Heart Rate Using the Fridericia Formula (QTcF) Interval to Week 24 (Based on Standard 12-lead ECG Data)', 'timeFrame': 'Baseline; Week 24', 'description': "* Baseline was the Day 1 value.\n* QTcF is corrected QT interval using Fridericia's formula. QTcF = QT/cube root (RR), where RR is in seconds.\n* AUC0-6 for QTcF was calculated using the trapezoidal rule, mean of triplicate values, and actual time (latest of triplicate times).\n* Mean daytime QTcF (AUC0-6/6) was computed by dividing AUC0-6 by the time from dosing to the 6 hour postdose time point."}], 'secondaryOutcomes': [{'measure': 'Change From Baseline in Mean Daytime QTcF Interval (AUC0-6/6) to Week 12 (Lead V5; Standard 12-lead ECG)', 'timeFrame': 'Baseline; Week 12', 'description': "* Baseline was the Day 1 value.\n* QTcF is corrected QT interval using Fridericia's formula. QTcF = QT/cube root (RR), where RR is in seconds.\n* AUC0-6 for QTcF was calculated using the trapezoidal rule, mean of triplicate values, and actual time (latest of triplicate times) .\n* Mean daytime QTcF (AUC0-6/6) was computed by dividing AUC0-6 by the time from dosing to the 6 hour postdose time point."}, {'measure': 'Change From Baseline in Mean Daily (Daytime and Nocturnal) QTcF Interval to Week 24 (Lead V5; Holter)', 'timeFrame': 'Baseline; Week 24', 'description': "* Baseline was the Day 1 value.\n* QTcF is corrected QT interval using Fridericia's formula. QTcF = QT/cube root (RR), where RR is in seconds.\n* Mean daytime QTcF (AUC0-6/6) was computed by dividing AUC0-6 by the time from the first nonmissing nominal time point to the last nonmissing nominal time point, from predose to 6 hours postdose. Mean nocturnal QTcF (AUC0-6/6) was computed by dividing AUC0-6 by the time from the first nonmissing nominal time point to the last nonmissing nominal time point, from midnight to 6:00 AM. Daily was computed as the average of daytime (AUC0-6/6) and nocturnal (AUC0-6/6), with both values required to compute the average."}, {'measure': 'Change From Baseline in Mean Nocturnal QTcF Interval to Week 24 (Lead V5; Holter)', 'timeFrame': 'Baseline; Week 24', 'description': "* Baseline was the Day 1 value.\n* QTcF is corrected QT interval using Fridericia's formula. QTcF = QT/cube root (RR), where RR is in seconds.\n* Mean nocturnal QTcF (AUC0-6/6) was computed by dividing AUC0-6 by the time from the first nonmissing nominal time point to the last nonmissing nominal time point, from midnight to 6:00 AM."}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Type 3 long QT syndrome (LQT3)', 'Long QT syndrome', 'Congenital long QT', 'Sudden cardiac death'], 'conditions': ['Long QT Syndrome Type 3']}, 'descriptionModule': {'briefSummary': 'The primary objective of this study is to evaluate the effect of oral eleclazine on mean daytime QTcF interval after 24 weeks of treatment with elecalzine in participants with long QT syndrome Type 3. During the single-blind treatment period (24 weeks), participants will receive eleclazine and/or eleclazine placebo. Following the single-blind treatment period, participants who have not permanently discontinued study drug will be eligible, at the discretion of the investigator, to continue receiving eleclazine during an open-label extension (OLE) phase.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '70 Years', 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Key Inclusion Criteria:\n\n* Individuals with an established diagnosis of LQT3 (by genotype testing)\n* Mean (of triplicate) QTc interval ≥ 480 msec (or ≥ 460 msec, for individuals who are currently taking ranolazine or Class I antiarrhythmic drugs such as mexiletine) at 3 or more time points, determined by standard 12-lead ECG, at screening\n\nKey Exclusion Criteria:\n\n* Known mutations associated with type 1 long QT syndrome (LQT1) or type 2 long QT syndrome (LQT2)\n* Known or suspected history of seizures or epilepsy\n* History of heart failure defined as New York Heart Association (NYHA) Class IV and/or known left ventricular ejection fraction (EF) ≤ 45%\n* Body mass index (BMI) ≥ 40 kg/m\\^2 at screening\n* Severe renal impairment at screening (defined as an estimated glomerular filtration rate (eGFR) \\< 30 mL/min/1.73m\\^2, using the 4 Variable Modification of Diet in Renal Disease (MDRD) equation, as determined by the study center)\n* Abnormal liver function tests at screening, defined as alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \\> 2 x upper limit of normal (ULN), or total bilirubin \\> 1.5 x ULN\n* An aborted cardiac arrest (ACA), implantable cardioverter-defibrillator (ICD) implantation, syncopal episode, or appropriate ICD therapy within 3 months prior to screening\n* Any other condition or circumstance that in the opinion of the investigator would preclude compliance with the study protocol\n\nNote: Other protocol defined Inclusion/Exclusion criteria may apply.'}, 'identificationModule': {'nctId': 'NCT02300558', 'briefTitle': 'Effect of Eleclazine on Shortening of the QT Interval, Safety, and Tolerability in Adults With Long QT Syndrome Type 3', 'organization': {'class': 'INDUSTRY', 'fullName': 'Gilead Sciences'}, 'officialTitle': 'A Phase 3, Single-Blind Study to Evaluate the Effect of Eleclazine (GS-6615) on Shortening of the QT Interval, Safety, and Tolerability in Subjects With Long QT Syndrome Type 3', 'orgStudyIdInfo': {'id': 'GS-US-372-1234'}, 'secondaryIdInfos': [{'id': '2014-000042-30', 'type': 'EUDRACT_NUMBER'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Eleclazine (Single-blind treatment phase)', 'description': 'Eleclazine and/or eleclazine placebo up to Week 24', 'interventionNames': ['Drug: Eleclazine', 'Drug: Eleclazine placebo']}, {'type': 'EXPERIMENTAL', 'label': 'Open-label Extension Phase', 'description': "Eligible participants will continue to receive open-label eleclazine until this drug is commercially available for the treatment of patients with LQT3, or until Gilead terminates development of eleclazine for the treatment of patients with LQT3, or the investigator deems it no longer in the participant's best interest.", 'interventionNames': ['Drug: Eleclazine']}], 'interventions': [{'name': 'Eleclazine', 'type': 'DRUG', 'otherNames': ['GS-6615'], 'description': 'Tablets administered orally', 'armGroupLabels': ['Eleclazine (Single-blind treatment phase)', 'Open-label Extension Phase']}, {'name': 'Eleclazine placebo', 'type': 'DRUG', 'description': 'Tablets administered orally', 'armGroupLabels': ['Eleclazine (Single-blind treatment phase)']}]}, 'contactsLocationsModule': {'locations': [{'zip': '55905', 'city': 'Rochester', 'state': 'Minnesota', 'country': 'United States', 'facility': 'Mayo Clinic Rochester', 'geoPoint': {'lat': 44.02163, 'lon': -92.4699}}, {'zip': '10016', 'city': 'New York', 'state': 'New York', 'country': 'United States', 'facility': 'NYU Langone Medical Center', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': '14642', 'city': 'Rochester', 'state': 'New York', 'country': 'United States', 'facility': 'University of Rochester Medical Center', 'geoPoint': {'lat': 43.15478, 'lon': -77.61556}}, {'zip': 'B3H 3A7', 'city': 'Halifax', 'state': 'Nova Scotia', 'country': 'Canada', 'facility': 'Nova Scotia Health Authority', 'geoPoint': {'lat': 44.64269, 'lon': -63.57688}}, {'zip': '44093', 'city': 'Nantes', 'country': 'France', 'facility': "L'institut Du Thorax Nantes", 'geoPoint': {'lat': 47.21725, 'lon': -1.55336}}, {'zip': '75877', 'city': 'Paris', 'country': 'France', 'facility': 'Groupe Hospitalier Bichat Claude Bernard', 'geoPoint': {'lat': 48.85341, 'lon': 2.3488}}, {'zip': '97410', 'city': 'Saint-Pierre', 'country': 'France', 'facility': 'CHU Réunion Sud', 'geoPoint': {'lat': 43.29282, 'lon': 5.40682}}, {'zip': '80336', 'city': 'München', 'country': 'Germany', 'facility': 'LMU Klinikum der Universität München', 'geoPoint': {'lat': 51.60698, 'lon': 13.31243}}, {'city': 'Tel Aviv', 'country': 'Israel', 'facility': 'Tel Aviv Sourasky Medical Center', 'geoPoint': {'lat': 32.08088, 'lon': 34.78057}}, {'zip': '27100', 'city': 'Pavia', 'country': 'Italy', 'facility': 'Fondazione Salvatore Maugeri IRCCS', 'geoPoint': {'lat': 45.19205, 'lon': 9.15917}}, {'zip': '1105 AZ', 'city': 'Amsterdam', 'country': 'Netherlands', 'facility': 'Academisch Medisch Centrum Amsterdam', 'geoPoint': {'lat': 52.37403, 'lon': 4.88969}}, {'zip': 'EC1M 6BQ', 'city': 'London', 'country': 'United Kingdom', 'facility': 'Barts and The London School of Medicine and Dentistry', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}], 'overallOfficials': [{'name': 'Gilead Study Director', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Gilead Sciences'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Gilead Sciences', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}