Clinical trial Recruiting at 2 sites Observational Ages 1 Year to 75

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 typeObservational
Ages1 Year to 75 Years
SexAll
Enrollment50 (estimated)
SponsorUniversity of Minnesota
Academic / other
Locations2 sites (Minneapolis, Minnesota and 1 other locations)
StatusRecruiting
StartedOctober 2010
Main results expectedJuly 31, 2030
Study endsJuly 31, 2030
Registry record updatedAugust 14, 2026
Trial IDNCT01581424 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

  • 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

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

How to take part

  1. Review the eligibility criteria above with your treating physician.
  2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
  3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.
Last reviewed 2026-11-14 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.