New hope for fabry patients with kidney failure: drug dosing study underway

NCT ID NCT04020055

Ongoing Disease control Sponsor: Amicus Therapeutics Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jul 07, 2026 · Updated 2 times

Summary

This study tests the safety and how the body processes migalastat in 14 adults with Fabry disease who have severe kidney impairment or are on dialysis. Participants take migalastat capsules, and researchers measure drug levels in blood and dialysate. The goal is to ensure proper dosing for this vulnerable group.

What this could mean

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

Active substance: migalastat (a drug that helps break down fatty substances in cells)
What this could lead to: If successful, this could confirm that migalastat is safe and properly dosed for Fabry patients with severe kidney problems, expanding treatment options for this group.
What could go wrong: This is a small, early-stage study (14 people) focused on safety and drug levels, not on whether the drug improves symptoms. It may not lead to a new treatment if safety issues arise.

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

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for FABRY DISEASE are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

Conditions

The condition(s) this trial relates to.

chronic kidney disease Fabry disease kidney disorder kidney failure

As listed by the trial registrant

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

FABRY DISEASE

Contacts and locations

Locations

Centro Hospitalar e Universitário de Coimbra (CHUC)

Coimbra, 3041-801, Portugal
Emory University

Atlanta, Georgia, 30322, United States
Hospital General Universitario Gregorio Marañon

Madrid, 28007, Spain
Hospital General Universitario de Elda

Elda, 03600, Spain
Hospital Universitari(o) de Bellvitge (HUB) Feixa Llarga

Barcelona, 08907, Spain
Lysosomal and Rare Disorders Research and Treatment Center, Inc

Fairfax, Virginia, 22030, United States
Osaka University Hospital

Suita, Osaka, 565-0871, Japan
Royal Melbourne Hospital

Parkville, Victoria, 3050, Australia
Royal Perth Hospital

Perth, Washington, 6000, Australia
Salford Royal Hospital

Salford, England, M6 8HD, United Kingdom
Shizuoka General Hospital

Shizuoka, Shizuoka, 420-8527, Japan
The Cleveland Clinic

Cleveland, Ohio, 44195, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.