Ignite Creation Date:
2025-12-25 @ 2:03 AM
Ignite Modification Date:
2025-12-27 @ 9:12 AM
Study NCT ID:
NCT04702360
Status:
APPROVED_FOR_MARKETING
Last Update Posted:
2021-07-15
First Post:
2021-01-06
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
ELX/TEZ/IVA Expanded Access Program for Cystic Fibrosis (CF) Patients With at Least One F508del Mutation
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D003550', 'term': 'Cystic Fibrosis'}], 'ancestors': [{'id': 'D010182', 'term': 'Pancreatic Diseases'}, {'id': 'D004066', 'term': 'Digestive System Diseases'}, {'id': 'D008171', 'term': 'Lung Diseases'}, {'id': 'D012140', 'term': 'Respiratory Tract Diseases'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D007232', 'term': 'Infant, Newborn, Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C000706587', 'term': 'elexacaftor, ivacaftor, tezacaftor drug combination'}, {'id': 'C545203', 'term': 'ivacaftor'}]}}, 'protocolSection': {'designModule': {'studyType': 'EXPANDED_ACCESS', 'expandedAccessTypes': {'treatment': True}, 'nPtrsToThisExpAccNctId': 1}, 'statusModule': {'overallStatus': 'APPROVED_FOR_MARKETING', 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2021-07', 'lastUpdateSubmitDate': '2021-07-08', 'studyFirstSubmitDate': '2021-01-06', 'studyFirstSubmitQcDate': '2021-01-06', 'lastUpdatePostDateStruct': {'date': '2021-07-15', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2021-01-08', 'type': 'ACTUAL'}}, 'conditionsModule': {'conditions': ['Cystic Fibrosis']}, 'descriptionModule': {'briefSummary': 'The purpose of this program is to provide elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) to CF patients in critical need who are 6 to 11 years of age with at least one F508del mutation in response to unsolicited physician requests.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD'], 'maximumAge': '11 Years', 'minimumAge': '6 Years', 'eligibilityCriteria': 'Key Inclusion Criteria:\n\n* Patients with confirmed diagnosis of CF who have an F/any genotype\n* Prior treatment for at least 6 months with an approved alternative CFTR modulator (if the patient is eligible for such treatment)\n* Patients with percent predicted forced expiratory volume in 1 second (ppFEV1) \\<40 for a minimum of 60 consecutive days before the date of completion of the request form\n\nKey Exclusion Criteria:\n\n* Patients with severe hepatic impairment\n\nOther protocol-defined inclusion/exclusion criteria may apply'}, 'identificationModule': {'nctId': 'NCT04702360', 'briefTitle': 'ELX/TEZ/IVA Expanded Access Program for Cystic Fibrosis (CF) Patients With at Least One F508del Mutation', 'organization': {'class': 'INDUSTRY', 'fullName': 'Vertex Pharmaceuticals Incorporated'}, 'officialTitle': 'ELX/TEZ/IVA Expanded Access Program for Patients 6 Through 11 Years of Age With Cystic Fibrosis Who Have At Least One F508del Mutation', 'orgStudyIdInfo': {'id': 'VX20-445-905'}}, 'armsInterventionsModule': {'interventions': [{'name': 'ELX/TEZ/IVA', 'type': 'DRUG', 'otherNames': ['elexacaftor/tezacaftor/ivacaftor', 'VX-445/VX-661/VX-770'], 'description': 'Fixed-dose combination tablet for oral administration.'}, {'name': 'IVA', 'type': 'DRUG', 'otherNames': ['ivacaftor', 'VX-770'], 'description': 'IVA monotablet for oral administration.'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Vertex Pharmaceuticals Incorporated', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}