Understanding kidney changes in Fabry disease
Official title: Natural History and Structural Functional Relationships in Fabry Renal Disease Natural History, Structural Functional Relationships and Determinants of Renal Structural Responses (Changes) With Enzyme Replacement Therapy in Fabry Disease
Run by University of Minnesota · NCT01581424 · Data updated 2026-11-12
This study looks at how kidney problems develop in people with Fabry disease before they start treatment to help understand the disease better and find ways to intervene early.
Quick facts
| Study type | Observational |
|---|---|
| Ages | 1 Year to 75 Years |
| Sex | All |
| Enrollment | 50 (estimated) |
| Sponsor | University of Minnesota Academic / other |
| Locations | 2 sites (Minneapolis, Minnesota and 1 other locations) |
| Status | Recruiting |
| Started | October 2010 |
| Main results expected | July 31, 2030 |
| Study ends | July 31, 2030 |
| Registry record updated | August 14, 2026 |
| Trial ID | NCT01581424 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates include patients diagnosed with Fabry disease who are undergoing kidney biopsies and have not received enzyme replacement therapy.
✕Probably not a fit
Patients with serum creatinine levels above 2.5 mg/dL or those with other renal diseases may not benefit from this study.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This observational study aims to investigate the natural history of kidney changes in patients with Fabry disease before they start enzyme replacement therapy. It will analyze kidney biopsies to identify microscopic structural changes and correlate these with kidney function metrics such as glomerular filtration rate and urinary albumin excretion. The study seeks to establish a clearer understanding of how kidney injury progresses in Fabry patients, potentially leading to better early intervention strategies. By focusing on untreated patients, the research will provide insights into the disease's progression and its impact on kidney health.
Why it matters
Potential benefit: If successful, this study could lead to improved early detection and intervention strategies for kidney dysfunction in Fabry disease patients.
How similar studies have gone: Other studies have shown success in understanding kidney function in Fabry disease, but this specific approach focusing on untreated patients is relatively novel.
Who can join: the full rules
Show full inclusion / exclusion 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.
Where it's running
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University of Minnesota
Minneapolis, Minnesota, United States Recruiting -
Universtity of Minnesota, Department of Pediatric Nephrology
Minneapolis, Minnesota, United States Recruiting
Study contacts
- Principal investigator: Chet Whitley, MD — University of Minnesota
- Study coordinator: Michael Mauer, MD
- Email: mauer002@umn.edu
- Phone: 612-626-2720
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How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.