Ignite Creation Date:
2025-12-24 @ 12:19 PM
Ignite Modification Date:
2026-01-01 @ 5:50 AM
Study NCT ID:
NCT05697861
Status:
RECRUITING
Last Update Posted:
2025-08-26
First Post:
2023-01-13
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Long-Term Follow-Up (LTFU) of Subjects Dosed With NTLA-2001
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D028227', 'term': 'Amyloid Neuropathies, Familial'}], 'ancestors': [{'id': 'D020271', 'term': 'Heredodegenerative Disorders, Nervous System'}, {'id': 'D019636', 'term': 'Neurodegenerative Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D017772', 'term': 'Amyloid Neuropathies'}, {'id': 'D010523', 'term': 'Peripheral Nervous System Diseases'}, {'id': 'D009468', 'term': 'Neuromuscular Diseases'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D028226', 'term': 'Amyloidosis, Familial'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}, {'id': 'D000686', 'term': 'Amyloidosis'}, {'id': 'D057165', 'term': 'Proteostasis Deficiencies'}]}}, 'protocolSection': {'designModule': {'bioSpec': {'retention': 'SAMPLES_WITHOUT_DNA', 'description': 'serum'}, 'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'CASE_ONLY'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 72}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2023-07-10', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-05', 'completionDateStruct': {'date': '2038-03', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-08-20', 'studyFirstSubmitDate': '2023-01-13', 'studyFirstSubmitQcDate': '2023-01-24', 'lastUpdatePostDateStruct': {'date': '2025-08-26', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2023-01-26', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2038-03', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Incidence of Treatment-related SAEs and Protocol-specified AESIs', 'timeFrame': 'up to 15 years', 'description': 'Long-term safety assessment of any treatment-related SAEs and protocol-specified AESIs'}], 'secondaryOutcomes': [{'measure': 'PD biomarkers for ATTR including serum TTR and serum prealbumin', 'timeFrame': 'up to 15 years', 'description': 'Long-term assessment of serum TTR and serum prealbumin'}]}, 'oversightModule': {'isUsExport': False, 'oversightHasDmc': False}, 'conditionsModule': {'conditions': ['Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy', 'Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy', 'Wild-Type Transthyretin Cardiac Amyloidosis']}, 'descriptionModule': {'briefSummary': 'This is a follow-up study of subjects who received NTLA-2001 in a previous clinical trial as an observational evaluation of the long-term effects of the investigational therapy.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'Participating sites in an Intellia-sponsored clinical study of NTLA-2001.', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n1. A subject has completed or discontinued from an Intellia-sponsored clinical study in which a complete or partial dose of NTLA-2001 was received.\n2. A subject has provided informed consent for the LTFU study.\n\nExclusion Criteria:\n\nNone'}, 'identificationModule': {'nctId': 'NCT05697861', 'briefTitle': 'Long-Term Follow-Up (LTFU) of Subjects Dosed With NTLA-2001', 'organization': {'class': 'INDUSTRY', 'fullName': 'Intellia Therapeutics'}, 'officialTitle': 'Long-Term Follow-Up of Subjects Treated With NTLA-2001', 'orgStudyIdInfo': {'id': 'ITL-2001-CL-999'}, 'secondaryIdInfos': [{'id': '2022-003405-30', 'type': 'EUDRACT_NUMBER'}]}, 'contactsLocationsModule': {'locations': [{'city': 'Paris', 'status': 'RECRUITING', 'country': 'France', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 48.85341, 'lon': 2.3488}}, {'city': 'Auckland', 'status': 'RECRUITING', 'country': 'New Zealand', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': -36.84853, 'lon': 174.76349}}, {'city': 'Umeå', 'status': 'RECRUITING', 'country': 'Sweden', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 63.82842, 'lon': 20.25972}}, {'city': 'London', 'status': 'RECRUITING', 'country': 'United Kingdom', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}], 'centralContacts': [{'name': 'Trial Manager at Intellia', 'role': 'CONTACT', 'email': 'medicalinformation@intelliatx.com', 'phone': '1-857-285-6200', 'phoneExt': 'Ext. 6'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Intellia Therapeutics', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}