Ignite Creation Date:
2025-12-24 @ 5:34 PM
Ignite Modification Date:
2025-12-31 @ 5:30 AM
Study NCT ID:
NCT05039268
Status:
COMPLETED
Last Update Posted:
2023-10-11
First Post:
2021-08-23
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
3K3A-APC for Treatment of Amyotrophic Lateral Sclerosis (ALS)
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D000690', 'term': 'Amyotrophic Lateral Sclerosis'}], 'ancestors': [{'id': 'D013118', 'term': 'Spinal Cord Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D016472', 'term': 'Motor Neuron Disease'}, {'id': 'D019636', 'term': 'Neurodegenerative Diseases'}, {'id': 'D057177', 'term': 'TDP-43 Proteinopathies'}, {'id': 'D009468', 'term': 'Neuromuscular Diseases'}, {'id': 'D057165', 'term': 'Proteostasis Deficiencies'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C000591161', 'term': '3K3A-APC protein'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'richard.gan@mq.edu.au', 'phone': '+61 2 9812 3739', 'title': 'Richard Gan', 'organization': 'Macquarie University'}, 'certainAgreement': {'piSponsorEmployee': True}, 'limitationsAndCaveats': {'description': 'The main limitation of the study is the small number of participants in the \\[18F\\]FEMPA PET analysis, which is one of the primary outcomes. In total, only 8 participants (5 in the 15mg group and 3 in the 30mg group) were analysed. The reasons for not including in the analysis are: PET scan data loss due to technical issues (1 participant), PET scan not being performed due to FEMPA production/QC failure (1 participant), and data being uninterpretable at analysis stage (6 participants).'}}, 'adverseEventsModule': {'timeFrame': 'Day-of-consent through end-of-participation, 15 days', 'description': 'Adverse events are continuously collected from day-of-consent through end-of-participation for each participant.', 'eventGroups': [{'id': 'EG000', 'title': '15mg Dose Group', 'description': 'Participants will receive a fixed dose regimen of five doses of 15mg.\n\n3K3A-APC Protein: 3K3A-APC with intravenous dosing of five doses of either 15mg or 30mg at 12 hourly interval. The first 8 patients will receive 15mg dose, and the next 8 patients will receive 30mg dose.', 'otherNumAtRisk': 8, 'deathsNumAtRisk': 8, 'otherNumAffected': 4, 'seriousNumAtRisk': 8, 'deathsNumAffected': 0, 'seriousNumAffected': 0}, {'id': 'EG001', 'title': '30mg Dose Group', 'description': 'Participants will receive a fixed dose regimen of five doses of 30mg.\n\n3K3A-APC Protein: 3K3A-APC with intravenous dosing of five doses of either 15mg or 30mg at 12 hourly interval. The first 8 patients will receive 15mg dose, and the next 8 patients will receive 30mg dose.', 'otherNumAtRisk': 8, 'deathsNumAtRisk': 8, 'otherNumAffected': 6, 'seriousNumAtRisk': 8, 'deathsNumAffected': 0, 'seriousNumAffected': 0}], 'otherEvents': [{'term': 'Fall', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (Unspecified)'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 3}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (Unspecified)'}, {'term': 'Constipation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (Unspecified)'}, {'term': 'Neutrophilia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 0}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (Unspecified)'}, {'term': 'Back pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (Unspecified)'}, {'term': 'Abdominal Pain Upper', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (Unspecified)'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (Unspecified)'}, {'term': 'Blood calcium increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 0}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (Unspecified)'}, {'term': 'Blood bicarbonate increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 0}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (Unspecified)'}, {'term': 'Supraventricular extrasystoles', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 0}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (Unspecified)'}, {'term': 'Herpes virus infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (Unspecified)'}, {'term': 'Arthralgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 0}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (Unspecified)'}, {'term': 'Catheter site extravasation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (Unspecified)'}], 'frequencyThreshold': '0'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Number of Participants Who Had Any Serious Adverse Events or Any Adverse Events With Severity Higher Than "Moderate".', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}, {'value': '8', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': '15mg Dose Group', 'description': 'Participants will receive a fixed dose regimen of five doses of 15mg.\n\n3K3A-APC Protein: 3K3A-APC with intravenous dosing of five doses of either 15mg or 30mg at 12 hourly interval. The first 8 patients will receive 15mg dose, and the next 8 patients will receive 30mg dose.'}, {'id': 'OG001', 'title': '30mg Dose Group', 'description': 'Participants will receive a fixed dose regimen of five doses of 30mg.\n\n3K3A-APC Protein: 3K3A-APC with intravenous dosing of five doses of either 15mg or 30mg at 12 hourly interval. The first 8 patients will receive 15mg dose, and the next 8 patients will receive 30mg dose.'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': '15 Days', 'description': 'Number of participants who had any serious adverse events or any adverse events with severity higher than "moderate", as determined by the Principal Investigator, using the composite safety assessment including clinical laboratory testing (full blood count, biochemistry, coagulation, iron study, CSF analysis and ECG), physical examination and self-reporting of adverse events. All clinical significant findings in the composite safety assessment were reported as adverse events.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'All dosed participants are included in the analysis.'}, {'type': 'PRIMARY', 'title': 'Percentage of Change in PERSI Score in the Motor Cortex Before and After Dosing', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': '15mg Dose Group', 'description': 'Participants will receive a fixed dose regimen of five doses of 15mg.\n\n3K3A-APC Protein: 3K3A-APC with intravenous dosing of five doses of either 15mg or 30mg at 12 hourly interval. The first 8 patients will receive 15mg dose, and the next 8 patients will receive 30mg dose.'}, {'id': 'OG001', 'title': '30mg Dose Group', 'description': 'Participants will receive a fixed dose regimen of five doses of 30mg.\n\n3K3A-APC Protein: 3K3A-APC with intravenous dosing of five doses of either 15mg or 30mg at 12 hourly interval. The first 8 patients will receive 15mg dose, and the next 8 patients will receive 30mg dose.'}], 'classes': [{'categories': [{'measurements': [{'value': '-0.14', 'groupId': 'OG000', 'lowerLimit': '-4.01', 'upperLimit': '2.63'}, {'value': '0.53', 'groupId': 'OG001', 'lowerLimit': '-0.96', 'upperLimit': '2.61'}]}]}], 'paramType': 'MEAN', 'timeFrame': '7 Days', 'description': 'PERSI (Parametric Estimation of Reference Signal Intensity) score is the measurement of microglial activation in the motor cortex utilising serial \\[18F\\]FEMPA PET imaging. The percentage of change in PERSI score before and after dosing in the two (2) dose cohorts is calculated.', 'unitOfMeasure': 'Percentage of change', 'dispersionType': 'Full Range', 'reportingStatus': 'POSTED', 'populationDescription': 'In total, 8 participants were included in the data analysis, with 5 participants in the 15mg group and 3 participants in the 30mg group.'}, {'type': 'SECONDARY', 'title': 'Diffusion Kurtosis Using MRI Scan', 'timeFrame': '7 Days', 'description': 'Change in diffusion kurtosis using MRI scan of the brain to determine whether the blood brain barrier integrity can be measured in ALS by Magnetic Resonance Imaging, and whether 3K3A-APC is able to repair it', 'reportingStatus': 'NOT_POSTED', 'denomUnitsSelected': 'Participants'}, {'type': 'SECONDARY', 'title': 'Monocyte Activation', 'timeFrame': '7 Days', 'description': 'Change in the level of monocyte activation in the peripheral blood utilising a novel method.', 'reportingStatus': 'NOT_POSTED', 'denomUnitsSelected': 'Participants'}, {'type': 'SECONDARY', 'title': 'Cytokine Level', 'timeFrame': '7 Days', 'description': 'Change in cytokine level in serum, plasma and CSF.', 'reportingStatus': 'NOT_POSTED', 'denomUnitsSelected': 'Participants'}, {'type': 'SECONDARY', 'title': 'Chemokine Level', 'timeFrame': '7 Days', 'description': 'Change in chemokine level in serum, plasma and CSF.', 'reportingStatus': 'NOT_POSTED', 'denomUnitsSelected': 'Participants'}, {'type': 'SECONDARY', 'title': 'Neurofilament Level', 'timeFrame': '7 Days', 'description': 'Change in neurofilament level in serum, plasma and CSF.', 'reportingStatus': 'NOT_POSTED', 'denomUnitsSelected': 'Participants'}, {'type': 'SECONDARY', 'title': 'Soluble CD14 Level', 'timeFrame': '7 Days', 'description': 'Change in soluble CD14 level in serum, plasma and CSF.', 'reportingStatus': 'NOT_POSTED', 'denomUnitsSelected': 'Participants'}, {'type': 'SECONDARY', 'title': 'Kynurenine Level', 'timeFrame': '7 Days', 'description': 'Change in kynurenine level in serum, plasma and CSF.', 'reportingStatus': 'NOT_POSTED', 'denomUnitsSelected': 'Participants'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': '15mg Dose Group', 'description': 'Participants will receive a fixed dose regimen of five doses of 15mg.\n\n3K3A-APC Protein: 3K3A-APC with intravenous dosing of five doses of either 15mg or 30mg at 12 hourly interval. The first 8 patients will receive 15mg dose, and the next 8 patients will receive 30mg dose.'}, {'id': 'FG001', 'title': '30mg Dose Group', 'description': 'Participants will receive a fixed dose regimen of five doses of 30mg.\n\n3K3A-APC Protein: 3K3A-APC with intravenous dosing of five doses of either 15mg or 30mg at 12 hourly interval. The first 8 patients will receive 15mg dose, and the next 8 patients will receive 30mg dose.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '8'}, {'groupId': 'FG001', 'numSubjects': '8'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '8'}, {'groupId': 'FG001', 'numSubjects': '8'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}]}]}]}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'BG000'}, {'value': '8', 'groupId': 'BG001'}, {'value': '16', 'groupId': 'BG002'}]}], 'groups': [{'id': 'BG000', 'title': '15mg Dose Group', 'description': 'Participants will receive a fixed dose regimen of five doses of 15mg.\n\n3K3A-APC Protein: 3K3A-APC with intravenous dosing of five doses of either 15mg or 30mg at 12 hourly interval. The first 8 patients will receive 15mg dose, and the next 8 patients will receive 30mg dose.'}, {'id': 'BG001', 'title': '30mg Dose Group', 'description': 'Participants will receive a fixed dose regimen of five doses of 30mg.\n\n3K3A-APC Protein: 3K3A-APC with intravenous dosing of five doses of either 15mg or 30mg at 12 hourly interval. The first 8 patients will receive 15mg dose, and the next 8 patients will receive 30mg dose.'}, {'id': 'BG002', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '6', 'groupId': 'BG000'}, {'value': '5', 'groupId': 'BG001'}, {'value': '11', 'groupId': 'BG002'}]}, {'title': '>=65 years', 'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '5', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '59.5', 'groupId': 'BG000', 'lowerLimit': '42', 'upperLimit': '73'}, {'value': '57.1', 'groupId': 'BG001', 'lowerLimit': '39', 'upperLimit': '71'}, {'value': '58.3', 'groupId': 'BG002', 'lowerLimit': '39', 'upperLimit': '73'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'FULL_RANGE'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '5', 'groupId': 'BG002'}]}, {'title': 'Male', 'measurements': [{'value': '6', 'groupId': 'BG000'}, {'value': '5', 'groupId': 'BG001'}, {'value': '11', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Ethnicity (NIH/OMB)', 'classes': [{'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '8', 'groupId': 'BG000'}, {'value': '8', 'groupId': 'BG001'}, {'value': '16', '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': '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': '8', 'groupId': 'BG000'}, {'value': '8', 'groupId': 'BG001'}, {'value': '16', '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': 'Australia', 'categories': [{'measurements': [{'value': '8', 'groupId': 'BG000'}, {'value': '8', 'groupId': 'BG001'}, {'value': '16', 'groupId': 'BG002'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}, {'title': 'ALSFRS-R Score', 'classes': [{'categories': [{'measurements': [{'value': '30.875', 'groupId': 'BG000', 'lowerLimit': '19', 'upperLimit': '45'}, {'value': '37.125', 'groupId': 'BG001', 'lowerLimit': '20', 'upperLimit': '46'}, {'value': '34.0', 'groupId': 'BG002', 'lowerLimit': '19', 'upperLimit': '46'}]}]}], 'paramType': 'MEAN', 'description': "ALSFRS-R measures 12 aspects of physical function, ranging from subject's ability to swallow and use utensils to climbing stairs and breathing. Each function is scored from 4 (normal) to 0 (no ability). The maximum score is 48 and the minimum is 0.", 'unitOfMeasure': 'units on a scale', 'dispersionType': 'FULL_RANGE'}]}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2022-03-17', 'size': 2556119, 'label': 'Study Protocol and Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'Prot_SAP_000.pdf', 'typeAbbrev': 'Prot_SAP', 'uploadDate': '2023-08-29T23:50', 'hasProtocol': True}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SEQUENTIAL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 16}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2021-11-25', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2022-09', 'completionDateStruct': {'date': '2022-09-12', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2023-09-21', 'studyFirstSubmitDate': '2021-08-23', 'resultsFirstSubmitDate': '2023-09-21', 'studyFirstSubmitQcDate': '2021-09-02', 'lastUpdatePostDateStruct': {'date': '2023-10-11', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2023-09-21', 'studyFirstPostDateStruct': {'date': '2021-09-09', 'type': 'ACTUAL'}, 'resultsFirstPostDateStruct': {'date': '2023-10-11', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2022-09-12', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of Participants Who Had Any Serious Adverse Events or Any Adverse Events With Severity Higher Than "Moderate".', 'timeFrame': '15 Days', 'description': 'Number of participants who had any serious adverse events or any adverse events with severity higher than "moderate", as determined by the Principal Investigator, using the composite safety assessment including clinical laboratory testing (full blood count, biochemistry, coagulation, iron study, CSF analysis and ECG), physical examination and self-reporting of adverse events. All clinical significant findings in the composite safety assessment were reported as adverse events.'}, {'measure': 'Percentage of Change in PERSI Score in the Motor Cortex Before and After Dosing', 'timeFrame': '7 Days', 'description': 'PERSI (Parametric Estimation of Reference Signal Intensity) score is the measurement of microglial activation in the motor cortex utilising serial \\[18F\\]FEMPA PET imaging. The percentage of change in PERSI score before and after dosing in the two (2) dose cohorts is calculated.'}], 'secondaryOutcomes': [{'measure': 'Diffusion Kurtosis Using MRI Scan', 'timeFrame': '7 Days', 'description': 'Change in diffusion kurtosis using MRI scan of the brain to determine whether the blood brain barrier integrity can be measured in ALS by Magnetic Resonance Imaging, and whether 3K3A-APC is able to repair it'}, {'measure': 'Monocyte Activation', 'timeFrame': '7 Days', 'description': 'Change in the level of monocyte activation in the peripheral blood utilising a novel method.'}, {'measure': 'Cytokine Level', 'timeFrame': '7 Days', 'description': 'Change in cytokine level in serum, plasma and CSF.'}, {'measure': 'Chemokine Level', 'timeFrame': '7 Days', 'description': 'Change in chemokine level in serum, plasma and CSF.'}, {'measure': 'Neurofilament Level', 'timeFrame': '7 Days', 'description': 'Change in neurofilament level in serum, plasma and CSF.'}, {'measure': 'Soluble CD14 Level', 'timeFrame': '7 Days', 'description': 'Change in soluble CD14 level in serum, plasma and CSF.'}, {'measure': 'Kynurenine Level', 'timeFrame': '7 Days', 'description': 'Change in kynurenine level in serum, plasma and CSF.'}]}, 'oversightModule': {'isUsExport': True, 'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['ALS', 'Amyotrophic Lateral Sclerosis', 'MND', 'Motor Neurone Disease', '3K3A-APC'], 'conditions': ['Amyotrophic Lateral Sclerosis']}, 'descriptionModule': {'briefSummary': 'Phase 2 open label trial to investigate the safety and potentially efficacy of 3K3A-APC in patients with Amyotrophic Lateral Sclerosis (ALS).', 'detailedDescription': 'This Phase 2 open label trial seeks to investigate whether a novel therapy named 3K3A-APC is safe and potentially effective in patients with Amyotrophic Lateral Sclerosis (ALS). A total of 16 patients with ALS will be enrolled into 2 dose cohorts with five doses of 15mg or 30mg doses given 12 hours apart in each cohort. The primary study outcomes are to ensure the safety and tolerability of 3K3AAPC in ALS patients, and to determine whether 3K3A-APC is able to reduce the pathological changes that might possibly cause ALS.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '75 Years', 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n1. Patients must have clinically definite ALS (Awaji Criteria)\n2. Male or female age 18 years and less than 75 years at time of ALS study\n3. Symptom onset less than 36 months before screening\n4. Diagnosis of ALS less than 24 months before screening\n5. Clinically definite Upper Motor Neuron signs\n\nExclusion Criteria:\n\n1. Current treatment with anticoagulants (e.g., warfarin, novel oral anticoagulants, heparin) that might preclude safe completion of the lumbar puncture\n2. Condition that precludes the safe performance of routine lumbar puncture, such as prohibitive lumbar spinal disease, bleeding diathesis, or clinically significant coagulopathy or thrombocytopenia\n3. Use of investigational drugs or devices within 60 days prior to Baseline (dietary supplements taken outside of a clinical trial are not exclusionary, e.g., coenzyme Q10)\n4. Prolonged prothrombin time or activated partial thromboplastin time \\>2xULN\n5. Severe hypertension or hypotension\n6. Glomerular filtration rate (GFR) \\<35 mL/min\n7. Forced vital capacity (FVC) at screening of \\<50% of predicted\n8. Prior exposure to any exogenous form of APC\n9. Inability to lie flat for procedures (MRI, PET, LP)\n10. Pregnant or lactating during the study period'}, 'identificationModule': {'nctId': 'NCT05039268', 'briefTitle': '3K3A-APC for Treatment of Amyotrophic Lateral Sclerosis (ALS)', 'organization': {'class': 'OTHER', 'fullName': 'Macquarie University, Australia'}, 'officialTitle': 'A Phase 2 Open Label Trial of 3K3A-APC in Amyotrophic Lateral Sclerosis', 'orgStudyIdInfo': {'id': 'ZZ-3K3A-202'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'ACTIVE_COMPARATOR', 'label': '15mg Dose Group', 'description': 'Participants will receive a fixed dose regimen of five doses of 15mg.', 'interventionNames': ['Drug: 3K3A-APC Protein']}, {'type': 'ACTIVE_COMPARATOR', 'label': '30mg Dose Group', 'description': 'Participants will receive a fixed dose regimen of five doses of 30mg.', 'interventionNames': ['Drug: 3K3A-APC Protein']}], 'interventions': [{'name': '3K3A-APC Protein', 'type': 'DRUG', 'otherNames': ['3K3A-APC'], 'description': '3K3A-APC with intravenous dosing of five doses of either 15mg or 30mg at 12 hourly interval. The first 8 patients will receive 15mg dose, and the next 8 patients will receive 30mg dose.', 'armGroupLabels': ['15mg Dose Group', '30mg Dose Group']}]}, 'contactsLocationsModule': {'locations': [{'zip': '2113', 'city': 'Macquarie Park', 'state': 'New South Wales', 'country': 'Australia', 'facility': 'Macquarie University', 'geoPoint': {'lat': -33.78105, 'lon': 151.12757}}], 'overallOfficials': [{'name': 'Dominic Rowe, PhD, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Macquarie University, Australia'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Macquarie University, Australia', 'class': 'OTHER'}, 'collaborators': [{'name': 'ZZ Biotech, LLC', 'class': 'INDUSTRY'}], 'responsibleParty': {'type': 'SPONSOR'}}}}