RecruitingNCT07201025

Imaging Assessments of ARPKD Kidney Disease Progression


Sponsor

The Cleveland Clinic

Enrollment

60 participants

Start Date

Dec 12, 2024

Study Type

OBSERVATIONAL

Conditions

Summary

Autosomal Recessive Polycystic Kidney Disease (ARPKD) is a potentially lethal genetic disorder for which there are currently no disease specific treatments. Clinical trials have been limited by the absence of robust measures of disease progression. The overall goal of this 5 year NIH-funded prospective, observational multicenter study conducted at CC (primary site) and Children's Hospital of Philadelphia (CHOP) (collaborating site) is establish a set of rapid, quantitative, and reproducible novel kidney MRI measures applicable to standard clinical MRI scanners, which could serve as potential biomarkers to measure response to therapy in patients across the disease spectrum and all ages. The investigators will recruit a total of 45 ARPKD patients and 15 healthy controls at the the two sites . All subjects will be \> or = 6yrs old with no contraindications to undergoing MRI (non contrast). ARPKD subjects will be recruited into one of 3 cohorts based on their estimated glomerular filtration rate (eGFR): early CKD (eGFR\> or =90ml/min/1.73m2), mild CKD (60-89ml/min/1.73m2) and moderate CKD (30-59ml/min/1.73m2). For ARPKD subjects, participation will last 3 years and consist of a baseline and 3 subsequent annual visits. Healthy controls will only have 1 study visit. Study visits for all participants will include collection of clinical and demographic data, clinical blood and urine tests (E.g. serum creatinine) and MRI imaging. ARPKD (but not healthy subjects) will also undergo a measured GFR test (iohexol clearance) to accurately assess their kidney function.


Eligibility

Min Age: 6 Years

Inclusion Criteria7

  • ALL SUBJECTS
  • Ages \>6 years old
  • Able to lay still for 30 minutes in an MRI scanner ARPKD Patients
  • Clinical and/or genetic diagnosis of ARPKD
  • eGFR (creatinine based) \>30ml/min/1.732 performed within the last year Healthy Controls
  • Otherwise healthy with no known structural or functional kidney disease
  • No history of hypertension or documented high blood pressure within the last year

Exclusion Criteria8

  • ALL SUBJECTS
  • Contraindications to MRI (e.g. metal implants)
  • Parental/patient refusal to sign an informed consent/ assent form ARPKD Patients
  • Prior kidney or other solid organ transplant
  • eGFR \<30ml/min/1.732 Healthy Controls
  • Systemic diseases known to place patient at risk for kidney disease (e.g. diabetes mellitus)
  • Obesity (BMI \>95th percentile for age for \<18 years old, or BMI\>30 for adults)
  • History of prematurity (\<32 weeks gestational age)

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Locations(2)

Cleveland Clinic

Cleveland, Ohio, United States

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

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NCT07201025


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