[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100627038":3},{"organization":4,"outcomesModule":7,"designInfo":67,"detailedDescription":66,"studyPopulation":66,"armGroups":73,"interventions":88,"overallOfficials":66,"centralContacts":103,"locations":112,"responsibleParty":155,"collaborators":66,"id":157,"slug":158,"hasResults":159,"nctId":160,"briefTitle":161,"officialTitle":161,"acronym":162,"eligibilityCriteria":163,"healthyVolunteers":159,"sex":164,"minAge":165,"maxAge":166,"enrollmentInfo":167,"targetDuration":66,"studyType":170,"phases":171,"briefSummary":173,"conditions":174,"keywords":66,"overallStatus":176,"whyStopped":66,"lastUpdateSubmitDate":177,"lastUpdatePostDateStruct":178,"startDateStruct":181,"completionDateStruct":183,"leadSponsor":185,"locationsCount":186},{"fullName":5,"class":6},"Assistance Publique - Hôpitaux de Paris","OTHER",{"primaryOutcomes":8,"secondaryOutcomes":13,"otherOutcomes":66},[9],{"measure":10,"description":11,"timeFrame":12},"Difference in forced vital capacity decline between the 2 groups at one year.","Evaluate the efficacy of triple immunomodulatory therapy (prednisone, azithromycin and hydroxychloroquine) for 12 months in patients with ILD associated with a variant of a surfactant-related gene.","12 Months",[14,18,21,24,27,30,32,35,37,39,42,44,46,48,50,52,54,56,58,60,62,64],{"measure":15,"description":16,"timeFrame":17},"tolerance of the triple therapy","Clinical and biological tolerance (occurrence of an adverse effect during treatment), ECG (including QTc prolongation) (at 3, 6, 9, 12 months after randomization) (only HCQ or AZI patients) and ophthalmological (at 6 months and 12 months after randomization)","12 months",{"measure":19,"description":20,"timeFrame":17},"correlation between the respiratory, radiological and clinical functional response,","Thoracic CT scan (progression criteria \\[99\\]) and PFT (Pulmonary function tests) at one year after randomization",{"measure":22,"description":23,"timeFrame":17},"Quality of life of the patients","Quality of life questionnaire (SF-36, scale from 0 to 100, where 0 represents the poorest health and 100 represents the best possible health) (EORTC QLQ-C30, v3.0 : a validated patient-reported questionnaire for assessing quality of life in cancer patients.) at one year after randomization,",{"measure":15,"description":25,"timeFrame":26},"Clinical and biological tolerance (occurrence of an adverse effect during treatment), ECG (including QTc prolongation) at 6, months after randomization (only HCQ or AZI patients) and ophthalmological (at one year after randomization)","6 months",{"measure":15,"description":28,"timeFrame":29},"Clinical and biological tolerance (occurrence of an adverse effect during treatment), ECG (including QTc prolongation) at 3 months after randomization (only HCQ or AZI patients) and ophthalmological (at one year after randomization)","3 months",{"measure":19,"description":31,"timeFrame":26},"Thoracic CT scan (progression criteria \\[99\\]) and PFT (Pulmonary function tests) at 6 months after randomization",{"measure":22,"description":33,"timeFrame":34},"Quality of life questionnaire (SF-36,scale from 0 to 100, where 0 represents the poorest health and 100 represents the best possible health) (EORTC QLQ-C30, v3.0 : a validated patient-reported questionnaire for assessing quality of life in cancer patients.) at 9 months after randomization,","9 months",{"measure":22,"description":36,"timeFrame":26},"Quality of life questionnaire (SF-36 scale from 0 to 100, where 0 represents the poorest health and 100 represents the best possible health) (EORTC QLQ-C30, v3.0 :a validated patient-reported questionnaire for assessing quality of life in cancer patients. ) at 6 months after randomization,",{"measure":22,"description":38,"timeFrame":29},"Quality of life questionnaire (SF-36, scale from 0 to 100, where 0 represents the poorest health and 100 represents the best possible health) (EORTC QLQ-C30, v3.0 :a validated patient-reported questionnaire for assessing quality of life in cancer patients ) at 3 months after randomization,",{"measure":40,"description":41,"timeFrame":17},"Overall survival","Collection of vital status, lung transplantation, hospitalization for pulmonary and non-pulmonary causes and episodes of exacerbation at each visit until the end of follow-up 12 months after randomization",{"measure":43,"description":41,"timeFrame":17},"Transplant-free survival",{"measure":45,"description":41,"timeFrame":17},"Exacerbation free-survival",{"measure":47,"description":41,"timeFrame":17},"Hospitalization-free survival",{"measure":15,"description":49,"timeFrame":34},"Clinical and biological tolerance (occurrence of an adverse effect during treatment),",{"measure":15,"description":51,"timeFrame":17},"ECG (including QTc prolongation) at 12 months after randomization (only HCQ or AZI patients)",{"measure":15,"description":53,"timeFrame":17},"Ophthalmological (at one year after randomization)",{"measure":15,"description":55,"timeFrame":34},"ECG (including QTc prolongation) at , 9 months after randomization (only HCQ or AZI patients)",{"measure":15,"description":57,"timeFrame":26},"ECG (including QTc prolongation) at 6 months after randomization (only HCQ or AZI patients)",{"measure":15,"description":59,"timeFrame":29},"ECG (including QTc prolongation) at 3 months after randomization (only HCQ or AZI patients)",{"measure":40,"description":61,"timeFrame":34},"Collection of vital status, lung transplantation, hospitalization for pulmonary and non-pulmonary causes and episodes of exacerbation at each visit until the end of follow-up 9 months after randomization",{"measure":40,"description":63,"timeFrame":26},"Collection of vital status, lung transplantation, hospitalization for pulmonary and non-pulmonary causes and episodes of exacerbation at each visit until the end of follow-up 6 months after randomization",{"measure":40,"description":65,"timeFrame":29},"Collection of vital status, lung transplantation, hospitalization for pulmonary and non-pulmonary causes and episodes of exacerbation at each visit until the end of follow-up 3 months after randomization",null,{"allocation":68,"interventionModel":69,"interventionModelDescription":66,"primaryPurpose":70,"observationalModel":66,"timePerspective":66,"maskingInfo":71},"RANDOMIZED","PARALLEL","TREATMENT",{"masking":72,"maskingDescription":66,"whoMasked":66},"NONE",[74,82],{"label":75,"type":76,"description":77,"interventionNames":78},"Prednisone 10 mg\u002Fday -Hydroxychloroquine 400 mg\u002Fday - Azithromycin 250mg*3","EXPERIMENTAL","The experimental treatment will be the combination of:\n\n* Prednisone 10 mg\u002Fday (1 tablet\u002Fday)\n* Hydroxychloroquine 400 mg\u002Fday (2 tablets\u002Fday)\n* Azithromycin 250 mg x3\u002Fweek (3 tablets\u002Fweek) Route of administration: oral route Duration of treatment: 12 months Market authorization: yes Use in their market authorization indication: no",[79,80,81],"Drug: Azithromycin 250 mg x3\u002Fweek (3 tablets\u002Fweek)","Drug: Prednisone 10 mg\u002Fday","Drug: Hydroxychloroquine 400 mg\u002Fday",{"label":83,"type":84,"description":85,"interventionNames":86},"Standard of care","ACTIVE_COMPARATOR","Standard of care: any symptomatic treatment to interstitial lung disease. No other experimental or off-label treatment (such as ivacaftor) will be allowed during the study.",[87],"Drug: Active Comparator: Standard of care",[89,94,97,100],{"type":90,"name":91,"description":92,"armGroupLabels":93,"otherNames":66},"DRUG","Azithromycin 250 mg x3\u002Fweek (3 tablets\u002Fweek)","Route of administration: oral route Duration of treatment: 12 months Market authorization: yes Use in their market authorization indication: no",[75],{"type":90,"name":95,"description":92,"armGroupLabels":96,"otherNames":66},"Prednisone 10 mg\u002Fday",[75],{"type":90,"name":98,"description":92,"armGroupLabels":99,"otherNames":66},"Hydroxychloroquine 400 mg\u002Fday",[75],{"type":90,"name":101,"description":85,"armGroupLabels":102,"otherNames":66},"Active Comparator: Standard of care",[83],[104,109],{"name":105,"role":106,"phone":107,"phoneExt":66,"email":108},"Annabelle METOIS","CONTACT","0140257939","annabelle\u002Fmetois@aphp.fr",{"name":110,"role":106,"phone":66,"phoneExt":66,"email":111},"BORIE Raphaël","raphael.borie@aphp.fr",[113,129,142],{"facility":114,"status":66,"city":115,"state":66,"zip":116,"country":117,"countryCode":118,"cosmosGeoPoint":119,"geoPoint":124,"contacts":125},"Pneumologie Louis Pradel","Bron","69500","France","FR",{"type":120,"coordinates":121},"Point",[122,123],4.91303,45.73865,{"lat":123,"lon":122},[126],{"name":127,"role":106,"phone":66,"phoneExt":66,"email":128},"COTTIN Vincent","vincent.cottin@chu-lyon.fr",{"facility":130,"status":66,"city":131,"state":66,"zip":132,"country":117,"countryCode":118,"cosmosGeoPoint":133,"geoPoint":137,"contacts":138},"Pneumologie Institu Coeur Poumon","Lille","59037",{"type":120,"coordinates":134},[135,136],3.05512,50.63391,{"lat":136,"lon":135},[139],{"name":140,"role":106,"phone":66,"phoneExt":66,"email":141},"WEMEAU Lidwine","lidwibne.wemeau@chru-lille.fr",{"facility":143,"status":66,"city":144,"state":66,"zip":145,"country":117,"countryCode":118,"cosmosGeoPoint":146,"geoPoint":150,"contacts":151},"Pneumologie Pontchaillou","Rennes","35033",{"type":120,"coordinates":147},[148,149],-1.67431,48.11109,{"lat":149,"lon":148},[152],{"name":153,"role":106,"phone":66,"phoneExt":66,"email":154},"JOUNEAU Stéphane","stephane.jouneau@chu-rennes.fr",{"type":156,"investigatorFullName":66,"investigatorTitle":66,"investigatorAffiliation":66,"oldNameTitle":66,"oldOrganization":66},"SPONSOR","100627038","phase-2-immunomodulatory-treatment-of-interstitial-lung-disease-associated-with-surfactant-related-gene-variants-100627038",false,"NCT07443436","Immunomodulatory Treatment of Interstitial Lung Disease Associated With Surfactant Related Gene Variants","TIPS","Inclusion Criteria:\n\n1. Age ≥ 18 years and \\\u003C80 years\n2. Carrier of a variant classified as pathogenic or probably pathogenic or considered as eligible by the genetic multidisciplinary discussion in SFTPA1, SFTPA2, SFTPC, NKX2-1, SFTPB or two variants classified as pathogenic or probably pathogenic or considered as eligible by the genetic multidisciplinary discussion in ABCA3 or SFTPB\n3. ILD whatever the pattern corresponding to a volume \\> 10% of the total lung on a CT scan of less than 2 years\n\nExclusion Criteria:\n\n1. Contraindication to azithromycin and hydroxychloroquine and prednisone (details in section 6.2)\n2. Pregnancy and breastfeeding\n3. Female of childbearing potential\\* not willing to use reliable contraception from inclusion and during 22 months\n4. Desire to have a child within 22 months\n5. Enrolment to another interventional study (clinical trial on medicinal product, medical device and interventional research involving human participants not concerning health product)\n6. Subject deprived of their liberty or subject that may have insufficient power, intelligence, education, resources, strength, or other needed attributes to protect their own interests; or unable to explicitly give consent.\n7. No affiliation to any health insurance system\n8. Refusal to participate to the study","ALL","18 Years","80 Years",{"count":168,"type":169},30,"ESTIMATED","INTERVENTIONAL",[172],"PHASE2","Scientific justification : Variants in surfactant-related genes (SRG) explain approximately 6% of familial pulmonary fibrosis (FPF).\n\nThe pathophysiology is unknown and seems to involve endoplasmic reticulum stress in type 2 alveolar epithelial cells.\n\nVariable improvement in the prognosis of childhood and adult interstitial lung disease (ILD) associated with a variant of a SRG, initially reported to be lethal within months of diagnosis, has been observed since the consensual use of prednisone, azithromycin and hydroxychloroquine targeting endoplasmic reticulum stress, without demonstration of the efficacy of any of these treatments alone or in combination.\n\nThe investigators hypothesize that a treatment combining prednisone, azithromycin and hydroxychloroquine is safe and could improve the prognosis of adult patients with ILD associated with SRG variant.\n\nMain objective and primary endpoint : Main objective:\n\nEvaluate the efficacy of triple immunomodulatory therapy (prednisone, azithromycin and hydroxychloroquine) for 12 months in patients with ILD associated with a variant of a surfactant-related gene.\n\nPrimary endpoint:\n\nDifference in forced vital capacity decline between the 2 groups at one year.\n\nSecondary objectives and endpoints : Secondary objectives:\n\n1. tolerance of the triple therapy,\n2. correlation between the respiratory, radiological and clinical functional response,\n3. quality of life of the patients,\n4. overall survival, transplant-free survival, exacerbation free-survival, hospitalization-free survival\n\nSecondary endpoints:\n\n1. Clinical and biological tolerance (occurrence of an adverse effect during treatment), ECG (at 3, 6, 9, 12 months after randomization) (only HCQ or AZI patients) and ophthalmological (at one year after randomization)\n2. Thoracic CT scan and PFT at 6 months and one year after randomization\n3. Quality of life questionnaire (EORTC QLQ-C30, v3.0) at 3 months, 6 months, 9 months and one year after randomization,\n4. Collection of vital status, lung transplantation, hospitalization for pulmonary and non-pulmonary causes and episodes of exacerbation at each visit until the end of follow-up 12 months after randomization.\n\nDesign of the study : Multicenter, randomized, controlled, two-arm, parallel, open-label superiority study comparing triple immunomodulatory therapy (prednisone, azithromycin, and hydroxychloroquine) to standard of care\n\nCategory : Category 2\n\nPopulation of study participants: Patients aged over 18 years with ILD and SRG variant\n\nNumber of participants included : 30\n\nDesign of the study : Multicenter, randomized, controlled, two-arm, parallel, open-label superiority study comparing triple immunomodulatory therapy (prednisone, azithromycin, and hydroxychloroquine) to standard of care.",[175],"Interstitial Lung Disease","NOT_YET_RECRUITING","2026-09-17",{"date":179,"type":180},"2026-09-22","ACTUAL",{"date":182,"type":169},"2026-11-01",{"date":184,"type":169},"2027-11-01",{"name":5,"class":6},3]