Ignite Creation Date:
2025-12-25 @ 12:39 AM
Ignite Modification Date:
2026-02-19 @ 5:33 PM
Study NCT ID:
NCT06300567
Status:
NOT_YET_RECRUITING
Last Update Posted:
2024-03-12
First Post:
2024-03-02
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Assessment of Patients Not Concluded After Neonatal Screening of Cystic Fibrosis.
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'interventionBrowseModule': {'meshes': [{'id': 'D003625', 'term': 'Data Collection'}], 'ancestors': [{'id': 'D004812', 'term': 'Epidemiologic Methods'}, {'id': 'D008919', 'term': 'Investigative Techniques'}, {'id': 'D017531', 'term': 'Health Care Evaluation Mechanisms'}, {'id': 'D011787', 'term': 'Quality of Health Care'}, {'id': 'D017530', 'term': 'Health Care Quality, Access, and Evaluation'}, {'id': 'D011634', 'term': 'Public Health'}, {'id': 'D004778', 'term': 'Environment and Public Health'}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'RETROSPECTIVE', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 400}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'NOT_YET_RECRUITING', 'startDateStruct': {'date': '2024-05-01', 'type': 'ESTIMATED'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2024-03', 'completionDateStruct': {'date': '2026-11-02', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2024-03-07', 'studyFirstSubmitDate': '2024-03-02', 'studyFirstSubmitQcDate': '2024-03-02', 'lastUpdatePostDateStruct': {'date': '2024-03-12', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2024-03-08', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2026-05-02', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'lung Imaging', 'timeFrame': 'within 3 months after visit', 'description': 'abnormal lung Imaging score, assessing the area of the lung with bronchiectasis, bronchial wall thickening, mucus plug and air trapping'}], 'secondaryOutcomes': [{'measure': 'lung infection', 'timeFrame': 'within 2 weeks after visit', 'description': 'sputum bacterial colonization (number of Colony Forming Unit) of bacteria, fungi'}, {'measure': 'pancreatic function', 'timeFrame': 'within 2 weeks after visit', 'description': 'fecal elastase in µg/g of feces'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['CFTR-RD']}, 'descriptionModule': {'briefSummary': 'Assessment of the outcome of patients not concluded fir cystic fibrosis, either symptomatic patients or asymptomatic children detected by newborn screening for cystic fibrosis. The aim is to identify patients potentially at risk of progressing to the clinical spectrum of cystic fibrosis', 'detailedDescription': 'Among patients not concluded for the diagnosis of cystic fibrosis, some are carriers of CFTR-RD mutations, therefore at risk of progressing to a symptomatic form, and may already have a subclinical involvement that it is important to identify. Among patients who do not carry the CFTR-RD genotype, other patients may be at risk of developing symptoms, if they are carriers of an undetected variant or wrongly classified as non-pathogenic. It is important to detect them to prevent the onset of symptoms'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'children of adults not concluded for the diagnosis of Cystic fibrosis', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\npatients not concluded for the diagnosis of Cystic fibrosis either because of intermediate sweat test or because extensive genetic study has identified 2 vrainats at least 1 being not CF causing\n\n\\-\n\nExclusion Criteria:\n\n* patients carrying 2 CF causing variants of the CFTR gene\n* patients carrying only 1 or no variants of CFTR'}, 'identificationModule': {'nctId': 'NCT06300567', 'briefTitle': 'Assessment of Patients Not Concluded After Neonatal Screening of Cystic Fibrosis.', 'organization': {'class': 'OTHER', 'fullName': 'Hôpital Necker-Enfants Malades'}, 'officialTitle': 'Assessment of Patients Not Concluded After Neonatal Screening of Cystic Fibrosis, CFTR-RD Misdiagnosis.', 'orgStudyIdInfo': {'id': 'CFTR-RD misdiagnosis'}}, 'armsInterventionsModule': {'armGroups': [{'label': 'patients with unconcluded diagnosis for cystic fibrosis', 'description': '1. symptomatic patientscarrying 2 variants of CFTR, including at least 1 non CF causing\n2. patients not concluded at neonatal screening of Cystic Fibrosis', 'interventionNames': ['Other: data collection']}], 'interventions': [{'name': 'data collection', 'type': 'OTHER', 'description': 'collection of data of generated by "real world" follow up', 'armGroupLabels': ['patients with unconcluded diagnosis for cystic fibrosis']}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Hôpital Necker-Enfants Malades', 'class': 'OTHER'}, 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'Professor', 'investigatorFullName': 'Isabelle Sermet-Gaudelus', 'investigatorAffiliation': 'Hôpital Necker-Enfants Malades'}}}}