Kidney Function in People With Cystic Fibrosis in the Era of HEMT

NCT06595420 | Recruiting

• 2 other identifiers: HSR231650005245A123
• Study Type: observational
• Target: 260
• Locations: 1 country
• Sites: 3

Brief Summary

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.

Conditions

Cystic Fibrosis (CF); Acute Kidney Injury; Chronic Kidney Disease(CKD)

Outcome Measures

Primary Outcomes (3)

• 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.

  Outpatient CF Cohort

  Enrollment and every 3 months for 24 months

• Correlation between recurrent hospitalizations and urinary kidney injury signature.

  Inpatient CF Cohort

  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.

• Relationship between recurrent hospitalization and change in slope of eGFR

  Inpatient CF Cohort

  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.

Secondary Outcomes (2)

• The correlation between changes in the urinary protein biomarker panel, neutrophil activation, and extracellular vesicles over time and eGFR.

  Enrollment and every 3 months for 24 months.

• Correlation between %FEV1 at admission or decline in %FEV1 and urinary kidney injury signature.

  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.

Study Arms (3)

Outpatient CF Cohort

Not hospitalized CF group: Diagnosis of CF, age \>30 y, with ongoing care at one of the 3 CF Centers (Dartmouth, UAB, UVA).

Inpatient CF Cohort

Hospitalized CF cohort: Diagnosis of CF, age \>7 y, being admitted for intravenous antibiotic treatment of pulmonary exacerbation.

Healthy Controls

Healthy Volunteers without CF

Eligibility Criteria

• Age: 7 Years+
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Participants will be derived from patients with CF with ongoing care at one of the 3 CF Centers (Dartmouth, UAB, UVA).

You may qualify if:

• Outpatient CF Cohort
• Diagnoses of Cystic Fibrosis
• Age \> 30 years old
• Able to provide informed consent
• Inpatient CF Cohort
• Diagnoses of Cystic Fibrosis
• Age \> 7 years old
• Able to provide informed consent and assent (where applicable)
• PwCF frequently hospitalized for a pulmonary exacerbation (\>1 hospital admission in the prior 12 months)
• PwCF sporadically hospitalized for a pulmonary exacerbation (no hospital admissions in the prior 12 months)
• Able to provide urine sample independently
• Healthy Controls
• Healthy, as per participant self-report
• Age between 30-50 years
• Able to provide informed consent

You may not qualify if:

• Outpatient CF Cohort
• History of any organ transplant
• History of immunodeficiency
• Previous or current cancer diagnoses
• Pregnant or breastfeeding
• On chronic dialysis
• Non-compliance (demonstrated by \<2 visits during the 12 months before enrollment)
• Inpatient CF Cohort
• The initiation of intravenous antibiotic therapy after hospital admission before obtaining the first blood and urine sample
• History of any organ transplant
• History of immunodeficiency
• Previous or current cancer diagnoses
• Pregnant or breastfeeding
• On chronic dialysis
• Healthy Controls

Sponsors & Collaborators

• University of Virginia: lead

Study Sites (3)

University of Alabama at Birmingham

Birmingham, Alabama, 35233, United States

RECRUITING

Dartmouth-Hitchcock Geisel School of Medicine at Dartmouth

Lebanon, New Hampshire, 03756, United States

RECRUITING

University of Virginia Children's Hospital

Charlottesville, Virginia, 22908, United States

RECRUITING

Related Publications (15)

• 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: 8853436 BACKGROUND

• Southern KW. Acute renal failure in people with cystic fibrosis. Thorax. 2007 Jun;62(6):472-3. doi: 10.1136/thx.2006.072355.

  PMID: 17536029 BACKGROUND

• 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: 15521084 BACKGROUND

• 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: 17234661 BACKGROUND

• 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.

  PMID: 21799076 BACKGROUND

• Stevanovic M, G.M. Primary Causes of ESRD in the US Cystic Fibrosis Population. JASN 33, 694

  BACKGROUND

• Stevanovic, M.G.M.L. Characteristics of US Individuals with Cystic Fibrosis and ESRD. JASN 33, 694

  BACKGROUND

• 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: 31767021 BACKGROUND

• 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: 29187303 BACKGROUND

• 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: 25151032 BACKGROUND

• 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: 23618617 BACKGROUND

• 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: 31205376 BACKGROUND

• 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: 24026256 BACKGROUND

• 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: 27230798 BACKGROUND

• 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.

  PMID: 35529086 BACKGROUND

Biospecimen

Retention: SAMPLES WITHOUT DNA

Bood, urine

MeSH Terms

Conditions

Cystic Fibrosis; Renal Insufficiency, Chronic; Acute Kidney Injury

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases; Renal Insufficiency; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Central Study Contacts

Agnieszka Swiatecka-Urban, MD

CONTACT

434-924-0946; AS6XX@uvahealth.org

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor of Pediatrics

Study Record Dates

• First Submitted: September 10, 2024
• First Posted: September 19, 2024
• Study Start: January 9, 2025
• Primary Completion (Estimated): December 1, 2027
• Study Completion (Estimated): December 1, 2027
• Last Updated: May 4, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (3)