Fabry disease
Clinical trials for Fabry disease explained in plain language.
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Can a new pill stop fatty buildup in fabry disease?
Disease control Not yet recruitingThis trial tests whether the drug lucerastat can reduce the buildup of a fatty substance called Gb3 in the kidneys of adult men with Fabry disease who have not received prior treatment. Participants will take lucerastat daily for 18 months and undergo kidney biopsies to measure c…
Matched conditions: FABRY DISEASE
Phase 3 • Sponsor: Idorsia Pharmaceuticals Ltd. • Aim: Disease control
Last updated Aug 23, 2026 00:00 UTC
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Fabry disease diagnosis differs between men and women, new study aims to find out how
Knowledge-focused Not yet recruitingThis study will survey 200 adults with Fabry disease to understand how men and women experience different paths to diagnosis. Researchers want to see if symptoms or family screening lead to diagnosis more often in one sex, and how long diagnosis takes. The goal is to identify pat…
Matched conditions: FABRY DISEASE
Sponsor: University Hospital, Toulouse • Aim: Knowledge-focused
Last updated Jun 27, 2026 11:03 UTC
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Could a PET scan spot heart trouble in fabry disease before It's too late?
Knowledge-focused Not yet recruitingThis study will test a special PET scan tracer and blood tests to better understand and predict heart damage in people with Fabry disease. Researchers will look at heart tissue samples and blood markers from 20 adult patients. The goal is to find new ways to diagnose and forecast…
Matched conditions: FABRY DISEASE
Sponsor: Núcleo de Apoio à Investigação Clínica - FMUP • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:12 UTC