[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100283112":3},{"organization":4,"outcomesModule":7,"designInfo":157,"detailedDescription":163,"studyPopulation":156,"armGroups":164,"interventions":178,"overallOfficials":184,"centralContacts":189,"locations":198,"responsibleParty":221,"collaborators":224,"id":229,"slug":156,"hasResults":230,"nctId":231,"briefTitle":232,"officialTitle":233,"acronym":234,"eligibilityCriteria":235,"healthyVolunteers":230,"sex":236,"minAge":156,"maxAge":156,"enrollmentInfo":237,"targetDuration":156,"studyType":240,"phases":241,"briefSummary":243,"conditions":244,"keywords":156,"overallStatus":201,"whyStopped":156,"lastUpdateSubmitDate":246,"lastUpdatePostDateStruct":247,"startDateStruct":250,"completionDateStruct":252,"leadSponsor":254,"locationsCount":255},{"fullName":5,"class":6},"Hôpital Necker-Enfants Malades","OTHER",{"primaryOutcomes":8,"secondaryOutcomes":13,"otherOutcomes":156},[9],{"measure":10,"description":11,"timeFrame":12},"Forced Expiratory Volume in 1 second","Respiratory Function test","initiation, 7 days, 1 month, 6 months, 1 year and every 6 months",[14,16,18,20,23,26,29,33,36,39,42,45,48,51,54,57,61,64,68,71,74,77,80,84,88,92,95,99,103,107,111,115,119,122,126,129,133,137,141,145,148,150,153],{"measure":15,"description":11,"timeFrame":12},"Forced Vital Capacity",{"measure":17,"description":11,"timeFrame":12},"Forced Expiratory Flow 25-75",{"measure":19,"description":11,"timeFrame":12},"Residual Volume",{"measure":21,"description":22,"timeFrame":12},"elastase in sputum","elastase activity in UI\u002Fg of sputum",{"measure":24,"description":25,"timeFrame":12},"calprotectin in sputum","calprotectin in µg\u002Fg of sputum",{"measure":27,"description":28,"timeFrame":12},"IL-8 in sputum","IL-8 in µg\u002Fg of sputum",{"measure":30,"description":31,"timeFrame":32},"Calprotectin in blood","calprotectin in µg\u002Fml of blood","initiation, 1 month, 3 Months, 6 months and every 6 months",{"measure":34,"description":35,"timeFrame":32},"IL-8 in blood","IL-8 in µg\u002Fml of blood",{"measure":37,"description":38,"timeFrame":32},"tumor necrosis factor in sputum","tumor necrosis factor in µg\u002Fg of sputum",{"measure":40,"description":41,"timeFrame":32},"liver function test in blood","serum glutamate oxaloacetate transaminase in ui\u002Fml",{"measure":43,"description":44,"timeFrame":32},"liver function test\u002FSGPT in blood","serum glutamate pyruvate transaminase in ui\u002Fml",{"measure":46,"description":47,"timeFrame":32},"liver function test\u002Fbilirubin in blood","Bilirubin in mg\u002Fml",{"measure":49,"description":50,"timeFrame":32},"liver function test, gammaGT in blood","GammaGT in UI\u002Fml",{"measure":52,"description":53,"timeFrame":32},"creatine phosphokinase in blood","CPK in mg\u002Fml",{"measure":55,"description":56,"timeFrame":32},"Amylase in blood","amylase in mg\u002Fml",{"measure":58,"description":59,"timeFrame":60},"Impedancemetry","ambulatory measurement of body composition","initiation , 1 month, 3 Months, 6 months and every 6 months",{"measure":62,"description":63,"timeFrame":60},"Dynamometry","ambulatory measurement of quadriceps strength",{"measure":65,"description":66,"timeFrame":67},"Sweat test","chloride concentration in sweat","initiation ,1 month",{"measure":69,"description":70,"timeFrame":12},"proteomics of sweat","measurement of proteins in sweat",{"measure":72,"description":73,"timeFrame":12},"metabolomics of sweat","measurement of métabolites in sweat",{"measure":75,"description":76,"timeFrame":12},"proteomics of exhalate","measurement of proteins in exhaled air",{"measure":78,"description":79,"timeFrame":60},"sputum bronchial microorganism colonization","bacterial, fungi and viral colonization",{"measure":81,"description":82,"timeFrame":83},"Lung MRI","Lung Imaging evaluation : number of bronchiectasis, number of mucus plugs","initiation, at 1 year and every year",{"measure":85,"description":86,"timeFrame":87},"osteodensitometry","Bone mineralization body composition","initiation, 1 year, and every year",{"measure":89,"description":90,"timeFrame":91},"fecal elastase","elastase feces in µg\u002Fg feces","initiation, at 6 months and every year",{"measure":93,"description":94,"timeFrame":91},"fecal calprotectin","calprotectin, concentraion in feces in µg\u002Fg",{"measure":96,"description":97,"timeFrame":98},"Chest CT scan","Lung Imaging: % versuys normal of lung parenchuma with bronchiectasis, airway wall thickening, mucus plugs, air trapping","initiation, 3 years and 5 years",{"measure":100,"description":101,"timeFrame":102},"glycemic Holter","glycemia monitoring","initiation, 1 year and every year",{"measure":104,"description":105,"timeFrame":106},"abdominal ultrasonography","presence of liver hyperechogenicity, fibrosis, as assessed by the radiologist","treatment initiation, 1 year and every year",{"measure":108,"description":109,"timeFrame":110},"proton density fat fraction","magnetic resonance Imaging of the Pancreas","treatment initiation, 1 year",{"measure":112,"description":113,"timeFrame":114},"patient quality of life","Score tolerance of the treatment, perception of respiratory, digestive symptoms, energy, body image as assessed by the \"Cystic Fibrosis Questionnaire\" score a better quality of life is indicated by an increase in the score value. Minimum value is 0, maximum is 100.","initiation, 1 month, 6 months, 1 year and every 6 months",{"measure":116,"description":117,"timeFrame":118},"CFTR activity in nasal cells\u002Fchloride","Chloride transport in primary nasal cell cultures obtained by nasal brushing and study in Ussing chamber (µA\u002Fcm2)","initiation of treatment and repeated if cell culture failure",{"measure":120,"description":121,"timeFrame":118},"CFTR activity in nasal cells\u002Fbicarbonat","Bicarbonate transport in primary nasal cell cultures obtained by nasal brushing and study in Ussing chamber (µA\u002Fcm2)",{"measure":123,"description":124,"timeFrame":125},"CFTR activity in intestinal epithelium\u002Fchloride","Chloride transport in intestinal primary culture and study in Ussing chamber (µA\u002Fcm2)","initiation of treatment and repeated of cell culture failure",{"measure":127,"description":128,"timeFrame":125},"CFTR activity in intestinal epithelium\u002Fbicarbonate","Bicarbonate transport in intestinal primary culture and study in Ussing chamber (µA\u002Fcm2)",{"measure":130,"description":131,"timeFrame":132},"sweat evaporimetry","quantity of sweat produced afer bet-adrenergic stimulation after subcutaneous injection","initiation and 1 month",{"measure":134,"description":135,"timeFrame":136},"Elasto MRI","measurement of liver fibrosis by MRI","initiation and 1 year",{"measure":138,"description":139,"timeFrame":140},"Lung Clearance Index","Capacity of the lung to washout pure Oxygen,","initiation, 6 months, 1 year and every 6 months",{"measure":142,"description":143,"timeFrame":144},"proteomics in blood","measurement of proteins in blood","initiation, 7 days, 1 month, 6 months, 1 year and every year",{"measure":146,"description":147,"timeFrame":144},"metabolomics in blood","measurement of métabolites in blood",{"measure":149,"description":143,"timeFrame":144},"proteomic in urine",{"measure":151,"description":152,"timeFrame":144},"metabolomics in urine","measurement of métabolites in urine",{"measure":154,"description":155,"timeFrame":144},"Exhaled air composition","Volatile organic compounds in exhaled air",null,{"allocation":158,"interventionModel":159,"interventionModelDescription":156,"primaryPurpose":160,"observationalModel":156,"timePerspective":156,"maskingInfo":161},"NON_RANDOMIZED","PARALLEL","BASIC_SCIENCE",{"masking":162,"maskingDescription":156,"whoMasked":156},"NONE","This study is based upon the hypothesis that clinical response of cystic fibrosis patients to CFTR modulators is correlated to in vitro responses to these drugs of epithelial cells derived from the patients, as assessed by CFTR-dependent Chloride secretion. Epithelial cells will be derived either from nasal or rectal epithelia, and consist both of cultured cells and organoids. The drugs tested will be Ivacaftor, or Lumacaftor\u002FIvacaftor, according to patient's treatment. Results of these assays will be compared with response to treatment at 6 and 12 months, assessed by clinical response and in vivo assay of CFTR function.",[165,170,174],{"label":166,"type":6,"description":167,"interventionNames":168},"Cystic fibrosis, treated","Cystic fibrosis patients treated either by Ivacaftor or by the association Ivacaftor-Lumacaftor",[169],"Procedure: Nasal swab; rectal biopsy.",{"label":171,"type":6,"description":172,"interventionNames":173},"Cystic fibrosis, non treated","Cystic fibrosis patients, non treated by a CFTR modulator",[169],{"label":175,"type":6,"description":176,"interventionNames":177},"Non-Cystic fibrosis","Patients in whom cystic fibrosis diagnosis has been suspected, but excluded by physiological and genetic investigations",[169],[179],{"type":180,"name":181,"description":182,"armGroupLabels":183,"otherNames":156},"PROCEDURE","Nasal swab; rectal biopsy.","Nasal epithelial cells will be obtained by nasal swabs from patients of the three arms; intestinal epithelial cells will be obtained, by rectal biopsy, only from patients treated by CFTR modulators.",[171,166,175],[185],{"name":186,"affiliation":187,"role":188},"Aleksander Edelman, phD","APHP","STUDY_DIRECTOR",[190,195],{"name":191,"role":192,"phone":193,"phoneExt":156,"email":194},"Isabelle Sermet, MD, PhD","CONTACT","33 1 44 49 48 87","isabelle.sermet@aphp.fr",{"name":196,"role":192,"phone":156,"phoneExt":156,"email":197},"Jean-Louis Pérignon, MD, PhD","jean-louis.perignon@aphp.fr",[199],{"facility":200,"status":201,"city":202,"state":156,"zip":203,"country":204,"countryCode":205,"cosmosGeoPoint":206,"geoPoint":211,"contacts":212},"Necker Hospital","RECRUITING","Paris","75014","France","FR",{"type":207,"coordinates":208},"Point",[209,210],2.3488,48.85341,{"lat":210,"lon":209},[213,217,219],{"name":214,"role":192,"phone":215,"phoneExt":156,"email":216},"SERMET Isabelle, Professor","01 44 49 48 87","isabelle.sermet@nck.aphp.fr",{"name":218,"role":192,"phone":215,"phoneExt":156,"email":216},"LE Bourgeois Muriel, MD",{"name":214,"role":220,"phone":156,"phoneExt":156,"email":156},"PRINCIPAL_INVESTIGATOR",{"type":220,"investigatorFullName":222,"investigatorTitle":223,"investigatorAffiliation":5,"oldNameTitle":156,"oldOrganization":156},"Isabelle Sermet-Gaudelus","Professor",[225,227],{"name":226,"class":6},"Association Mucoviscidose-ABCF2",{"name":228,"class":6},"Vaincre la Mucoviscidose","100283112",false,"NCT02965326","Surrogate Markers of Response to New Therapies in Cystic Fibrosis Patients","Personalized Therapy of Cystic Fibrosis: Set-up of Response Markers","BIO-CFTR","Inclusion Criteria:\n\n* Cystic fibrosis patients treated by CFTR modulators (Ivacaftor or the association Ivacaftor-Lumacaftor)\n* Cystic fibrosis patients non treated by CFTR modulators\n* Patients in whom cystic fibrosis diagnosis has been suspected, but excluded by physiological and genetic investigations\n\nExclusion Criteria:\n\n* pregnant or lactating women\n* contraindication to nasal swab\n* contraindication to rectal biopsy","ALL",{"count":238,"type":239},75,"ESTIMATED","INTERVENTIONAL",[242],"NA","The purpose of this study is to determine which biological marker, or association of biological markers, best predict clinical response of cystic fibrosis patients to CFTR modulators.",[245],"Cystic Fibrosis","2024-03-08",{"date":248,"type":249},"2024-03-12","ACTUAL",{"date":251,"type":249},"2016-05",{"date":253,"type":239},"2026-10",{"name":5,"class":6},1]