Ignite Creation Date:
2025-12-24 @ 7:59 PM
Ignite Modification Date:
2026-01-01 @ 6:53 PM
Study NCT ID:
NCT03636204
Status:
COMPLETED
Last Update Posted:
2020-07-27
First Post:
2018-07-12
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
A First in Human Study in Healthy Volunteers and in Participants With Frontotemporal Dementia With Granulin (GRN) Mutation
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'removedCountries': ['Netherlands']}, 'conditionBrowseModule': {'meshes': [{'id': 'D057180', 'term': 'Frontotemporal Dementia'}], 'ancestors': [{'id': 'D057174', 'term': 'Frontotemporal Lobar Degeneration'}, {'id': 'D003704', 'term': 'Dementia'}, {'id': 'D001927', 'term': 'Brain Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D057177', 'term': 'TDP-43 Proteinopathies'}, {'id': 'D019636', 'term': 'Neurodegenerative Diseases'}, {'id': 'D057165', 'term': 'Proteostasis Deficiencies'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}, {'id': 'D019965', 'term': 'Neurocognitive Disorders'}, {'id': 'D001523', 'term': 'Mental Disorders'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 64}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2018-09-14', 'type': 'ACTUAL'}, 'statusVerifiedDate': '2020-07', 'completionDateStruct': {'date': '2019-12-31', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2020-07-23', 'studyFirstSubmitDate': '2018-07-12', 'studyFirstSubmitQcDate': '2018-08-15', 'lastUpdatePostDateStruct': {'date': '2020-07-27', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2018-08-17', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2019-12-31', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Evaluation of safety and tolerability of AL001 measured by number of subjects with adverse events and Dose Limiting Adverse Event (DLAEs)', 'timeFrame': '85 days', 'description': 'Incidence of adverse events and dose limiting Adverse Events during the DLAE observation period and/or study treatment periods.'}], 'secondaryOutcomes': [{'measure': 'Pharmacokinetics (PK) of AL001', 'timeFrame': '85 days', 'description': 'Serum and cerebrospinal fluid (CSF) concentration of AL001 at specified time points'}, {'measure': 'Maximum plasma concentration (Cmax) for AL001', 'timeFrame': '85 days', 'description': 'Evaluate Cmax for serum and CSF concentration of AL001 at specified time points'}, {'measure': 'Area under the curve concentration (AUC) for AL001', 'timeFrame': '85 days', 'description': 'Evaluate AUC for serum and CSF concentration of AL001 at specified time points'}]}, 'oversightModule': {'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Healthy', 'Frontotemporal Dementia']}, 'referencesModule': {'references': [{'pmid': '37322482', 'type': 'DERIVED', 'citation': 'Kurnellas M, Mitra A, Schwabe T, Paul R, Arrant AE, Roberson ED, Ward M, Yeh F, Long H, Rosenthal A. Latozinemab, a novel progranulin-elevating therapy for frontotemporal dementia. J Transl Med. 2023 Jun 15;21(1):387. doi: 10.1186/s12967-023-04251-y.'}]}, 'descriptionModule': {'briefSummary': 'A first in human phase 1 study in healthy volunteers and participants with Granulin (GRN) mutation causative of frontotemporal dementia (FTD) to assess AL001 safety, tolerability, pharmacokinetics, and pharmacodynamics', 'detailedDescription': 'This is a first-in-human Phase 1 study designed to investigate the safety, tolerability, PK and PD of intravenously administered AL001 in Healthy Volunteers and FTD-GRN mutation carriers.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '80 Years', 'minimumAge': '18 Years', 'healthyVolunteers': True, 'eligibilityCriteria': "Inclusion Criteria:\n\n* BMI 18.0-35.0 kg/m2\n* 45-120 kg, inclusive\n* At screening, females must be non-pregnant and non-lactating, or of nonchildbearing potential (either surgically sterilized or physiologically incapable of becoming pregnant, or at least 1-year postmenopausal (amenorrhoea duration of 12 consecutive months); nonpregnancy will be confirmed for all females by a pregnancy test conducted at screening, (each) admission, and at follow-up.\n* Female participants of child-bearing potential must agree to use adequate contraception from screening until 90 days after the follow-up visit.\n* In good physical health on the basis of no clinically significant findings from medical history, physical examination (PE), laboratory tests, 12 lead ECG, and vital signs, as judged by the Investigator.\n* Willingness and able to comply with the study protocol, in the investigator's judgement.\n\nExclusion Criteria:\n\n* Known history of severe allergic, anaphylactic, or other hypersensitivity reactions to chimeric, human, or humanized antibodies or fusion proteins.\n* Positive drug or alcohol at screening and prior to first dose\n* History of alcohol abuse or substance abuse"}, 'identificationModule': {'nctId': 'NCT03636204', 'briefTitle': 'A First in Human Study in Healthy Volunteers and in Participants With Frontotemporal Dementia With Granulin (GRN) Mutation', 'organization': {'class': 'INDUSTRY', 'fullName': 'Alector Inc.'}, 'officialTitle': 'A First in Human Phase 1 Study in Healthy Volunteers and in Participants With Frontotemporal Dementia (FTD) With Granulin Mutation', 'orgStudyIdInfo': {'id': 'AL001-1'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'AL001', 'description': 'Up to six single ascending doses of AL001', 'interventionNames': ['Biological: AL001']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Saline Solution', 'description': 'Saline solution will be administered as a single infusion for each cohort in a ratio of 6 active and 2 placebo subjects', 'interventionNames': ['Other: Placebo']}], 'interventions': [{'name': 'AL001', 'type': 'BIOLOGICAL', 'description': 'Active dose of AL001', 'armGroupLabels': ['AL001']}, {'name': 'Placebo', 'type': 'OTHER', 'description': 'Saline solution administered as a single infusion as palcebo.', 'armGroupLabels': ['Saline Solution']}]}, 'contactsLocationsModule': {'locations': [{'zip': '35294', 'city': 'Birmingham', 'state': 'Alabama', 'country': 'United States', 'facility': 'University of Alabama', 'geoPoint': {'lat': 33.52066, 'lon': -86.80249}}, {'zip': '94158', 'city': 'San Francisco', 'state': 'California', 'country': 'United States', 'facility': 'UCSF', 'geoPoint': {'lat': 37.77493, 'lon': -122.41942}}, {'zip': '32806', 'city': 'Orlando', 'state': 'Florida', 'country': 'United States', 'facility': 'Study site', 'geoPoint': {'lat': 28.53834, 'lon': -81.37924}}, {'zip': '55905', 'city': 'Rochester', 'state': 'Minnesota', 'country': 'United States', 'facility': 'Mayo Clinic', 'geoPoint': {'lat': 44.02163, 'lon': -92.4699}}, {'zip': '19104', 'city': 'Philadelphia', 'state': 'Pennsylvania', 'country': 'United States', 'facility': 'University of Pennsylvania', 'geoPoint': {'lat': 39.95238, 'lon': -75.16362}}, {'city': 'London', 'state': 'Ontario', 'country': 'Canada', 'facility': "Lawson Health Research Institute, St. Joseph's", 'geoPoint': {'lat': 42.98339, 'lon': -81.23304}}, {'city': 'Toronto', 'country': 'Canada', 'facility': 'Sunnybrook Health Sciences Centre', 'geoPoint': {'lat': 43.70643, 'lon': -79.39864}}, {'zip': 'WC1N 3BG', 'city': 'London', 'country': 'United Kingdom', 'facility': 'University College London', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}], 'overallOfficials': [{'name': 'George Stoica', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Bioclinica Research'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Alector Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}