One-Time gene therapy could change fabry disease treatment
NCT ID NCT04046224
First seen Apr 23, 2026 · Last updated Jun 23, 2026 · Updated 11 times
Summary
This trial tested a new gene therapy called ST-920 for Fabry disease, a rare genetic condition. The therapy uses a harmless virus to deliver a working gene that helps the body produce an enzyme it's missing. 36 adults with Fabry disease received a single intravenous dose and were monitored for a year to check safety and tolerability. The goal is to see if this one-time treatment can provide long-term benefits.
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Locations
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Addenbrooke's Hospital
Cambridge, CB2 0QQ, United Kingdom
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Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611, United States
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Azienda Ospedaliero-Universitaria Careggi
Florence, Tuscany, 50134, Italy
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Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
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Emory University School of Medicine
Atlanta, Georgia, 30322, United States
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Lysosomal and Rare Disorders Research and Treatment Center (LDRTC)
Fairfax, Virginia, 22030, United States
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M.A.G.I.C. Clinic Ltd.
Calgary, Alberta, T2E 7Z4, Canada
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Mt. Sinai School of Medicine
New York, New York, 10029, United States
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National Taiwan University Hospital
Taipei, Taiwan
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Queen Elizabeth Hospital
Birmingham, B15 2TH, United Kingdom
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Royal Free Hospital
London, United Kingdom
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The Royal Melbourne Hospital
Parkville, Victoria, 3050, Australia
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University Hospital of Würzburg
Würzburg, Germany
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University Medical Center Hamburg-Eppendorf
Hamburg, Germany
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University of California, Irvine
Irvine, California, 92697, United States
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University of Iowa Hospital and Clinics
Iowa City, Iowa, 52242, United States
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University of Minnesota Medical Center
Minneapolis, Minnesota, 55455, United States
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University of South Florida
Tampa, Florida, 33620, United States
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
ST-920 (a gene therapy using a modified virus to deliver a working copy of the alpha-galactosidase A gene)
What this could lead to
If successful, this could provide a long-term treatment option for Fabry disease, potentially reducing or eliminating the need for regular enzyme replacement therapy.
What could go wrong
This is an early-phase trial (Phase 1/2) with only 36 participants, so safety and effectiveness are not yet proven. Gene therapies can have unexpected side effects, and long-term benefits are uncertain.
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.