Cystic Fibrosis (CF)NCT06595420
Kidney Function in People With Cystic Fibrosis in the Era of HEMT
An observational study of Cystic Fibrosis (CF) and Chronic Kidney Disease(CKD), run by University of Virginia at 3 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 7 years and olderHealthy volunteers can take part
- Conditions the study is for
- Cystic Fibrosis (CF), Chronic Kidney Disease(CKD), Acute Kidney Injury
- Type of treatment
- Injection or infusion
- How many people
- 260 participants
- Where
- 3 sitesUnited States
- Study dates
- 9 Jan 2025to Dec 2027
- Phase
- Observational
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 7 years and older
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
Study population: Participants will be derived from patients with CF with ongoing care at one of the 3 CF Centers (Dartmouth, UAB, UVA).
Who may be able to join
- Outpatient CF Cohort
- Diagnoses of Cystic Fibrosis
- Age \> 30 years old
- Able to provide informed consent
- Inpatient CF Cohort
- Diagnoses of Cystic Fibrosis
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- Age \> 7 years old
- Able to provide informed consent and assent (where applicable)
- 55 PwCF frequently hospitalized for a pulmonary exacerbation (\>1 hospital admission in the prior 12 months)
- 55 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
Who cannot join
- Outpatient CF Cohort
- History of any organ transplant
- History of immunodeficiency
- Previous or current cancer diagnoses
- Pregnant or breastfeeding
- On chronic dialysis
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- 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
- History or current kidney disease, organ transplantation, cancer, or any other chronic illness
- Current use of antibiotics
- Urinary symptoms or UTI (dysuria, frequency, urgency)
- Pregnant women
- Menstruating on the study visit day
- Blood relatives of PwCF
Trial locations and contacts
3 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States3 sites, 3 recruiting
- Recruiting
University of Alabama at Birmingham
Birmingham, Alabama, 35233
William T Harris, MD, 205-638-9583, wtharris@uabmc.edu
- Recruiting
University of Virginia Children's Hospital
Charlottesville, Virginia, 22908
Agnieszka Swiatecka-Urban, MD, 434-924-0946, AS6XX@uvahealth.org
- Recruiting
Dartmouth-Hitchcock Geisel School of Medicine at Dartmouth
Lebanon, New Hampshire, 03756
Sladjana Skopelja-Gardner, PhD, 603-667-8259, Sladjana.Skopelja-Gardner@dartmouth.edu
Alix Ashare, MD
About this study
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.
Read the detailed description
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.
The 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 2/week thereafter during the admission, and then after hospital discharge at each subsequent clinical encounter for 24 months.
These 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.
- Medical subject headings
- Cystic Fibrosis, Renal Insufficiency, Chronic, Acute Kidney Injury
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 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 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: Enrollment and every 3 months for 24 months
Correlation between recurrent hospitalizations and urinary kidney injury signature.
Inpatient CF Cohort
Measured: 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
Measured: 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.
Outpatient CF Cohort
Measured: Enrollment and every 3 months for 24 months.
Correlation between %FEV1 at admission or decline in %FEV1 and urinary kidney injury signature.
Inpatient CF Cohort
Measured: 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.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Sep 2024
- Study start9 Jan 2025
- Primary completionDec 2027 (estimated)
- Study completionDec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (15)Papers the sponsor has linked to this study.
- Other trials for Cystic Fibrosis (CF)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 260 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Virginia
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Agnieszka Swiatecka-Urban, University of Virginia
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06595420
- Sponsor's protocol ID
- HSR231650
- Other IDs
- 005245A123
- Status last verified
- Apr 2026
- First submitted
- 10 Sep 2024
- Last updated
- 4 May 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
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.
PubMed 8853436Southern KW. Acute renal failure in people with cystic fibrosis. Thorax. 2007 Jun;62(6):472-3. doi: 10.1136/thx.2006.072355.
PubMed 17536029Al-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.
PubMed 15521084Bertenshaw 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.
PubMed 17234661Quon 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.
PubMed 21799076Stevanovic M, G.M. Primary Causes of ESRD in the US Cystic Fibrosis Population. JASN 33, 694
Stevanovic, M.G.M.L. Characteristics of US Individuals with Cystic Fibrosis and ESRD. JASN 33, 694
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.
PubMed 31767021Berg 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.
PubMed 29187303Alicandro 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.
PubMed 25151032Nazareth 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.
PubMed 23618617van 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.
PubMed 31205376Kovesdy 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.
PubMed 24026256Breyer 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.
PubMed 27230798Kovesdy 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.
PubMed 35529086