NCT01581424 Natural History and Structural Functional Relationships in Fabry Renal Disease Treatment Outcomes(Changes)in Fabry Renal Disease Study
| NCT ID | NCT01581424 |
| Status | Recruiting |
| Phase | — |
| Sponsor | University of Minnesota |
| Condition | Fabry Disease |
| Study Type | OBSERVATIONAL |
| Enrollment | 50 participants |
| Start Date | 2010-10 |
| Primary Completion | 2030-07-31 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
This is an observational study. You will not receive an experimental treatment; researchers will collect data based on your existing condition or standard treatment.
This trial targets 50 participants in total. It began in 2010-10 with a primary completion date of 2030-07-31.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
The investigators will perform a study with two major components. The first is a natural history study of untreated Fabry patients. This study component will detail kidney microscopic structural changes in Fabry patients before starting enzyme replacement therapy and will correlate these changes with kidney function, including glomerular filtration rate and urinary albumin excretion rate. The investigators will perform studies on samples obtained at baseline, or before enzyme replacement therapy is initiated. The goal of our study is to find kidney microscopic changes in the biopsies that are associated with kidney disfunction. Our hypotheses for this study are: 1. Much of the natural history of Fabry renal structural changes will occur without detectable renal functional alterations. 2. Structural changes associated with the initial onset of proteinuria and those associated with the subsequent progressive loss of filtration function will differ and will be best described by non-linear models. 3. There will be sufficient precision of Fabry renal structural-functional relationships to support renal structure as an acceptable clinical trial surrogate endpoint for later renal functional deterioration. The second component examines the effects of age and gender at start of enzyme replacement therapy (ERT), as well as dosage levels of ERT on the renal cellular clearance of GL3 from Fabry patients by comparing baseline to follow-up kidney biopsies performed 5, 11, and 60 months later, with all comparisons matched for ERT treatment duration. Our hypotheses for this component of the study are as follows: 1. Enzyme Replacement Therapy(ERT) instituted at younger ages is more effective in reducing podocytes(PC),distal tubular cells(DTC),and arterial smooth muscle cells (ASMC)GL-3 than in older Fabry patients. 2. Earlier institution of ERT will stabilize PC numbers while later ERT institution, especially in proteinuric adults, may not prevent progressive decline in PC numbers and associated glomerular sclerosis, tubulointerstitial injury, and GFR loss. 3. Whereas lower ERT dose may effectively clear GL-3 from endothelial and mesangial cells, it will be less effective in clearing GL-3 from PC and also from DTC and ASMC. 4. Affected cells will be cleared of GL-3 equivalently in females and males.
Eligibility Criteria
Inclusion Criteria: * Patients diagnosed with Fabry disease who have/have not received enzyme replacement therapy where a clinical decision has been made to obtain a kidney biopsy, a GFR, and urinary albumin studies or where patients have previously completed clinical trials which included measures of renal function and renal biopsies. Exclusion Criteria: * Patients with serum creatinine more than 2.5 mg/dL or known to have a renal disease other than Fabry.
Contact & Investigator
Chet Whitley, MD
PRINCIPAL INVESTIGATOR
University of Minnesota
Frequently Asked Questions
Who can join the NCT01581424 clinical trial?
This trial is open to participants of all sexes, aged 1 Year or older, up to 75 Years, studying Fabry Disease. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT01581424 currently recruiting?
Yes, NCT01581424 is actively recruiting participants. Contact the research team at mauer002@umn.edu for enrollment information.
Where is the NCT01581424 trial being conducted?
This trial is being conducted at Minneapolis, United States, Minneapolis, United States.
Who is sponsoring the NCT01581424 clinical trial?
NCT01581424 is sponsored by University of Minnesota. The principal investigator is Chet Whitley, MD at University of Minnesota. The trial plans to enroll 50 participants.