One-Time gene therapy could change fabry disease treatment
NCT ID NCT04046224
First seen Apr 23, 2026 · Last updated May 15, 2026 · Updated 5 times
Summary
This study tested a new gene therapy called ST-920 for people with 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 received a single intravenous dose and were followed for one year to check safety and how well the treatment works. The goal is to reduce harmful substances in the body and improve symptoms.
Disclaimer
Read more
Show less
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.
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.
By submitting, you agree to our Terms of use
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
By submitting, you agree to our Terms of use
Locations
-
Addenbrooke's Hospital
Cambridge, CB2 0QQ, United Kingdom
-
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611, United States
-
Azienda Ospedaliero-Universitaria Careggi
Florence, Tuscany, 50134, Italy
-
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
-
Emory University School of Medicine
Atlanta, Georgia, 30322, United States
-
Lysosomal and Rare Disorders Research and Treatment Center (LDRTC)
Fairfax, Virginia, 22030, United States
-
M.A.G.I.C. Clinic Ltd.
Calgary, Alberta, T2E 7Z4, Canada
-
Mt. Sinai School of Medicine
New York, New York, 10029, United States
-
National Taiwan University Hospital
Taipei, Taiwan
-
Queen Elizabeth Hospital
Birmingham, B15 2TH, United Kingdom
-
Royal Free Hospital
London, United Kingdom
-
The Royal Melbourne Hospital
Parkville, Victoria, 3050, Australia
-
University Hospital of Würzburg
Würzburg, Germany
-
University Medical Center Hamburg-Eppendorf
Hamburg, Germany
-
University of California, Irvine
Irvine, California, 92697, United States
-
University of Iowa Hospital and Clinics
Iowa City, Iowa, 52242, United States
-
University of Minnesota Medical Center
Minneapolis, Minnesota, 55455, United States
-
University of South Florida
Tampa, Florida, 33620, United States
Conditions
Explore the condition pages connected to this study.