Ignite Creation Date:
2025-12-24 @ 9:11 PM
Ignite Modification Date:
2026-02-03 @ 9:40 AM
Study NCT ID:
NCT03664804
Status:
COMPLETED
Last Update Posted:
2025-01-03
First Post:
2018-09-07
Is Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Study to Measure Cerebrospinal Fluid Mutant Huntingtin Protein in Participants With Early Manifest Stage I or Stage II Huntington's Disease
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D006816', 'term': 'Huntington Disease'}], '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': 'D003704', 'term': 'Dementia'}, {'id': 'D002819', 'term': 'Chorea'}, {'id': 'D020820', 'term': 'Dyskinesias'}, {'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': 'D003072', 'term': 'Cognition Disorders'}, {'id': 'D019965', 'term': 'Neurocognitive Disorders'}, {'id': 'D001523', 'term': 'Mental Disorders'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'genentech@druginfo.com', 'phone': '800 821-8590', 'title': 'Medical Communications', 'organization': 'Hoffmann-La Roche'}, 'certainAgreement': {'otherDetails': "The Study being conducted under this Agreement is part of the Overall Study. Investigator is free to publish in reputable journals or to present at professional conferences the results of the Study, but only after the first publication or presentation that involves the Overall Study. The Sponsor may request that Confidential Information be deleted and/or the publication be postponed in order to protect the Sponsor's intellectual property rights", 'restrictionType': 'OTHER', 'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': 'AE timeframe: From Baseline to 15 Months', 'description': 'There was no Serious Adverse Event to be reported. Non-Serious Adverse Events at the frequency threshold of 5% cut-off are reported below.\n\nOut of 95 recruited participants, 2 were withdrawn before the study intervention, therefore exluded.', 'eventGroups': [{'id': 'EG000', 'title': 'NHS Participants With Early Manifest Stage I or II HD', 'description': 'Participants with Early Manifest Stage I or II HD', 'otherNumAtRisk': 93, 'deathsNumAtRisk': 93, 'otherNumAffected': 29, 'seriousNumAtRisk': 93, 'deathsNumAffected': 29, 'seriousNumAffected': 0}], 'otherEvents': [{'term': 'Puncture site pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 93, 'numEvents': 5, 'numAffected': 5}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'Medra v 26'}, {'term': 'Post lumbar puncture syndrome', 'stats': [{'groupId': 'EG000', 'numAtRisk': 93, 'numEvents': 19, 'numAffected': 14}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'Medra v 26'}, {'term': 'Procedural pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 93, 'numEvents': 9, 'numAffected': 7}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'Medra v 26'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 93, 'numEvents': 13, 'numAffected': 9}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'Medra v 26'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Change From Baseline in the Following Clinical Endpoints at 3, 9, and 15 Months: cUHDRS, TFC, TMS, SDMT, SWR Test and IS', 'denoms': [{'units': 'Participants', 'counts': [{'value': '93', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': "Participants With Early Manifest Stage I or II Huntington's Disease (HD)", 'description': 'No study drug was administered in this study'}], 'classes': [{'title': 'Composite UHDRS (cUHDRS) Month 3', 'denoms': [{'units': 'Participants', 'counts': [{'value': '89', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.19', 'spread': '1.08', 'groupId': 'OG000'}]}]}, {'title': 'Composite UHDRS (cUHDRS) Month 9', 'denoms': [{'units': 'Participants', 'counts': [{'value': '88', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.12', 'spread': '1.24', 'groupId': 'OG000'}]}]}, {'title': 'Composite UHDRS (cUHDRS) Month 15', 'denoms': [{'units': 'Participants', 'counts': [{'value': '62', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.76', 'spread': '1.49', 'groupId': 'OG000'}]}]}, {'title': 'UHDRS Total Functional Capacity (TFC) Month 3', 'denoms': [{'units': 'Participants', 'counts': [{'value': '89', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.24', 'spread': '0.89', 'groupId': 'OG000'}]}]}, {'title': 'UHDRS Total Functional Capacity (TFC) Month 9', 'denoms': [{'units': 'Participants', 'counts': [{'value': '88', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.33', 'spread': '1.31', 'groupId': 'OG000'}]}]}, {'title': 'UHDRS Total Functional Capacity (TFC) Month 15', 'denoms': [{'units': 'Participants', 'counts': [{'value': '62', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-1.10', 'spread': '1.60', 'groupId': 'OG000'}]}]}, {'title': 'UHDRS Total Motor Score (TMS) Month 3', 'denoms': [{'units': 'Participants', 'counts': [{'value': '89', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1.38', 'spread': '5.73', 'groupId': 'OG000'}]}]}, {'title': 'UHDRS Total Motor Score (TMS) Month 9', 'denoms': [{'units': 'Participants', 'counts': [{'value': '88', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '2.78', 'spread': '8.36', 'groupId': 'OG000'}]}]}, {'title': 'UHDRS Total Motor Score (TMS) Month 15', 'denoms': [{'units': 'Participants', 'counts': [{'value': '62', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '6.53', 'spread': '9.93', 'groupId': 'OG000'}]}]}, {'title': 'Symbol Digit Modalities Test (SDMT) Month 3', 'denoms': [{'units': 'Participants', 'counts': [{'value': '90', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '2.83', 'spread': '7.16', 'groupId': 'OG000'}]}]}, {'title': 'Symbol Digit Modalities Test (SDMT) Month 9', 'denoms': [{'units': 'Participants', 'counts': [{'value': '88', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '2.07', 'spread': '7.33', 'groupId': 'OG000'}]}]}, {'title': 'Symbol Digit Modalities Test (SDMT) Month 15', 'denoms': [{'units': 'Participants', 'counts': [{'value': '62', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1.92', 'spread': '6.97', 'groupId': 'OG000'}]}]}, {'title': 'Stroop Word Reading Test (SWRT) Month 3', 'denoms': [{'units': 'Participants', 'counts': [{'value': '90', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '2.52', 'spread': '12.58', 'groupId': 'OG000'}]}]}, {'title': 'Stroop Word Reading Test (SWRT) Month 9', 'denoms': [{'units': 'Participants', 'counts': [{'value': '88', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.24', 'spread': '11.86', 'groupId': 'OG000'}]}]}, {'title': 'Stroop Word Reading Test (SWRT) Month 15', 'denoms': [{'units': 'Participants', 'counts': [{'value': '62', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-3.16', 'spread': '11.67', 'groupId': 'OG000'}]}]}, {'title': 'UHDRS Independence Scale (IS) Month 3', 'denoms': [{'units': 'Participants', 'counts': [{'value': '89', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-1.24', 'spread': '5.45', 'groupId': 'OG000'}]}]}, {'title': 'UHDRS Independence Scale (IS) Month 9', 'denoms': [{'units': 'Participants', 'counts': [{'value': '88', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-2.56', 'spread': '7.07', 'groupId': 'OG000'}]}]}, {'title': 'UHDRS Independence Scale (IS) Month 15', 'denoms': [{'units': 'Participants', 'counts': [{'value': '62', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-4.27', 'spread': '8.34', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline to 15 Months', 'description': "The reported data are as follows: cUHDRS = composite Unified Huntington's Disease Rating Scale; TFC = Total Functional; Capacity Scale; TMS = Total Motor; Scale; SDMT = Symbol Digit Modalities Test; SWR = Stroop Word Reading; IS = Independence Scale.\n\ncUHDRS: score range from -3.06 (worst) to not defined maximum (best); Stroop Word Reading Test: score range not defined, higher scores indicate better cognitive performance; Symbol Digit Modalities Test: score range from 0 (worst) to 110 (best); Total Functional Capacity: score range from 0 (worst) to 13 (best); Total Motor Scale: score range from 0 (best) to 124 (worst).\n\nData at Month 3, 9, and 15 are reported respectively", 'unitOfMeasure': 'Scores on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'ITT Population'}, {'type': 'PRIMARY', 'title': 'Change From Baseline in Biomarkers of Neuronal Injury (CSF NfL and Tau) at 3, 9, and 15 Months', 'denoms': [{'units': 'Participants', 'counts': [{'value': '93', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': "Participants With Early Manifest Stage I or II Huntington's Disease (HD)", 'description': 'No study drug was administered in this study'}], 'classes': [{'title': 'Tau Month 3', 'denoms': [{'units': 'Participants', 'counts': [{'value': '89', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1.759', 'groupId': 'OG000', 'lowerLimit': '-2.927', 'upperLimit': '6.671'}]}]}, {'title': 'Tau Month 9', 'denoms': [{'units': 'Participants', 'counts': [{'value': '86', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '3.731', 'groupId': 'OG000', 'lowerLimit': '-1.960', 'upperLimit': '9.751'}]}]}, {'title': 'Tau Month 15', 'denoms': [{'units': 'Participants', 'counts': [{'value': '60', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '10.097', 'groupId': 'OG000', 'lowerLimit': '1.283', 'upperLimit': '19.678'}]}]}, {'title': 'NfL Month 3', 'denoms': [{'units': 'Participants', 'counts': [{'value': '89', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.052', 'groupId': 'OG000', 'lowerLimit': '-2.614', 'upperLimit': '2.578'}]}]}, {'title': 'NfL Month 9', 'denoms': [{'units': 'Participants', 'counts': [{'value': '86', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '4.035', 'groupId': 'OG000', 'lowerLimit': '-0.149', 'upperLimit': '8.395'}]}]}, {'title': 'NfL Month 15', 'denoms': [{'units': 'Participants', 'counts': [{'value': '61', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '5.971', 'groupId': 'OG000', 'lowerLimit': '1.629', 'upperLimit': '10.499'}]}]}, {'title': 'YKL-40 Month 3', 'denoms': [{'units': 'Participants', 'counts': [{'value': '89', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.763', 'groupId': 'OG000', 'lowerLimit': '-3.268', 'upperLimit': '1.806'}]}]}, {'title': 'YKL-40 Month 9', 'denoms': [{'units': 'Participants', 'counts': [{'value': '86', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1.026', 'groupId': 'OG000', 'lowerLimit': '-2.112', 'upperLimit': '4.263'}]}]}, {'title': 'YKL-40 Month 15', 'denoms': [{'units': 'Participants', 'counts': [{'value': '61', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '6.590', 'groupId': 'OG000', 'lowerLimit': '0.162', 'upperLimit': '13.431'}]}]}], 'paramType': 'GEOMETRIC_MEAN', 'timeFrame': 'Baseline to 15 Months', 'description': 'The reported date appreciations are as follows: CSF = Cerebrospinal Fluid; NfL = Neurofilament Light Chain. An overview of percentage change from baseline in geometric means for CSF tau and CSF NfL, and CSF YKL-40 are reported', 'unitOfMeasure': 'Percent Change', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'ITT Population'}, {'type': 'PRIMARY', 'title': 'Change From Baseline in Brain Atrophy Endpoints (Whole Brain Volume Decline, Caudate Volume Decline) as Determined by Brain MRI, at 3, 9, and 15 Months', 'denoms': [{'units': 'Participants', 'counts': [{'value': '93', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': "Participants With Early Manifest Stage I or II Huntington's Disease (HD)", 'description': 'No study drug was administered in this study'}], 'classes': [{'title': 'Caudate Volume Month 3', 'denoms': [{'units': 'Participants', 'counts': [{'value': '75', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1.345', 'groupId': 'OG000', 'lowerLimit': '0.875', 'upperLimit': '1.815'}]}]}, {'title': 'Caudate Volume Month 9', 'denoms': [{'units': 'Participants', 'counts': [{'value': '77', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '2.811', 'groupId': 'OG000', 'lowerLimit': '2.285', 'upperLimit': '3.337'}]}]}, {'title': 'Caudate Volume Month 15', 'denoms': [{'units': 'Participants', 'counts': [{'value': '43', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '4.806', 'groupId': 'OG000', 'lowerLimit': '4.057', 'upperLimit': '5.554'}]}]}, {'title': 'Ventricular Volume Month 3', 'denoms': [{'units': 'Participants', 'counts': [{'value': '86', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '2.662', 'groupId': 'OG000', 'lowerLimit': '2.016', 'upperLimit': '3.308'}]}]}, {'title': 'Ventricular Volume Month 9', 'denoms': [{'units': 'Participants', 'counts': [{'value': '83', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '5.124', 'groupId': 'OG000', 'lowerLimit': '4.132', 'upperLimit': '6.115'}]}]}, {'title': 'Ventricular Volume Month 15', 'denoms': [{'units': 'Participants', 'counts': [{'value': '47', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '9.622', 'groupId': 'OG000', 'lowerLimit': '8.195', 'upperLimit': '11.050'}]}]}, {'title': 'Whole Brain Volume Month 3', 'denoms': [{'units': 'Participants', 'counts': [{'value': '79', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.304', 'groupId': 'OG000', 'lowerLimit': '0.175', 'upperLimit': '0.434'}]}]}, {'title': 'Whole Brain Volume Month 9', 'denoms': [{'units': 'Participants', 'counts': [{'value': '79', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.513', 'groupId': 'OG000', 'lowerLimit': '0.346', 'upperLimit': '0.681'}]}]}, {'title': 'Whole Brain Volume Month 15', 'denoms': [{'units': 'Participants', 'counts': [{'value': '31', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1.332', 'groupId': 'OG000', 'lowerLimit': '1.062', 'upperLimit': '1.603'}]}]}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'Baseline to 15 Months', 'description': 'Data for Least Square (LS) mean percentage change from baseline to Months 3, 9, and 15 for ventricular volume, caudate volume, and whole brain volume, based on boundary shift integrals (BSIs) are reported', 'unitOfMeasure': 'Percentage change', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'ITT Population'}, {'type': 'SECONDARY', 'title': 'Within-Participant Change From Baseline in CSF mHTT Levels at 3, 9, and 15 Months', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': "Participants With Early Manifest Stage I or II Huntington's Disease (HD)", 'description': 'No study drug was administered in this study'}], 'timeFrame': 'Baseline to 15 Months', 'description': 'mHTT=Mutant Huntingtin Protein. New stability information has revealed that all samples for this outcome measure were out of stability, leaving no valid data points. Therefore there is no data to report.', 'reportingStatus': 'POSTED', 'populationDescription': "No participants' data to report"}, {'type': 'SECONDARY', 'title': 'Association of Change From Baseline in Cerebrospinal Fluid (FSF) mHTT With Change From Baseline in Clinical Measure', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': "Participants With Early Manifest Stage I or II Huntington's Disease (HD)", 'description': 'No study drug was administered in this study'}], 'timeFrame': 'Baseline to 15 Months', 'description': 'New stability information has revealed that all samples for this outcome measure were out of stability, leaving no valid data points. Therefore there is no data to report.', 'reportingStatus': 'POSTED', 'populationDescription': "No participants' data available"}, {'type': 'SECONDARY', 'title': 'Association of Change From Baseline in Biomarkers of Neuronal Injury', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': "Participants With Early Manifest Stage I or II Huntington's Disease (HD)", 'description': 'No study drug was administered in this study'}], 'timeFrame': 'Baseline to 15 Months', 'description': 'New stability information has revealed that all samples for this outcome measure were out of stability, leaving no valid data points. Therefore there is no data to report.', 'reportingStatus': 'POSTED', 'populationDescription': "No participants' data available"}, {'type': 'SECONDARY', 'title': 'Association of Change From Baseline in Brain Atrophy Endpoints, as Determined by Brain MRI', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': "Participants With Early Manifest Stage I or II Huntington's Disease (HD)", 'description': 'No study drug was administered in this study'}], 'timeFrame': 'Baseline to 15 Months', 'description': 'New stability information has revealed that all samples for this outcome measure were out of stability, leaving no valid data points. Therefore there is no data to report.', 'reportingStatus': 'POSTED', 'populationDescription': "No participants' data available"}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': "Participants With Early Manifest Stage I or II Huntington's Disease (HD)", 'description': 'No study drug was administered in this study'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '95'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '88'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '7'}]}], 'dropWithdraws': [{'type': 'Withdrawal by Subject', 'reasons': [{'groupId': 'FG000', 'numSubjects': '7'}]}]}], 'recruitmentDetails': 'A total of 93 patients (97.9%) were included in the safety population and the intent-to-treat (ITT) population. Two patients were excluded from both the analysis populations as the patients had discontinued the study.', 'preAssignmentDetails': 'Out of 95 participants recruited, 2 were withdrawn before the study intervention.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '95', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': "Participants With Early Manifest Stage I or II Huntington's Disease (HD)", 'description': 'No study drug was administered in this study'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '48.1', 'spread': '9.9', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'Years', 'dispersionType': 'STANDARD_DEVIATION', 'populationDescription': 'Out of 95 recruited baseline population, 2 were withdrawn before the study intervention'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '36', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '59', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants', 'populationDescription': 'Out of 95 recruited participants, 2 were withdrawn before the study intervention.'}, {'title': 'Ethnicity (NIH/OMB)', 'classes': [{'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '3', 'groupId': 'BG000'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '92', 'groupId': 'BG000'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants', 'populationDescription': 'Out of 95 recruited participants, 2 were withdrawn before the study intervention.'}, {'title': 'Race (NIH/OMB)', 'classes': [{'categories': [{'title': 'American Indian or Alaska Native', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Asian', 'measurements': [{'value': '1', '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': '89', 'groupId': 'BG000'}]}, {'title': 'More than one race', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '4', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants', 'populationDescription': 'Out of 95 recruited participants, 2 were withdrawn before the study intervention.'}]}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2019-12-18', 'size': 805700, 'label': 'Study Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_000.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2022-04-09T06:32', 'hasProtocol': True}, {'date': '2020-09-19', 'size': 1118048, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_001.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2022-04-09T06:32', 'hasProtocol': False}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'OTHER', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 95}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2018-12-05', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2024-11', 'completionDateStruct': {'date': '2022-05-07', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2024-11-14', 'studyFirstSubmitDate': '2018-09-07', 'resultsFirstSubmitDate': '2022-04-11', 'studyFirstSubmitQcDate': '2018-09-07', 'lastUpdatePostDateStruct': {'date': '2025-01-03', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2024-11-14', 'studyFirstPostDateStruct': {'date': '2018-09-11', 'type': 'ACTUAL'}, 'resultsFirstPostDateStruct': {'date': '2025-01-03', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2021-05-07', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Change From Baseline in the Following Clinical Endpoints at 3, 9, and 15 Months: cUHDRS, TFC, TMS, SDMT, SWR Test and IS', 'timeFrame': 'Baseline to 15 Months', 'description': "The reported data are as follows: cUHDRS = composite Unified Huntington's Disease Rating Scale; TFC = Total Functional; Capacity Scale; TMS = Total Motor; Scale; SDMT = Symbol Digit Modalities Test; SWR = Stroop Word Reading; IS = Independence Scale.\n\ncUHDRS: score range from -3.06 (worst) to not defined maximum (best); Stroop Word Reading Test: score range not defined, higher scores indicate better cognitive performance; Symbol Digit Modalities Test: score range from 0 (worst) to 110 (best); Total Functional Capacity: score range from 0 (worst) to 13 (best); Total Motor Scale: score range from 0 (best) to 124 (worst).\n\nData at Month 3, 9, and 15 are reported respectively"}, {'measure': 'Change From Baseline in Biomarkers of Neuronal Injury (CSF NfL and Tau) at 3, 9, and 15 Months', 'timeFrame': 'Baseline to 15 Months', 'description': 'The reported date appreciations are as follows: CSF = Cerebrospinal Fluid; NfL = Neurofilament Light Chain. An overview of percentage change from baseline in geometric means for CSF tau and CSF NfL, and CSF YKL-40 are reported'}, {'measure': 'Change From Baseline in Brain Atrophy Endpoints (Whole Brain Volume Decline, Caudate Volume Decline) as Determined by Brain MRI, at 3, 9, and 15 Months', 'timeFrame': 'Baseline to 15 Months', 'description': 'Data for Least Square (LS) mean percentage change from baseline to Months 3, 9, and 15 for ventricular volume, caudate volume, and whole brain volume, based on boundary shift integrals (BSIs) are reported'}], 'secondaryOutcomes': [{'measure': 'Within-Participant Change From Baseline in CSF mHTT Levels at 3, 9, and 15 Months', 'timeFrame': 'Baseline to 15 Months', 'description': 'mHTT=Mutant Huntingtin Protein. New stability information has revealed that all samples for this outcome measure were out of stability, leaving no valid data points. Therefore there is no data to report.'}, {'measure': 'Association of Change From Baseline in Cerebrospinal Fluid (FSF) mHTT With Change From Baseline in Clinical Measure', 'timeFrame': 'Baseline to 15 Months', 'description': 'New stability information has revealed that all samples for this outcome measure were out of stability, leaving no valid data points. Therefore there is no data to report.'}, {'measure': 'Association of Change From Baseline in Biomarkers of Neuronal Injury', 'timeFrame': 'Baseline to 15 Months', 'description': 'New stability information has revealed that all samples for this outcome measure were out of stability, leaving no valid data points. Therefore there is no data to report.'}, {'measure': 'Association of Change From Baseline in Brain Atrophy Endpoints, as Determined by Brain MRI', 'timeFrame': 'Baseline to 15 Months', 'description': 'New stability information has revealed that all samples for this outcome measure were out of stability, leaving no valid data points. Therefore there is no data to report.'}]}, 'oversightModule': {'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ["Huntington's Disease"]}, 'descriptionModule': {'briefSummary': "The study is designed as a multi-site, prospective, 15-month longitudinal, cohort study measuring CSF mHTT in participants with early manifest Stage I or Stage II Huntington's Disease (HD)."}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '65 Years', 'minimumAge': '25 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* Capacity to consent to participate in the study as assessed using the Evaluation to Sign Consent tool and investigator judgment\n* Age 25 to 65 years, inclusive, at the time of signing Informed Consent Form\n* Early manifest, Stage I or Stage II HD (defined as TFC of 7-13, inclusive)\n* Genetically confirmed disease (CAG repeat length ≥ 36 in huntingtin gene by direct DNA testing)\n* Body mass index ≥18 and ≤32 kg/m2; total body weight \\>50 kg\n* Ability to undergo and tolerate MRI scans\n* Ability to tolerate blood draws and lumbar puncture\n* Ability and willingness to comply with all aspects of the protocol, including completion of interviews and questionnaires and carrying/wearing of a digital monitoring device\n* Stable medical, psychiatric, and neurological status for at least 12 weeks prior to screening and at the time of enrollment\n* Signed study companion consent for participation, if a study companion is available\n* For women of childbearing potential: agreement to remain abstinent or use acceptable contraceptive methods during the observational period\n\nExclusion Criteria:\n\n* Any condition, including severe chorea, that would prevent either writing or performing pen and paper or smartphone-based tasks\n* History of attempted suicide or suicidal ideation with plan (i.e., active suicidal ideation) that required hospital visit and/or change in level of care within 12 months prior to screening\n* Current active psychosis, confusional state, or violent behavior\n* Any serious medical condition or clinically significant laboratory, vital sign, or electrocardiogram abnormalities at screening that, in the investigator's judgement, precludes the participant's safe participation in and completion of the study\n* Pregnant or breastfeeding, or intending to become pregnant during the study\n* Positive for hepatitis C virus antibody or hepatitis B surface antigen at screening\n* Known HIV infection\n* Current or previous use of an antisense oligonucleotide (including small interfering RNA)\n* Current use of antipsychotics prescribed for psychosis, cholinesterase inhibitors, memantine, amantadine, or riluzole including use within 12 weeks of enrollment\n* Treatment with an investigational drug within 30 days prior to screening or 5 half-lives of the investigational drug, whichever is longer\n* Antiplatelet or anticoagulant therapy within the 14 days prior to screening or anticipated use during the study, including, but not limited, to aspirin (unless ≤81mg/day), clopidogrel, dipyridamole, warfarin, dabigatran, rivaroxaban, and apixaban\n* History of bleeding diathesis or coagulopathy; platelet count \\< lower limit of normal unless stable and assessed by the Investigator and Sponsor Medical Monitor to be not clinically significant\n* Malignancy within 5 years prior to screening, except basal or squamous cell carcinoma of the skin or carcinoma in situ of the cervix that has been successfully treated\n* History of gene therapy or cell transplantation or any other experimental brain surgery\n* Concurrent or planned concurrent participation in any clinical study without approval of the Medical Monitor\n* Presence of implanted shunt for the drainage of CSF or an implanted CNS catheter\n* Pre-existing structural brain lesion as assessed by MRI scan"}, 'identificationModule': {'nctId': 'NCT03664804', 'briefTitle': "Study to Measure Cerebrospinal Fluid Mutant Huntingtin Protein in Participants With Early Manifest Stage I or Stage II Huntington's Disease", 'organization': {'class': 'INDUSTRY', 'fullName': 'Hoffmann-La Roche'}, 'officialTitle': "A Multi-Site, Prospective, Longitudinal, Cohort Study Measuring Cerebrospinal Fluid-Mutant Huntingtin Protein in Patients With Huntington's Disease", 'orgStudyIdInfo': {'id': 'BN40422'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'OTHER', 'label': 'Participants with Early Manifest Stage I or II HD', 'description': 'No study drug was administered in this study', 'interventionNames': ['Other: No Study Drug was Administered in this Study']}], 'interventions': [{'name': 'No Study Drug was Administered in this Study', 'type': 'OTHER', 'description': 'No study drug was administered in this study', 'armGroupLabels': ['Participants with Early Manifest Stage I or II HD']}]}, 'contactsLocationsModule': {'locations': [{'zip': '80113', 'city': 'Englewood', 'state': 'Colorado', 'country': 'United States', 'facility': 'CenExel Rocky Mountain Clinical Research, LLC', 'geoPoint': {'lat': 39.64777, 'lon': -104.98776}}, {'zip': '20007', 'city': 'Washington D.C.', 'state': 'District of Columbia', 'country': 'United States', 'facility': 'Georgetown University; 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