Ignite Creation Date:
2025-12-25 @ 2:24 AM
Ignite Modification Date:
2026-02-25 @ 8:19 PM
Study NCT ID:
NCT03732534
Status:
TERMINATED
Last Update Posted:
2022-04-19
First Post:
2018-11-05
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Rollover Study for Continuing NBI-98854 Administration in Pediatric Subjects With Tourette Syndrome
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'submissionTracking': {'firstMcpInfo': {'postDateStruct': {'date': '2022-02-23', 'type': 'ACTUAL'}}}}, 'conditionBrowseModule': {'meshes': [{'id': 'D005879', 'term': 'Tourette Syndrome'}], 'ancestors': [{'id': 'D001480', 'term': 'Basal Ganglia Diseases'}, {'id': 'D001927', 'term': 'Brain Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D013981', 'term': 'Tic Disorders'}, {'id': 'D009069', 'term': 'Movement Disorders'}, {'id': 'D020271', 'term': 'Heredodegenerative Disorders, Nervous System'}, {'id': 'D019636', 'term': 'Neurodegenerative Diseases'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D065886', 'term': 'Neurodevelopmental Disorders'}, {'id': 'D001523', 'term': 'Mental Disorders'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C000603978', 'term': 'valbenazine'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'medinfo@neurocrine.com', 'phone': '877-641-3461', 'title': 'Neurocrine Medical Information', 'organization': 'Neurocrine Biosciences'}, 'certainAgreement': {'otherDetails': 'Generally, the PI has the right to publish results provided such publication does not violate confidentiality or IP provisions within the contract with the Sponsor. Prior to submission for publication or presentation of results, the PI must provide the Sponsor time for review. The Sponsor can request the PI to withhold or remove information from all publications. For a multi-center study, any publication of results by the PI shall not be made before the first multi-center publication.', 'restrictionType': 'OTHER', 'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': 'Up to 16 weeks', 'eventGroups': [{'id': 'EG000', 'title': 'Valbenazine', 'description': 'Participants received valbenazine once daily for up to 96 weeks. The starting dose was 20 mg for participants \\<50 kg at baseline and 40 mg for participants ≥50 kg at baseline, and could be escalated in increments of 20 mg every 2 weeks to a maximum of 60 mg for participants \\<50 kg and 80 mg for participants ≥50 kg to achieve an optimal dose of valbenazine for each participant.', 'otherNumAtRisk': 6, 'deathsNumAtRisk': 6, 'otherNumAffected': 2, 'seriousNumAtRisk': 6, 'deathsNumAffected': 0, 'seriousNumAffected': 0}], 'otherEvents': [{'term': 'Somnolence', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numAffected': 2}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}, {'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 6, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.0'}], 'frequencyThreshold': '0'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Number of Participants With Treatment-Emergent Adverse Events (TEAEs)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Valbenazine', 'description': 'Participants received valbenazine once daily for up to 96 weeks. The starting dose was 20 mg for participants \\<50 kg at baseline and 40 mg for participants ≥50 kg at baseline, and could be escalated in increments of 20 mg every 2 weeks to a maximum of 60 mg for participants \\<50 kg and 80 mg for participants ≥50 kg to achieve an optimal dose of valbenazine for each participant.'}], 'classes': [{'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Up to 16 Weeks', 'description': 'A TEAE is an adverse event not present prior to the initiation of study drug dosing, or is an already present event that worsens either in intensity or frequency following the initiation of study drug dosing.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Valbenazine', 'description': 'Participants received valbenazine once daily for up to 96 weeks. The starting dose was 20 mg for participants \\<50 kg at baseline and 40 mg for participants ≥50 kg at baseline, and could be escalated in increments of 20 mg every 2 weeks to a maximum of 60 mg for participants \\<50 kg and 80 mg for participants ≥50 kg to achieve an optimal dose of valbenazine for each participant.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '6'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '6'}]}], 'dropWithdraws': [{'type': 'Study terminated by Sponsor', 'reasons': [{'groupId': 'FG000', 'numSubjects': '6'}]}]}], 'recruitmentDetails': 'Approximately 240 participants who completed the Phase 2 Study NBI-98854-TS2004 or were to be dosed for at least 16 weeks in Phase 2 Study NBI-98854-TS2005 were planned for this study. However, only 6 participants were enrolled and received study drug because the study was terminated early by the Sponsor.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'Valbenazine', 'description': 'Participants received valbenazine once daily for up to 96 weeks. The starting dose was 20 mg for participants \\<50 kg at baseline and 40 mg for participants ≥50 kg at baseline, and could be escalated in increments of 20 mg every 2 weeks to a maximum of 60 mg for participants \\<50 kg and 80 mg for participants ≥50 kg to achieve an optimal dose of valbenazine for each participant.'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '11.8', 'spread': '2.5', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '1', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '5', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Ethnicity (NIH/OMB)', 'classes': [{'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '2', 'groupId': 'BG000'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '4', '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': '0', 'groupId': 'BG000'}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Black or African American', 'measurements': [{'value': '1', 'groupId': 'BG000'}]}, {'title': 'White', 'measurements': [{'value': '5', 'groupId': 'BG000'}]}, {'title': 'More than one race', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}]}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2018-12-03', 'size': 25641972, 'label': 'Study Protocol and Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'Prot_SAP_000.pdf', 'typeAbbrev': 'Prot_SAP', 'uploadDate': '2022-01-26T18:49', 'hasProtocol': True}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 6}}, 'statusModule': {'whyStopped': 'Study stopped early due to investigational drug not meeting primary endpoint in the main study T-Force GOLD (NBI-98854-TS2003); no safety concerns identified.', 'overallStatus': 'TERMINATED', 'startDateStruct': {'date': '2018-10-17', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2022-03', 'dispFirstSubmitDate': '2020-01-28', 'completionDateStruct': {'date': '2019-02-18', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2022-03-25', 'studyFirstSubmitDate': '2018-11-05', 'dispFirstSubmitQcDate': '2022-03-25', 'resultsFirstSubmitDate': '2022-01-29', 'studyFirstSubmitQcDate': '2018-11-05', 'dispFirstPostDateStruct': {'date': '2022-04-19', 'type': 'ACTUAL'}, 'lastUpdatePostDateStruct': {'date': '2022-04-19', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2022-03-25', 'studyFirstPostDateStruct': {'date': '2018-11-06', 'type': 'ACTUAL'}, 'resultsFirstPostDateStruct': {'date': '2022-04-19', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2019-02-18', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of Participants With Treatment-Emergent Adverse Events (TEAEs)', 'timeFrame': 'Up to 16 Weeks', 'description': 'A TEAE is an adverse event not present prior to the initiation of study drug dosing, or is an already present event that worsens either in intensity or frequency following the initiation of study drug dosing.'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Tourette Syndrome']}, 'descriptionModule': {'briefSummary': 'This is an open-label, rollover study to collect long-term safety, tolerability, and investigator- and participant-reported pharmacodynamic (PD) data after chronic administration of NBI-98854 in pediatric participants with Tourette Syndrome (TS), as well as to provide open-label access to NBI-98854 for the treatment of TS for pediatric participants who have taken part in a Phase 2 NBI-98854 study.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '18 Years', 'minimumAge': '6 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n1. Have a clinical diagnosis of Tourette Syndrome (TS)\n2. Have participated in the NBI-98854-TS2004 or NBI 98854-TS2005 Phase 2 studies\n3. If using maintenance medication(s) for TS and/or TS spectrum diagnoses (e.g. obsessive-compulsive disorder \\[OCD\\], Attention-Deficit Hyperactivity Disorder \\[ADHD\\]), be on stable doses\n4. Be in good general health\n5. Subjects of childbearing potential who do not practice total abstinence must agree to use hormonal or two forms of nonhormonal contraception (dual contraception) consistently during the screening, treatment and follow-up periods of the study\n\nExclusion Criteria:\n\n1. Have an active, clinically significant unstable medical condition within 1 month before screening\n2. Have a known history of long QT syndrome or cardiac arrhythmia\n3. Have a known history of neuroleptic malignant syndrome\n4. Have a cancer diagnosis within 3 years prior to screening (some exceptions allowed)\n5. Have an allergy, hypersensitivity, or intolerance to VMAT2 inhibitors\n6. Have a blood loss ≥250 mL or donated blood within 56 days, or donated plasma within 7 days, before the start of the study\n7. Have a known history of substance dependence, substance (drug) or alcohol abuse within 3 months before the start of the study\n8. Have a significant risk of suicidal or violent behavior\n9. Have received an investigational drug within 30 days before the start of the study or plan to use an investigational drug (other than NBI-98854) during the study\n10. Are currently participating in another NBI-98854 clinical study\n11. Are pregnant (for females)'}, 'identificationModule': {'nctId': 'NCT03732534', 'briefTitle': 'Rollover Study for Continuing NBI-98854 Administration in Pediatric Subjects With Tourette Syndrome', 'organization': {'class': 'INDUSTRY', 'fullName': 'Neurocrine Biosciences'}, 'officialTitle': 'Open-Label Rollover Study for Continuing NBI-98854 Administration for the Treatment of Pediatric Subjects With Tourette Syndrome', 'orgStudyIdInfo': {'id': 'NBI-98854-TS2007'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'NBI-98854', 'description': 'NBI-98854 administered once daily for up to 96 weeks', 'interventionNames': ['Drug: NBI-98854']}], 'interventions': [{'name': 'NBI-98854', 'type': 'DRUG', 'otherNames': ['Valbenazine'], 'description': 'vesicular monoamine transporter 2 (VMAT2) inhibitor', 'armGroupLabels': ['NBI-98854']}]}, 'contactsLocationsModule': {'locations': [{'zip': '92805', 'city': 'Anaheim', 'state': 'California', 'country': 'United States', 'facility': 'Neurocrine Clinical Site', 'geoPoint': {'lat': 33.83529, 'lon': -117.9145}}, {'zip': '92108', 'city': 'San Diego', 'state': 'California', 'country': 'United States', 'facility': 'Neurocrine Clinical Site', 'geoPoint': {'lat': 32.71571, 'lon': -117.16472}}, {'zip': '32561', 'city': 'Gulf Breeze', 'state': 'Florida', 'country': 'United States', 'facility': 'Neurocrine Clinical Site', 'geoPoint': {'lat': 30.35714, 'lon': -87.16386}}, {'zip': '33013', 'city': 'Hialeah', 'state': 'Florida', 'country': 'United States', 'facility': 'Neurocrine Clinical Site', 'geoPoint': {'lat': 25.8576, 'lon': -80.27811}}, {'zip': '60634', 'city': 'Chicago', 'state': 'Illinois', 'country': 'United States', 'facility': 'Neurocrine Clinical Site', 'geoPoint': {'lat': 41.85003, 'lon': -87.65005}}, {'zip': '60563', 'city': 'Naperville', 'state': 'Illinois', 'country': 'United States', 'facility': 'Neurocrine Clinical Site', 'geoPoint': {'lat': 41.78586, 'lon': -88.14729}}, {'zip': '68526', 'city': 'Lincoln', 'state': 'Nebraska', 'country': 'United States', 'facility': 'Neurocrine Clinical Site', 'geoPoint': {'lat': 40.8, 'lon': -96.66696}}, {'zip': '75243', 'city': 'Dallas', 'state': 'Texas', 'country': 'United States', 'facility': 'Neurocrine Clinical Site', 'geoPoint': {'lat': 32.78306, 'lon': -96.80667}}, {'zip': '77058', 'city': 'Houston', 'state': 'Texas', 'country': 'United States', 'facility': 'Neurocrine Clinical Site', 'geoPoint': {'lat': 29.76328, 'lon': -95.36327}}, {'zip': '00926', 'city': 'San Juan', 'state': 'PR', 'country': 'Puerto Rico', 'facility': 'Neurocrine Clinical Site', 'geoPoint': {'lat': 18.46633, 'lon': -66.10572}}], 'overallOfficials': [{'name': 'Clinical Development Lead', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Neurocrine Biosciences'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Neurocrine Biosciences', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}