Ignite Creation Date:
2025-12-24 @ 11:49 PM
Ignite Modification Date:
2025-12-25 @ 9:43 PM
Study NCT ID:
NCT06977451
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2025-07-25
First Post:
2025-05-09
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Personalized Antisense Oligonucleotide for a Single Participant With CHCHD10 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': '2024-06-24', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-05', 'completionDateStruct': {'date': '2026-06', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-07-22', 'studyFirstSubmitDate': '2025-05-09', 'studyFirstSubmitQcDate': '2025-05-09', 'lastUpdatePostDateStruct': {'date': '2025-07-25', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2025-05-18', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2026-06', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Clinical Functioning', 'timeFrame': 'Baseline to 12 months', 'description': 'Change from baseline at 12-months post nL-CHCHD-001 administration in scores on Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R). A maximum score of 48 represents normal functioning, and the minimum score is 0.'}, {'measure': 'Motor Functioning', 'timeFrame': 'Baseline to 12 months', 'description': 'Change from baseline at 12-months post nL-CHCHD-001 administration in scores on Slow Vital Capacity (SVC) Performance.'}, {'measure': 'Motor Functioning', 'timeFrame': 'Baseline to 12 months', 'description': 'Change from baseline at 12-months post nL-CHCHD-001 administration in scores on Handheld Dynamometry (HHD).'}, {'measure': 'Clinical Functioning', 'timeFrame': 'Baseline to 12 months', 'description': 'Change from baseline at 12-months post nL-CHCHD-001 administration in scores on Edinburgh Cognitive and Behavioral ALS Screen (ECAS).'}, {'measure': 'Clinical Functioning', 'timeFrame': 'Baseline to 12 months', 'description': 'Change from baseline at 12-months post nL-CHCHD-001 administration in scores on ALS Cognitive Behavioral Screen (ALS-CBS)'}, {'measure': 'Clinical Functioning', 'timeFrame': 'Baseline to 12 months', 'description': 'Change from baseline at 12-months post nL-CHCHD-001 administration in scores on 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-CHCHD-001 administration in serum and cerebrospinal fluid 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'}, {'measure': 'Safety and Efficacy', 'timeFrame': 'Baseline to 12 months', 'description': 'Emergent abnormalities in neurological exam'}, {'measure': 'Safety and Efficacy', 'timeFrame': 'Baseline to 12 months', 'description': 'Emergent abnormalities in safety labs (CSF, chemistry, hematology, coagulation, and urinalysis)'}]}, 'oversightModule': {'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 CHCHD10'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT'], 'maximumAge': '63 Years', 'minimumAge': '63 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 stie 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': 'NCT06977451', 'briefTitle': 'Personalized Antisense Oligonucleotide for a Single Participant With CHCHD10 ALS', 'organization': {'class': 'OTHER', 'fullName': 'n-Lorem Foundation'}, 'officialTitle': 'An Open-Label Single Center, Single Participant Study of an Experimental Antisense Oligonucleotide Treatment for Amyotrophic Lateral Sclerosis (ALS) Due to an Arg15Leu Pathogenic Variant in CHCHD10', 'orgStudyIdInfo': {'id': 'AAAV1774'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Open Label', 'interventionNames': ['Drug: nL-CHCHD-001']}], 'interventions': [{'name': 'nL-CHCHD-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}}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'n-Lorem Foundation', 'class': 'OTHER'}, 'collaborators': [{'name': 'Columbia University', 'class': 'OTHER'}], 'responsibleParty': {'type': 'SPONSOR'}}}}