Ignite Creation Date:
2025-12-24 @ 7:56 PM
Ignite Modification Date:
2025-12-25 @ 5:31 PM
Study NCT ID:
NCT00928304
Status:
COMPLETED
Last Update Posted:
2014-03-05
First Post:
2009-06-24
Is NOT Gene Therapy:
False
Has Adverse Events:
True
Brief Title:
Phase II Study of Florbetaben (BAY94-9172) PET Imaging for Detection/Exclusion of Cerebral β-amyloid.
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D004314', 'term': 'Down Syndrome'}, {'id': 'D058225', 'term': 'Plaque, Amyloid'}], 'ancestors': [{'id': 'D008607', 'term': 'Intellectual Disability'}, {'id': 'D019954', 'term': 'Neurobehavioral Manifestations'}, {'id': 'D009461', 'term': 'Neurologic Manifestations'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D000015', 'term': 'Abnormalities, Multiple'}, {'id': 'D000013', 'term': 'Congenital Abnormalities'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D025063', 'term': 'Chromosome Disorders'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D020763', 'term': 'Pathological Conditions, Anatomical'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C527756', 'term': "4-(N-methylamino)-4'-(2-(2-(2-fluoroethoxy)ethoxy)ethoxy)stilbene"}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'juergen.hirschfeld@piramal.com', 'phone': '49 30 461 1246 15', 'title': 'Dr. Juergen Hirschfeld', 'organization': 'Piramal Imaging'}, 'certainAgreement': {'restrictionType': 'LTE60', 'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': '8 days', 'description': 'Adverse event data were collected from Day 1 (baseline) until Day 8 (secondary follow-up visit)', 'eventGroups': [{'id': 'EG000', 'title': 'Down Syndrome Group', 'description': '39 Down Syndrome subjects received Florbetaben (BAY94-9172): 300 MBq single IV injection of 2 to 10 mL', 'otherNumAtRisk': 39, 'otherNumAffected': 2, 'seriousNumAtRisk': 39, 'seriousNumAffected': 0}, {'id': 'EG001', 'title': 'Healthy Volunteer Group', 'description': '70 healthy volunteer subjects received Florbetaben (BAY94-9172): 300 MBq single IV injection of 2 to 10 mL', 'otherNumAtRisk': 70, 'otherNumAffected': 5, 'seriousNumAtRisk': 70, 'seriousNumAffected': 0}], 'otherEvents': [{'term': 'Injection site hematoma', 'stats': [{'groupId': 'EG000', 'numAtRisk': 39, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 70, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.1)'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 39, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 70, 'numEvents': 5, 'numAffected': 5}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (13.1)'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Sensitivity of the Independent Visual Assessment of Detecting Cerebral Amyloid-beta in Individuals With Down Syndrome and Specificity in Subjects Without Down Syndrome', 'denoms': [{'units': 'Participants', 'counts': [{'value': '109', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Majority Read', 'description': 'Majority read of the visual assessment made by 3 independent readers of PET images from all subjects.'}], 'classes': [{'title': 'Sensitivity', 'categories': [{'measurements': [{'value': '46.15', 'groupId': 'OG000', 'lowerLimit': '30.51', 'upperLimit': '61.80'}]}]}, {'title': 'Specificity', 'categories': [{'measurements': [{'value': '100.00', 'groupId': 'OG000', 'lowerLimit': '100.00', 'upperLimit': '100.00'}]}]}], 'paramType': 'NUMBER', 'timeFrame': '100-120 min', 'description': 'The primary variables are sensitivity (percentage of DS subjects positive for cerebral beta-amyloid) and specificity (percentage of healthy volunteers negative for cerebral beta-amyloid) of brain uptake of florbetaben based on the majority read of the visual assessment by three independent blinded readers of PET images obtained 100 to 120 minutes post-injection of florbetaben.', 'unitOfMeasure': 'percentage of subjects', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'All subjects in the down syndrome population and healthy volunteers were included in this analysis.'}, {'type': 'SECONDARY', 'title': 'Sensitivity Results in the Down Syndrome Age Subgroups', 'denoms': [{'units': 'Participants', 'counts': [{'value': '21', 'groupId': 'OG000'}, {'value': '18', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Majority Read (DS-Young)', 'description': 'Majority Read results for Down Syndrome subjects with age \\<= 46 yrs'}, {'id': 'OG001', 'title': 'Majority Read (DS-Old)', 'description': 'Majority Read results for Down Syndrome subjects with age \\>46 yrs'}], 'classes': [{'categories': [{'measurements': [{'value': '23.81', 'groupId': 'OG000', 'lowerLimit': '5.59', 'upperLimit': '42.03'}, {'value': '72.22', 'groupId': 'OG001', 'lowerLimit': '51.53', 'upperLimit': '92.91'}]}]}], 'paramType': 'NUMBER', 'timeFrame': '100 - 120 min', 'description': 'The majority read sensitivity (percentage of DS subjects positive for cerebral beta-amyloid by majority read) was computed for the group of DS subjects with age equal to or below the median age (DS-young) and for DS subjects with age above the median age (DS-old). The median age was 46 yrs, with 3 subjects being exactly 46 yrs old. As defined, these subjects were assigned to the DS-young group.', 'unitOfMeasure': 'percentage of subjects', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'All Down Syndrome subjects (n=39) were analyzed for this outcome.'}, {'type': 'SECONDARY', 'title': 'Quantitative Parameters Standard Uptake Value Ratio', 'denoms': [{'units': 'Participants', 'counts': [{'value': '39', 'groupId': 'OG000'}, {'value': '70', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Down Syndrome Group', 'description': 'Subjects with Down Syndrome enrolled in the study'}, {'id': 'OG001', 'title': 'Healthy Volunteer Group', 'description': 'Healthy volunteers enrolled in the study'}], 'classes': [{'title': 'frontal cortex', 'categories': [{'measurements': [{'value': '1.396', 'spread': '0.2488', 'groupId': 'OG000'}, {'value': '1.146', 'spread': '0.0749', 'groupId': 'OG001'}]}]}, {'title': 'posterior cingulate', 'categories': [{'measurements': [{'value': '1.525', 'spread': '0.2549', 'groupId': 'OG000'}, {'value': '1.329', 'spread': '0.1118', 'groupId': 'OG001'}]}]}, {'title': 'lateral temporal cortex', 'categories': [{'measurements': [{'value': '1.350', 'spread': '0.2265', 'groupId': 'OG000'}, {'value': '1.132', 'spread': '0.0623', 'groupId': 'OG001'}]}]}, {'title': 'parietal cortex', 'categories': [{'measurements': [{'value': '1.391', 'spread': '0.2594', 'groupId': 'OG000'}, {'value': '1.138', 'spread': '0.0742', 'groupId': 'OG001'}]}]}, {'title': 'cerebellum, white matter', 'categories': [{'measurements': [{'value': '1.730', 'spread': '0.2115', 'groupId': 'OG000'}, {'value': '1.769', 'spread': '0.1302', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': '100 - 120 min p.i.', 'description': 'The Standard Uptake Value Ratios for florbetaben signal in the frontal cortex, posterior cingulate, lateral temporal cortex, parietal cortex, and cerebellum (white matter) were determined as a quantitative measure of tracer uptake. The SUV is defined as the ratio of (1) the tissue radioactivity concentration c (in MBq/kg) at time point t, and (2) the injected activity (in MBq, extrapolated to the same time t) divided by the body weight (in kg). These SUV numbers from regions of interest were then used to derive SUV ratios (SUVR) using the SUV from the cerebellar cortex as reference.', 'unitOfMeasure': 'SUVR', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'All Down Syndrome subjects and healthy volunteers enrolled in the study were included in this analysis'}, {'type': 'SECONDARY', 'title': 'Consistency Between Visual and Quantitative Efficacy', 'denoms': [{'units': 'Participants', 'counts': [{'value': '18', 'groupId': 'OG000'}, {'value': '21', 'groupId': 'OG001'}, {'value': '70', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'Down Syndrome PET-positive (Majority Read)', 'description': 'Down Syndrome subjects positive for cerebral beta-amyloid as determined by majority read'}, {'id': 'OG001', 'title': 'Down Syndrome PET-negative (Majority Read)', 'description': 'Down Syndrome subjects negative for cerebral beta-amyloid as determined by majority read'}, {'id': 'OG002', 'title': 'Healthy Volunteer Group', 'description': 'All healthy volunteers enrolled in the study'}], 'classes': [{'title': 'frontal cortex', 'categories': [{'measurements': [{'value': '1.566', 'spread': '0.2372', 'groupId': 'OG000'}, {'value': '1.249', 'spread': '0.1451', 'groupId': 'OG001'}, {'value': '1.146', 'spread': '0.0749', 'groupId': 'OG002'}]}]}, {'title': 'posterior cingulate', 'categories': [{'measurements': [{'value': '1.626', 'spread': '0.2372', 'groupId': 'OG000'}, {'value': '1.438', 'spread': '0.2421', 'groupId': 'OG001'}, {'value': '1.329', 'spread': '0.1118', 'groupId': 'OG002'}]}]}, {'title': 'lateral temporal cortex', 'categories': [{'measurements': [{'value': '1.498', 'spread': '0.2396', 'groupId': 'OG000'}, {'value': '1.223', 'spread': '0.1092', 'groupId': 'OG001'}, {'value': '1.132', 'spread': '0.0623', 'groupId': 'OG002'}]}]}, {'title': 'parietal cortex', 'categories': [{'measurements': [{'value': '1.557', 'spread': '0.2567', 'groupId': 'OG000'}, {'value': '1.250', 'spread': '0.1620', 'groupId': 'OG001'}, {'value': '1.138', 'spread': '0.0742', 'groupId': 'OG002'}]}]}, {'title': 'cerebellum, white matter', 'categories': [{'measurements': [{'value': '1.698', 'spread': '0.2396', 'groupId': 'OG000'}, {'value': '1.758', 'spread': '0.1856', 'groupId': 'OG001'}, {'value': '1.769', 'spread': '0.1302', 'groupId': 'OG002'}]}]}], 'paramType': 'MEAN', 'timeFrame': '100 - 120 min', 'description': 'For a comparison of the results of the visual assessment with the quantitative assessment, descriptive Standardized Uptake Value Ratio (SUVR) statistics were computed separately for DS subjects with an abnormal/normal majority read of the PET scan (DS-PET+/DS-PET-). The SUV is defined as the ratio of (1) the tissue radioactivity concentration c (in MBq/kg) at time point t, and (2) the injected activity (in MBq, extrapolated to the same time t) divided by the body weight (in kg). These SUV numbers from regions of interest were then used to derive SUV ratios (SUVR) using the SUV from the cerebellar cortex as reference.', 'unitOfMeasure': 'SUVR', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Down Syndrome Group', 'description': '39 Down Syndrome subjects received Florbetaben (BAY94-9172) : 300 megabecquerels (MBq) single IV injection of 2 to 10 mL'}, {'id': 'FG001', 'title': 'Healthy Volunteer Group', 'description': '70 healthy volunteer subjects received Florbetaben (BAY94-9172): 300 MBq single IV injection of 2 to 10 mL'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '39'}, {'groupId': 'FG001', 'numSubjects': '70'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '39'}, {'groupId': 'FG001', 'numSubjects': '70'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}]}]}], 'recruitmentDetails': 'The study was conducted at two sites in the US.', 'preAssignmentDetails': 'A total of 130 subjects were screened. 21 subjects were considered as screen failures and were not enrolled into the study. 9 subjects did not meet the inclusion/exclusion criteria. 10 subjects withdrew their consent. 1 subject was lost to follow-up and 1 subjected exceeded the weight limit for the camera.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '109', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'Subjects Enrolled in Study', 'description': 'All Down Syndrome and healthy volunteer subjects'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '109', 'groupId': 'BG000'}]}, {'title': '>=65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Age, Continuous', 'classes': [{'title': 'Down Syndrome Group', 'categories': [{'measurements': [{'value': '46.3', 'spread': '4.7', 'groupId': 'BG000'}]}]}, {'title': 'Healthy Volunteer Group', 'categories': [{'measurements': [{'value': '27.7', 'spread': '5.1', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '60', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '49', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'United States', 'categories': [{'measurements': [{'value': '109', 'groupId': 'BG000'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}], 'populationDescription': 'All Down Syndrome subjects and healthy volunteers enrolled into the study were included in the Baseline Analysis Population'}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'DIAGNOSTIC', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 109}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2009-06'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2014-01', 'dispFirstSubmitDate': '2013-05-09', 'completionDateStruct': {'date': '2011-01', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2014-01-29', 'studyFirstSubmitDate': '2009-06-24', 'dispFirstSubmitQcDate': '2013-05-09', 'resultsFirstSubmitDate': '2013-12-09', 'studyFirstSubmitQcDate': '2009-06-24', 'dispFirstPostDateStruct': {'date': '2013-05-16', 'type': 'ESTIMATED'}, 'lastUpdatePostDateStruct': {'date': '2014-03-05', 'type': 'ESTIMATED'}, 'resultsFirstSubmitQcDate': '2013-12-09', 'studyFirstPostDateStruct': {'date': '2009-06-25', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2014-01-28', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2011-01', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Sensitivity of the Independent Visual Assessment of Detecting Cerebral Amyloid-beta in Individuals With Down Syndrome and Specificity in Subjects Without Down Syndrome', 'timeFrame': '100-120 min', 'description': 'The primary variables are sensitivity (percentage of DS subjects positive for cerebral beta-amyloid) and specificity (percentage of healthy volunteers negative for cerebral beta-amyloid) of brain uptake of florbetaben based on the majority read of the visual assessment by three independent blinded readers of PET images obtained 100 to 120 minutes post-injection of florbetaben.'}], 'secondaryOutcomes': [{'measure': 'Sensitivity Results in the Down Syndrome Age Subgroups', 'timeFrame': '100 - 120 min', 'description': 'The majority read sensitivity (percentage of DS subjects positive for cerebral beta-amyloid by majority read) was computed for the group of DS subjects with age equal to or below the median age (DS-young) and for DS subjects with age above the median age (DS-old). The median age was 46 yrs, with 3 subjects being exactly 46 yrs old. As defined, these subjects were assigned to the DS-young group.'}, {'measure': 'Quantitative Parameters Standard Uptake Value Ratio', 'timeFrame': '100 - 120 min p.i.', 'description': 'The Standard Uptake Value Ratios for florbetaben signal in the frontal cortex, posterior cingulate, lateral temporal cortex, parietal cortex, and cerebellum (white matter) were determined as a quantitative measure of tracer uptake. The SUV is defined as the ratio of (1) the tissue radioactivity concentration c (in MBq/kg) at time point t, and (2) the injected activity (in MBq, extrapolated to the same time t) divided by the body weight (in kg). These SUV numbers from regions of interest were then used to derive SUV ratios (SUVR) using the SUV from the cerebellar cortex as reference.'}, {'measure': 'Consistency Between Visual and Quantitative Efficacy', 'timeFrame': '100 - 120 min', 'description': 'For a comparison of the results of the visual assessment with the quantitative assessment, descriptive Standardized Uptake Value Ratio (SUVR) statistics were computed separately for DS subjects with an abnormal/normal majority read of the PET scan (DS-PET+/DS-PET-). The SUV is defined as the ratio of (1) the tissue radioactivity concentration c (in MBq/kg) at time point t, and (2) the injected activity (in MBq, extrapolated to the same time t) divided by the body weight (in kg). These SUV numbers from regions of interest were then used to derive SUV ratios (SUVR) using the SUV from the cerebellar cortex as reference.'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'keywords': ['Amyloid beta-protein', 'Down Syndrome'], 'conditions': ['Down Syndrome', 'Amyloid Beta-protein']}, 'referencesModule': {'references': [{'pmid': '33773598', 'type': 'DERIVED', 'citation': 'Bullich S, Roe-Vellve N, Marquie M, Landau SM, Barthel H, Villemagne VL, Sanabria A, Tartari JP, Sotolongo-Grau O, Dore V, Koglin N, Muller A, Perrotin A, Jovalekic A, De Santi S, Tarraga L, Stephens AW, Rowe CC, Sabri O, Seibyl JP, Boada M. Early detection of amyloid load using 18F-florbetaben PET. Alzheimers Res Ther. 2021 Mar 27;13(1):67. doi: 10.1186/s13195-021-00807-6.'}]}, 'descriptionModule': {'briefSummary': 'To determine the sensitivity of the visual assessment of BAY94-9172 PET images in detecting cerebral β-amyloid in individuals with Down Syndrome (DS) and specificity in individuals without DS. Given that individuals with Down Syndrome develop β-amyloid pathology over the age of 40, the clinical diagnosis of Down Syndrome will serve as the standard of truth.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '21 Years', 'healthyVolunteers': True, 'eligibilityCriteria': 'Inclusion Criteria:\n\nStudy participants were individuals with DS and healthy volunteers (HVs).\n\n* Main inclusion criteria for individuals without DS\n\n * \\>=21 and \\<= 40 years of age\n * Mini-Mental State Examination (MMSE) \\>= 28\n * Clinical Dementia Rating (CDR) of 0\n* Main inclusion criteria for individuals with DS\n\n * \\>= 40 years of age\n\nExclusion Criteria:\n\n* Main exclusion criteria for both groups\n\n * Unstable medical or psychiatric condition, study specific screening procedures with the understanding that the patient has the right to withdraw from the study at any time'}, 'identificationModule': {'nctId': 'NCT00928304', 'briefTitle': 'Phase II Study of Florbetaben (BAY94-9172) PET Imaging for Detection/Exclusion of Cerebral β-amyloid.', 'organization': {'class': 'INDUSTRY', 'fullName': 'Life Molecular Imaging SA'}, 'officialTitle': 'An Open-Label, Non-Randomized Study to Evaluate the Efficacy and Safety of BAY94-9172 (ZK 6013443) Positron Emission Tomography (PET) for Detection of Cerebral ß-Amyloid in Individuals With Down Syndrome Compared to Individuals Without Down Syndrome', 'orgStudyIdInfo': {'id': '14311'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Florbetaben (BAY94-9172)', 'interventionNames': ['Drug: Florbetaben (BAY94-9172)']}], 'interventions': [{'name': 'Florbetaben (BAY94-9172)', 'type': 'DRUG', 'description': '300 megabecquerels (MBq) as single IV injection of 2 to 10 mL', 'armGroupLabels': ['Florbetaben (BAY94-9172)']}]}, 'contactsLocationsModule': {'locations': [{'zip': '85351', 'city': 'Sun City', 'state': 'Arizona', 'country': 'United States', 'geoPoint': {'lat': 33.59754, 'lon': -112.27182}}, {'zip': '06510', 'city': 'New Haven', 'state': 'Connecticut', 'country': 'United States', 'geoPoint': {'lat': 41.30815, 'lon': -72.92816}}], 'overallOfficials': [{'name': 'Bayer Study Director', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Bayer'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Life Molecular Imaging SA', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}