Ignite Creation Date:
2025-12-25 @ 4:07 AM
Ignite Modification Date:
2026-02-26 @ 2:22 AM
Study NCT ID:
NCT02939820
Status:
APPROVED_FOR_MARKETING
Last Update Posted:
2024-05-20
First Post:
2016-10-18
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Expanded Access Protocol of Patisiran for Patients With Hereditary ATTR Amyloidosis (hATTR)
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'removedCountries': ['United States']}, 'conditionBrowseModule': {'meshes': [{'id': 'D028226', 'term': 'Amyloidosis, Familial'}, {'id': 'D028227', 'term': 'Amyloid Neuropathies, Familial'}, {'id': 'D017772', 'term': 'Amyloid Neuropathies'}, {'id': 'C567782', 'term': 'Amyloidosis, Hereditary, Transthyretin-Related'}, {'id': 'D000686', 'term': 'Amyloidosis'}], 'ancestors': [{'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}, {'id': 'D057165', 'term': 'Proteostasis Deficiencies'}, {'id': 'D020271', 'term': 'Heredodegenerative Disorders, Nervous System'}, {'id': 'D019636', 'term': 'Neurodegenerative Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D010523', 'term': 'Peripheral Nervous System Diseases'}, {'id': 'D009468', 'term': 'Neuromuscular Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C000606954', 'term': 'patisiran'}]}}, 'protocolSection': {'designModule': {'studyType': 'EXPANDED_ACCESS', 'nPtrsToThisExpAccNctId': 2}, 'statusModule': {'overallStatus': 'APPROVED_FOR_MARKETING', 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2024-05', 'lastUpdateSubmitDate': '2024-05-16', 'studyFirstSubmitDate': '2016-10-18', 'studyFirstSubmitQcDate': '2016-10-18', 'lastUpdatePostDateStruct': {'date': '2024-05-20', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2016-10-20', 'type': 'ESTIMATED'}}, 'conditionsModule': {'keywords': ['RNAi therapeutic', 'FAP', 'Familial Amyloid Polyneuropathies', 'TTR', 'Transthyretin', 'Amyloidosis'], 'conditions': ['TTR-mediated Amyloidosis', 'Amyloidosis, Hereditary', 'Amyloid Neuropathies, Familial', 'Familial Amyloid Polyneuropathies', 'Amyloid Neuropathies', 'Amyloidosis, Hereditary, Transthyretin-Related']}, 'referencesModule': {'references': [{'pmid': '31215818', 'type': 'DERIVED', 'citation': 'Miah KM, Hyde SC, Gill DR. Emerging gene therapies for cystic fibrosis. Expert Rev Respir Med. 2019 Aug;13(8):709-725. doi: 10.1080/17476348.2019.1634547. Epub 2019 Jun 27.'}], 'seeAlsoLinks': [{'url': 'https://ghr.nlm.nih.gov/condition/transthyretin-amyloidosis', 'label': 'https://ghr.nlm.nih.gov/condition/transthyretin-amyloidosis'}, {'url': 'https://www.nlm.nih.gov/medlineplus/amyloidosis.html', 'label': 'https://www.nlm.nih.gov/medlineplus/amyloidosis.html'}]}, 'descriptionModule': {'briefSummary': 'The purpose of this study is to provide expanded access of patisiran to patients with hereditary transthyretin-mediated amyloidosis (hATTR).', 'detailedDescription': 'Choosing to participate in an expanded access program is an important personal decision. Talk with your doctor and family members or friends about deciding to join a research study. To learn more about this study, please have your doctor contact the study research staff using the Contacts provided. For general information, see the links provided in More Information.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Male or female greater than or equal to 18 years of age\n* Have a diagnosis of hATTR\n* Meet Karnofsky performance status and Polyneuropathy Disability (PND) score requirements\n* Have adequate complete blood counts, liver function tests and coagulation tests\n\nExclusion Criteria:\n\n* Participated in an interventional hATTR amyloidosis clinical trial involving RNA interference (RNAi) therapeutics within the last 12 months\n* Are currently eligible to participate in or currently enrolled in an ongoing interventional hATTR amyloidosis clinical trial\n* Have inadequate cardiac function\n* Known primary amyloidosis (AL amyloidosis) or leptomeningeal amyloidosis\n* Have known serious comorbidities or considered unfit for the program by the investigator\n* Prior or planned liver or heart transplantation'}, 'identificationModule': {'nctId': 'NCT02939820', 'briefTitle': 'Expanded Access Protocol of Patisiran for Patients With Hereditary ATTR Amyloidosis (hATTR)', 'organization': {'class': 'INDUSTRY', 'fullName': 'Alnylam Pharmaceuticals'}, 'officialTitle': 'Expanded Access Protocol of Patisiran for Patients With Hereditary Transthyretin-Mediated Amyloidosis (hATTR Amyloidosis) With Polyneuropathy', 'orgStudyIdInfo': {'id': 'ALN-TTR02-007'}}, 'armsInterventionsModule': {'interventions': [{'name': 'patisiran (ALN-TTR02)', 'type': 'DRUG', 'description': 'patisiran (ALN-TTR02) administered by intravenous (IV) infusion'}]}, 'contactsLocationsModule': {'overallOfficials': [{'name': 'Medical Director', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Alnylam Pharmaceuticals'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Alnylam Pharmaceuticals', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}