Ignite Creation Date:
2025-12-24 @ 11:14 PM
Ignite Modification Date:
2025-12-25 @ 8:50 PM
Study NCT ID:
NCT04301856
Status:
RECRUITING
Last Update Posted:
2021-03-22
First Post:
2020-02-21
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Response to CFTR Modulators in CF Patients Under 18 Years
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'interventionBrowseModule': {'meshes': [{'id': 'C000599212', 'term': 'lumacaftor, ivacaftor drug combination'}]}}, 'protocolSection': {'designModule': {'bioSpec': {'retention': 'SAMPLES_WITHOUT_DNA', 'description': 'Excess bronchial secretions and serum'}, 'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 600}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2020-01-01', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2021-03', 'completionDateStruct': {'date': '2026-01-01', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2021-03-19', 'studyFirstSubmitDate': '2020-02-21', 'studyFirstSubmitQcDate': '2020-03-05', 'lastUpdatePostDateStruct': {'date': '2021-03-22', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2020-03-10', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2025-07-01', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Lung Imaging', 'timeFrame': 'at initiation, as part of national guidelines', 'description': 'Lung structural injury assessed by Low Dose CT, as part of routine care'}, {'measure': 'Lung Imaging', 'timeFrame': 'at 1 year, as part of national guidelines', 'description': 'Lung structural injury assessed by Low Dose CT, as part of routine care'}, {'measure': 'Lung Imaging', 'timeFrame': 'at 3 years, as part of national guidelines', 'description': 'Lung structural injury assessed by Low Dose CT, as part of routine care'}, {'measure': 'Lung Imaging', 'timeFrame': 'at 5 years, as part of national guidelines', 'description': 'Lung structural injury assessed by Low Dose CT, as part of routine care'}], 'secondaryOutcomes': [{'measure': 'weight in kilogrammes', 'timeFrame': 'longitudinal monitoring of assessments carried out as part of routine care during 5 yrs', 'description': 'weight in kilogrammes (associated with a retrospective collection in the year prior to treatment)'}, {'measure': 'height in meters', 'timeFrame': 'longitudinal monitoring of assessments carried out as part of routine care during 5 yrs', 'description': 'height in meters (associated with a retrospective collection in the year prior to treatment)'}, {'measure': 'pubertal evolution', 'timeFrame': 'longitudinal monitoring of assessments carried out as part of routine care during 5 yrs', 'description': 'pubertal evolution (associated with a retrospective collection in the year prior to treatment)'}, {'measure': 'bronchial infectious exacerbations', 'timeFrame': 'longitudinal monitoring of assessments carried out as part of routine care during 5 yrs', 'description': 'bronchial infectious exacerbations (associated with a retrospective collection in the year prior to treatment)'}, {'measure': 'Forced Expiratory Volume in 1 second(FEV1)', 'timeFrame': 'longitudinal monitoring of assessments carried out as part of routine care during 5 yrs', 'description': 'Forced Expiratory Volume in 1 second(FEV1) in liter'}, {'measure': 'Forced Vital Capacity (FVC)', 'timeFrame': 'longitudinal monitoring of assessments carried out as part of routine care during 5 yrs', 'description': 'Forced Vital Capacity (FVC) in liter'}, {'measure': 'Force Expiratory Flow 50 (FEV50)', 'timeFrame': 'longitudinal monitoring of assessments carried out as part of routine care during 5 yrs', 'description': 'Force Expiratory Flow 50 (FEV50) in liter'}, {'measure': 'Forced Expiratory Flow 25-75 (FEV25-75)', 'timeFrame': 'longitudinal monitoring of assessments carried out as part of routine care during 5 yrs', 'description': 'Forced Expiratory Flow 25-75 (FEV25-75) in liter'}, {'measure': 'Residual Volume (RV)', 'timeFrame': 'longitudinal monitoring of assessments carried out as part of routine care during 5 yrs', 'description': 'Residual Volume (RV) in liter'}, {'measure': 'Total Pulmonary Capacity', 'timeFrame': 'longitudinal monitoring of assessments carried out as part of routine care during 5 yrs', 'description': 'Total Pulmonary Capacity in liter'}, {'measure': 'Lung Clearance Index - Lung Clearance Index', 'timeFrame': 'longitudinal monitoring of assessments carried out as part of routine care during 5 yrs', 'description': 'Lung clearance of nitrogen'}, {'measure': 'colonization of bronchial secretions', 'timeFrame': 'longitudinal monitoring of assessments carried out as part of routine care during 5 yrs', 'description': 'bacteria, fungi, mycobacteria'}, {'measure': 'quality of life questionnaire', 'timeFrame': 'longitudinal monitoring of assessments carried out as part of routine care during 5 yrs', 'description': 'CFQ questionnaire for children above 8 years: worse 0, better 100'}, {'measure': 'ENT quality of life questionnaire', 'timeFrame': 'longitudinal monitoring of assessments carried out as part of routine care during 5 yrs', 'description': 'SN-score: better score 1, worse 7'}, {'measure': 'Abdominal quality of life questionnaire', 'timeFrame': 'longitudinal monitoring of assessments carried out as part of routine care during 5 yrs', 'description': 'better score: 0, worse: 25'}, {'measure': 'liver ultrasound', 'timeFrame': 'longitudinal monitoring of assessments carried out as part of routine care during 5 yrs', 'description': 'liver ultrasound'}, {'measure': 'elastometry (data available in centers with the necessary equipment)', 'timeFrame': 'longitudinal monitoring of assessments carried out as part of routine care during 5 yrs', 'description': 'elastometry (data available in centers with the necessary equipment)'}, {'measure': 'sweat test', 'timeFrame': 'longitudinal monitoring of assessments carried out as part of routine care during 5 yrs', 'description': 'sweat collection'}, {'measure': 'serum and fecal pancreatic biological markers', 'timeFrame': 'longitudinal monitoring of assessments carried out as part of routine care during 5 yrs', 'description': 'immunoreactive trypsin, lipase, amylase, vitamin A and E, Prothrombin time, and fecal (fecal elastase'}, {'measure': 'bone biological markers', 'timeFrame': 'longitudinal monitoring of assessments carried out as part of routine care during 5 yrs', 'description': '25OHvitD, Ca, P, PTH, Osteocalcin, IgF1, IgF1BP3, CTX'}, {'measure': 'bone maturation', 'timeFrame': 'longitudinal monitoring of assessments carried out as part of routine care during 5 yrs', 'description': 'Zscore (in relation to height and sex and weight) (data available in centers with the necessary equipment)'}, {'measure': 'puberty', 'timeFrame': 'longitudinal monitoring of assessments carried out as part of routine care during 5 yrs', 'description': 'serum dosage of FSH, LH, Estradiol, testosterone Pelvic ultrasound if puberty initiated in girls'}, {'measure': 'intestine inflammation', 'timeFrame': 'longitudinal monitoring of assessments carried out as part of routine care during 5 yrs', 'description': 'fecal Calprotectine'}, {'measure': 'glycemic regulation', 'timeFrame': 'longitudinal monitoring of assessments carried out as part of routine care during 5 yrs', 'description': 'monitoring of glycemic dysregulation ( as routinely done in the centers)'}, {'measure': 'side effects: declarative collection and monitoring', 'timeFrame': 'longitudinal monitoring of assessments carried out as part of routine care during 5 yrs', 'description': 'declarative collection and monitoring'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Cystic Fibrosis in Children']}, 'referencesModule': {'references': [{'pmid': '41138737', 'type': 'DERIVED', 'citation': 'Sermet-Gaudelus I, Letierce A, Berteloot L, Bonnel AS, Chen Y, Makani P, Kelly-Aubert M, Kanoun F, Penalva L, Bouleghem N, Bihouee T, Bui S, Corvol H, Houdouin V, Mittaine M, Tatopoulos A, Weiss L, Wizla N, Kapel N, Bessou A, Tiddens HAWM, Caudri D, Reix P, Marguet C; MODUL-CF study group. Effect of elexacaftor-tezacaftor-ivacaftor on bronchial dilatations in adolescents with cystic fibrosis: a multicentre prospective observational study. Lancet Respir Med. 2025 Oct 22:S2213-2600(25)00248-6. doi: 10.1016/S2213-2600(25)00248-6. Online ahead of print.'}]}, 'descriptionModule': {'briefSummary': 'CFTR modulators should improve the prognosis of Cystic Fibrosis. Identifying patients under the age of 18 responding to CFTR modulators as well as detecting possible toxicity is an important medical objective given the potential side effects and the high cost of these molecules.\n\nThis observational follow-up cohort study is carried out as part of routine care.\n\nThe main objective is to assess the evolution of pulmonary structural impairment by low-dose CF scan at the end of the first year of CFTR modulator therapy.\n\nThe secondary objectives are to evaluate structural impairment at low dose scan at 3 years and 5 years of CFTR modulator treatment, the evolution of respiratory functional parameters, growth, puberty, lung infection, sweat test, quality of life and pancreatic function, as well as tolerance of modulators including liver toxicity.', 'detailedDescription': "Cystic fibrosis (CF) is a deadly disease. This is due to overinfected chronic obstructive pulmonary disease that progresses to end-stage respiratory failure. CFTR modulators should improve the prognosis of CF, as they may slow the progression of patients' lung disease. Assessing their impact in the paediatric population is becoming a major issue. Children and adolescents under the age of 18 are a target cohort because they have a lung disease that is still poorly developed. Early prescription of CFTR modulators is therefore a priority but requires evidence of absence of toxicity. Identifying patients under the age of 18 responding to CFTR modulators as well as detecting possible toxicity, is an important medical objective given the potential side effects and the high cost of these molecules.\n\nThe outcomes previously used in Phase III studies (FEV1, frequency of exacerbations, nutritional status) are insufficiently sensitive in this population.\n\nOther criteria need to be analyzed to identify the response to CFTR modulators in the short and medium term. The investigators hypothesize that the assessment of pulmonary structural impairment by low-dose lung CT-scan as part of routine care could be a much more sensitive criterion for the development of lung disease under CFTR modulators.\n\nThis observational follow-up cohort study is carried out as part of routine care. It does not involve a specific collection for research. Excess bronchial secretions and blood will be kept instead of being discarded in the event of a possible requalification for research.\n\nThe main objective is to assess the evolution of pulmonary structural impairment by low-dose CF scan at the end of the first year of CFTR modulator therapy The secondary objectives are to assess following criteria\n\n* Tolerance of modulators in this age group, including screening for bronchial reactivity at treatment, early liver toxicity\n* Longitudinal evolution of pulmonary structural impairment by low dose scan at 3 years and 5 years of CFTR modulator treatment\n* Evolution of respiratory functional parameters\n* Measurement by spirometry and plethysmography\n* Lung clearance index (if possible)\n* Longitudinal evolution of bacterial colonization, compared to the year prior to modulating treatment\n* Exacerbations: number, duration, days of antibiotics, hospitalizations, return to stable condition\n* Colonization of bronchial secretions\n* Changes in quality of life\n* Evolution of the sweat test\n* Longitudinal evaluation of pancreatic function\n* Longitudinal evaluation of growth and puberty compared to the year prior to CFTR modulator\n* Growth speed, and bone age\n* Bone mineralization, body composition (if possible)\n* Pubertal markers from 9 years in girls and 10 years in boys\n* Evaluation of glycemic dysregulation if present\n* Preservation of samples taken as part of routine care (serum, bronchial secretions) for possible research use"}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '18 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'Children with cystic fibrosis under the age of 18 eligible for CFTR modulator thérapies according to french national recommandations', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\nChildren with cystic fibrosis under the age of 18 under CFTR modulator therapy\n\nExclusion Criteria:\n\n* Patients with cystic fibrosis without indication for CFTR modulator therapy\n* Patients over the age of 18\n* Pregnant or lactating women'}, 'identificationModule': {'nctId': 'NCT04301856', 'acronym': 'MODUL-CF', 'briefTitle': 'Response to CFTR Modulators in CF Patients Under 18 Years', 'organization': {'class': 'OTHER', 'fullName': 'Societe Francaise de la Mucoviscidose'}, 'officialTitle': 'Evaluation of the Response to CFTR Modulators in Patients With Cystic Fibrosis Less Than 18 Years of Age', 'orgStudyIdInfo': {'id': '0011928'}}, 'armsInterventionsModule': {'armGroups': [{'label': 'CF children treated with CFTR modul', 'description': 'Cystic fibrosis patients under 18 years treated with CFTR modulators according to french health recommendations observational cohort study', 'interventionNames': ['Drug: CFTR Modulators']}], 'interventions': [{'name': 'CFTR Modulators', 'type': 'DRUG', 'otherNames': ['Orkambi, Kalydeko...'], 'description': 'CFTR modulators as recommended in routine care by the french health authorities', 'armGroupLabels': ['CF children treated with CFTR modul']}]}, 'contactsLocationsModule': {'locations': [{'zip': '75015', 'city': 'Paris', 'status': 'RECRUITING', 'country': 'France', 'contacts': [{'name': 'Isabelle Sermet-Gaudelus, MD PhD', 'role': 'CONTACT', 'email': 'isabelle.sermet@aphp.fr', 'phone': '0033687078403'}], 'facility': 'Sermet-Gaudelus Isabelle', 'geoPoint': {'lat': 48.85341, 'lon': 2.3488}}], 'centralContacts': [{'name': 'Stéphane Mazur, PhD', 'role': 'CONTACT', 'email': 'stephane.mazur@chu-lyon.fr', 'phone': '0033427855043'}, {'name': 'Anne-Sophie Bonnel, MD', 'role': 'CONTACT', 'email': 'asbonnel@ch-versailles.fr', 'phone': 'OO33144494887'}], 'overallOfficials': [{'name': 'Isabelle Sermet-Gaudelus, MD PhD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Société Francaise de la Mucoviscidose'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Societe Francaise de la Mucoviscidose', 'class': 'OTHER'}, 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'Professor Isabelle Sermet-Gaudelus', 'investigatorFullName': 'Isabelle SERMET-GAUDELUS', 'investigatorAffiliation': 'Societe Francaise de la Mucoviscidose'}}}}