Can early enzyme therapy save kidneys in fabry disease?

NCT ID NCT01581424

Recruiting now Knowledge-focused Sponsor: University of Minnesota Source: ClinicalTrials.gov ↗

First seen Aug 14, 2026 · Last updated Aug 14, 2026

Summary

This study investigates how Fabry disease damages the kidneys over time, even before routine tests show problems. Researchers will examine kidney biopsies and function in 50 patients, some of whom receive enzyme replacement therapy. They aim to see if starting treatment earlier or at higher doses better clears harmful fat deposits from kidney cells, potentially preserving kidney function.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: Enzyme replacement therapy (ERT) for Fabry disease
What this could lead to: If successful, this could help doctors predict kidney decline earlier and tailor enzyme replacement therapy to protect kidneys better in Fabry disease.
What could go wrong: This is an observational and exploratory study, not a large clinical trial. Findings may not apply to all patients, and the long-term benefits of earlier or higher-dose therapy remain uncertain.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

Fabry disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

FABRY DISEASE

Contacts and locations

Locations

University of Minnesota
RECRUITING

Minneapolis, Minnesota, 55455, United States
Universtity of Minnesota, Department of Pediatric Nephrology
RECRUITING

Minneapolis, Minnesota, 55455, United States

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