Ignite Creation Date:
2025-12-25 @ 4:08 AM
Ignite Modification Date:
2026-01-01 @ 2:26 PM
Study NCT ID:
NCT06595420
Status:
RECRUITING
Last Update Posted:
2025-04-15
First Post:
2024-09-10
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Kidney Function in People With Cystic Fibrosis in the Era of HEMT
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D003550', 'term': 'Cystic Fibrosis'}, {'id': 'D051436', 'term': 'Renal Insufficiency, Chronic'}, {'id': 'D058186', 'term': 'Acute Kidney Injury'}], 'ancestors': [{'id': 'D010182', 'term': 'Pancreatic Diseases'}, {'id': 'D004066', 'term': 'Digestive System Diseases'}, {'id': 'D008171', 'term': 'Lung Diseases'}, {'id': 'D012140', 'term': 'Respiratory Tract Diseases'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D007232', 'term': 'Infant, Newborn, Diseases'}, {'id': 'D051437', 'term': 'Renal Insufficiency'}, {'id': 'D007674', 'term': 'Kidney Diseases'}, {'id': 'D014570', 'term': 'Urologic Diseases'}, {'id': 'D052776', 'term': 'Female Urogenital Diseases'}, {'id': 'D005261', 'term': 'Female Urogenital Diseases and Pregnancy Complications'}, {'id': 'D000091642', 'term': 'Urogenital Diseases'}, {'id': 'D052801', 'term': 'Male Urogenital Diseases'}, {'id': 'D002908', 'term': 'Chronic Disease'}, {'id': 'D020969', 'term': 'Disease Attributes'}, {'id': 'D010335', 'term': 'Pathologic Processes'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}]}}, 'protocolSection': {'designModule': {'bioSpec': {'retention': 'SAMPLES_WITHOUT_DNA', 'description': 'Bood, urine'}, 'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 260}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2025-01-09', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-04', 'completionDateStruct': {'date': '2027-12', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-04-14', 'studyFirstSubmitDate': '2024-09-10', 'studyFirstSubmitQcDate': '2024-09-10', 'lastUpdatePostDateStruct': {'date': '2025-04-15', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2024-09-19', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2027-12', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Examine whether trajectories of eGFR (calculated from serum creatinine and cystatin C) correlate with urinary kidney injury signatures detected in different urine fractions, or urinary neutrophil levels/activation.', 'timeFrame': 'Enrollment and every 3 months for 24 months', 'description': 'Outpatient CF Cohort'}, {'measure': 'Correlation between recurrent hospitalizations and urinary kidney injury signature.', 'timeFrame': 'On admission before the initiation of intravenous antibiotic therapy, every 48 hrs during the hospitalization, after discharge at each subsequent routine CF care visit for 24 months.', 'description': 'Inpatient CF Cohort'}, {'measure': 'Relationship between recurrent hospitalization and change in slope of eGFR', 'timeFrame': 'On admission before the initiation of intravenous antibiotic therapy, every 48 hrs during the hospitalization, after discharge at each subsequent routine CF care visit for 24 months.', 'description': 'Inpatient CF Cohort'}], 'secondaryOutcomes': [{'measure': 'The correlation between changes in the urinary protein biomarker panel, neutrophil activation, and extracellular vesicles over time and eGFR.', 'timeFrame': 'Enrollment and every 3 months for 24 months.', 'description': 'Outpatient CF Cohort'}, {'measure': 'Correlation between %FEV1 at admission or decline in %FEV1 and urinary kidney injury signature.', 'timeFrame': 'On admission before the initiation of intravenous antibiotic therapy, every 48 hrs during the hospitalization, after discharge at each subsequent routine CF care visit for 24 months.', 'description': 'Inpatient CF Cohort'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Cystic Fibrosis (CF)', 'Chronic Kidney Disease(CKD)', 'Acute Kidney Injury']}, 'referencesModule': {'references': [{'pmid': '8853436', 'type': 'BACKGROUND', 'citation': 'Devuyst O, Burrow CR, Schwiebert EM, Guggino WB, Wilson PD. Developmental regulation of CFTR expression during human nephrogenesis. Am J Physiol. 1996 Sep;271(3 Pt 2):F723-35. doi: 10.1152/ajprenal.1996.271.3.F723.'}, {'pmid': '17536029', 'type': 'BACKGROUND', 'citation': 'Southern KW. Acute renal failure in people with cystic fibrosis. Thorax. 2007 Jun;62(6):472-3. doi: 10.1136/thx.2006.072355.'}, {'pmid': '15521084', 'type': 'BACKGROUND', 'citation': 'Al-Aloul M, Miller H, Alapati S, Stockton PA, Ledson MJ, Walshaw MJ. Renal impairment in cystic fibrosis patients due to repeated intravenous aminoglycoside use. Pediatr Pulmonol. 2005 Jan;39(1):15-20. doi: 10.1002/ppul.20138.'}, {'pmid': '17234661', 'type': 'BACKGROUND', 'citation': 'Bertenshaw C, Watson AR, Lewis S, Smyth A. Survey of acute renal failure in patients with cystic fibrosis in the UK. Thorax. 2007 Jun;62(6):541-5. doi: 10.1136/thx.2006.067595. Epub 2007 Jan 18.'}, {'pmid': '21799076', 'type': 'BACKGROUND', 'citation': 'Quon BS, Mayer-Hamblett N, Aitken ML, Smyth AR, Goss CH. Risk factors for chronic kidney disease in adults with cystic fibrosis. Am J Respir Crit Care Med. 2011 Nov 15;184(10):1147-52. doi: 10.1164/rccm.201105-0932OC. Epub 2011 Jul 28.'}, {'type': 'BACKGROUND', 'citation': 'Stevanovic M, G.M. Primary Causes of ESRD in the US Cystic Fibrosis Population. JASN 33, 694'}, {'type': 'BACKGROUND', 'citation': 'Stevanovic, M.G.M.L. Characteristics of US Individuals with Cystic Fibrosis and ESRD. JASN 33, 694'}, {'pmid': '31767021', 'type': 'BACKGROUND', 'citation': 'Lai S, Mazzaferro S, Mitterhofer AP, Bonci E, Marotta PG, Pelligra F, Murciano M, Celani C, Troiani P, Cimino G, Palange P. Renal involvement and metabolic alterations in adults patients affected by cystic fibrosis. J Transl Med. 2019 Nov 25;17(1):388. doi: 10.1186/s12967-019-02139-4.'}, {'pmid': '29187303', 'type': 'BACKGROUND', 'citation': 'Berg KH, Ryom L, Faurholt-Jepsen D, Pressler T, Katzenstein TL. Prevalence and characteristics of chronic kidney disease among Danish adults with cystic fibrosis. J Cyst Fibros. 2018 Jul;17(4):478-483. doi: 10.1016/j.jcf.2017.11.001. Epub 2017 Dec 1.'}, {'pmid': '25151032', 'type': 'BACKGROUND', 'citation': 'Alicandro G, Frova L, Di Fraia G, Colombo C. Cystic fibrosis mortality trend in Italy from 1970 to 2011. J Cyst Fibros. 2015 Mar;14(2):267-74. doi: 10.1016/j.jcf.2014.07.010. Epub 2014 Aug 20.'}, {'pmid': '23618617', 'type': 'BACKGROUND', 'citation': 'Nazareth D, Walshaw M. A review of renal disease in cystic fibrosis. J Cyst Fibros. 2013 Jul;12(4):309-17. doi: 10.1016/j.jcf.2013.03.005. Epub 2013 Apr 22.'}, {'pmid': '31205376', 'type': 'BACKGROUND', 'citation': 'van Duijl TT, Ruhaak LR, de Fijter JW, Cobbaert CM. Kidney Injury Biomarkers in an Academic Hospital Setting: Where Are We Now? Clin Biochem Rev. 2019 May;40(2):79-97. doi: 10.33176/AACB-18-00017.'}, {'pmid': '24026256', 'type': 'BACKGROUND', 'citation': 'Kovesdy CP, Bleyer AJ, Molnar MZ, Ma JZ, Sim JJ, Cushman WC, Quarles LD, Kalantar-Zadeh K. Blood pressure and mortality in U.S. veterans with chronic kidney disease: a cohort study. Ann Intern Med. 2013 Aug 20;159(4):233-42. doi: 10.7326/0003-4819-159-4-201308200-00004.'}, {'pmid': '27230798', 'type': 'BACKGROUND', 'citation': 'Breyer MD, Susztak K. The next generation of therapeutics for chronic kidney disease. Nat Rev Drug Discov. 2016 Aug;15(8):568-88. doi: 10.1038/nrd.2016.67. Epub 2016 May 27.'}, {'pmid': '35529086', 'type': 'BACKGROUND', 'citation': 'Kovesdy CP. Epidemiology of chronic kidney disease: an update 2022. Kidney Int Suppl (2011). 2022 Apr;12(1):7-11. doi: 10.1016/j.kisu.2021.11.003. Epub 2022 Mar 18.'}]}, 'descriptionModule': {'briefSummary': '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.', 'detailedDescription': 'The prevalence of chronic kidney disease is significantly increased in patients with cystic fibrosis (PwCF) with a major impact on morbidity and medication tolerance as people age. Although expressed in both the proximal and distal tubules, the specific contribution of CFTR dysfunction to renal disease remains uncertain. PwCF often are exposed to renal toxins such as frequent aminoglycosides, systemic inflammation, and activated leukocytes, but it is unknown if CFTR dysfunction predisposes to amplified tubular injury. Conventional measures of kidney function, such as serum creatinine, are insensitive to detecting early injury, limiting an opportunity to prevent CKD. This study will address the gaps in early detection and mechanisms of renal dysfunction in CF. The investigators will define the triggers and targetable mechanistic pathways of kidney injury in CF and discover novel strategies for renal protection. The central hypothesis of this study is that CFTR dysfunction alters renal development and increases the inflammatory and fibrogenic responses to nephrotoxic stimuli.\n\nThe study involves prospective evaluation of biospecimens (blood and urine) and clinical data. The study analyzes biospecimens in CF outpatients (n=110), CF inpatients (n=110), and healthy subjects (n=40). In the outpatient cohort, biospecimens will be collected at the time of each routine care visit every 3 months for 24 months. PwCF admitted for intravenous (IV) antibiotics will have biospecimens collected on admission and every 48 hrs thereafter during the admission, and then after hospital discharge at each subsequent clinical encounter for 24 months.\n\nThese biospecimens will be analyzed for biomarkers, fibrogenic analysis, inflammatory signals, and extracellular vesicles. Clinical data will be examined from chart review and correlated with biospecimen result.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '7 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'Participants will be derived from patients with CF with ongoing care at one of the 3 CF Centers (Dartmouth, UAB, UVA).', 'healthyVolunteers': True, 'eligibilityCriteria': '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'}, 'identificationModule': {'nctId': 'NCT06595420', 'briefTitle': 'Kidney Function in People With Cystic Fibrosis in the Era of HEMT', 'organization': {'class': 'OTHER', 'fullName': 'University of Virginia'}, 'officialTitle': 'Kidney Function in People With Cystic Fibrosis in the Era of HEMT', 'orgStudyIdInfo': {'id': 'HSR231650'}, 'secondaryIdInfos': [{'id': '005245A123', 'type': 'OTHER_GRANT', 'domain': 'Cystic Fibrosis Foundation'}]}, 'armsInterventionsModule': {'armGroups': [{'label': 'Outpatient CF Cohort', 'description': 'Not hospitalized CF group: Diagnosis of CF, age \\>30 y, with ongoing care at one of the 3 CF Centers (Dartmouth, UAB, UVA).'}, {'label': 'Inpatient CF Cohort', 'description': 'Hospitalized CF cohort: Diagnosis of CF, age \\>7 y, being admitted for intravenous antibiotic treatment of pulmonary exacerbation.'}, {'label': 'Healthy Controls', 'description': 'Healthy Volunteers without CF'}]}, 'contactsLocationsModule': {'locations': [{'zip': '35233', 'city': 'Birmingham', 'state': 'Alabama', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'William T Harris, MD', 'role': 'CONTACT', 'email': 'wtharris@uabmc.edu', 'phone': '205-638-9583'}], 'facility': 'University of Alabama at Birmingham', 'geoPoint': {'lat': 33.52066, 'lon': -86.80249}}, {'zip': '03756', 'city': 'Lebanon', 'state': 'New Hampshire', 'status': 'NOT_YET_RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Sladjana Skopelja-Gardner, PhD', 'role': 'CONTACT', 'email': 'Sladjana.Skopelja-Gardner@dartmouth.edu', 'phone': '603-667-8259'}], 'facility': 'Dartmouth-Hitchcock Geisel School of Medicine at Dartmouth', 'geoPoint': {'lat': 43.64229, 'lon': -72.25176}}, {'zip': '22908', 'city': 'Charlottesville', 'state': 'Virginia', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Agnieszka Swiatecka-Urban, MD', 'role': 'CONTACT', 'email': 'AS6XX@uvahealth.org', 'phone': '434-924-0946'}], 'facility': 'University of Virginia Children's Hospital', 'geoPoint': {'lat': 38.02931, 'lon': -78.47668}}], 'centralContacts': [{'name': 'Agnieszka Swiatecka-Urban, MD', 'role': 'CONTACT', 'email': 'AS6XX@uvahealth.org', 'phone': '434-924-0946'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'University of Virginia', 'class': 'OTHER'}, 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'Professor of Pediatrics', 'investigatorFullName': 'Agnieszka Swiatecka-Urban', 'investigatorAffiliation': 'University of Virginia'}}}}