[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"cystic-fibrosis-cf\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:cystic-fibrosis-cf":28},{"pageToken":4,"total":5,"offset":6,"count":7,"results":8},null,43,0,25,[9,44,75,106,132,156,179,205,231,261,295,321,342,368,387,417,448,472,497,517,535,560,583,608,638],{"id":10,"slug":11,"hasResults":12,"nctId":13,"briefTitle":14,"officialTitle":15,"acronym":4,"eligibilityCriteria":16,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":25,"conditions":26,"keywords":29,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":32,"lastUpdatePostDateStruct":33,"startDateStruct":36,"completionDateStruct":38,"leadSponsor":40,"locationsCount":43},"100627585","phase-2-phase-2-study-to-assess-the-safety-and-efficacy-of-ang003-100627585",false,"NCT07450547","Phase 2 Study to Assess the Safety and Efficacy of ANG003","A Phase 2, Multicenter, Randomized, Active-controlled Study to Assess the Safety and Efficacy of ANG003 at Two Different Dose Levels in Subjects With Exocrine Pancreatic Insufficiency Due to Cystic Fibrosis","Inclusion Criteria:\n\n1. Male and female subjects aged ≥12 years from the date of informed consent in the US and aged ≥18 years in the EU\n2. Confirmed and documented diagnosis of CF\n3. Diagnosed with severe EPI as defined by a fecal elastase ≤50 μg\u002Fg stool measured at Screening by a central laboratory.\n4. Subject has controlled EPI and taking a stable dose of pancreatic enzyme replacement therapy (PERT) for 90 days prior to Screening.\n5. Adequate nutritional status measured by body mass index (BMI) defined by:\n\n   1. BMI ≥25th percentile for children aged 12-17 years\n   2. BMI ≥18.5 kg\u002Fm2 for ≥18 years of age\n\nExclusion Criteria:\n\n1. History of fibrosing colonopathy or recurring distal intestinal obstructive syndrome within 6 months of Screening.\n2. History of lung or liver transplant, listing for organ transplant or significant bowel resection within the last 6 months. Subjects with a history of resection that does not result in short bowel syndrome are eligible.\n3. Known hypersensitivity or other severe reaction to any ingredient of the investigational product (IP), Creon, or the stool marker (FD\\&C Blue #2).\n4. Any chronic diarrheal illness unrelated to pancreatic insufficiency, actively being treated for small intestinal bacterial overgrowth, requiring use of naso-gastric, J-tube, G-tube, and\u002For enteral feeding for the study duration, Clostridioides difficile infection within 6 months prior to Screening, or severe constipation defined as \\\u003C1 bowel movement\u002Fweek.","ALL","12 Years",{"count":20,"type":21},113,"ESTIMATED","INTERVENTIONAL",[24],"PHASE2","In this study, ANG003, a pancreatic enzyme replacement therapy (PERT; commonly called \"enzymes\"), is being investigated as a potential treatment for exocrine pancreatic insufficiency (EPI). People with EPI due to Cystic Fibrosis (CF) may be eligible to participate in this study. The primary objective of this study is to evaluate the safety of ANG003 and see if it works as well compared to Creon, an approved PERT.",[27,28],"Exocrine Pancreatic Insufficiency (EPI)","Cystic Fibrosis (CF)",[28,30],"EPI","RECRUITING","2026-07-01",{"date":34,"type":35},"2026-07-02","ACTUAL",{"date":37,"type":35},"2026-04-24",{"date":39,"type":21},"2027-07",{"name":41,"class":42},"Anagram Therapeutics, Inc.","INDUSTRY",26,{"id":45,"slug":46,"hasResults":12,"nctId":47,"briefTitle":48,"officialTitle":49,"acronym":50,"eligibilityCriteria":51,"healthyVolunteers":12,"sex":17,"minAge":52,"maxAge":4,"enrollmentInfo":53,"targetDuration":4,"studyType":55,"phases":4,"briefSummary":56,"conditions":57,"keywords":59,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":32,"lastUpdatePostDateStruct":66,"startDateStruct":67,"completionDateStruct":69,"leadSponsor":71,"locationsCount":74},"100616985","study-to-enable-new-diagnostics-for-pulmonary-microbes-in-people-with-cf-100616985","NCT07312734","Study to Enable New Diagnostics for Pulmonary Microbes in People With CF","Study to Enable New Diagnostics for Pulmonary Microbes in People With CF (SEND-CF)","SEND-CF","Inclusion Criteria:\n\n* ≥ 16 years of age on day of study visit\n* Documentation of CF Diagnosis\n* Able to expectorate sputum\n* Percent predicted FEV1 ≥ 30%\n\nExclusion Criteria:\n\n* History of solid organ transplantation\n* History of active malignancy (or treatment for malignancy) in 12 months prior to the study visit\n* Pregnant","16 Years",{"count":54,"type":21},300,"OBSERVATIONAL","Sputum culture has been the best approach to detect harmful bacteria in the lungs of people with cystic fibrosis (CF). With the widespread use of new CF therapies (like Trikafta and Alyftrak), it is more difficult for people with CF to produce sputum even though they still have harmful bacteria in their lungs. The SEND-CF study is being done to see if there are other ways to detect harmful bacteria in the lungs.",[28,58],"New Diagnostics",[60,61,62,63,64,65],"Cystic Fibrosis","CF","Breath collection","Specimen collection","Detection of pathogens","New diagnostics",{"date":34,"type":35},{"date":68,"type":35},"2026-03-30",{"date":70,"type":21},"2027-08-01",{"name":72,"class":73},"Chris Goss","OTHER",9,{"id":76,"slug":77,"hasResults":12,"nctId":78,"briefTitle":79,"officialTitle":79,"acronym":80,"eligibilityCriteria":81,"healthyVolunteers":12,"sex":17,"minAge":82,"maxAge":83,"enrollmentInfo":84,"targetDuration":4,"studyType":22,"phases":86,"briefSummary":88,"conditions":89,"keywords":91,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":32,"lastUpdatePostDateStruct":98,"startDateStruct":99,"completionDateStruct":101,"leadSponsor":103,"locationsCount":105},"100610105","gastrointestinal-response-of-pediatric-cystic-fibrosis-patients-on-mediterranean-diet-100610105","NCT07223255","Gastrointestinal Response of Pediatric Cystic Fibrosis Patients on Mediterranean Diet","MedDietCF","Inclusion Criteria:\n\n* Male and female pediatric patient with cystic fibrosis age 3 and older\n* Nutritional status defined as a BMI Z-score of at least -1 or above\n* Confirm diagnosis of CF defined by 2 CF causing mutations on genetic testing or sweat chloride greater than 60 mEq\u002FL\n* Children with pancreatic insufficient CF and on PERT\n* Children with pancreatic sufficient CF not on PERT\n* Child must be on a full, solids based diet\n* Family willing to child adhere to an exclusive Mediterranean style diet for a period of 6 months\n* Child must be able to follow-up at regular CF clinic visits and attend any additional study visits if necessary\n\nExclusion Criteria:\n\n* Children with malnutrition\n* Children who require nutritional supplementation via any type of feeding tube\n* Children with poorly controlled CF lung disease\n* Children with advanced CF liver disease\n* Children with a comorbid gastrointestinal disease such as celiac disease, Crohn's disease or other malabsorptive process to be reviewed by PI\n* Children with significant food allergies or other gastrointestinal allergy\n* Family is unwilling to adhere to prescribed dietary intervention","3 Years","18 Years",{"count":85,"type":21},20,[87],"NA","There is no study to date that has evaluated the impact or effect of a Mediterranean diet in children with CF. The goal of this study would be to help provide better guidance around questions the investigators, as CF care providers continue to receive from patients and families about how to best promote overall health in pediatric cystic fibrosis from a dietary perspective. Currently, the updated nutritional recommendations are variable and broad. Parents continue to search for more concrete guidance about how best to promote long-term health given the ever-increasing life expectancy of cystic fibrosis patients in this new area of advanced therapeutics. Given the changing landscape of the CF care in general, children are less likely to struggle with early life malnutrition, and it is becoming increasingly clear that high fat, high calorie diets are not beneficial nor are necessary for all children with CF.",[28,90],"Cystic Fibrosis Gastrointestinal Disease",[92,93,94,95,96,97],"cfmed","meddiet","meddiet cf","mediterannean diet","cystic fibrosis","CF with Mediterranean diet",{"date":34,"type":35},{"date":100,"type":21},"2026-09-01",{"date":102,"type":21},"2027-06",{"name":104,"class":73},"Dartmouth-Hitchcock Medical Center",2,{"id":107,"slug":108,"hasResults":12,"nctId":109,"briefTitle":110,"officialTitle":110,"acronym":4,"eligibilityCriteria":111,"healthyVolunteers":12,"sex":17,"minAge":83,"maxAge":112,"enrollmentInfo":113,"targetDuration":4,"studyType":55,"phases":4,"briefSummary":115,"conditions":116,"keywords":120,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":123,"lastUpdatePostDateStruct":124,"startDateStruct":125,"completionDateStruct":127,"leadSponsor":129,"locationsCount":131},"100591807","evaluation-of-ventilation-defects-downstream-of-mucus-plugs-in-patients-with-muco-obstructive-lung-disease-100591807","NCT06985225","Evaluation of Ventilation Defects Downstream of Mucus Plugs in Patients With Muco-Obstructive Lung Disease","Inclusion Criteria:\n\n* Adequate completion of informed consent process with written documentation\n* Patients 18 - 65 years old\n* • Physician diagnosis of muco-obstructive pulmonary disease, including cystic fibrosis, severe asthma, chronic obstructive pulmonary disease, or non-cystic fibrosis bronchiectasis for \\> 1 year\n* Able to perform reproducible spirometry according to ATS criteria based on clinical PFTs.\n\nExclusion Criteria:\n\n* Respiratory tract infection within the 4 weeks prior to Visit 1\n* Body mass index (BMI) \\> 30 at Visit 1\n* One-time doses such as intra-articular injections require a 4-week washout prior to Visit 1\n* ER visit related to pulmonary condition within the previous 4 weeks of Visit 1\n* Significant concomitant medical illness, including (but not limited to) heart disease, cancer, uncontrolled diabetes, other chronic lung diseases (determined by the Investigator.)\n* Resting O2 saturation \\\u003C90% with maximum supplemental O2 delivered by nasal canula\n* Positive urine pregnancy test\n* Participation in an intervention study (including bronchoscopy) or use of investigative drugs within the past 30 days or plans to enroll in such a trial during the study\n* Unable or unlikely to complete study assessments in the opinion of the Investigator\n* Study intervention poses undue risk to patient in the opinion of the Investigator\n* Conditions that will prohibit MRI scanning determined by the MRI safety screening.","65 Years",{"count":114,"type":21},8,"In this study, xenon MRI will be used to evaluate regional functional consequences of mucus plugs in the lungs of patients with muco-obstructive pulmonary disease. Mucus plugs will be identified using CT imaging, and xenon MRI will be used to evaluate ventilation and gas exchange impairments in regions of the lungs corresponding to the airways downstream of mucus plugs.",[117,118,119,28],"Severe Asthma","COPD","Non-CF Bronchiectasis",[121,122,117,118,119],"Xenon MRI","Mucus Plug","2026-06-26",{"date":32,"type":35},{"date":126,"type":35},"2025-06-01",{"date":128,"type":21},"2026-12",{"name":130,"class":73},"University of Kansas Medical Center",1,{"id":133,"slug":134,"hasResults":12,"nctId":135,"briefTitle":136,"officialTitle":136,"acronym":137,"eligibilityCriteria":138,"healthyVolunteers":12,"sex":17,"minAge":139,"maxAge":140,"enrollmentInfo":141,"targetDuration":4,"studyType":22,"phases":143,"briefSummary":144,"conditions":145,"keywords":146,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":148,"lastUpdatePostDateStruct":149,"startDateStruct":151,"completionDateStruct":153,"leadSponsor":154,"locationsCount":131},"100563491","personalized-mobile-health-platform-to-promote-physical-activity-in-adolescents-and-young-adults-with-cystic-fibrosis-100563491","NCT06616857","Personalized Mobile Health Platform to Promote Physical Activity in Adolescents and Young Adults With Cystic Fibrosis","NUDGE","Inclusion Criteria:\n\n1. 13-25 years old\n2. Has a verified CF diagnosis or CF-related disorder\n3. Medically stable (i.e., FEV1\\>30%, not experiencing a CF-related exacerbation)\n4. Speaks and reads English\n\nExclusion Criteria:\n\n1. Have a comorbidity limiting PA participation (e.g., neurological condition)\n2. Have a significant cognitive impairment that interferes with study completion\n3. Have any oxygen, CPAP or BiPAP requirement","13 Years","25 Years",{"count":142,"type":21},30,[87],"The goal of this clinical trial is to help adolescents and young adults between the ages of 13-25 with Cystic Fibrosis (CF), medically stable, able to speak and read English, and are not experiencing a CF - related exacerbation, who are already active to remain, or gradually encourage them to increase their levels of physical activity\n\nParticipants will be asked to utilize a smartphone program, called NUDGE that we have developed. NUDGE is a chatbot with evidence-based features known to help teens make progress toward health goal:\n\n* Set and review goals\n* Self-monitor progress\n* Provide feedback on goal attainment\n* Revise future goals",[28],[147],"physical activity","2026-06-22",{"date":150,"type":35},"2026-06-23",{"date":152,"type":35},"2025-02-18",{"date":128,"type":21},{"name":155,"class":73},"Nemours Children's Clinic",{"id":157,"slug":158,"hasResults":12,"nctId":159,"briefTitle":160,"officialTitle":160,"acronym":161,"eligibilityCriteria":162,"healthyVolunteers":12,"sex":17,"minAge":163,"maxAge":83,"enrollmentInfo":164,"targetDuration":4,"studyType":55,"phases":4,"briefSummary":166,"conditions":167,"keywords":168,"overallStatus":170,"whyStopped":4,"lastUpdateSubmitDate":171,"lastUpdatePostDateStruct":172,"startDateStruct":174,"completionDateStruct":175,"leadSponsor":177,"locationsCount":4},"100644126","home-air-pollution-in-children-with-cystic-fibrosis-study-100644126","NCT07665203","Home Air Pollution in Children With Cystic Fibrosis Study","HEROIC-CF","Inclusion Criteria:\n\n* Age 6 to 18 years old\n* Hispanic of any race or non-Hispanic white\n* Diagnosed with cystic fibrosis\n\nExclusion Criteria:\n\n* Cannot perform spirometry\n* Planning to move in next 12 months\n* Spends \\\u003C4 nights a week in one residence\n* Active smoking in the home","6 Years",{"count":165,"type":21},200,"Cystic Fibrosis (CF) is a devastating chronic pulmonary disease that continues to cause significant morbidity and mortality despite great advances in therapies. Hispanic children with CF have worse outcomes, including higher mortality and more severe pulmonary disease, than non-Hispanic white children with CF. It is not known why Hispanic children with CF have more severe disease as it is not explained by CFTR genetic severity, diagnosis age, or socioeconomic status. The health disparities have worsened, not improved, for Hispanic children with CF since the development of new disease-altering therapeutics, CFTR modulators. It is critical to determine what is contributing to lung disease severity in Hispanic children with CF. Non-genetic factors, including environmental exposures, are estimated to account for 50% of lung disease severity variability in CF. Air pollution exposure during early childhood is associated with lower pulmonary function in healthy children and severe lung disease in children with asthma. However, air pollution exposure is vastly understudied in other chronic pulmonary diseases of childhood, such as CF. Investigating air pollution exposure in CF may provide vital information about the drivers of health disparities in Hispanic children with CF and about the environmental exposures influencing lung disease severity across all children with CF. To investigate air pollution exposure in children with CF, the investigators have assembled a multidisciplinary team of international experts in air pollution exposure, CF lung disease, health disparities, and pulmonary microbiome. The investigators will use two phenomenally rich databases, the CF Foundation Patient Registry and the University of Washington Spatiotemporal Air Pollution Exposure Model, to investigate the first aim: 1A) To determine whether neighborhood-level ambient air pollution exposure during childhood differs between 1500 Hispanic and 8500 non-Hispanic white cwCF in the CF Foundation Patient Registry, and 1B) To determine if neighborhood-level ambient air pollution exposure is associated with lung disease severity in Hispanic and non-Hispanic white cwCF. Across six geographically diverse clinical research CF centers, the investigators will enroll 100 Hispanic and 100 non-Hispanic children with CF to investigate the following aims: 2) To assess differences in residential indoor and ambient air pollution exposures by ethnicity in 200 cwCF, as well as the association between such exposure and pulmonary function by ethnicity, 3) To investigate the association of indoor and ambient air pollution exposure on airway inflammation and microbiome diversity and composition in Hispanic and non-Hispanic white cwCF using metatranscriptomic RNA sequencing. The HEROIC-CF Study is poised to advance the knowledge of the effect of air pollution exposure on not only CF lung disease severity, but may be a model to understand environmental exposures on disease severity in other chronic pulmonary diseases of childhood.",[28],[96,169],"air pollution","NOT_YET_RECRUITING","2026-06-17",{"date":173,"type":35},"2026-06-24",{"date":100,"type":21},{"date":176,"type":21},"2031-04-30",{"name":178,"class":73},"Seattle Children's Hospital",{"id":180,"slug":181,"hasResults":12,"nctId":182,"briefTitle":183,"officialTitle":184,"acronym":4,"eligibilityCriteria":185,"healthyVolunteers":12,"sex":17,"minAge":83,"maxAge":186,"enrollmentInfo":187,"targetDuration":4,"studyType":22,"phases":189,"briefSummary":190,"conditions":191,"keywords":192,"overallStatus":170,"whyStopped":4,"lastUpdateSubmitDate":196,"lastUpdatePostDateStruct":197,"startDateStruct":199,"completionDateStruct":201,"leadSponsor":203,"locationsCount":131},"100605481","nutrition-supplement-for-cystic-fibrosis-100605481","NCT07163078","Nutrition Supplement for Cystic Fibrosis","A Medical Nutrition Supplement for Cystic Fibrosis","Inclusion Criteria:\n\n* Diagnosed with cystic fibrosis\n* Diagnosed with exocrine pancreatic insufficiency\n* 18 years old or older\n* Currently on modulator\n* Normal liver enzyme labs\n\nExclusion Criteria: :\n\n* Non-English-speaking participants\n* Acute health crisis\n* Persistent elevation of liver enzymes \\>6 months (E2) (ALT \\>80 U\u002FL)\n* History of liver abnormalities\n* If patients are currently taking Category A or Category B in the LiverTox categorization system\n* Recent vitamin D supplementation of 30 mcg\u002Fday or higher, vitamin E supplements of 200 IU\u002Fday or higher, or copper at 2 mg\u002Fday or higher\n* Any other concern by investigator that the subject is inappropriate for inclusion\n* Patients who is on a reduced dose of a CFTR modulator\n* Patients on azole antifungals (voriconazole, itraconazole, posaconzole, \\>7 days of fluconazole, etc.).\n* Patients who binge drink EtOH - for men more than 2 drinks\u002Fday, for women more than 1 drink\u002F day\n* Patients that are on other medications that are sensitive CYP3A4 substrates - such as tacrolimus for example\n* Patients on sensitive CYP3A4 substrates including but not limited to tacrolimus, sirolimus, and cyclosporine","50 Years",{"count":188,"type":21},60,[87],"The goal of this study is to learn if one nutrition supplement formula works better than a different formula in adults with cystic fibrosis. The main question being addressed is: Will certain atypical versions of certain nutrients outperform typical versions of these nutrients? This will be determined by examining blood measures of nutrient levels and\u002For indications of nutrient function indicators pre- and post-intervention. Participants will take the supplements for 6 weeks with a blood draw before and after that time.",[28],[60,193,194,195],"Vitamins","Copper","Choline","2026-06-05",{"date":198,"type":35},"2026-06-09",{"date":200,"type":21},"2026-06",{"date":202,"type":21},"2027-01",{"name":204,"class":73},"Ohio State University",{"id":206,"slug":207,"hasResults":12,"nctId":208,"briefTitle":209,"officialTitle":209,"acronym":210,"eligibilityCriteria":211,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":212,"targetDuration":4,"studyType":55,"phases":4,"briefSummary":213,"conditions":214,"keywords":217,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":223,"lastUpdatePostDateStruct":224,"startDateStruct":225,"completionDateStruct":227,"leadSponsor":229,"locationsCount":131},"100640399","pharmacokinetics-of-antibiotics-in-patients-with-cystic-fibrosis-trated-with-elexacaftortezacaftorivacaftor-eti-100640399","NCT07629986","Pharmacokinetics of Antibiotics in Patients With Cystic Fibrosis Trated With Elexacaftor\u002FTezacaftor\u002FIvacaftor (ETI)","PKCF","Inclusion Criteria:\n\n* Diagnosis of cystic fibrosis confirmed by sweat test and\u002For genetic testing\n* Treatment with elexacaftor\u002Ftezacaftor\u002Fivacaftor (ETI) for at least 3 months\n* Age 12 years or older\n* Patient informed and not objecting to participation; for minors, parents\u002Flegal guardians informed and not objecting to participation\n* Clinical indication for antibiotic therapy for pulmonary exacerbation or respiratory infection according to treating physician\n* Affiliation to a social security system\n\nExclusion Criteria:\n\n* Lung transplantation or heart-lung transplantation\n* Patients under guardianship or curatorship\n* Pregnant or breastfeeding women",{"count":142,"type":21},"Cystic fibrosis (CF) is associated with major pharmacokinetic and pharmacodynamic alterations affecting antibiotic exposure, including changes in absorption, distribution, metabolism, and elimination. Historically, these alterations justified the use of higher antibiotic doses in CF patients in order to achieve therapeutic concentrations and improve pulmonary outcomes.\n\nThe advent of highly effective CFTR modulators, particularly the triple combination elexacaftor\u002Ftezacaftor\u002Fivacaftor (ETI), has substantially improved pulmonary function, nutritional status, inflammatory burden, and quality of life in patients with CF. ETI therapy also appears to modify respiratory microbiology and reduce the frequency of pulmonary exacerbations.\n\nThese clinical and physiological improvements may alter antibiotic pharmacokinetics and pharmacodynamics in patients with CF, potentially making current high-dose antibiotic recommendations less appropriate for some patients. Since repeated exposure to high-dose antibiotics is associated with cumulative toxicities, particularly aminoglycoside-related ototoxicity and nephrotoxicity, reassessment of antibiotic dosing strategies is warranted.\n\nThe PKCF study is a multicenter, prospective, observational, non-interventional study designed to characterize the pharmacokinetic profiles of intravenous antibiotics administered during pulmonary exacerbations in adolescents and adults with cystic fibrosis receiving ETI therapy.",[28,215,216],"Pulmonary Exacerbation","Respiratory Infection Bacterial",[60,218,219,220,221,222,215],"CFTR modulators","Elexacaftor\u002FTezacaftor\u002FIvacaftor","Antibiotics","Pharmacokinetics","Therapeutic drug monitoring","2026-06-01",{"date":196,"type":35},{"date":226,"type":35},"2026-03-19",{"date":228,"type":21},"2028-05",{"name":230,"class":73},"Fondation Ildys",{"id":232,"slug":233,"hasResults":12,"nctId":234,"briefTitle":235,"officialTitle":236,"acronym":237,"eligibilityCriteria":238,"healthyVolunteers":12,"sex":17,"minAge":82,"maxAge":4,"enrollmentInfo":239,"targetDuration":4,"studyType":22,"phases":241,"briefSummary":243,"conditions":244,"keywords":245,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":251,"lastUpdatePostDateStruct":252,"startDateStruct":254,"completionDateStruct":256,"leadSponsor":258,"locationsCount":260},"100604378","phase-4-ensuring-access-to-optimal-therapy-in-cf-the-enact-study-100604378","NCT07148739","Ensuring Access to Optimal Therapy in CF: The ENACT Study","Ensuring Access to Optimal Therapy in Cystic Fibrosis: The ENACT Study","ENACT","Inclusion Criteria:\n\n* documentation of CF diagnosis per CFF diagnostic criteria and known CFTR genotype\n* age 2 years and older\n* ability to provide written informed consent and\u002For assent (by subject and\u002For legal guardian)\n* on a stable dose of triple combination CFTR modulator therapy for at least two weeks prior to Visit 1\n* clinically stable lung disease, defined as no documented acute decrease in FEV1 \\> 10%, OR use of additional antibiotics (intravenous \\[IV\\] or oral \\[PO\\]) within 4 weeks prior to screening\n\nExclusion Criteria:\n\n* recent significant unintentional weight loss, as determined by the investigator, in the 4 weeks prior to screening\n* pregnant or breastfeeding female\n* history of alcohol or substance abuse in the 6 months prior to screening\n* participation in a study involving an investigational intervention within 28 days (or 5 half-lives, whichever is longer) prior to screening\n* in the opinion of the Investigator, medical or psychiatric illness, or other conditions that would interfere with participation",{"count":240,"type":21},100,[242],"PHASE4","This clinical trial is examining the action and effects of several new drugs in the treatment of cystic fibrosis in children. In addition, several genetic factors are examined. The hope is that the ability to determine prior to treatment those individuals who will or will not respond to existing therapies will avoid needless risk of side effects and the high cost of a potentially ineffective treatment regimen. Understanding the way these drugs work in the body and the best way to study them is critical to expanding the use of these drugs to all patients with cystic fibrosis (CF).",[28],[60,61,246,247,248,249,250],"CFTR modulator","Pediatric CF patients","Elexacaftor","Tezacaftor","Ivacaftor","2026-05-29",{"date":253,"type":35},"2026-06-02",{"date":255,"type":35},"2025-06-10",{"date":257,"type":21},"2030-12",{"name":259,"class":73},"Arkansas Children's Hospital Research Institute",3,{"id":262,"slug":263,"hasResults":12,"nctId":264,"briefTitle":265,"officialTitle":265,"acronym":266,"eligibilityCriteria":267,"healthyVolunteers":268,"sex":17,"minAge":83,"maxAge":269,"enrollmentInfo":270,"targetDuration":4,"studyType":55,"phases":4,"briefSummary":271,"conditions":272,"keywords":4,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":286,"lastUpdatePostDateStruct":287,"startDateStruct":289,"completionDateStruct":291,"leadSponsor":293,"locationsCount":131},"100599366","routine-validation-and-reproducibility-testing-of-laboratory-assays-and-research-techniques-used-for-endocrine-cardiometabolic-and-musculoskeletal-disorder-research-vald-100599366","NCT07083557","Routine Validation and Reproducibility Testing of Laboratory Assays and Research Techniques Used for Endocrine, Cardiometabolic, and Musculoskeletal Disorder Research (VALD)","VALD","Inclusion Criteria:\n\n* ≥18 and ≤100 years of age\n* body mass index ≥16.0 and ≤60 kg\u002Fm2\n\nExclusion Criteria:\n\n* \\\u003C18 and \\>100 years of age\n* body mass index \\\u003C16.0 or \\>60 kg\u002Fm2\n* allergies, intolerances, or dietary restrictions to meal ingredients, vegans or vegetarians\n* use of medications or dietary supplements (e.g., anti-inflammatories, immune modulators, etc) that could interfere with the particular assay\u002Ftechniques being evaluated\n* engaged in regular structured exercise \\>150 min per week unless needed for validation of the assay\u002Ftechnique being evaluated\n* significant organ system dysfunction or diseases, except those that are sought for validation of the assay\u002Ftechnique being evaluated\n* alcohol use disorder as defined by the National Institute of Alcohol Abuse and Alcoholism or use of controlled substances unless alcohol use disorder is required for validation of the assay\u002Ftechnique being evaluated\n* pregnant women, persons who smoke, prisoners, and inability to grant voluntary informed consent.",true,"100 Years",{"count":240,"type":21},"The purpose of this research study is to validate (check the accuracy of) laboratory assays, intravenous catheter insertion, and equipment or devices and their reproducibility, which is necessary to perform high quality research on chronic diseases, nutrition, and metabolism (the process by which a substance is handled in the body) at the University of Missouri. As technology changes and uses new testing methods, it is necessary to compare results from old tests, equipment and devices and new tests, equipment, or devices and the reproducibility of these measurements to make sure the results are accurate. Reproducibility means performing the same test more than once to see if the same results can be achieved each time. This study will look at the validation and reproducibility of tests and laboratory assays in participants who are healthy or affected by relevant endocrine, cardiometabolic, and musculoskeletal disorders.",[273,274,275,276,277,278,279,280,281,282,28,283,284,285],"Obesity and Obesity-related Medical Conditions","Diabetes","Atherosclerotic Disease","Heart Failure","MASH","Sarcopenia","Osteoporosis","Hyperparathyroidism","Hypoparathyroidism","Ischemic Heart Disease","Chronic Kidney Disease(CKD)","Osteopenia","Cachexia","2026-05-05",{"date":288,"type":35},"2026-05-07",{"date":290,"type":21},"2027-01-01",{"date":292,"type":21},"2030-07-01",{"name":294,"class":73},"Bettina Mittendorfer",{"id":296,"slug":297,"hasResults":12,"nctId":298,"briefTitle":299,"officialTitle":300,"acronym":301,"eligibilityCriteria":302,"healthyVolunteers":268,"sex":17,"minAge":83,"maxAge":112,"enrollmentInfo":303,"targetDuration":4,"studyType":22,"phases":304,"briefSummary":306,"conditions":307,"keywords":308,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":312,"lastUpdatePostDateStruct":313,"startDateStruct":315,"completionDateStruct":317,"leadSponsor":319,"locationsCount":131},"100636725","early-phase-1-proof-of-principle-study-for-an-efficacy-trial-of-linaclotide-for-cystic-fibrosis-100636725","NCT07569419","Proof of Principle Study for an Efficacy Trial of Linaclotide for Cystic Fibrosis","A Randomised, Placebo-controlled Crossover Study Defining the Mode of Action of Linaclotide in Healthy Volunteers Using MRI","MODEL","Inclusion Criteria:\n\nParticipant is willing and able to give informed consent for participation in the study\n\nNot currently taking any medications (except for selective serotonin reuptake inhibitors, low dose tricyclic antidepressants, antihistamines, and oral contraceptive pill).\n\nAged between 18-60 years.\n\nAbility to conform to the study protocol, including overnight fasting, dietary and lifestyle restriction, administering linaclotide and placebo intervention, MRI scanning, consuming the rice pudding\u002Fblue dye meal, and rating stool frequency and appearance.\n\nExclusion Criteria:\n\nContraindication to MRI scanning (i.e. metallic implants, pacemakers, history of metallic foreign body in eye(s) and penetrating eye injury, unable to lie flat and relatively still for less than 5 minutes.)\n\nPregnancy, lactating, or planning pregnancy during the investigation declared by candidate.\n\nHistory declared by the candidate of pre-existing gastrointestinal disorder that may affect bowel function.\n\nReported history of previous resection of the oesophagus, stomach, or intestine (excluding appendix).\n\nIntestinal stoma.\n\nAny medical condition that may potentially compromise participation in the study e.g., known food intolerance to rice pudding, known contraindication to the oral administration of linaclotide or placebo.\n\nHas a body mass index (BMI) value less than 18.5 or greater than 35.\n\nWill not agree to follow dietary and lifestyle restrictions required.\n\nUnable to stop drugs known to alter GI motility including mebeverine, opiates, monoamine oxidase inhibitors, phenothiazines, benzodiazepines, calcium channel antagonists for the duration of the study.\n\nParticipants who are currently (or in the past 3 months) taking antibiotics or probiotics as these may impact GI function.\n\nParticipation in night shift work the week prior to the study day. Night work is defined as working between midnight and 6.00 AM.\n\nAnyone who in the opinion of the investigator is unlikely to be able to comply with the protocol e.g., cognitive dysfunction, chaotic lifestyle related to substance abuse.\n\nHaving taken part in a research study in the last 3 months involving invasive procedures or an inconvenience allowance\n\n\\-",{"count":43,"type":21},[305],"EARLY_PHASE1","Linaclotide is a medicine used to treat constipation and irritable bowel syndrome with constipation (IBS-C). It works by acting on the surface of the gut lining, where it increases the movement of salt and water into the bowel. This softens stools, makes them easier to pass, and can also reduce gut pain\n\nOne advantage of linaclotide is that, unlike some natural substances in the gut, it is stable and can act throughout the intestine. Studies in animals show that it has the strongest effect in the upper small intestine, but it may act in other parts of the bowel as well. In people, however, it is not yet clear whether linaclotide mainly works in the small intestine or in the large intestine (colon). Knowing this is important, because it could help the investigators understand whether linaclotide might also be useful in other conditions, such as cystic fibrosis, where the gut does not handle fluid properly.\n\nLinaclotide is taken as a capsule, but less than 1% is absorbed into the bloodstream. Instead, it stays in the gut, where it is broken down into smaller active parts. This means both the small intestine and colon may be exposed to its effects.\n\nUntil now, it has been hard to study this because traditional methods only measure one part of the gut at a time. A team at the University of Nottingham has developed MRI scanning methods that can safely and non-invasively measure water content in the small intestine and colon.\n\nThe aim of this pilot study is to use MRI in healthy volunteers to see exactly where linaclotide acts. This knowledge will help optimise future studies in conditions such as cystic fibrosis.",[28],[309,96,310,311],"linaclotide","gastrointestinal","MRI","2026-04-28",{"date":314,"type":35},"2026-05-06",{"date":316,"type":35},"2026-03-09",{"date":318,"type":21},"2026-10",{"name":320,"class":73},"University of Nottingham",{"id":322,"slug":323,"hasResults":12,"nctId":324,"briefTitle":325,"officialTitle":325,"acronym":4,"eligibilityCriteria":326,"healthyVolunteers":268,"sex":17,"minAge":327,"maxAge":4,"enrollmentInfo":328,"targetDuration":4,"studyType":55,"phases":4,"briefSummary":330,"conditions":331,"keywords":4,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":333,"lastUpdatePostDateStruct":334,"startDateStruct":336,"completionDateStruct":338,"leadSponsor":340,"locationsCount":260},"100561842","kidney-function-in-people-with-cystic-fibrosis-in-the-era-of-hemt-100561842","NCT06595420","Kidney Function in People With Cystic Fibrosis in the Era of HEMT","Inclusion Criteria:\n\n1. Outpatient CF Cohort\n\n   * Diagnoses of Cystic Fibrosis\n   * Age \\> 30 years old\n   * Able to provide informed consent\n2. Inpatient CF Cohort\n\n   * Diagnoses of Cystic Fibrosis\n   * Age \\> 7 years old\n   * Able to provide informed consent and assent (where applicable)\n   * 55 PwCF frequently hospitalized for a pulmonary exacerbation (\\>1 hospital admission in the prior 12 months)\n   * 55 PwCF sporadically hospitalized for a pulmonary exacerbation (no hospital admissions in the prior 12 months)\n   * Able to provide urine sample independently\n3. Healthy Controls\n\n   * Healthy, as per participant self-report\n   * Age between 30-50 years\n   * Able to provide informed consent\n\nExclusion Criteria:\n\n1. Outpatient CF Cohort\n\n   * History of any organ transplant\n   * History of immunodeficiency\n   * Previous or current cancer diagnoses\n   * Pregnant or breastfeeding\n   * On chronic dialysis\n   * Non-compliance (demonstrated by \\<2 visits during the 12 months before enrollment)\n2. Inpatient CF Cohort\n\n   * The initiation of intravenous antibiotic therapy after hospital admission before obtaining the first blood and urine sample\n   * History of any organ transplant\n   * History of immunodeficiency\n   * Previous or current cancer diagnoses\n   * Pregnant or breastfeeding\n   * On chronic dialysis\n3. Healthy Controls\n\n   * History or current kidney disease, organ transplantation, cancer, or any other chronic illness\n   * Current use of antibiotics\n   * Urinary symptoms or UTI (dysuria, frequency, urgency)\n   * Pregnant women\n   * Menstruating on the study visit day\n   * Blood relatives of PwCF","7 Years",{"count":329,"type":21},260,"The purpose of this study is to find out what causes kidney disease in people with CF. The investigators will study biomarkers in the blood and urine that can either predict who is at risk or detect kidney damage early before it becomes permanent. The study will compare these markers in people with CF over time and during the treatment of lung flare-ups. It will also compare the blood and urine samples obtained from people without CF. The comparison aims to better understand the impact of cystic fibrosis and its treatment on the kidneys, as well as to develop improved methods for preventing, diagnosing, and treating kidney issues associated with CF.",[28,283,332],"Acute Kidney Injury","2026-04-27",{"date":335,"type":35},"2026-05-04",{"date":337,"type":35},"2025-01-09",{"date":339,"type":21},"2027-12",{"name":341,"class":73},"University of Virginia",{"id":343,"slug":344,"hasResults":12,"nctId":345,"briefTitle":346,"officialTitle":347,"acronym":348,"eligibilityCriteria":349,"healthyVolunteers":12,"sex":17,"minAge":83,"maxAge":350,"enrollmentInfo":351,"targetDuration":4,"studyType":22,"phases":353,"briefSummary":354,"conditions":355,"keywords":358,"overallStatus":170,"whyStopped":4,"lastUpdateSubmitDate":359,"lastUpdatePostDateStruct":360,"startDateStruct":362,"completionDateStruct":364,"leadSponsor":366,"locationsCount":142},"100635937","phase-2-a-study-to-evaluate-safety-and-explore-efficacy-of-new-lipase-nhs7108-in-adult-participants-with-exocrine-pancreatic-insufficiency-100635937","NCT07559175","A Study to Evaluate Safety and Explore Efficacy of New Lipase NHS7108 in Adult Participants With Exocrine Pancreatic Insufficiency.","A Phase 2a, Randomized, Open-Label, Active-Controlled, Crossover Study to Evaluate Safety and Explore Efficacy of Different Doses of New Lipase NHS7108 Administered Orally in Participants With Exocrine Pancreatic Insufficiency- EPIC","EPIC","Inclusion Criteria: Participants are eligible to be included in the study only if all of the following criteria apply:\n\n1. Participant is a male or female between 18 to 85 years of age, inclusive, at the time of signing the informed consent.\n2. Participants with clinically confirmed\\* chronic established (not due to a transient clinical diagnosis, i.e., acute pancreatitis) EPI and with a clinical indication for PERT.\n3. CFA off PERT of \\\u003C80% at screening\n4. Normal body mass index (BMI) by age and sex to ensure participant's EPI is adequately controlled in the opinion of the investigator (BMI of at least 17.0 and no greater than 35.0 kg\u002Fm²)\n5. Standard-of-care medications are allowed (antibiotics, mucolytic agents, aerosols, antacids), if on stable doses for at least 1 month prior to baseline CFA and CNA assessments and must not be altered in dose or stopped during the study.\n6. Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) modulators are allowed if on stable doses for at least 3 months prior to randomization. Participants should not start taking CFTR modulators during the duration of the study.\n7. Contraceptive use by men and women must be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.\n\n   Male Participants:\n   * Non-sterilized male participants are eligible to participate if they agree to one of the following starting at screening and continuing throughout the clinical study period, and until the end-of-study safety follow-up: Must agree to stay abstinent (where abstinence is the preferred and usual lifestyle of the participant), OR male participants with a female partner of childbearing potential must agree to use highly effective contraception consisting of 2 forms of birth control (1 of which must be a barrier method). Male participants with a pregnant or breastfeeding partner must agree to remain abstinent from penile-vaginal intercourse or use a male condom during each episode of penile penetration. These requirements also apply to participants in a same sex relationship.\n   * Male participants must agree not to donate sperm starting at screening and continuing throughout the clinical study period, and until the end-of-study safety follow-up.\n\n   Female Participants:\n   * Female participants of childbearing potential (as defined in Appendix 4) are eligible to participate if they meet the following criteria: Agree not to become pregnant during the clinical study period and until the end-of-study safety follow-up. Have a negative serum pregnancy test at screening and a negative urine pregnancy test at Day 1. If heterosexually active, must agree to consistently use a form of highly effective birth control, in combination with a barrier method starting at screening and continuing throughout the clinical study period, and until the end-of-study safety follow-up, OR agree to stay abstinent (where abstinence is the preferred and usual lifestyle of the participant), starting at screening and continuing throughout the clinical study period, and until the end-of-study safety follow-up. These requirements do not apply to participants in a same sex relationship.\n   * Female participants of nonchildbearing potential are eligible to participate if one of the following conditions apply: Must have a confirmed clinical history of sterility (documented hysterectomy, bilateral salpingectomy, or bilateral oophorectomy, as confirmed by review of the participant's medical records, medical examination, or medical history interview). Must be postmenopausal as defined as: amenorrhea for at least 1 year prior to screening and a laboratory confirmed serum follicle-stimulating hormone (FSH) level ≥ 40 mIU\u002FmL. Female participants on hormone replacement therapy (HRT) and whose menopausal status is in doubt will be required to use 1 of the non-estrogen hormonal highly effective contraception methods from screening until at and continuing throughout the clinical study period, and until the end-of-study safety follow-up if they wish to continue their HRT during the study.\n   * Female participants must agree not to donate ova starting at screening and continuing throughout the clinical study period, and until the end-of-study safety follow-up.\n   * For WOCBP only, serum pregnancy test will be performed at screening and urine pregnancy tests will be performed at the timepoints outlined in the SoA.\n   * The Investigator is responsible for review of medical history, menstrual history, and recent sexual activity to decrease the risk for inclusion of a woman with an early undetected pregnancy.\n8. Participants are capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.\n9. Participants agree not to participate in another interventional study while participating in the study.\n\nExclusion Criteria:\n\nParticipants are excluded from the study if any of the following criteria apply:\n\n1. Female participants who are breastfeeding at the time of screening.\n2. Previous GI surgery, except for non-invasive neonatal or early childhood procedures such as correction of hypertrophic pyloric stenosis, surgical correction of Meckel's diverticulum, volvulus, or intestinal intussusception. Hernia repair, appendectomy, cesarean section, tubal ligation, hysterectomy, polypectomy, hemorrhoidectomy, or cholecystectomy are allowed if performed at least 2 years before randomization and have no impact on intestinal transit or absorption.\n3. Participants on enteral feeding.\n4. Participants with celiac disease.\n5. Known allergy or adverse reaction history to any component of NHS7108, Zenpep®, omega-3 oil, and\u002For to any other product administered during the study, including the blue dye.\n6. Concurrent clinically significant, at the discretion of the Investigator, hematological, renal, endocrine, pulmonary, cardiovascular, hepatic, psychiatric, neurologic, or allergic disease (including drug allergies, but excluding untreated, asymptomatic, and seasonal allergies and childhood asthma) or any other disorder that in the opinion of the Investigator may put the participant at greater safety risk, influence response to study intervention, or interfere with study assessments.\n7. Concurrent conditions having a clinically significant impact on GI motility function, with the exception of pancreatic insufficiency due to pancreatectomy or CP.\n8. Any significant clinical\u002Flaboratory\u002Fradiological sign of unstable or unexpectedly deteriorating respiratory disease during the study duration, at the discretion of the Investigator.\n9. Omega-3 supplements are prohibited during all the study periods and should be stopped at least 7 days prior to the baseline off-treatment fat absorption assessments.\n10. Participants starting new medications or changing dose within 1 month before baseline off-treatment screening assessments.\n11. Acute use of oral or intravenous antibiotics within 2 weeks prior to the first dose of study intervention.\n12. Treatment with any investigational drug or device\u002Ftreatment within the 30 days or 5 half-lives of the drug (whichever is longer) prior to first dose of study intervention.\n13. Participant has any clinically significant abnormalities in hematology, coagulation, clinical chemistry, or urinalysis at screening as judged by the Investigator OR as detailed below:\n\n    * Estimated glomerular filtration rate \\\u003C 60 mL\u002Fmin at screening visit.\n    * Total bilirubin \\> 1.5 × upper limit of normal (ULN) at screening visit.\n    * Serum alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \\> 2 × ULN at screening visit.\n14. Positive human immunodeficiency virus (HIV) antibody test.\n15. Presence of hepatitis B surface antigen (HBsAg) or hepatitis B core antibody (HBcAb) at screening or within 3 months prior to first dose of study intervention.\n16. Positive hepatitis C virus (HCV) antibody test result at screening or within 3 months prior to first dose of study intervention AND positive on reflex to hepatitis C RNA test.\n17. Concurrent drug or alcohol addiction that in the opinion of the Investigator would preclude participation and compliance with the study procedures.","85 Years",{"count":352,"type":21},66,[24],"The purpose of this study is to measure the safety and explore the efficacy of 4 different doses of the new lipase NHS7108 in participants with EPI. In this study, all participants will take NHS7108 daily for 14 days and a matching dose of standard-of-care, pancrelipase (Zenpep®) for 14 days according to Treatment Sequence assignment. Both NHS7108 and Zenpep® are oral capsules that will be taken with each of the daily 3 meals and 2 snacks. Participants will interrupt all of their usual pancrelipase\u002Fpancreatin treatment for up to 8 days during screening and for the entire 2 treatment periods, where participants will take either the new lipase NHS7108 or a matching dose of the standard-of-care pancrelipase (Zenpep®). Participants will be asked to stay in a setting that allows controlled diet and 72-hour stool collection for approximately 7 days during the screening period and again for approximately 7 days at the end of each treatment period. During these 3 supervised periods, participants will receive a standardized diet with a predefined amount of fat and protein, stools will be collected in special containers and during the last day of the treatment period, blood samples will be obtained to measure fat absorption. These are essential to ensure valid assessment of participants' fat and protein absorption. Outside the 3 supervised periods, participants will be provided with guidelines and recommendations to create their own home-controlled meals and snacks according to their preferences for the remainder of the study duration.\n\nNumber of Participants:\n\nThe aim is to have 56 participants completing the study. Assuming approximately 14% drop-out rate, approximately 66 participants will be randomized to study intervention.\n\nStudy Arms and Duration:\n\nThe total study duration for each participant will be about 100 days (approximately 14 weeks), including:\n\n* A screening period of up to approximately 28 days (might be extended up to a total of 56 days) prior to the first dose administration.\n* A crossover treatment period (2 treatment periods: approximately 14 days each, with no washout in between). For each treatment period, study intervention will be administered 5 times a day (with 3 main meals and 2 snacks). After completion of Treatment Period 1, the participant will receive and start the new treatment for Treatment Period 2.\n* An end of treatment\u002Fearly discontinuation visit within approximately 7 days of the last study intervention dose.\n* An end-of-study safety follow-up visit at 14 (±2) days after the last dose administration.\n\nVery low dose group: 10 mg NHS7108 (approximately 25,000 LU)\u002Fmain meal and snack; 25,000 LU Zenpep\u002Fmain meal and snack (50 mg NHS7108 \\[approximately 125,000 LU\\] per day; 125,000 LU Zenpep per day)\n\nLow dose group: 20 mg NHS7108 (approximately 50,000 LU)\u002Fmain meal and 10 mg NHS7108 (approximately 25,000 LU)\u002Fsnack; 50,000 LU Zenpep\u002Fmain meal and 25,000 LU Zenpep\u002Fsnack (80 mg NHS7108 \\[approximately 200,000 LU\\] per day; 200,000 LU Zenpep per day)\n\nMedium dose group: 40 mg NHS7108 (approximately 100,000 LU)\u002Fmain meal and 20 mg (approximately 50,000 LU) NHS7108\u002Fsnack; 100,000 LU Zenpep\u002Fmain meal and 50,000 LU Zenpep\u002Fsnack (160 mg \\[approximately 400,000 LU\\] NHS7108 per day; 400,000 LU Zenpep per day)\n\nHigh dose group: 60 mg NHS7108 (approximately 150,000 LU)\u002Fmain meal and 30 mg NHS7108 (approximately 75,000 LU)\u002Fsnack; 150,000 LU Zenpep\u002Fmain meal and 75,000 LU Zenpep\u002Fsnack (240 mg NHS7108 \\[approximately 600,000 LU\\] per day; 600,000 LU Zenpep per day).\n\nThe dose for participants \\\u003C 60 kg who are assigned to the high dose group will need to be weight-adjusted to ensure that they receive no more than 10,000 LU\u002Fkg\u002Fday.",[27,28,356,357],"Pancreatic Enzyme Abnormality","Pancreatic Diseases",[348],"2026-04-23",{"date":361,"type":35},"2026-04-30",{"date":363,"type":21},"2026-04",{"date":365,"type":21},"2027-09",{"name":367,"class":42},"Aimmune Nestlé Health Science US R&D, LLC",{"id":369,"slug":370,"hasResults":12,"nctId":371,"briefTitle":372,"officialTitle":372,"acronym":373,"eligibilityCriteria":374,"healthyVolunteers":12,"sex":17,"minAge":375,"maxAge":4,"enrollmentInfo":376,"targetDuration":4,"studyType":55,"phases":4,"briefSummary":377,"conditions":378,"keywords":4,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":359,"lastUpdatePostDateStruct":379,"startDateStruct":381,"completionDateStruct":383,"leadSponsor":385,"locationsCount":131},"100607755","magnify---pulmonary-magnetic-resonance-imaging-for-cystic-fibrosis-100607755","NCT07192679","MAGNIFY - Pulmonary Magnetic Resonance Imaging for Cystic Fibrosis","MAGNIFY","General Inclusion criteria\n\nFor eligibility into MAGNIFY, subjects should meet all of the following criteria:\n\n1. A confirmed clinical diagnosis of CF, consisting of 2 confirmed disease-causing CFTR mutations along with either positive sweat chloride (\\>60mmol\u002FL, measured before starting CFTR modulator therapy) or a clinical picture consistent with CF as judged by a senior CF physician. Patients will be under one of named regional CF centres above.\n2. Be able to attend the local facility for scans (Royal Hallamshire Hospital, Sheffield).\n\nFor eligibility into 129Xe-MRI and lung function (cohort 1,2 and 3)\n\n1. Aged 5 years and above\n2. FEV1 \\>30% predicted (best in the previous 6 months) For eligibility for cohort 1\n\n1\\. Previous participation in the MMAVIC study, with at least one prior visit where lung ventilation MRI was successfully measured.\n\nFor eligibility into cohort 4 for 1H MRI only:\n\n1\\. Aged between 1 and 5 years of age\n\nGeneral Exclusion criteria\n\nPatients who meet any of the following criteria will be excluded from the study. Further exclusions may be applied at the discretion of the principal investigators.\n\n1. Previous lung transplant.\n2. Infection with organisms of the Burkholderia cepacia complex, MRSA or Mycobacterium abscessus.\n3. Pregnancy.\n4. Resting SpO2 \\\u003C 90% in room air.\n5. Inability to comfortably lie supine for more than 60 minutes.\n6. Any contraindication(s) to MRI scanning as per the MRI questionnaire used in clinical practice by the University of Sheffield MRI unit, Royal Hallamshire Hospital.\n\nResearch visit (temporary) exclusion criteria\n\n1. Pulmonary exacerbation within 4 weeks as defined by no new treatments in that time, no clinically significant change in their symptoms or spirometry (as judged by attending physician).\n2. Pregnancy. Patients who become pregnant prior to consent or during the study can remain in the study. However, no research visits will take place during pregnancy.","1 Year",{"count":188,"type":21},"This research study is looking at new ways of measuring the function of the lungs in patients with cystic fibrosis. This study is using the most advanced methods for measuring lung function including 2 tests called hyperpolarised gas magnetic resonance imaging (HP MRI) and multiple breath washout (MBW), to better understand changes in the lungs over time.\n\nHP MRI involves taking pictures of the air in your lungs after breathing in a harmless gas (xenon). MBW is a breathing test used to calculate something called the lung clearance index (LCI).\n\nBy measuring these tests on the same day, alongside standard lung function tests, we aim to understand lung function in greater detail than ever before.",[28],{"date":380,"type":35},"2026-04-29",{"date":382,"type":35},"2024-02-21",{"date":384,"type":21},"2029-06-30",{"name":386,"class":73},"Sheffield Teaching Hospitals NHS Foundation Trust",{"id":388,"slug":389,"hasResults":12,"nctId":390,"briefTitle":391,"officialTitle":391,"acronym":392,"eligibilityCriteria":393,"healthyVolunteers":12,"sex":17,"minAge":83,"maxAge":4,"enrollmentInfo":394,"targetDuration":4,"studyType":22,"phases":396,"briefSummary":397,"conditions":398,"keywords":400,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":408,"lastUpdatePostDateStruct":409,"startDateStruct":411,"completionDateStruct":413,"leadSponsor":415,"locationsCount":131},"100617100","prevalence-of-exercise-induced-ventilatory-limitation-and-associated-factors-in-patients-with-cystic-fibrosis-receiving-elexacaftor-tezacaftor-ivacaftor-100617100","NCT07314229","Prevalence of Exercise-induced Ventilatory Limitation and Associated Factors in Patients With Cystic Fibrosis Receiving Elexacaftor-Tezacaftor-Ivacaftor","MUCOLIMEX","Inclusion Criteria:\n\n* Male or female\n* Adult aged 18 or over\n* Suffering from cystic fibrosis\n* Treated at the CRCM in Lille and Créteil\n* Treated by ETI\n* Be covered by social security\n* Be able to understand the requirements of the study, provide written informed consent, and comply with the study's data collection procedures\n\nExclusion Criteria:\n\n* Medical contraindication or inability to perform a stress test according to ERS recommendations\n\n  * Absolute contraindications\n  * Relative contraindications:\n* Exacerbation of the condition in the 4 weeks preceding the V1 visit (27).\n* Pregnant or breastfeeding women\n* Administrative reasons\n* Persons deprived of their liberty\n* Minors or protected adults\n* Persons who have refused or are unable to give informed consent\n* Persons in emergency situations",{"count":395,"type":21},130,[87],"Cystic fibrosis is a genetic disorder affecting the entire body and associated with respiratory exacerbations, impaired quality of life and reduced life expectancy. The therapeutic management of cystic fibrosis has been profoundly changed by the recent arrival of a combination of highly effective cystic fibrosis transmembrane conductance regulator (CFTR) modulators, Elexacaftor-Tezacaftor-Ivacaftor (ETI), which improve quality of life, respiratory function and reducing the number of exacerbations. The impact of these treatments on exercise adaptation has not been clearly identified.\n\nThe main objective is to estimate the prevalence of ventilatory reserve amputation during submaximal exercise testing assessed by the 6-minute walk test (6MWT) in patients with cystic fibrosis treated with ETIs.",[28,399],"Mucoviscidosis",[401,147,402,403,404,405,406,407],"Cystic fibrosis","CFTR modulator treatment","exercise limitation","respiratory physiology","ventilatory limitation","reduction in ventilatory reserve","dynamic distension","2026-04-21",{"date":410,"type":35},"2026-04-22",{"date":412,"type":35},"2025-12-17",{"date":414,"type":21},"2027-04-04",{"name":416,"class":73},"University Hospital, Lille",{"id":418,"slug":419,"hasResults":12,"nctId":420,"briefTitle":421,"officialTitle":422,"acronym":4,"eligibilityCriteria":423,"healthyVolunteers":12,"sex":17,"minAge":83,"maxAge":4,"enrollmentInfo":424,"targetDuration":4,"studyType":22,"phases":426,"briefSummary":428,"conditions":429,"keywords":431,"overallStatus":170,"whyStopped":4,"lastUpdateSubmitDate":439,"lastUpdatePostDateStruct":440,"startDateStruct":442,"completionDateStruct":444,"leadSponsor":446,"locationsCount":105},"100614154","phase-1-bacteriophages-for-adults-with-cystic-fibrosis-and-chronic-achromobacter-lung-infection-100614154","NCT07275905","Bacteriophages for Adults With Cystic Fibrosis and Chronic Achromobacter Lung Infection","A Phase 1, Open-label, Randomized, Pilot and Feasibility Trial to Evaluate the Safety and Tolerability of the Achromobacter-targeting Bacteriophage Cocktail, AchromoPhage, Among Persons With Cystic Fibrosis and Chronic Achromobacter Lung Infections: The AchromoPhage Trial","Inclusion Criteria:\n\n* In order to be eligible to participate in this study, an individual must meet all of the following criteria:\n\n  1. Adults of any gender, age 18 years or greater at the time of enrollment.\n  2. Weight of 40 kg or greater.\n  3. Diagnosed with cystic fibrosis (CF).\n  4. Stable respiratory symptoms within 30 days prior to screening.\n  5. Chronic Achromobacter respiratory infection, defined as isolation by culture of Achromobacter species from two or more respiratory, oral, and\u002For nasopharyngeal samples provided by the participant within 24 months before the date of pre-screening.\n  6. At least one Achromobacter isolate cultured from a respiratory, oral, and\u002For nasopharyngeal sample provided by the participant no more than 60 days before the date of planned enrollment must be susceptible to at least 1 phage in AchromoPhage cocktail using study assays.\n  7. FEV1 ≥ (greater than or equal to) 40% of predicted at time of screening\n  8. Ability and willingness to provide informed consent or, if applicable, the ability and willingness of legal guardian\u002Frepresentative to provide informed consent.\n  9. Willingness to comply with study procedures.\n  10. Ability to travel to the University of Pittsburgh or the University of California San Diego for phage administration and in-person visits.\n  11. Agreement not to enroll in other bacteriophage studies or receive bacteriophages for clinical care during the study, except in life-saving situations.\n  12. For females of reproductive potential, a negative urine pregnancy test at the time of consent (before randomization and dosing).\n  13. Willingness to use highly effective contraception or other preventive measures to avoid conception during the study and for 6 months after the last phage dose.\n\nNote, criteria 5 and 6 are the microbiologic criteria.\n\nExclusion Criteria:\n\n* An individual who meets any of the following criteria will be excluded from participation in this study:\n\n  1. Serious medical illness requiring systemic treatment or hospitalization within 30 days prior to screening (unless medically stable and approved by the PI).\n  2. Acute pulmonary exacerbation requiring systemic antibiotics within 30 days prior to screening.\n  3. Acute respiratory illness, including viral infection, within 30 days prior to screening.\n  4. Grade 3 or higher alanine aminotransferase (ALT) or aspartate aminotransferase (AST) at or within 30 days prior to screening, defined as ALT or AST values \\>5.0 x upper limit of normal (ULN).\n  5. Currently breastfeeding, pregnant, or planning to become pregnant within 6 months.\n  6. Hemoglobin \\\u003C 8 g\u002FdL\n  7. Absolute neutrophil count \\\u003C 1000 cells\u002FuL\n  8. Intolerance to inhaled therapies.\n  9. Known allergy or sensitivity to components of the AchromoPhage cocktail.\n  10. Any other condition that, in the PI's opinion, would interfere with the conduct of the study or would not be in the participant's best interest.",{"count":425,"type":21},12,[427],"PHASE1","The goal of this clinical trial is to learn if a new treatment called AchromoPhage is safe and well tolerated in adults with cystic fibrosis (CF) who have long-term lung infections caused by Achromobacter bacteria. AchromoPhage is a mixture of four naturally occurring viruses, called phages, that are designed to target and kill Achromobacter.\n\nThis study will include 12 participants. People will be randomly assigned to one of three groups to receive AchromoPhage in different ways: by inhalation only, by intravenous (IV) infusion only, or by inhalation followed by IV infusion.\n\nParticipants will:\n\n* Receive the study drug during clinic visits over a period of three weeks.\n* Provide blood, sputum, nasal, and oral samples so researchers can measure how the phages move through the body, how long they stay, and whether the body develops a response against them.\n* Complete breathing tests and quality-of-life questionnaires.\n\nThe main question this study will answer is whether AchromoPhage causes any serious or treatment-limiting side effects in the first 42 days after dosing. Researchers will also look at changes in lung function, quality of life, phage levels in the body, and how the treatment affects Achromobacter and other bacteria in the lungs.\n\nThe study is being run at the University of Pittsburgh (Pittsburgh, PA) and the University of California San Diego (San Diego, CA).",[28,430],"Achromobacter Infection",[432,433,434,435,436,437,96,438],"Bacteriophage Therapy","Phage Therapy","AchromoPhage","Multidrug-Resistant Infections","Lung Infection","Pulmonary Infection","Achromobacter","2026-04-02",{"date":441,"type":35},"2026-04-07",{"date":443,"type":21},"2026-05-01",{"date":445,"type":21},"2028-02",{"name":447,"class":73},"Ghady Haidar",{"id":449,"slug":450,"hasResults":12,"nctId":451,"briefTitle":452,"officialTitle":453,"acronym":4,"eligibilityCriteria":454,"healthyVolunteers":12,"sex":17,"minAge":163,"maxAge":83,"enrollmentInfo":455,"targetDuration":4,"studyType":22,"phases":457,"briefSummary":458,"conditions":459,"keywords":460,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":465,"lastUpdatePostDateStruct":466,"startDateStruct":467,"completionDateStruct":469,"leadSponsor":470,"locationsCount":131},"100632070","exercises-effect-on-muscle-strength-aerobic-capacity-and-respiratory-functions-in-cystic-fibrosis-100632070","NCT07508904","Exercises' Effect on Muscle Strength, Aerobic Capacity and Respiratory Functions in Cystic Fibrosis","Effects of Upper and Lower Limb Exercises on Muscle Strength, Aerobic Capacity and Respiratory Functions in Children With Cystic Fibrosis","Inclusion Criteria:\n\n* Children aged 6-18 years\n* Diagnosed with cystic fibrosis\n* Clinically stable\n* Able to perform exercise\n* Parental\u002Fguardian consent provided\n\nExclusion Criteria:\n\n* Other severe pulmonary diseases\n* Cardiovascular or orthopedic limitations\n* Surgery or hospitalization within last 1 month\n* Inability to follow instructions\n* Oxygen-dependent or ventilator-dependent patients",{"count":456,"type":21},22,[87],"This research aims to evaluate the effects of upper and lower limb exercises on muscle strength and pulmonary function in children diagnosed with CF. While aerobic training is a known component of CF management, resistance training focused on specific limb groups has gained attention for its additional benefits. Upper limb exercises may aid respiratory muscle endurance and thoracic mobility, enhancing pulmonary mechanics. In contrast, lower limb exercises (such as cycling or squats) are associated with improved oxygen consumption (VO₂ peak), enhanced mobility, and greater lower body strength.\n\nThis randomized clinical trial will be conducted using a non-probability convenience sampling technique. The study will take place at the pediatric cystic fibrosis centers of Gulab Devi Chest Hospital and The Children's Hospital, Lahore. The targeted population includes children aged 6 to 18 years who have been diagnosed with cystic fibrosis and referred to the physiotherapy department. The study duration will be ten months following the approval of the synopsis. Eligible participants will be children aged between 6 and 18 years, clinically diagnosed with cystic fibrosis, currently stable with no history of hospitalization or significant lung infection in the past month. They should be physically able to participate in exercise and capable of following instructions, with informed consent obtained from their parents or guardians. Children will be excluded if they suffer from other severe lung diseases, cardiovascular or orthopedic conditions that restrict exercise, recent surgery or hospitalization within the last month, or if they are unable to understand instructions. Those dependent on oxygen therapy or ventilator support at all times will also be excluded.\n\nThe primary outcome measures will include lung function assessed by spirometry (FVC, FEV1), sputum production recorded through a sputum diary, and aerobic capacity measured by the Incremental Shuttle Walk Test (ISWT). Muscle strength will be evaluated for both upper and lower limbs, using a handheld dynamometer for the upper limbs and the Sit-to-Stand Test for the lower limb",[28],[60,461,462,463,464],"Muscle Strength","Respiratory Function","Upper and Lower Limb Exercises","Pediatric Rehabilitation","2026-03-27",{"date":439,"type":35},{"date":468,"type":35},"2025-12-01",{"date":100,"type":21},{"name":471,"class":73},"Riphah International University",{"id":473,"slug":474,"hasResults":12,"nctId":475,"briefTitle":476,"officialTitle":477,"acronym":4,"eligibilityCriteria":478,"healthyVolunteers":12,"sex":17,"minAge":163,"maxAge":83,"enrollmentInfo":479,"targetDuration":4,"studyType":55,"phases":4,"briefSummary":481,"conditions":482,"keywords":484,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":489,"lastUpdatePostDateStruct":490,"startDateStruct":491,"completionDateStruct":493,"leadSponsor":495,"locationsCount":131},"100591765","physical-impairments-in-children-with-cystic-fibrosis-100591765","NCT06984679","Physical Impairments in Children With Cystic Fibrosis","Comparison of Dyspnea, Functional Capacity, Muscle Strength, Urinary Incontinence and Quality of Life Between Children and Adolescents With Cystic Fibrosis and Healthy Children","Inclusion Criteria for Children and Adolescents with Cystic Fibrosis:\n\n* To be between the ages of 6-18\n* To have been diagnosed with cystic fibrosis\n* To be clinically stable for at least 3 weeks\n* To have the necessary cooperation for the measurements\n* To volunteer to participate in the study\n\nInclusion Criteria for Healthy Children and Adolescents:\n\n* To be between the ages of 6-18\n* To be in a similar average and ratio with the group of children and adolescents with cystic fibrosis in terms of age and gender\n* To have the necessary cooperation for the measurements\n* To volunteer to participate in the study\n\nExclusion Criteria for Children and Adolescents with Cystic Fibrosis:\n\n* Having any orthopedic, neurological, psychological or cardiovascular problem that may prevent the measurements from being performed in the last 6 months\n* Smoking or quitting smoking\n\nExclusion Criteria for Healthy Children and Adolescents:\n\n* Having any orthopedic, neurological, psychological or cardiovascular problem that may prevent the measurements from being performed in the last 6 months\n* Having any chronic disease\n* Smoking or quitting smoking",{"count":480,"type":21},50,"It is aimed to reveal impairments regarding urinary incontinence, dyspnea, muscle strength, functional capacity or quality of life in children and adolescents with cystic fibrosis compared to healthy children and adolescents.",[28,483],"Cystic Fibrosis in Children",[60,485,486,461,487,488],"Urinary Incontinence","Dyspnea","Quality of Life","Exercise Test","2026-03-23",{"date":465,"type":35},{"date":492,"type":35},"2025-09-15",{"date":494,"type":21},"2027-03-03",{"name":496,"class":73},"Izmir Democracy University",{"id":498,"slug":499,"hasResults":12,"nctId":500,"briefTitle":501,"officialTitle":501,"acronym":502,"eligibilityCriteria":503,"healthyVolunteers":12,"sex":17,"minAge":52,"maxAge":4,"enrollmentInfo":504,"targetDuration":4,"studyType":55,"phases":4,"briefSummary":505,"conditions":506,"keywords":4,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":226,"lastUpdatePostDateStruct":508,"startDateStruct":510,"completionDateStruct":512,"leadSponsor":514,"locationsCount":516},"100630202","understanding-inflammation-infection-and-interventions-in-severe-exacerbations-of-cystic-fibrosis-100630202","NCT07484607","Understanding Inflammation, InFection and Interventions in Severe Exacerbations of Cystic Fibrosis","UNIFIED-CF","Inclusion Criteria:\n\n1. Confirmed diagnosis of cystic fibrosis (CF), defined as presence of two pathogenic CF-causing CFTR mutations AND clinical features consistent with a diagnosis of CF, OR presence of at least one pathogenic CF-causing CFTR mutation AND sweat chloride (before use of CFTR modulators) \\>60mmol\u002FL AND clinical features consistent with a diagnosis of CF.\n2. Receiving care from a UK Adult Cystic Fibrosis Centre taking part in the study.\n3. EITHER:\n\n   • Have had at least 1 previous exacerbation of CF lung disease, treated with intravenous antibiotics, in the previous 12 months.\n\n   OR\n\n   • Enrolled in the CF-Tracker study (IRAS ID 338539) within the last 24 months (dated from date of completion of baseline Tracker visit)\n4. In case of treatment for an exacerbation, likely to be treated with a ß-lactam or an anti-pseudomonal penicillin, combined with tobramycin or colistin, per CF Trust and NICE guidelines for 1st-line CF therapies.\n5. Able to produce sputum (spontaneous or induced) at baseline visit.\n6. Able to understand the patient information sheet, willing to consent to study protocol.\n\nExclusion Criteria:\n\n1. When attending for the baseline visit participants should be clinically stable at the time of the visit. This is defined as no acute change in their baseline symptoms or presence of new viral symptoms. They should not be on additional antibiotics or anti-viral therapies for any reason (above their usual medications), and should have completed any such additional therapies at least 4 weeks prior.\n2. Extensive antibiotic allergies or intolerances that mean they could not be treated with standard CF antibiotic regimens, as outlined in section 5.6.\n3. Subjects with infection with Mycobacteria tuberculosis\n4. Subjects with active ABPA, defined as receiving treatment for ABPA currently or within the last 4 months, or those considered at risk of requiring treatment for ABPA in the next 12 months.\n5. Subjects receiving long term oral steroids at an equivalent dose of 10mg or more per day of prednisolone.\n6. Subjects receiving any other form of long term immune-suppressant therapy.\n7. Subjects with non-tuberculous mycobacteria (NTM) infection who are undergoing active eradication therapy. Subjects with chronic NTM infection who are not on eradication therapy, and not expecting to start this within the next 12 months, are not excluded.\n8. Any other condition, co-morbidity or other feature that, in the opinion of the investigator would render the subject unable to complete the protocol or unsuitable for inclusion.\n9. Planning on participating in a clinical trial of a novel experimental investigational medical product in the next 12 months.",{"count":165,"type":21},"The UNIFIED-CF study is an observational study designed to investigate the impacts of treatment given for severe pulmonary exacerbations in people living with cystic fibrosis (pwCF). Exacerbations are episodes when pwCF become more unwell, typically characterised by increased cough, sputum, and breathlessness and treated with a combination of oral and\u002For intravenous antibiotics. Severe exacerbations require treatment with intravenous antibiotics and impart considerable morbidity on pwCF.\n\nIn this study, the investigators will recruit people at risk of severe CF exacerbations when they are well and if\u002Fwhen they are subsequently admitted for treatment of an exacerbation, the investigators will track symptoms and lung function during recovery, and collect blood, sputum and stool samples to allow us to explore the biological mechanisms of exacerbations and how they relate to different treatment responses.\n\nThe study is event driven and will complete recruitment once 125 participants have completed treatment and follow-up for a severe exacerbation event.\n\nThis study is funded by the Cystic Fibrosis Trust. This study is part of a wider programme of research, led by the PULSE-CF Innovation Hub (and hosted by the University of Manchester). The aim of the Hub is that the data from the UNIFIED-CF study will ultimately support the design of a platform clinical trial to test exacerbation-prevention interventions in CF.",[28,507],"Cystic Fibrosis Pulmonary Exacerbation",{"date":509,"type":35},"2026-03-24",{"date":511,"type":35},"2025-10-09",{"date":513,"type":21},"2029-12-31",{"name":515,"class":73},"Alexander Horsley",6,{"id":518,"slug":519,"hasResults":12,"nctId":520,"briefTitle":521,"officialTitle":521,"acronym":522,"eligibilityCriteria":523,"healthyVolunteers":268,"sex":17,"minAge":524,"maxAge":4,"enrollmentInfo":525,"targetDuration":4,"studyType":55,"phases":4,"briefSummary":526,"conditions":527,"keywords":4,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":528,"lastUpdatePostDateStruct":529,"startDateStruct":530,"completionDateStruct":532,"leadSponsor":533,"locationsCount":534},"100588371","identifying-the-causes-and-risk-factors-of-pulmonary-exacerbations-in-cystic-fibrosis-100588371","NCT06940531","Identifying the Causes and Risk Factors of Pulmonary Exacerbations in Cystic Fibrosis","CF-Tracker","Inclusion Criteria:\n\nFor Adult Participants\n\n1. Confirmed diagnosis of cystic fibrosis (CF), defined as presence of two pathogenic CF-causing CFTR mutations AND clinical features consistent with a diagnosis of CF, OR presence of at least one pathogenic CF-causing CFTR mutation AND sweat chloride (before use of CFTR modulators) \\>60mmol\u002FL AND clinical features consistent with a diagnosis of CF.\n2. Age ≥ 16 years and receiving care from a UK Adult Cystic Fibrosis Centre for main study. 5-16yrs for Paediatric pilot study (see below).\n3. Have had at least 1 previous exacerbation of CF lung disease, treated with oral or intravenous antibiotics, in the previous 12 months.\n4. Able to understand the patient information sheet, willing to consent to study protocol and to returning home samples\n5. Has a home spirometry device and able to use this\n\n   For those taking part in Group-B, additional inclusion criteria include\n6. Willing to attend for additional face to face visits at 4 weeks, 26 weeks, and if they become unwell\n\n   For those taking part in home monitoring (as part of Group-B at Manchester)\n7. Has wireless internet at home\n8. Willing to allow to home access to set up monitoring devices, collect these back in at end of study, and to carry out other visits to perform calibration or intermittent home air sampling.\n\nFor Paediatric Participants\n\n1. Confirmed diagnosis of cystic fibrosis (CF), defined as presence of two pathogenic CF-causing CFTR mutations AND clinical features consistent with a diagnosis of CF, OR presence of at least one pathogenic CF-causing CFTR mutation AND sweat chloride (before use of CFTR modulators) \\>60mmol\u002FL AND clinical features consistent with a diagnosis of CF.\n2. Receiving care from an eligible Paediatric CF Centre.\n3. Age 5-16 years\n4. Have had at least 1 previous exacerbation of CF lung disease, treated with antibiotics.\n5. Able to understand the study and\u002For willing to assent to study protocol.\n6. Parents or guardians able to understand the study and willing to consent to take part, including helping with home sampling\n7. Has a home spirometry device and able to use this\n\nFor Healthy Volunteers\n\n1. Healthy subject, male or female, aged 16-65 years\n2. No active lung condition, chronic inflammatory disorder or infection\n3. Not been on antibiotics or anti-inflammatory agents of any sort (including inhaled or systemic corticosteroids) for at least 90 days.\n4. No recent (defined as within the previous 4 weeks) acute viral symptoms\n5. Willing to sign the consent form and provide the samples.\n\nExclusion Criteria:\n\n1. Unable to produce sputum, spontaneous or induced, at visit 1. If subject is normally able to produce sputum and still wishes to take part, visit 1 can be repeated on up to two additional occasions if this is needed to obtain sputum sample.\n2. For the first visit, participants should be clinically stable at the time of the visit. This is defined as no acute change in their baseline symptoms or presence of new viral symptoms. They should not be on additional antibiotics or anti-viral therapies for any reason (above their usual medications), and should have completed any such additional therapies at least 4 weeks prior to visit 1.\n3. Subjects with infection with Mycobacteria tuberculosis\n4. Subjects with active ABPA, defined as receiving treatment for ABPA currently or within the last 12 months, or those considered at risk of requiring treatment for ABPA in the next 12 months.\n5. Subjects receiving long term oral steroids at an equivalent dose of 10mg or more per day of prednisolone.\n6. Subjects receiving any other form of long term immune-suppressant therapy.\n7. Subjects with non-tuberculous mycobacteria (NTM) infection who are undergoing active eradication therapy. Subjects with chronic NTM infection who are not on eradication therapy, and not expecting to start this within the next 12 months, are not excluded.\n8. Subjects who are unable to complete home spirometry who have previously been shown poor adherence to home monitoring requests\n9. Any other condition, co-morbidity or other feature that, in the opinion of the investigator would render the subject unable to complete the protocol or unsuitable for inclusion.\n10. For home monitoring, any subject where the investigator or their team has concern about staff safety when performing home visits.\n\nPatients taking part in other long term trials or observational studies are eligible to take part in CF-Tracker. Local investigators should judge whether the burden of additional research visits will be manageable.","5 Years",{"count":54,"type":21},"The CF-Tracker study is a community surveillance study, designed to understand the causes of exacerbations in people with cystic fibrosis (CF) (pwCF). These are episodes when pwCF become more unwell, typically characterised by increased cough, sputum, and breathlessness, and requiring prolonged courses of oral or intravenous antibiotics.\n\nThis observational study applies a two-tiered approach over 12 months. It will recruit 200 pwCF to Group A, and an additional 100 pwCF to Group B, which follows the same format but includes additional in-clinic sampling.\n\nParticipants will provide longitudinal clinical data and biological samples. Group B will be offered at 5 specialist CF centres (Manchester, Cardiff, Newcastle, Leeds, Liverpool), will include additional sampling methods at clinic visits, and additional scheduled clinic visits at 1 month and 6 months. Group B participants will be offered an in-person visit if they become unwell, so that samples can be collected before they start antibiotics. In Group B, those attending the Manchester clinic will have the option of taking part in a 12 month home environmental and pollution monitoring, and sleep monitoring (both optional arms).\n\nA pilot study will test the practicalities of running the same protocol in a paediatric population. This will consist of up to 25 children with CF (5-15 years) attending a paediatric clinic in one of the four core centres. Up to 40 healthy volunteers will be recruited to provide samples on a single occasion as controls.\n\nThis study is funded by the Cystic Fibrosis Trust. This study is part of a wider programme of research, led by the PULSE-CF Innovation Hub (and hosted by the University of Manchester, www.pulse-cf.com). The aim of the Hub is that the data from CF-Tracker will support the delivery of a platform clinical trial to test exacerbation-prevention interventions in CF.",[28,507],"2026-03-16",{"date":226,"type":35},{"date":531,"type":35},"2025-05-16",{"date":513,"type":21},{"name":515,"class":73},18,{"id":536,"slug":537,"hasResults":12,"nctId":538,"briefTitle":539,"officialTitle":540,"acronym":541,"eligibilityCriteria":542,"healthyVolunteers":268,"sex":17,"minAge":163,"maxAge":83,"enrollmentInfo":543,"targetDuration":4,"studyType":55,"phases":4,"briefSummary":545,"conditions":546,"keywords":547,"overallStatus":170,"whyStopped":4,"lastUpdateSubmitDate":552,"lastUpdatePostDateStruct":553,"startDateStruct":555,"completionDateStruct":557,"leadSponsor":558,"locationsCount":131},"100627903","mri-assessment-of-lung-airways-in-cystic-fibrosis-evaluate-mris-ability-to-detect-changes-in-airway-structure--100627903","NCT07454681","MRI Assessment of Lung Airways in Cystic Fibrosis: Evaluate MRI's Ability to Detect Changes in Airway Structure .","Development of Magnetic Resonance Imaging Airway Segmentation to Assess and Monitor Cystic Fibrosis Lung Disease","UTE Airway MR","Group 1\n\nInclusion Criteria:\n\n* Participants must be greater than or equal to 6 years of age and not greater than 18 years of age.\n* Informed consent by patient or parent\u002Fguardian consent and participant assent when appropriate.\n* Able to perform reproducible spirometry\n\nExclusion Criteria:\n\n* Medical instability that would preclude the ability to undergo the required investigations\n* FEV1 % predicted \\\u003C 40%\n* Severe claustrophobia\n* Does not meet MRI screening criteria\n* Usage of oral antibiotics within 3 weeks prior to study visit\n* Known pulmonary disease\n\nGroup 2 Inclusion Criteria\n\n* Diagnosis of CF as evidenced by one or more clinical feature consistent with the CF phenotype or positive CF newborn screen\n* Participants must be greater than or equal to 6 years of age and not greater than 18 years of age.\n* Informed consent by patient or parent\u002Fguardian consent and participant assent when appropriate.\n* Able to perform reproducible spirometry\n\nExclusion Criteria\n\n* Medical instability that would preclude the ability to undergo the required investigations\n* FEV1 % predicted \\\u003C 40%\n* Severe claustrophobia\n* Does not meet MRI screening criteria\n* Worsening cough and\u002For sputum production within the past 3 days prior to study visit\n* The use of new oral and\u002For inhaled antibiotics within 3 weeks prior to study visit\n* Received intravenous antibiotics within 2 weeks prior to study visit\n* The use of supplementary oxygen\n* Status of post lung or another organ transplant",{"count":544,"type":21},76,"This study is being done to determine whether MRI can produce high quality lung and airway images in healthy and CF patients and if MRI can be used to evaluate size and shape of the airways with computer assistance. This study will also repeat MRI experiments two years after the initial MRI scan to see if changes to airway size and shape are seen over time. In a subset of participants, we will investigate whether MRI results are repeatable and reproducible in the short-term one week after the initial MRI visit. This study will help understand if MRI based measurements of airway size and shape can be used as a monitoring tool that does not use x-ray radiation in patients with CF.",[28],[60,548,549,550,551],"Magnetic Resonance Imaging","Ultrashort Echo Time","Multiple Breath Washout","Airways","2026-03-03",{"date":554,"type":35},"2026-03-06",{"date":556,"type":21},"2026-04-15",{"date":513,"type":21},{"name":559,"class":73},"The Hospital for Sick Children",{"id":561,"slug":562,"hasResults":12,"nctId":563,"briefTitle":564,"officialTitle":564,"acronym":4,"eligibilityCriteria":565,"healthyVolunteers":12,"sex":17,"minAge":83,"maxAge":4,"enrollmentInfo":566,"targetDuration":4,"studyType":22,"phases":567,"briefSummary":568,"conditions":569,"keywords":572,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":575,"lastUpdatePostDateStruct":576,"startDateStruct":577,"completionDateStruct":579,"leadSponsor":581,"locationsCount":131},"100626493","act-with-cf-self-help-toolkit-100626493","NCT07436351","ACT With CF Self-Help Toolkit","Inclusion Criteria:\n\n* 18 years or older\n* Existing diagnosis of cystic fibrosis\n* PHQ-9 score \\>4 or GAD-7 score \\>4\n\nExclusion Criteria:\n\n* History of suicide attempts or acute suicidal ideation on clinical assessment\n* Presence of psychotic disorder or symptoms\n* Presence of psychiatric disorders that interfere with participation in the study, judged by the study or treating clinician\n* Presence of other medical conditions that interfere with participation in the study, judged by the study or treating clinician",{"count":188,"type":21},[87],"Acceptance and Commitment Therapy (ACT) tailored to meet the needs of adults living with cystic fibrosis (ACT with CF) is a newer form of talk therapy that has been shown to reduce anxiety \\& depression and improve psychological flexibility, and value-based living. The investigators are now trying to find out whether a self-help version of this treatment (ACT with CF - Self Help Toolkit) is also effective in reducing anxiety and depression and improving psychological flexibility and value-based living in adults with CF.\n\nAdults with cystic fibrosis are at increased risk for anxiety and depression. This study examines whether a patient-facing therapy, ACT with CF - Self Help Toolkit can help to reduce anxiety and depression among adults with CF. This treatment can be accessed on the participant's smartphone.",[28,570,571],"Depressive and Anxiety Disorders","Psychological Flexibility",[96,573,61,574],"ACT","chronic illness","2026-02-27",{"date":552,"type":35},{"date":578,"type":35},"2026-01-20",{"date":580,"type":21},"2027-04",{"name":582,"class":73},"Thomas Jefferson University",{"id":584,"slug":585,"hasResults":12,"nctId":586,"briefTitle":587,"officialTitle":587,"acronym":588,"eligibilityCriteria":589,"healthyVolunteers":12,"sex":17,"minAge":590,"maxAge":591,"enrollmentInfo":592,"targetDuration":4,"studyType":55,"phases":4,"briefSummary":594,"conditions":595,"keywords":596,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":599,"lastUpdatePostDateStruct":600,"startDateStruct":602,"completionDateStruct":604,"leadSponsor":606,"locationsCount":131},"100616285","population-pharmacokinetics-of-elexacaftor-tezacaftor-ivacaftor-in-a-paediatric-population-100616285","NCT07303621","Population Pharmacokinetics of Elexacaftor-tezacaftor-ivacaftor in a Paediatric Population","IMPROVED","Inclusion Criteria:\n\n* Children aged 2 to 17 years old\n* Having Cystic Fibrosis\n* Treated by Elexacaftor\u002FTezacaftor and Ivacaftor (Trikafta® or Kaftrio®)\n\nExclusion Criteria:\n\n* Allergy to previous CFTR modulator association (Ivacaftor, lumacaftor)\n* Pregnant women\n* Patient already enrolled in another study with CYP3A4 inhibitor\n* Pulmonary transplant recipient","2 Years","17 Years",{"count":593,"type":21},150,"Cystic fibrosis is a rare, progressive genetic disease caused by a mutation in the CFTR (cystic fibrosis transmembrane conductance regulator) gene. Respiratory and nutritional effects are crucial to patients' prognosis. Since the early years of 2010, etiological treatment has been based on the use of CFTRm (CFTR modulator), which aim to restore the function of the mutated protein. Initially used as monotherapy and targeting a limited number of patients, CFTRm has gradually been extended to a larger number of patients, to the point where it now concerns 9 out of 10 patients, through the use of triple therapy with Elexacaftor-Tezacaftor-Ivacaftro (ETI) or Kaftrio(R).\n\nThe efficacy of triple therapy is spectacular, revolutionizing the prognosis of the disease. However, the potential for neuropsychological side-effects (20-50% depending on age, but more frequent in young children under 5) and hepatic side-effects (hepatic cytolysis) must be taken into account. A better understanding of pharmacokinetic variability in children, as well as the relationship between exposure to therapeutic effects and adverse reactions, is therefore particularly important.\n\nThe aim of this study is to measure the association between the pharmacokinetic parameters of Elexacaftor, Tezacaftor and Ivacaftor (plasma clearance and volume of distribution) and therapeutic or adverse effects in pediatric patients with cystic fibrosis treated with the combination.",[28],[597,248,250,60,598],"Trikafta","pharmacokinetics","2026-02-18",{"date":601,"type":35},"2026-02-20",{"date":603,"type":35},"2026-02-09",{"date":605,"type":21},"2027-08-09",{"name":607,"class":73},"Hospices Civils de Lyon",{"id":609,"slug":610,"hasResults":12,"nctId":611,"briefTitle":612,"officialTitle":613,"acronym":614,"eligibilityCriteria":615,"healthyVolunteers":268,"sex":17,"minAge":52,"maxAge":4,"enrollmentInfo":616,"targetDuration":4,"studyType":55,"phases":4,"briefSummary":618,"conditions":619,"keywords":621,"overallStatus":170,"whyStopped":4,"lastUpdateSubmitDate":629,"lastUpdatePostDateStruct":630,"startDateStruct":632,"completionDateStruct":634,"leadSponsor":636,"locationsCount":131},"100623884","a-prospective-study-of-advanced-diagnostics-in-people-with-an-unclear-diagnosis-of-cystic-fibrosis-100623884","NCT07402434","A Prospective Study of Advanced Diagnostics in People With an Unclear Diagnosis of Cystic Fibrosis","Advanced Diagnostic Validation and Novel Clinical Evaluation Across the CFTR Spectrum (ADVANCE-CFTR): a Prospective Study of CFTR Functional Assays, Including Rectal Organoids, to Improve the Accuracy of Diagnosing Cystic Fibrosis.","ADVANCE-CFTR","Inclusion Criteria:\n\nGroup A: Difficult Cystic Fibrosis Diagnosis Cohort\n\nInclusion criteria:\n\n1. Patients with difficult-to-diagnose CF\\*\n2. Adults (≥16y)\n3. Willing to undergo rectal biopsy\n4. Able to comply with the study\n\n   * Definition: Non-diagnostic first line CFTR testing (individuals who do not fulfil CF diagnostic criteria \\[2 CF-causing CFTR variants and\u002For sweat chloride concentration ≥60 mmol\u002FL\\]). Of note, there are some individuals with \\\u003C2 CF-causing CFTR variants after extended CFTR genetic analysis but with a SCC \\>60mmol\u002FL. Expansion of the patient pool to include these is permissible.\n\nGroup B: Cystic Fibrosis Patient Cohort\n\nInclusion criteria:\n\n1. Patients with confirmed Cystic Fibrosis\n2. Adults (≥16y)\n3. Willing to undergo rectal biopsy for this study\n4. Able to comply with the study\n\nGroup C: Non-Cystic Fibrosis Patient Cohort\n\nInclusion criteria:\n\n1. Patients without Cystic Fibrosis or CFTR-related disorder\n2. Adults (≥16y)\n3. Willing to undergo rectal biopsy for this study (if undergoing a lower GI endoscopy for other purposes)\n4. Able to comply with the study\n\nExclusion Criteria:\n\nGroup A: Difficult Cystic Fibrosis Diagnosis Cohort\n\nExclusion criteria:\n\n1. Contra-indication to rectal biopsy (e.g. significant bleeding diathesis)\n2. History of lung transplantation\n3. Receiving CFTR modulator treatment\n4. Current participation (or participation within one month of enrolment) in a clinical trial of an investigational medicinal product which affects CFTR function (e.g. CFTR modulators, genetic therapies)\n5. Female who is pregnant or breastfeeding\n6. Unable to provide informed consent\n\nGroup B: Cystic Fibrosis Patient Cohort\n\nExclusion criteria:\n\n1. Contra-indication to rectal biopsy (e.g. significant bleeding diathesis)\n2. Current participation (or participation within one month of enrolment) in a clinical trial of an investigational medicinal product which affects CFTR function (e.g. CFTR modulators, genetic therapies)\n3. Female who is pregnant or breastfeeding\n4. Unable to provide informed consent\n5. Exclude all subjects where diagnosis of CF is in doubt\n\nGroup C: Non-Cystic Fibrosis Patient Cohort\n\nExclusion criteria:\n\n1. Contra-indication to rectal biopsy (e.g. significant bleeding diathesis)\n2. Female who is pregnant or breastfeeding\n3. Unable to provide informed consent",{"count":617,"type":21},80,"Sometimes it is very difficult to tell if someone has cystic fibrosis (CF), especially when they have rare CF genes. Without this certainty, they are unlikely to get the correct treatment so their health may be affected. More accurate ways to test for CF are therefore needed in this situation. The aim of this study is to develop a more accurate test using what are called \"organoids\" or \"mini organs.\" Organoids are grown in the laboratory from a small piece of gut tissue. As they have the person's exact genes, they can show if the CF gene (\"CFTR\") is working correctly or not and thus if the person has CF. The investigators will compare the organoid response with the current more established tests, such as the sweat test and CF genetics, and other recognised specialist tests called nasal potential difference (NPD) and intestinal current measurement (ICM). The gut tissue is usually taken by a quick, relatively painless, outpatient procedure (rectal biopsy). The additional benefit of organoids is that they can also help us to work out the best treatment for that individual by testing how well the gut tissue responds to different drugs in the laboratory. The investigators wish to carry out this research to prove that gut organoids are a better way to test for CF in this situation. The goal will be to diagnose people faster and for them to get better treatment quicker, both key for leading a longer and healthier life.",[28,620],"CFTR-related Disorders",[622,623,624,28,625,626,627,628],"CFTR-related disorders","Advanced Diagnostics","Personalised medicine","Nasal potential difference (NPD)","Intestinal current measurements (ICM)","Organoids","Sweat chloride concentration (SCC)","2026-02-12",{"date":631,"type":35},"2026-02-17",{"date":633,"type":21},"2026-02-02",{"date":635,"type":21},"2028-09-01",{"name":637,"class":73},"Royal Brompton & Harefield NHS Foundation Trust",{"id":639,"slug":640,"hasResults":12,"nctId":641,"briefTitle":642,"officialTitle":643,"acronym":4,"eligibilityCriteria":644,"healthyVolunteers":12,"sex":17,"minAge":83,"maxAge":4,"enrollmentInfo":645,"targetDuration":4,"studyType":22,"phases":646,"briefSummary":647,"conditions":648,"keywords":652,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":656,"lastUpdatePostDateStruct":657,"startDateStruct":659,"completionDateStruct":661,"leadSponsor":663,"locationsCount":260},"100598425","cf-wellness-program-100598425","NCT07071324","CF Wellness Program","CF Wellness Program: ORBIT Phase 2 Pilot RCT","Inclusion Criteria:\n\n1. ≥18 years old\n2. Documentation of CF diagnosis in the medical record\n3. Score of \\>4 on the Fatigue Severity Scale\n4. Access to a smartphone, tablet, and\u002For computer with access to internet\n5. Ability to understand\u002Fread\u002Fspeak English\n6. Receives CF care at a participating CF Center\n\nExclusion Criteria:\n\n1. Pulmonary exacerbation (physician determined and may include oral antibiotics, IV antibiotics, hospitalization) ±14 days of enrollment\n2. Pregnant or \\\u003C6 months post-partum (self-reported)\n3. Contraindication to light physical activity (as determined by the treating physician and may include pulmonary, cardiovascular, or musculoskeletal contraindications)\n4. Participated in the CFWP Feasibility Study\n5. Currently enrolled in another interventional trial\n6. Unavailable to complete coaching sessions within the study timeframe",{"count":617,"type":21},[87],"This study is a pilot randomized control trial (RCT; N=80) comparing the Cystic Fibrosis Wellness Program (CFWP) to usual care (UC) to evaluate (1) Intervention Adherence (completion of the CFWP Coaching Sessions) (2) Study Retention (completion of the Week 15 assessment) and (3) Data Quality (valid daytime and nighttime fitness tracker data). A secondary aim is to gather preliminary data to determine if the CFWP has a clinically significant signal over usual care to improve fatigue, sleep, and physical activity (PA) and reduce sedentary behavior.",[28,649,650,651],"Fatigue","Sleep Quality","Insomnia",[28,649,650,653,651,654,655],"Physical Activity","Cognitive Behavioral Therapy","Sedentary","2026-02-11",{"date":658,"type":35},"2026-02-13",{"date":660,"type":35},"2026-02-03",{"date":662,"type":21},"2028-06-30",{"name":664,"class":73},"Johns Hopkins University"]