Ignite Creation Date:
2025-12-26 @ 10:46 PM
Ignite Modification Date:
2025-12-26 @ 10:46 PM
Study NCT ID:
NCT07095712
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2025-07-31
First Post:
2025-05-12
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Personalized Antisense Oligonucleotide Therapy for A Single Participant With TARDBP ALS
Sponsor:
Organization:
Raw JSON
{'hasResults': False, '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'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1', 'PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 1}}, 'statusModule': {'overallStatus': 'ACTIVE_NOT_RECRUITING', 'startDateStruct': {'date': '2024-11-25', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-05', 'completionDateStruct': {'date': '2025-11', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-07-23', 'studyFirstSubmitDate': '2025-05-12', 'studyFirstSubmitQcDate': '2025-07-23', 'lastUpdatePostDateStruct': {'date': '2025-07-31', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2025-07-31', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2025-11', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Clinical Functioning', 'timeFrame': 'Baseline to 12 months', 'description': 'Change from baseline at 12-months post nL-TARD-001 administration in scores on Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R).'}, {'measure': 'Clinical Functioning', 'timeFrame': 'Baseline to 12 months', 'description': 'Change from baseline at 12-months post nL-TARD-001 administration in Slow Vital Capacity (SVC)'}, {'measure': 'Clinical Functioning', 'timeFrame': 'Baseline to 12 months', 'description': 'Change from baseline at 12-months post nL-TARD-001 administration in Edinburgh Cognitive and Behavioral ALS Screen (ECAS) score'}, {'measure': 'Clinical Functioning', 'timeFrame': 'Baseline to 12 months', 'description': 'Change from baseline at 12-months post nL-TARD-001 administration in ALS Cognitive Behavioral Screen (ALS-CBS) score'}, {'measure': 'Clinical Functioning', 'timeFrame': 'Baseline to 12 months', 'description': 'Change from baseline at 12-months post nL-TARD-001 administration in Amyotrophic Lateral Sclerosis Assessment Questionnaire 5 (ALSAQ-5)'}], 'secondaryOutcomes': [{'measure': 'Disease Biomarkers', 'timeFrame': 'Baseline to 12 months', 'description': 'Change from baseline at 12-months post nL-TARD-001 administration in serum/plasma and CSF neurofilament light chain levels'}, {'measure': 'Safety and Tolerability', 'timeFrame': 'Baseline to 12 months', 'description': 'Incidence and severity of adverse events'}, {'measure': 'Safety and Tolerability', 'timeFrame': 'Baseline to 12 months', 'description': 'Emergent abnormalities in physical exam (changes in appearance, skin, neck, ears, nose, throat, heart/lungs, abdomen, lymph nodes, and extremities compared to baseline)'}, {'measure': 'Safety and Tolerability', 'timeFrame': 'Baseline to 12 months', 'description': 'Emergent abnormalities in neurological exam (changes in mental status, gait, cerebellar, cranial nerve, motor, reflex, and sensations compared to baseline)'}, {'measure': 'Safety and Tolerability', 'timeFrame': 'Baseline to 12 months', 'description': 'Emergent abnormalities in laboratory analyses (results outside of normal range for CSF, chemistry, hematology, coagulation, and urinalysis)'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Amyotrophic Lateral Sclerosis']}, 'descriptionModule': {'briefSummary': 'This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for a single participant with amyotrophic lateral sclerosis (ALS) due to a pathogenic variant in TARDBP.', 'detailedDescription': 'This is an interventional study to evaluate the safety and efficacy of treatment with an individualized antisense oligonucleotide (ASO) treatment in a single participant with amyotrophic lateral sclerosis (ALS) due to a pathogenic variant in TARDBP'}, 'eligibilityModule': {'sex': 'FEMALE', 'stdAges': ['ADULT'], 'maximumAge': '49 Years', 'minimumAge': '49 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* Informed consent/assent provided by the participant (when appropriate), and/or participant's parent(s) or legally authorized representative(s)\n* Ability to travel to the study site and adhere to study-related follow-up examinations and/or procedures and provide access to participant's medical records\n* Genetically confirmed neurological disorder\n\nExclusion Criteria:\n\n* Participant has any condition that in the opinion of the Site Investigator would ultimately prevent the completion of study procedures\n* Use of an investigational medication within less than 5 half-lives of the drug at enrollment"}, 'identificationModule': {'nctId': 'NCT07095712', 'briefTitle': 'Personalized Antisense Oligonucleotide Therapy for A Single Participant With TARDBP ALS', 'organization': {'class': 'OTHER', 'fullName': 'n-Lorem Foundation'}, 'officialTitle': 'An Open-label Multicenter, Single Participant Study of an Experimental Antisense Oligonucleotide Treatment for Amyotrophic Lateral Sclerosis (ALS) Due to TARDBP (TDP-43) Genetic Mutation', 'orgStudyIdInfo': {'id': 'PRO00038978'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Open Label', 'interventionNames': ['Drug: nL-TARD-001']}], 'interventions': [{'name': 'nL-TARD-001', 'type': 'DRUG', 'description': 'Personalized antisense oligonucleotide', 'armGroupLabels': ['Open Label']}]}, 'contactsLocationsModule': {'locations': [{'zip': '10032', 'city': 'New York', 'state': 'New York', 'country': 'United States', 'facility': 'Columbia University, Irving Medical Center', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': '77030', 'city': 'Houston', 'state': 'Texas', 'country': 'United States', 'facility': 'Houston Methodist', 'geoPoint': {'lat': 29.76328, 'lon': -95.36327}}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'n-Lorem Foundation', 'class': 'OTHER'}, 'collaborators': [{'name': 'The Methodist Hospital Research Institute', 'class': 'OTHER'}, {'name': 'Columbia University', 'class': 'OTHER'}], 'responsibleParty': {'type': 'SPONSOR'}}}}