Can a single gene infusion rewrite the story of fabry disease?

NCT ID NCT04040049

Terminated ⭐️ CURE ⭐️ Sponsor: Spur Therapeutics Source: ClinicalTrials.gov ↗

First seen Aug 06, 2026 · Last updated Aug 07, 2026 · Updated 1 time

Summary

This trial is testing a gene therapy called FLT190 in adult men with classic Fabry disease, a genetic condition that causes harmful fat buildup in cells. The therapy uses a modified virus to deliver a working copy of the faulty gene, potentially enabling the body to produce the missing enzyme. The study aims to see if a single dose is safe and can reduce disease symptoms.

What this could mean

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

Active substance: FLT190, a gene therapy delivered via a modified virus
What this could lead to: If successful, a single infusion of FLT190 could provide a long-term or permanent fix for Fabry disease, reducing or eliminating the need for regular enzyme replacement therapy.
What could go wrong: This is an early-stage trial with a small number of participants, so safety and effectiveness are not yet proven. Gene therapy carries risks such as immune reactions or the treatment not working as intended.

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 lysosomal storage disease

As listed by the trial registrant

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

FABRY DISEASE LYSOSOMAL STORAGE DISEASES

Contacts and locations

Locations

Charité - Universitätsmedizin Berlin

Berlin, Germany
Columbia University

New York, New York, 10032, United States
Haukeland University Hospital

Bergen, Norway
Kaiser Permanente

Los Angeles, California, 90027, United States
Lysosomal and Rare Disorders Research and Treatment Center

Fairfax, Virginia, 22030, United States
Medical University of Vienna

Vienna, Austria
Metabolics and Genetics in Calgary (MAGIC Clinic)

Calgary, Toronto, T2E 7Z4, Canada
Royal Free Hospital

London, United Kingdom
Salford Royal NHS Foundation Trust

Salford, United Kingdom
UKEA University Hospital Hamburg

Hamburg, Germany
UPMC Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, 15224, United States
Universita Federico II di Napoli

Naples, Italy
University of Würzburg

Würzburg, Germany

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