Ignite Creation Date:
2025-12-24 @ 6:50 PM
Ignite Modification Date:
2026-01-02 @ 1:46 PM
Study NCT ID:
NCT05882357
Status:
COMPLETED
Last Update Posted:
2025-10-06
First Post:
2023-05-19
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Evaluation of Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) in Cystic Fibrosis (CF) Participants 12 to Less Than 24 Months of Age
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': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SEQUENTIAL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 70}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2023-06-27', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-10', 'completionDateStruct': {'date': '2025-09-04', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2025-10-03', 'studyFirstSubmitDate': '2023-05-19', 'studyFirstSubmitQcDate': '2023-05-19', 'lastUpdatePostDateStruct': {'date': '2025-10-06', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2023-05-31', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2025-09-04', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Part A: Observed Pre-dose Concentration (Ctrough) of ELX, TEZ, IVA, and their Relevant Metabolites', 'timeFrame': 'Day 1 up to Day 15'}, {'measure': 'Part A: Safety and Tolerability as Assessed by Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)', 'timeFrame': 'Day 1 up to Day 43'}, {'measure': 'Part B: Safety and Tolerability as Assessed by Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)', 'timeFrame': 'Day 1 up to Week 28'}], 'secondaryOutcomes': [{'measure': 'Part B: Observed Pre-dose Concentration (Ctrough) of ELX, TEZ, IVA, and their Relevant Metabolites', 'timeFrame': 'Day 15 up to Week 16'}, {'measure': 'Part B: Absolute Change in Sweat Chloride (SwCl)', 'timeFrame': 'From Baseline Through Week 24'}]}, 'oversightModule': {'isUsExport': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Cystic Fibrosis']}, 'descriptionModule': {'briefSummary': 'This study will evaluate the pharmacokinetics (PK), safety, tolerability, pharmacodynamics (PD), and efficacy of ELX/TEZ/IVA in CF subjects 12 to less than (\\<) 24 months of age.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD'], 'maximumAge': '24 Months', 'minimumAge': '12 Months', 'healthyVolunteers': False, 'eligibilityCriteria': 'Key Inclusion Criteria:\n\nParticipants who have at least 1 F508del mutation in the CF transmembrane conductance regulator (CFTR) gene or another ELX/TEZ/IVA-responsive CFTR mutation\n\nKey Exclusion Criteria:\n\nHistory of any illness or any clinical condition that, in the opinion of the investigator, might either confound the results of the study or pose an additional risk in administering study drug(s) to the participant\n\nOther protocol defined Inclusion/Exclusion criteria may apply.'}, 'identificationModule': {'nctId': 'NCT05882357', 'briefTitle': 'Evaluation of Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) in Cystic Fibrosis (CF) Participants 12 to Less Than 24 Months of Age', 'organization': {'class': 'INDUSTRY', 'fullName': 'Vertex Pharmaceuticals Incorporated'}, 'officialTitle': 'A Phase 3, Open-label Study Evaluating the Pharmacokinetics, Safety, and Tolerability of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis Subjects 12 to Less Than 24 Months of Age', 'orgStudyIdInfo': {'id': 'VX22-445-122'}, 'secondaryIdInfos': [{'id': '2023-503230-49-00', 'type': 'OTHER', 'domain': 'EU CT'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Part A', 'description': 'Participants will receive ELX/TEZ/IVA in the morning and IVA in the evening.', 'interventionNames': ['Drug: ELX/TEZ/IVA', 'Drug: IVA']}, {'type': 'EXPERIMENTAL', 'label': 'Part B', 'description': 'Participants will receive ELX/TEZ/IVA in the morning and IVA in the evening with the dose(s) to be based on the outcome of Part A.', 'interventionNames': ['Drug: ELX/TEZ/IVA', 'Drug: IVA']}], 'interventions': [{'name': 'ELX/TEZ/IVA', 'type': 'DRUG', 'otherNames': ['VX-445/VX-661/VX-770', 'elexacaftor/tezacaftor/ivacaftor'], 'description': 'Fixed-dose combination granules for oral administration.', 'armGroupLabels': ['Part A', 'Part B']}, {'name': 'IVA', 'type': 'DRUG', 'otherNames': ['VX-770', 'ivacaftor'], 'description': 'Granules for oral administration', 'armGroupLabels': ['Part A', 'Part B']}]}, 'contactsLocationsModule': {'locations': [{'city': 'Nedlands', 'country': 'Australia', 'facility': 'Telethon Kids Institute', 'geoPoint': {'lat': -31.98184, 'lon': 115.8073}}, {'city': 'Parkville', 'country': 'Australia', 'facility': "The Royal Children's Hospital", 'geoPoint': {'lat': -37.78333, 'lon': 144.95}}, {'city': 'South Brisbane', 'country': 'Australia', 'facility': "Queensland Children's Hospital", 'geoPoint': {'lat': -27.48034, 'lon': 153.02049}}, {'city': 'Westmead', 'country': 'Australia', 'facility': "The Children's Hospital at Westmead", 'geoPoint': {'lat': -33.80383, 'lon': 150.98768}}, {'city': 'Toronto', 'country': 'Canada', 'facility': 'The Hospital for Sick Children', 'geoPoint': {'lat': 43.70643, 'lon': -79.39864}}, {'city': 'Vancouver', 'country': 'Canada', 'facility': "British Columbia Children's Hospital", 'geoPoint': {'lat': 49.24966, 'lon': -123.11934}}, {'city': 'Copenhagen', 'country': 'Denmark', 'facility': 'Juliane Marie Center, Rigshospitalet', 'geoPoint': {'lat': 55.67594, 'lon': 12.56553}}, {'city': 'Berlin', 'country': 'Germany', 'facility': 'Charite Paediatric Pulmonology Department', 'geoPoint': {'lat': 52.52437, 'lon': 13.41053}}, {'city': 'Essen', 'country': 'Germany', 'facility': 'Kinderklinik III, Abt. fur Pneumologie', 'geoPoint': {'lat': 51.45657, 'lon': 7.01228}}, {'city': 'Hanover', 'country': 'Germany', 'facility': 'Medizinische Hochschule Hannover', 'geoPoint': {'lat': 52.37052, 'lon': 9.73322}}, {'city': 'Rotterdam', 'country': 'Netherlands', 'facility': "Erasmus Medical Center / Sophia Children's Hospital", 'geoPoint': {'lat': 51.9225, 'lon': 4.47917}}, {'city': 'Bern', 'country': 'Switzerland', 'facility': 'Inselspital - Universitaetsspital Bern', 'geoPoint': {'lat': 46.94809, 'lon': 7.44744}}, {'city': 'Zurich', 'country': 'Switzerland', 'facility': 'Kinderspital Zuerich', 'geoPoint': {'lat': 47.36667, 'lon': 8.55}}, {'city': 'Cardiff', 'country': 'United Kingdom', 'facility': 'Children and Young Adults Research Unit', 'geoPoint': {'lat': 51.48, 'lon': -3.18}}, {'city': 'Leeds', 'country': 'United Kingdom', 'facility': 'Leeds General Infirmary', 'geoPoint': {'lat': 53.79648, 'lon': -1.54785}}, {'city': 'Liverpool', 'country': 'United Kingdom', 'facility': "Alder Hey Children's NHS Foundation Trust", 'geoPoint': {'lat': 53.41058, 'lon': -2.97794}}, {'city': 'London', 'country': 'United Kingdom', 'facility': 'Great Ormond Street Hospital for Children', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}, {'city': 'London', 'country': 'United Kingdom', 'facility': 'Royal Brompton Hospital', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}, {'city': 'Southampton', 'country': 'United Kingdom', 'facility': 'Southampton General Hospital', 'geoPoint': {'lat': 50.90395, 'lon': -1.40428}}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO', 'description': 'Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/independent-research/clinical-trial-data-sharing'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Vertex Pharmaceuticals Incorporated', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}