Ignite Creation Date:
2025-12-24 @ 10:18 PM
Ignite Modification Date:
2025-12-28 @ 1:33 PM
Study NCT ID:
NCT05053035
Status:
TERMINATED
Last Update Posted:
2025-06-18
First Post:
2021-09-01
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
A Phase 2 Study to Evaluate AL001 in C9orf72-Associated ALS
Sponsor:
Organization:
Raw JSON
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Information', 'organization': 'Alector'}, 'certainAgreement': {'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': '32 Weeks', 'eventGroups': [{'id': 'EG000', 'title': 'AL001', 'description': 'AL001 every 4 weeks\n\nAL001: Administered via intravenous (IV) infusion', 'otherNumAtRisk': 3, 'deathsNumAtRisk': 3, 'otherNumAffected': 2, 'seriousNumAtRisk': 3, 'deathsNumAffected': 0, 'seriousNumAffected': 0}, {'id': 'EG001', 'title': 'Placebo', 'description': 'Placebo every 4 weeks\n\nPlacebo: Administered via intravenous (IV) infusion', 'otherNumAtRisk': 2, 'deathsNumAtRisk': 2, 'otherNumAffected': 2, 'seriousNumAtRisk': 2, 'deathsNumAffected': 0, 'seriousNumAffected': 0}], 'otherEvents': [{'term': 'Constipation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 2, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Diarrhea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 2, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Dyspepsia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 2, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 2, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Catheter site irritation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 2, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Fatigue', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 2, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Medical device site hemorrhage', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 2, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 2, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 2, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 2, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Urinary tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 2, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Post lumbar puncture syndrome', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 2, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Procedural pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 2, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Stoma site erythema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 2, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Stoma site hypergranulation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 2, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Hepatic enzyme increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 2, 'numAffected': 0}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 2, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Paraesthesia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 2, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Urinary incontinence', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 2, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Epistaxis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 2, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Pneumonitis aspiration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 2, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Throat irritation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 2, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Drug eruption', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 2, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}, {'term': 'Rash', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 2, 'numAffected': 0}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 25.0'}], 'frequencyThreshold': '0'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Evaluation of Safety and Tolerability of AL001 Measured by Number of Subjects With Adverse Events', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'AL001', 'description': 'AL001 every 4 weeks\n\nAL001: Administered via intravenous (IV) infusion'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Placebo every 4 weeks\n\nPlacebo: Administered via intravenous (IV) infusion'}], 'classes': [{'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': '24 weeks', 'description': 'Count of participants with adverse events during the study treatment period', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Safety population'}, {'type': 'PRIMARY', 'title': 'Immunogenicity of AL001', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'AL001', 'description': 'AL001 every 4 weeks\n\nAL001: Administered via intravenous (IV) infusion'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Placebo every 4 weeks\n\nPlacebo: Administered via intravenous (IV) infusion'}], 'classes': [{'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Week 24', 'description': 'Count of participants positive for Anti-drug Antibodies (ADAs) to AL001 at week 24', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Due to early termination of the study and insufficient sample size, no population-level analysis (within-group and between-group) was performed.'}, {'type': 'PRIMARY', 'title': 'Pharmacokinetics (PK) of AL001 in Serum', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'AL001', 'description': 'AL001 every 4 weeks\n\nAL001: Administered via intravenous (IV) infusion'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Placebo every 4 weeks\n\nPlacebo: Administered via intravenous (IV) infusion'}], 'classes': [{'categories': [{'measurements': [{'value': '705792.5', 'spread': '87387.7916', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Week 24', 'description': 'Concentration of AL001 in Serum at week 24', 'unitOfMeasure': 'ng/mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Due to early termination of the study and insufficient sample size, no population-level analysis (within-group and between-group) was performed.'}, {'type': 'PRIMARY', 'title': 'Pharmacokinetics (PK) of AL001 in CSF', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'AL001', 'description': 'AL001 every 4 weeks\n\nAL001: Administered via intravenous (IV) infusion'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Placebo every 4 weeks\n\nPlacebo: Administered via intravenous (IV) infusion'}], 'classes': [{'categories': [{'measurements': [{'value': '547.5', 'spread': '395.2727', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Week 24', 'description': 'Concentration of AL001 in Cerebrospinal fluid (CSF) at week 24', 'unitOfMeasure': 'ng/mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Due to early termination of the study and insufficient sample size, no population-level analysis (within-group and between-group) was performed.'}, {'type': 'PRIMARY', 'title': 'Change From Baseline in Plasma Progranulin', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'AL001', 'description': 'AL001 every 4 weeks\n\nAL001: Administered via intravenous (IV) infusion'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Placebo every 4 weeks\n\nPlacebo: Administered via intravenous (IV) infusion'}], 'classes': [{'categories': [{'measurements': [{'value': '175', 'spread': '2.8284', 'groupId': 'OG000'}, {'value': '-24.4', 'spread': '9.3338', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': '24 weeks', 'description': 'Evaluate the change from baseline to week 24 in plasma progranulin levels', 'unitOfMeasure': 'ng/mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Due to early termination of the study and insufficient sample size, no population-level analysis (within-group and between-group) was performed.'}, {'type': 'PRIMARY', 'title': 'Change From Baseline in CSF Progranulin', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'AL001', 'description': 'AL001 every 4 weeks\n\nAL001: Administered via intravenous (IV) infusion'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Placebo every 4 weeks\n\nPlacebo: Administered via intravenous (IV) infusion'}], 'classes': [{'categories': [{'measurements': [{'value': '2.865', 'spread': '1.0677', 'groupId': 'OG000'}, {'value': '-0.385', 'spread': '0.3323', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': '24 weeks', 'description': 'Evaluate the change from baseline to week 24 in Cerebrospinal fluid (CSF) progranulin levels', 'unitOfMeasure': 'ng/mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Due to early termination of the study and insufficient sample size, no population-level analysis (within-group and between-group) was performed.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Plasma Neurofilament Light Chain', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'AL001', 'description': 'AL001 every 4 weeks\n\nAL001: Administered via intravenous (IV) infusion'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Placebo every 4 weeks\n\nPlacebo: Administered via intravenous (IV) infusion'}], 'classes': [{'categories': [{'measurements': [{'value': '-0.35', 'spread': '3.3234', 'groupId': 'OG000'}, {'value': '1.9', 'spread': '2.8284', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': '24 weeks', 'description': 'Evaluate the change from baseline to week 24 in plasma neurofilament light chain levels', 'unitOfMeasure': 'pg/mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Due to early termination of the study and insufficient sample size, no population-level analysis (within-group and between-group) was performed.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in CSF Neurofilament Light Chain', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'AL001', 'description': 'AL001 every 4 weeks\n\nAL001: Administered via intravenous (IV) infusion'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Placebo every 4 weeks\n\nPlacebo: Administered via intravenous (IV) infusion'}], 'classes': [{'categories': [{'measurements': [{'value': '995.5', 'spread': '95.4594', 'groupId': 'OG000'}, {'value': '-1250.5', 'spread': '825.1936', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': '24 weeks', 'description': 'Evaluate change from baseline to week 24 in Cerebrospinal fluid (CSF) neurofilament light chain levels', 'unitOfMeasure': 'pg/mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Due to early termination of the study and insufficient sample size, no population-level analysis (within-group and between-group) was performed.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'AL001', 'description': 'AL001 every 4 weeks\n\nAL001: Administered via intravenous (IV) infusion'}, {'id': 'FG001', 'title': 'Placebo', 'description': 'Placebo every 4 weeks\n\nPlacebo: Administered via intravenous (IV) infusion'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '3'}, {'groupId': 'FG001', 'numSubjects': '2'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '2'}, {'groupId': 'FG001', 'numSubjects': '2'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '0'}]}], 'dropWithdraws': [{'type': 'Withdrawal by Subject', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '0'}]}]}]}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '5', 'groupId': 'BG002'}]}], 'groups': [{'id': 'BG000', 'title': 'AL001', 'description': 'AL001 every 4 weeks\n\nAL001: Administered via intravenous (IV) infusion'}, {'id': 'BG001', 'title': 'Placebo', 'description': 'Placebo every 4 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[{'value': '2', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '2', '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': '3', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '5', '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': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'United States', 'categories': [{'measurements': [{'value': '3', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '5', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}]}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2022-02-11', 'size': 1204907, 'label': 'Study Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_000.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2024-11-22T02:16', 'hasProtocol': True}, {'date': '2022-11-29', 'size': 326090, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_001.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2024-11-22T02:16', 'hasProtocol': False}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 5}}, 'statusModule': {'whyStopped': 'The Sponsor is considering a subsequent study in ALS, potentially with different inclusion/exclusion criteria.', 'overallStatus': 'TERMINATED', 'startDateStruct': {'date': '2021-09-02', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-05', 'completionDateStruct': {'date': '2022-10-28', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2025-05-30', 'studyFirstSubmitDate': '2021-09-01', 'resultsFirstSubmitDate': '2025-01-29', 'studyFirstSubmitQcDate': '2021-09-13', 'lastUpdatePostDateStruct': {'date': '2025-06-18', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2025-05-30', 'studyFirstPostDateStruct': {'date': '2021-09-22', 'type': 'ACTUAL'}, 'resultsFirstPostDateStruct': {'date': '2025-06-18', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2022-10-28', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Evaluation of Safety and Tolerability of AL001 Measured by Number of Subjects With Adverse Events', 'timeFrame': '24 weeks', 'description': 'Count of participants with adverse events during the study treatment period'}, {'measure': 'Immunogenicity of AL001', 'timeFrame': 'Week 24', 'description': 'Count of participants positive for Anti-drug Antibodies (ADAs) to AL001 at week 24'}, {'measure': 'Pharmacokinetics (PK) of AL001 in Serum', 'timeFrame': 'Week 24', 'description': 'Concentration of AL001 in Serum at week 24'}, {'measure': 'Pharmacokinetics (PK) of AL001 in CSF', 'timeFrame': 'Week 24', 'description': 'Concentration of AL001 in Cerebrospinal fluid (CSF) at week 24'}, {'measure': 'Change From Baseline in Plasma Progranulin', 'timeFrame': '24 weeks', 'description': 'Evaluate the change from baseline to week 24 in plasma progranulin levels'}, {'measure': 'Change From Baseline in CSF Progranulin', 'timeFrame': '24 weeks', 'description': 'Evaluate the change from baseline to week 24 in Cerebrospinal fluid (CSF) progranulin levels'}], 'secondaryOutcomes': [{'measure': 'Change From Baseline in Plasma Neurofilament Light Chain', 'timeFrame': '24 weeks', 'description': 'Evaluate the change from baseline to week 24 in plasma neurofilament light chain levels'}, {'measure': 'Change From Baseline in CSF Neurofilament Light Chain', 'timeFrame': '24 weeks', 'description': 'Evaluate change from baseline to week 24 in Cerebrospinal fluid (CSF) neurofilament light chain levels'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Amyotrophic Lateral Sclerosis']}, 'descriptionModule': {'briefSummary': 'A phase 2 double-blind, placebo-controlled study of AL001 in participants with C9orf72-associated ALS.', 'detailedDescription': 'This is a phase 2 double-blind, placebo-controlled trial to test the safety, tolerability, pharmacokinetics, and pharmacodynamics of AL001 in participants with C9orf72-associated Amyotrophic Lateral Sclerosis.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Confirmation of C9orf72 mutation\n* Diagnosis of ALS by revised El Escorial criteria\n* Time since onset of muscle weakness due to ALS ≤36 months at the time of the Screening Visit\n* Slow Vital Capacity (VC) ≥50% of predicted capacity at the time of the Screening Visit\n* If taking riluzole, must be on a stable dose of riluzole for at least 30 days prior to the Screening Visit. Riluzole naive participants are allowed.\n* If taking edaravone, must have completed at least one cycle of edaravone prior to the Screening Visit and plan to continue edaravone during the study. Edaravone naive participants are allowed.\n* Females must not be pregnant, breastfeeding or planning to conceive within the study period. Males must agree to use acceptable contraception\n* Capable of providing informed consent at the Screening visit and complying with study procedures throughout the study\n\nExclusion Criteria:\n\n* Clinically significant, unstable, medical condition (other than ALS)\n* Clinically significant heart disease, liver disease or kidney disease\n* Cognitive impairment or dementia\n* Current uncontrolled hypertension\n* History of unresolved cancer\n* Any experimental gene therapy\n* Any experimental vaccine (any vaccine against COVID-19 either approved or administered under an Emergency Use Authorization is allowed)'}, 'identificationModule': {'nctId': 'NCT05053035', 'briefTitle': 'A Phase 2 Study to Evaluate AL001 in C9orf72-Associated ALS', 'organization': {'class': 'INDUSTRY', 'fullName': 'Alector Inc.'}, 'officialTitle': 'A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AL001 in C9orf72-Associated Amyotrophic Lateral Sclerosis', 'orgStudyIdInfo': {'id': 'AL001-ALS-201'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'AL001', 'description': 'AL001 every 4 weeks', 'interventionNames': ['Drug: AL001']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Placebo', 'description': 'Placebo every 4 weeks', 'interventionNames': ['Drug: Placebo']}], 'interventions': [{'name': 'AL001', 'type': 'DRUG', 'description': 'Administered via intravenous (IV) infusion', 'armGroupLabels': ['AL001']}, {'name': 'Placebo', 'type': 'DRUG', 'description': 'Administered via intravenous (IV) infusion', 'armGroupLabels': ['Placebo']}]}, 'contactsLocationsModule': {'locations': [{'zip': '33612', 'city': 'Tampa', 'state': 'Florida', 'country': 'United States', 'facility': 'University of South Florida', 'geoPoint': {'lat': 27.94752, 'lon': -82.45843}}, {'zip': '02114', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': 'Massachusetts General Hospital', 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '63110', 'city': 'St Louis', 'state': 'Missouri', 'country': 'United States', 'facility': 'Washington University School of Medicine', 'geoPoint': {'lat': 38.62727, 'lon': -90.19789}}], 'overallOfficials': [{'name': 'Sabrina Paganoni, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Massachusetts General Hospital'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Alector Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}