Imaging Assessments of ARPKD Kidney Disease Progression

Status: Recruiting
Location: See all (2) locations...
Study Type: Observational
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
Participation Requirements
Sex: All
Minimum Age: 6
Healthy Volunteers: t
View:

⁃ 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

Locations
United States
Ohio
Cleveland Clinic
RECRUITING
Cleveland
Pennsylvania
Children's Hospital of Philadelphia
RECRUITING
Philadelphia
Contact Information
Primary
Katherine M Dell, MD
pedsresearch@ccf.org
216-444-6123
Backup
Research Coordinator
pedsresearch@ccf.org
216-386-9360
Time Frame
Start Date: 2024-12-12
Estimated Completion Date: 2029-02-28
Participants
Target number of participants: 60
Treatments
Early CKD Patients
eGFR \> or = to 90 ml/min/1.73m2
Mild CKD Patients
eGFR 60-89 ml/min/1.73m2
Moderate CKD Patients
eGFR 30-59 ml/min/1.73m2
Healthy Controls
Sponsors
Collaborators: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Leads: The Cleveland Clinic

This content was sourced from clinicaltrials.gov