Imaging Assessments of ARPKD Kidney Disease Progression
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age6+
SponsorThe Cleveland Clinic
About this trial
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 criteria
This trial accepts healthy volunteersQualifiers
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
Disqualifiers
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
Trial population
Inclusion criteria: 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 \>30ml/min/1.732 performed within the last year Healthy Controls * Otherwise healthy with no known structural or functional kidney disease * Normal documented blood pressure within the last year Exclusion criteria: 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 and/or anticipated dialysis or kidney transplant within the next 3 years 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)
Trial design
Design model
Cohort
Time perspective
Prospective
Treatments tested in this trial
Not listed
Trial groups
60 Participants
are grouped into 4 trial groupsGroup A: Early CKD Patients
Group B: Mild CKD Patients
Group C: Moderate CKD Patients
Group D: Healthy Controls
Trial outcomes
Primary outcomes
1
The diagnostic performance (AUROC, sensitivity, specificity) of the multimodal MRI assessments MRF (mean kidney T1 and T2), ASL (mean kidney perfusion), and MRE (mean kidney stiffness), alone or in combination to differentiate the 4 study cohorts
Time frame
3 Years2
The relationship between the imaging biomarkers, MRF (mean kidney T1 and T2), ASL (mean kidney perfusion), and MRE (mean kidney stiffness) alone or in combination, to baseline and change in measured GFR over 3 years in the 3 ARPKD cohorts
Time frame
3 Years3
The level of agreement between breath-hold and free breathing kidney MRF assessments in the 4 cohorts
Time frame
3 YearsSecondary outcomes
1
Repeatability of multimodal MRI assessments in ARPKD and healthy control subjects
Time frame
3 years2
Reproducibility of findings at the two study sites
Time frame
3 YearsSponsors and contacts
Click on the lead sponsor to view all of their trials.
The Cleveland Clinic
Lead sponsor
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Collaborator
Source ClinicalTrials.gov