[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"cftr-gene-mutation\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:cftr-gene-mutation":28},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,2,0,[8,45],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":25,"conditions":26,"keywords":29,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":33,"lastUpdatePostDateStruct":34,"startDateStruct":37,"completionDateStruct":39,"leadSponsor":41,"locationsCount":44},"100573559","phase-2-safety-tolerability-and-efficacy-study-of-arct-032-in-people-with-cystic-fibrosis-100573559",false,"NCT06747858","Safety, Tolerability and Efficacy Study of ARCT-032 in People With Cystic Fibrosis","A Phase 2, Open-label, Multiple Ascending-Dose Study to Evaluate the Safety, Tolerability and Efficacy of ARCT-032 in People With Cystic Fibrosis","LunairCF","Key Inclusion Criteria:\n\n1. Confirmed diagnosis of Cystic Fibrosis\n2. Not eligible for CFTR modulator therapy or not taking CFTR modulators for at least 60 days prior to dosing (e.g. due to intolerance, poor response, or lack of access to modulators)\n3. FEV1 between 40% to 100% (cohorts 1-3) and 45% to 90% (cohort 4, inclusive) of predicted value for age, sex and height\n\nExclusion Criteria:\n\n1. History of illness or medical condition that might pose an additional risk or may confound study results\n2. Recent moderate or severe hemoptysis\n3. Recent major surgery\n4. Solid organ or hematologic transplant\n5. Requirement of supplemental oxygen while awake or \\> 2L per minute while sleeping.\n6. Chronic maintenance systemic corticosteroids exceeding equivalent of daily 15 mg oral prednisone or 30 mg every other day\n7. Adequate liver and kidney function as determined by lab tests","ALL","18 Years",{"count":20,"type":21},33,"ESTIMATED","INTERVENTIONAL",[24],"PHASE2","ARCT-032-02 is a Phase 2, open-label, multicenter, multiple-ascending dose study of ARCT-032 in adults with CF who are not eligible for CFTR modulator therapy or are not taking CFTR modulators due to drug intolerance, poor response, or lack of access to modulators.",[27,28],"Cystic Fibrosis","CFTR Gene Mutation",[30,31],"CFTR","mRNA","RECRUITING","2026-05-06",{"date":35,"type":36},"2026-05-08","ACTUAL",{"date":38,"type":36},"2024-12-12",{"date":40,"type":21},"2027-06",{"name":42,"class":43},"Arcturus Therapeutics, Inc.","INDUSTRY",13,{"id":46,"slug":47,"hasResults":11,"nctId":48,"briefTitle":49,"officialTitle":49,"acronym":4,"eligibilityCriteria":50,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":51,"targetDuration":4,"studyType":22,"phases":53,"briefSummary":55,"conditions":56,"keywords":4,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":58,"lastUpdatePostDateStruct":59,"startDateStruct":61,"completionDateStruct":63,"leadSponsor":65,"locationsCount":68},"100502135","cystic-fibrosis-in-the-kidney-monitoring-the-effectiveness-of-elexacaftortezacaftorivacaftor-in-urine-after-a-short-pause-of-therapy-100502135","NCT05818319","Cystic Fibrosis in the Kidney: Monitoring the Effectiveness of Elexacaftor\u002Ftezacaftor\u002Fivacaftor in Urine After a Short Pause of Therapy","Inclusion Criteria:\n\n* Adult (age \\>17 years) CF patients.\n* Normal kidney function estimated by eGFR\\>90.\n* Adults capable of understanding and voluntarily consenting.\n\nExclusion Criteria:\n\n* Critical acute illness.\n* Severe lung disease (ppFEV1\\\u003C40%).\n* Adults not capable of understanding and voluntarily consenting.",{"count":52,"type":21},30,[54],"NA","In cystic fibrosis (CF) renal base excretion is impaired, due to mutations in the Cystic Fibrosis Transmembrane Regulator (CFTR) gene, since CFTR function is crucial in regulation of the kidney's HCO3- excretion.\n\nThe investigators suggest that challenged urine HCO3- excretion is a biomarker of CFTR function, which can be used to evaluate the extent of CFTR dysfunction and the possible correcting effects of CFTR modulating therapy.\n\nThis study aims to evaluate changes in challenged urine HCO3- excretion in CF patients, who are currently in treatment with the triple CFTR modulator combination therapy, Elexacaftor\u002Ftezacaftor\u002Fivacaftor (ETI), before, during, and after a short treatment pause.",[57,28],"Cystic Fibrosis (CF)","2025-02-17",{"date":60,"type":36},"2025-02-19",{"date":62,"type":36},"2023-06-01",{"date":64,"type":21},"2025-12",{"name":66,"class":67},"University of Aarhus","OTHER",1]