Single gene shot could tame rare enzyme disorder
NCT ID NCT06818838
First seen Jan 23, 2026 · Last updated May 23, 2026 · Updated 13 times
Summary
This study tests a one-time gene therapy injection for adults with Gaucher disease type 1, a rare genetic condition that causes organ enlargement and bone problems. The therapy uses a harmless virus to deliver a working gene to the liver, so the body can produce the missing enzyme. The trial will enroll 12 participants to check safety and whether the treatment improves blood counts and reduces organ size.
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 GAUCHER DISEASE, TYPE 1 are added.
Genom att skicka in godkänner du våra Användarvillkor
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
Genom att skicka in godkänner du våra Användarvillkor
Study contacts
-
Contact
Phone: •••-•••-•••• Email: •••••@•••••
-
Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
-
Guangzhou First People's Hospital
RECRUITINGGuangzhou, Guangdong, 510000, China
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
-
Hematology Hospital, Chinese Academy of Medical Sciences
RECRUITINGTianjin, Tianjin Municipality, 300011, China
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
-
Shanxi Bethune Hospital
RECRUITINGTaiyuan, Shanxi, 030000, China
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
Conditions
Explore the condition pages connected to this study.