Ignite Creation Date:
2025-12-24 @ 6:52 PM
Ignite Modification Date:
2025-12-28 @ 12:38 PM
Study NCT ID:
NCT05236257
Status:
COMPLETED
Last Update Posted:
2024-12-10
First Post:
2022-02-02
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
A Study Called EPI VITRAKVI to Compare Treatment Results in Patients With Infantile Fibrosarcoma (IFS), a Type of Connective Soft Tissue Cancer, Who Received a Treatment Called Larotrectinib From a Study Called SCOUT With Patient Data From an External Database
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'interventionBrowseModule': {'meshes': [{'id': 'C000609083', 'term': 'larotrectinib'}, {'id': 'D059039', 'term': 'Standard of Care'}], 'ancestors': [{'id': 'D019984', 'term': 'Quality Indicators, Health Care'}, {'id': 'D011787', 'term': 'Quality of Health Care'}, {'id': 'D006298', 'term': 'Health Services Administration'}, {'id': 'D017530', 'term': 'Health Care Quality, Access, and Evaluation'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'clinical-trials-contact@bayer.com', 'phone': '(+) 1-888-8422937', 'title': 'Therapeutic Area Head', 'organization': 'Bayer'}, 'certainAgreement': {'otherDetails': 'The disclosure agreement is that the publication is restricted for external partners.', 'restrictionType': 'OTHER', 'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': 'up to 5.5 years for participants in SCOUT study and 22.5 years for participants in external historical control', 'description': 'As it was stated in the protocol of the study, this retrospective observational study used secondary data collection from a previous clinical trial (SCOUT study) and from eligible databases used to select external historical control cohorts. Therefore, no new AEs or adverse drug reactions occurred besides the ones already described during the conduct of the initial clinical trial.', 'eventGroups': [{'id': 'EG000', 'title': 'Larotrectinib', 'description': 'Pediatric patients (up to 21 years old) with Infantile fibrosarcoma (IFS) harboring an NTRK gene fusion who have been enrolled in the SCOUT study (Bayer Study ID: 20290; NCT02637687) and treated with larotrectinib.', 'otherNumAtRisk': 51, 'deathsNumAtRisk': 51, 'otherNumAffected': 0, 'seriousNumAtRisk': 51, 'deathsNumAffected': 1, 'seriousNumAffected': 1}, {'id': 'EG001', 'title': 'External Controls', 'description': 'External historical control patients treated with at least one chemotherapy-based regimen, pooled from the Institut Curie (CURIE) database (N=18) and the Cooperative Weichteilsarkom Studiengruppe (CWS) database (N=24).', 'otherNumAtRisk': 42, 'deathsNumAtRisk': 42, 'otherNumAffected': 0, 'seriousNumAtRisk': 42, 'deathsNumAffected': 3, 'seriousNumAffected': 3}], 'seriousEvents': [{'term': 'Death', 'stats': [{'groupId': 'EG000', 'numAtRisk': 51, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 42, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}], 'frequencyThreshold': '0'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Time to Medical Treatment Failure', 'denoms': [{'units': 'Participants', 'counts': [{'value': '51', 'groupId': 'OG000'}, {'value': '42', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Larotrectinib', 'description': 'Pediatric patients (up to 21 years old) with Infantile fibrosarcoma (IFS) harboring an NTRK gene fusion who have been enrolled in the SCOUT study (Bayer Study ID: 20290; NCT02637687) and treated with larotrectinib.'}, {'id': 'OG001', 'title': 'External Controls', 'description': 'External historical control patients treated with at least one chemotherapy-based regimen, pooled from the Institut Curie (CURIE) database (N=18) and the Cooperative Weichteilsarkom Studiengruppe (CWS) database (N=24).'}], 'classes': [{'title': 'Unweighted sample', 'categories': [{'measurements': [{'value': 'NA', 'comment': 'median time to medical treatment failure was not estimable due to censored data', 'groupId': 'OG000', 'lowerLimit': 'NA', 'upperLimit': 'NA'}, {'value': 'NA', 'comment': 'median time to medical treatment failure was not estimable due to censored data', 'groupId': 'OG001', 'lowerLimit': '57.1', 'upperLimit': 'NA'}]}]}, {'title': 'Weighted sample', 'categories': [{'measurements': [{'value': 'NA', 'comment': 'median time to medical treatment failure was not estimable due to censored data', 'groupId': 'OG000', 'lowerLimit': 'NA', 'upperLimit': 'NA'}, {'value': '24.0', 'comment': 'upper limit of median time to medical treatment failure was not estimable due to censored data', 'groupId': 'OG001', 'lowerLimit': '3.0', 'upperLimit': 'NA'}]}]}], 'analyses': [{'pValue': '0.0058', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Hazard Ratio (HR)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '0.21', 'ciLowerLimit': '0.07', 'ciUpperLimit': '0.63', 'statisticalMethod': 'Cox Proportional Hazards model', 'nonInferiorityType': 'OTHER', 'statisticalComment': 'Unweighted'}, {'pValue': '0.0023', 'groupIds': ['OG000', 'OG001'], 'statisticalMethod': 'Log Rank', 'nonInferiorityType': 'OTHER', 'statisticalComment': 'Unweighted'}], 'paramType': 'MEDIAN', 'timeFrame': 'up to 5.5 years for participants in SCOUT study and 22.5 years for participants in external historical control', 'description': 'Time to medical treatment failure was defined as the time (months) from the start of treatment to the date of the earliest event from: subsequent systemic treatment, radiation therapy, mutilating surgery or death due to any cause.', 'unitOfMeasure': 'months', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Time to Subsequent Systemic Treatment', 'denoms': [{'units': 'Participants', 'counts': [{'value': '51', 'groupId': 'OG000'}, {'value': '42', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Larotrectinib', 'description': 'Pediatric patients (up to 21 years old) with Infantile fibrosarcoma (IFS) harboring an NTRK gene fusion who have been enrolled in the SCOUT study (Bayer Study ID: 20290; NCT02637687) and treated with larotrectinib.'}, {'id': 'OG001', 'title': 'External Controls', 'description': 'External historical control patients treated with at least one chemotherapy-based regimen, pooled from the Institut Curie (CURIE) database (N=18) and the Cooperative Weichteilsarkom Studiengruppe (CWS) database (N=24).'}], 'classes': [{'title': 'Unweighted Sample', 'categories': [{'measurements': [{'value': 'NA', 'comment': 'median time to subsequent systemic treatment was not estimable due to censored data', 'groupId': 'OG000', 'lowerLimit': 'NA', 'upperLimit': 'NA'}, {'value': 'NA', 'comment': 'median time to subsequent systemic treatment was not estimable due to censored data', 'groupId': 'OG001', 'lowerLimit': '13.0', 'upperLimit': 'NA'}]}]}, {'title': 'weighted Sample', 'categories': [{'measurements': [{'value': 'NA', 'comment': 'median time to subsequent systemic treatment was not estimable due to censored data', 'groupId': 'OG000', 'lowerLimit': 'NA', 'upperLimit': 'NA'}, {'value': '24.0', 'comment': 'Upper limit of median time to subsequent systemic treatment was not estimable due to censored data', 'groupId': 'OG001', 'lowerLimit': '24.0', 'upperLimit': 'NA'}]}]}], 'analyses': [{'pValue': '0.0703', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Hazard Ratio (HR)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '0.23', 'ciLowerLimit': '0.05', 'ciUpperLimit': '1.13', 'statisticalMethod': 'Cox Proportional Hazards model', 'nonInferiorityType': 'OTHER', 'statisticalComment': 'Unweighted'}, {'pValue': '0.0486', 'groupIds': ['OG000', 'OG001'], 'statisticalMethod': 'Log Rank', 'nonInferiorityType': 'OTHER', 'statisticalComment': 'Unweighted'}], 'paramType': 'MEDIAN', 'timeFrame': 'up to 5.5 years for participants in SCOUT study and 22.5 years for participants in external historical control', 'description': 'Time to subsequent systemic treatment was defined as the time from start date of Larotrectinib (for SCOUT) or start date of chemotherapy (for historical control cohorts) till the start date of a post-treatment systemic anti-cancer therapy, if any', 'unitOfMeasure': 'months', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Time to Mutilating Surgery Including Limb Amputation', 'denoms': [{'units': 'Participants', 'counts': [{'value': '51', 'groupId': 'OG000'}, {'value': '42', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Larotrectinib', 'description': 'Pediatric patients (up to 21 years old) with Infantile fibrosarcoma (IFS) harboring an NTRK gene fusion who have been enrolled in the SCOUT study (Bayer Study ID: 20290; NCT02637687) and treated with larotrectinib.'}, {'id': 'OG001', 'title': 'External Controls', 'description': 'External historical control patients treated with at least one chemotherapy-based regimen, pooled from the Institut Curie (CURIE) database (N=18) and the Cooperative Weichteilsarkom Studiengruppe (CWS) database (N=24).'}], 'classes': [{'title': 'Unweighted Sample', 'categories': [{'measurements': [{'value': 'NA', 'comment': 'median time to mutilating surgery including limb amputation was not estimable due to censored data', 'groupId': 'OG000', 'lowerLimit': 'NA', 'upperLimit': 'NA'}, {'value': 'NA', 'comment': 'median time to mutilating surgery including limb amputation was not estimable due to censored data', 'groupId': 'OG001', 'lowerLimit': 'NA', 'upperLimit': 'NA'}]}]}, {'title': 'Weighted Sample', 'categories': [{'measurements': [{'value': 'NA', 'comment': 'median time to mutilating surgery including limb amputation was not estimable due to censored data', 'groupId': 'OG000', 'lowerLimit': 'NA', 'upperLimit': 'NA'}, {'value': 'NA', 'comment': 'median time to mutilating surgery including limb amputation was not estimable due to censored data', 'groupId': 'OG001', 'lowerLimit': 'NA', 'upperLimit': 'NA'}]}]}], 'analyses': [{'pValue': '0.696', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Hazard Ratio (HR)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '0.23', 'ciLowerLimit': '0.05', 'ciUpperLimit': '1.12', 'statisticalMethod': 'Cox Proportional Hazards model', 'nonInferiorityType': 'OTHER', 'statisticalComment': 'Unweighted'}, {'pValue': '0.0476', 'groupIds': ['OG000', 'OG001'], 'statisticalMethod': 'Log Rank', 'nonInferiorityType': 'OTHER', 'statisticalComment': 'Unweighted'}], 'paramType': 'MEDIAN', 'timeFrame': 'up to 5.5 years for participants in SCOUT study and 22.5 years for participants in external historical control', 'description': 'Time to mutilating surgery including limb amputation was defined as the time from start date of Larotrectinib (for SCOUT) or start date of chemotherapy (for historical control cohorts) till the start date of a mutilating surgery (including limb amputation)', 'unitOfMeasure': 'months', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Time to First Radiation Therapy', 'denoms': [{'units': 'Participants', 'counts': [{'value': '51', 'groupId': 'OG000'}, {'value': '42', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Larotrectinib', 'description': 'Pediatric patients (up to 21 years old) with Infantile fibrosarcoma (IFS) harboring an NTRK gene fusion who have been enrolled in the SCOUT study (Bayer Study ID: 20290; NCT02637687) and treated with larotrectinib.'}, {'id': 'OG001', 'title': 'External Controls', 'description': 'External historical control patients treated with at least one chemotherapy-based regimen, pooled from the Institut Curie (CURIE) database (N=18) and the Cooperative Weichteilsarkom Studiengruppe (CWS) database (N=24).'}], 'classes': [{'title': 'Unweighted Sample', 'categories': [{'measurements': [{'value': 'NA', 'comment': 'median time to first radiation therapy was not estimable due to censored data', 'groupId': 'OG000', 'lowerLimit': 'NA', 'upperLimit': 'NA'}, {'value': 'NA', 'comment': 'median time to first radiation therapy was not estimable due to censored data', 'groupId': 'OG001', 'lowerLimit': 'NA', 'upperLimit': 'NA'}]}]}, {'title': 'Weighted Sample', 'categories': [{'measurements': [{'value': 'NA', 'comment': 'median time to first radiation therapy was not estimable due to censored data', 'groupId': 'OG000', 'lowerLimit': 'NA', 'upperLimit': 'NA'}, {'value': 'NA', 'comment': 'median time to first radiation therapy was not estimable due to censored data', 'groupId': 'OG001', 'lowerLimit': 'NA', 'upperLimit': 'NA'}]}]}], 'paramType': 'MEDIAN', 'timeFrame': 'up to 5.5 years for participants in SCOUT study and 22.5 years for participants in external historical control', 'description': 'Time to radiation therapy was defined as the time from start date of Larotrectinib (for SCOUT) or start date of chemotherapy(for historical control cohorts) till the start date of a radiation therapy, if any', 'unitOfMeasure': 'months', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Time to Complete Surgical Resection', 'denoms': [{'units': 'Participants', 'counts': [{'value': '51', 'groupId': 'OG000'}, {'value': '42', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Larotrectinib', 'description': 'Pediatric patients (up to 21 years old) with Infantile fibrosarcoma (IFS) harboring an NTRK gene fusion who have been enrolled in the SCOUT study (Bayer Study ID: 20290; NCT02637687) and treated with larotrectinib.'}, {'id': 'OG001', 'title': 'External Controls', 'description': 'External historical control patients treated with at least one chemotherapy-based regimen, pooled from the Institut Curie (CURIE) database (N=18) and the Cooperative Weichteilsarkom Studiengruppe (CWS) database (N=24).'}], 'classes': [{'title': 'Unweighted Sample', 'categories': [{'measurements': [{'value': 'NA', 'comment': 'median time to complete surgical resection was not estimable due to censored data', 'groupId': 'OG000', 'lowerLimit': 'NA', 'upperLimit': 'NA'}, {'value': 'NA', 'comment': 'median time to complete surgical resection was not estimable due to censored data', 'groupId': 'OG001', 'lowerLimit': 'NA', 'upperLimit': 'NA'}]}]}, {'title': 'Weighted Sample', 'categories': [{'measurements': [{'value': 'NA', 'comment': 'median time to complete surgical resection was not estimable due to censored data', 'groupId': 'OG000', 'lowerLimit': 'NA', 'upperLimit': 'NA'}, {'value': '6.1', 'comment': 'upper limit of median time to complete surgical resection was not estimable due to censored data', 'groupId': 'OG001', 'lowerLimit': '5.1', 'upperLimit': 'NA'}]}]}], 'analyses': [{'pValue': '0.5713', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Hazard Ratio (HR)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '0.79', 'ciLowerLimit': '0.36', 'ciUpperLimit': '1.77', 'statisticalMethod': 'Cox Proportional Hazards model', 'nonInferiorityType': 'OTHER', 'statisticalComment': 'Unweighted'}, {'pValue': '0.5695', 'groupIds': ['OG000', 'OG001'], 'statisticalMethod': 'Log Rank', 'nonInferiorityType': 'OTHER', 'statisticalComment': 'Unweighted'}], 'paramType': 'MEDIAN', 'timeFrame': 'up to 5.5 years for participants in SCOUT study and 22.5 years for participants in external historical control', 'description': 'Time to complete surgical resection (excluding amputation) was defined as the time from start date of Larotrectinib (for SCOUT) or start date of chemotherapy (for historical control cohorts) till the start date of a complete surgical resection (excluding amputation), if any', 'unitOfMeasure': 'months', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Overall Survival', 'denoms': [{'units': 'Participants', 'counts': [{'value': '51', 'groupId': 'OG000'}, {'value': '42', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Larotrectinib', 'description': 'Pediatric patients (up to 21 years old) with Infantile fibrosarcoma (IFS) harboring an NTRK gene fusion who have been enrolled in the SCOUT study (Bayer Study ID: 20290; NCT02637687) and treated with larotrectinib.'}, {'id': 'OG001', 'title': 'External Controls', 'description': 'External historical control patients treated with at least one chemotherapy-based regimen, pooled from the Institut Curie (CURIE) database (N=18) and the Cooperative Weichteilsarkom Studiengruppe (CWS) database (N=24).'}], 'classes': [{'title': 'Dead', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}]}, {'title': 'Alive', 'categories': [{'measurements': [{'value': '50', 'groupId': 'OG000'}, {'value': '39', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.3200', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Hazard Ratio (HR)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '0.32', 'ciLowerLimit': '0.03', 'ciUpperLimit': '3.06', 'statisticalMethod': 'Cox Proportional Hazards model', 'nonInferiorityType': 'OTHER', 'statisticalComment': 'Unweighted'}, {'pValue': '0.2940', 'groupIds': ['OG000', 'OG001'], 'statisticalMethod': 'Log Rank', 'nonInferiorityType': 'OTHER', 'statisticalComment': 'Unweighted'}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'up to 5.5 years for participants in SCOUT study and 22.5 years for participants in external historical control', 'description': 'Number of participants with death due to any cause, alive, or lost to follow-up.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Number of Participants With Treatment Discontinuation Due to Treatment Emergent Adverse Events', 'denoms': [{'units': 'Participants', 'counts': [{'value': '51', 'groupId': 'OG000'}, {'value': '42', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Larotrectinib', 'description': 'Pediatric patients (up to 21 years old) with Infantile fibrosarcoma (IFS) harboring an NTRK gene fusion who have been enrolled in the SCOUT study (Bayer Study ID: 20290; NCT02637687) and treated with larotrectinib.'}, {'id': 'OG001', 'title': 'External Controls', 'description': 'External historical control patients treated with at least one chemotherapy-based regimen, pooled from the Institut Curie (CURIE) database (N=18) and the Cooperative Weichteilsarkom Studiengruppe (CWS) database (N=24).'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'up to 5.5 years for participants in SCOUT study and 22.5 years for participants in external historical control', 'description': 'Larotrectinib treatment for the SCOUT study and chemotherapy (first line) for the external historical control cohort(s).', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Larotrectinib', 'description': 'Pediatric patients (up to 21 years old) with Infantile fibrosarcoma (IFS) harboring an NTRK gene fusion who have been enrolled in the SCOUT study (Bayer Study ID: 20290; NCT02637687) and treated with larotrectinib.'}, {'id': 'FG001', 'title': 'External Controls', 'description': 'External historical control patients treated with at least one chemotherapy-based regimen, pooled from the Institut Curie (CURIE) database (N=18) and the Cooperative Weichteilsarkom Studiengruppe (CWS) database (N=24).'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '51'}, {'groupId': 'FG001', 'numSubjects': '42'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '51'}, {'groupId': 'FG001', 'numSubjects': '42'}]}], 'dropWithdraws': [{'type': 'Ongoing with study treatment', 'reasons': [{'groupId': 'FG000', 'numSubjects': '29'}, {'groupId': 'FG001', 'numSubjects': '1'}]}, {'type': 'Discontinued study treatment', 'reasons': [{'groupId': 'FG000', 'numSubjects': '22'}, {'groupId': 'FG001', 'numSubjects': '41'}]}]}], 'recruitmentDetails': 'Study was conducted between 10-Mar-2022 (start retrospective observational study) and 13-Sep-2022 (table listing filles final) using secondary data.\n\nThree data sources were used in the study including SCOUT study (18 countries worldwide including France) and external historical control cohorts from the Institut Curie database and the Cooperative Weichteilsarkom Studiengruppe (CWS) database.', 'preAssignmentDetails': 'In total, 93 patients were included in this study (IFS population). The larotrectinib arm of the study included 51 patients from the SCOUT study. The single comparator arm of patients that received conventional chemotherapy included in total 42 external control patients, pooled from the Institut Curie database (N=18) and the CWS database (N=24).'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '51', 'groupId': 'BG000'}, {'value': '42', 'groupId': 'BG001'}, {'value': '93', 'groupId': 'BG002'}]}], 'groups': [{'id': 'BG000', 'title': 'Larotrectinib', 'description': 'Pediatric patients (up to 21 years old) with Infantile fibrosarcoma (IFS) harboring an NTRK gene fusion who have been enrolled in the SCOUT study (Bayer Study ID: 20290; NCT02637687) and treated with larotrectinib.'}, {'id': 'BG001', 'title': 'External Controls', 'description': 'External historical control patients treated with at least one chemotherapy-based regimen, pooled from the Institut Curie (CURIE) database (N=18) and the Cooperative Weichteilsarkom Studiengruppe (CWS) database (N=24).'}, {'id': 'BG002', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '2.01', 'spread': '3.49', 'groupId': 'BG000'}, {'value': '0.73', 'spread': '1.81', 'groupId': 'BG001'}, {'value': '1.43', 'spread': '2.91', 'groupId': 'BG002'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '21', 'groupId': 'BG000'}, {'value': '15', 'groupId': 'BG001'}, {'value': '36', 'groupId': 'BG002'}]}, {'title': 'Male', 'measurements': [{'value': '30', 'groupId': 'BG000'}, {'value': '27', 'groupId': 'BG001'}, {'value': '57', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race (NIH/OMB)', 'classes': [{'categories': [{'title': 'American Indian or Alaska Native', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Asian', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Black or African American', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'White', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'More than one race', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '51', 'groupId': 'BG000'}, {'value': '42', 'groupId': 'BG001'}, {'value': '93', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}]}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2021-07-27', 'size': 1117321, 'label': 'Study Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_000.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2023-09-11T02:56', 'hasProtocol': True}, {'date': '2022-07-28', 'size': 1117350, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_001.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2023-09-10T22:28', 'hasProtocol': False}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'RETROSPECTIVE', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 93}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2022-03-10', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2024-12', 'completionDateStruct': {'date': '2022-09-13', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2024-12-08', 'studyFirstSubmitDate': '2022-02-02', 'resultsFirstSubmitDate': '2023-09-11', 'studyFirstSubmitQcDate': '2022-02-02', 'lastUpdatePostDateStruct': {'date': '2024-12-10', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2024-12-08', 'studyFirstPostDateStruct': {'date': '2022-02-11', 'type': 'ACTUAL'}, 'resultsFirstPostDateStruct': {'date': '2024-12-10', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2022-09-13', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Time to Medical Treatment Failure', 'timeFrame': 'up to 5.5 years for participants in SCOUT study and 22.5 years for participants in external historical control', 'description': 'Time to medical treatment failure was defined as the time (months) from the start of treatment to the date of the earliest event from: subsequent systemic treatment, radiation therapy, mutilating surgery or death due to any cause.'}], 'secondaryOutcomes': [{'measure': 'Time to Subsequent Systemic Treatment', 'timeFrame': 'up to 5.5 years for participants in SCOUT study and 22.5 years for participants in external historical control', 'description': 'Time to subsequent systemic treatment was defined as the time from start date of Larotrectinib (for SCOUT) or start date of chemotherapy (for historical control cohorts) till the start date of a post-treatment systemic anti-cancer therapy, if any'}, {'measure': 'Time to Mutilating Surgery Including Limb Amputation', 'timeFrame': 'up to 5.5 years for participants in SCOUT study and 22.5 years for participants in external historical control', 'description': 'Time to mutilating surgery including limb amputation was defined as the time from start date of Larotrectinib (for SCOUT) or start date of chemotherapy (for historical control cohorts) till the start date of a mutilating surgery (including limb amputation)'}, {'measure': 'Time to First Radiation Therapy', 'timeFrame': 'up to 5.5 years for participants in SCOUT study and 22.5 years for participants in external historical control', 'description': 'Time to radiation therapy was defined as the time from start date of Larotrectinib (for SCOUT) or start date of chemotherapy(for historical control cohorts) till the start date of a radiation therapy, if any'}, {'measure': 'Time to Complete Surgical Resection', 'timeFrame': 'up to 5.5 years for participants in SCOUT study and 22.5 years for participants in external historical control', 'description': 'Time to complete surgical resection (excluding amputation) was defined as the time from start date of Larotrectinib (for SCOUT) or start date of chemotherapy (for historical control cohorts) till the start date of a complete surgical resection (excluding amputation), if any'}, {'measure': 'Overall Survival', 'timeFrame': 'up to 5.5 years for participants in SCOUT study and 22.5 years for participants in external historical control', 'description': 'Number of participants with death due to any cause, alive, or lost to follow-up.'}, {'measure': 'Number of Participants With Treatment Discontinuation Due to Treatment Emergent Adverse Events', 'timeFrame': 'up to 5.5 years for participants in SCOUT study and 22.5 years for participants in external historical control', 'description': 'Larotrectinib treatment for the SCOUT study and chemotherapy (first line) for the external historical control cohort(s).'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Advanced solid tumors', 'Infantile fibrosarcoma (IFS)', 'Neurotrophic tyrosine receptor kinase (NTRK)', 'Fusion Positive', 'Tropomyosin Receptor Kinase (TRK) fusion', 'Larotrectinib', 'External control'], 'conditions': ['Locally Advanced or Metastatic Infantile Fibrosarcoma Harboring an NTRK Gene Fusion', 'Infantile Fibrosarcoma']}, 'referencesModule': {'references': [{'pmid': '37133249', 'type': 'DERIVED', 'citation': 'Carton M, Del Castillo JP, Colin JB, Kurtinecz M, Feuilly M, Pierron G, Arvis P, Khadir SK, Sparber-Sauer M, Orbach D. Larotrectinib versus historical standard of care in patients with infantile fibrosarcoma: protocol of EPI-VITRAKVI. Future Oncol. 2023 Aug;19(24):1645-1653. doi: 10.2217/fon-2023-0114. Epub 2023 May 3.'}], 'seeAlsoLinks': [{'url': 'https://filehosting-v2.pharmacm.com/api/Attachment/Download?tenantId=80217261&parentIdentifier=21767&attachmentIdentifier=ecffefac-d54c-4afb-9c04-46093c1d3330&fileName=21767_Patient_Information_Sheet_2.0.pdf&versionIdentifier=', 'label': 'VITRAKVI Patient Information'}, {'url': 'http://clinicaltrials.bayer.com', 'label': 'Click here to find information for studies related to Bayer products. To find this study enter the NCT number or Bayer Study ID in the search field.'}]}, 'descriptionModule': {'briefSummary': 'This is an observational study in which data from the past of children and young people with a specific cancer, called NTRK gene fusion positive infantile fibrosarcoma (IFS) is studied.\n\nIFS is a rare type of childhood cancer that commonly affects legs and arms. IFS cancers typically have specific changes in their building plans (genes) called NTRK gene fusion. NTRK stands for the specific gene that has been altered, the neurotrophic tyrosine kinase (NTRK) gene.\n\nThis change to the building plan leads to the creation of an altered protein known as a TRK fusion protein, which can cause cancer cells to grow and to survive. The specific cancer is therefore also called TRK (tropomyosin receptor kinase) fusion-positive IFS.\n\nThe study drug, larotrectinib (also called BAY2757556) works by blocking the altered TRK fusion protein. Larotrectinib is already available in Europe and in many other countries and is approved for doctors to prescribe to patients with NTRK gene fusion cancer which has spread to nearby tissues and/or lymph nodes or to other parts of the body.\n\nIn France, HAS (the French authority in charge of evaluating health products and technologies) gave a positive opinion for the reimbursement of larotrectinib but only in the pediatric patients with IFS or another STS harboring a NTRK gene fusion, which is locally advanced or metastatic, and refractory or in relapse mainly due to the lack of comparative evidence.\n\nThe main purpose of this study is to collect more data to learn how well larotrectinib works compared with current standard of care chemotherapy in people up to 21 years of age with NTRK gene fusion positive IFS that has spread to nearby tissues and/or lymph nodes (locally advanced) or other parts of the body (metastatic).\n\nTo see how well larotrectinib works, researchers will make a comparison between\n\n* how long larotrectinib works well and\n* how long the standard of care works well.\n\nWorking well means that the treatments can prevent the following from happening:\n\n* need for a new treatment for the cancer\n* need for radiation therapy for the cancer\n* need for surgery to treat the cancer, but which causes major damage to body parts\n* death.\n\nIn addition to the above, data about medical problems related to the treatments in both groups and that may have required to stop the treatment will be compared.\n\nThe data for the comparison will come from\n\n* an ongoing international study called SCOUT which was started in December 2015 (larotrectinib group)\n* international databases (standard of care chemotherapy group). Data will be from the year 2000 up to the present.\n\nThere will be no required visits with a study doctor or required tests in this study.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '21 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'The study population comprises of all pediatric patients in the SCOUT study and the eligible historical cohort(s) with a diagnosis of locally advanced or metastatic IFS harboring an NTRK gene fusion.', 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Age ≤ 21 years old.\n* Locally advanced or metastatic Infantile Fibrosarcoma (IFS).\n* Identification of an NTRK gene fusion by a molecular biology assay.\n* Patients with available information on clinical, radiological characteristics of their tumor, therapies administered and outcomes.\n* Patients receiving larotrectinib in the SCOUT trial.\n* Patients receiving at least chemotherapy drugs in the historical control cohort(s).\n* No opposition from the patients and/or representatives for data use.\n\nExclusion Criteria:\n\n* Patients treated with TRK inhibitors in the historical control cohort(s).\n* Patients with documented absence of NTRK gene fusion.\n* Patients participating in an investigational program with interventions outside of routine clinical practice.'}, 'identificationModule': {'nctId': 'NCT05236257', 'acronym': 'EPI VITRAKVI', 'briefTitle': 'A Study Called EPI VITRAKVI to Compare Treatment Results in Patients With Infantile Fibrosarcoma (IFS), a Type of Connective Soft Tissue Cancer, Who Received a Treatment Called Larotrectinib From a Study Called SCOUT With Patient Data From an External Database', 'organization': {'class': 'INDUSTRY', 'fullName': 'Bayer'}, 'officialTitle': 'A Comparison of Clinical Outcomes in Infantile Fibrosarcoma (IFS) Patients Treated With Larotrectinib in the Phase I/II SCOUT Study Versus (an) External Historical Cohort(s)', 'orgStudyIdInfo': {'id': '21767'}}, 'armsInterventionsModule': {'armGroups': [{'label': 'Larotrectinib', 'description': 'Pediatric patients with IFS harboring an NTRK gene fusion who have been enrolled in the SCOUT study.', 'interventionNames': ['Drug: Larotrectinib (Vitrakvi, BAY2757556)']}, {'label': 'Standard care', 'description': 'Pediatric patients with IFS harboring an NTRK gene fusion in the eligible external cohort(s).', 'interventionNames': ['Drug: Standard of Care']}], 'interventions': [{'name': 'Larotrectinib (Vitrakvi, BAY2757556)', 'type': 'DRUG', 'description': 'Pediatric patients with IFS harboring an NTRK gene fusion.', 'armGroupLabels': ['Larotrectinib']}, {'name': 'Standard of Care', 'type': 'DRUG', 'description': 'Standard of care for the patients from the eligible external cohorts.', 'armGroupLabels': ['Standard care']}]}, 'contactsLocationsModule': {'locations': [{'city': 'Multiple Locations', 'country': 'France', 'facility': 'Multiple Locations'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO', 'description': 'Availability of this study\'s data will later be determined according to Bayer\'s commitment to the EFPIA/PhRMA "Principles for responsible clinical trial data sharing". This pertains to scope, timepoint and process of data access. As such, Bayer commits to sharing upon request from qualified researchers patient-level clinical trial data, study-level clinical trial data, and protocols from clinical trials in patients for medicines and indications approved in the US and EU as necessary for conducting legitimate research. This applies to data on new medicines and indications that have been approved by the EU and US regulatory agencies on or after January 01, 2014.\n\nInterested researchers can use www.vivli.org to request access to anonymized patient-level data and supporting documents from clinical studies to conduct research. Information on the Bayer criteria for listing studies and other relevant information is provided in the member section of the portal.'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Bayer', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}