Gene therapy could free gaucher patients from lifelong infusions
NCT ID NCT07223944
First seen Nov 03, 2025 · Last updated May 06, 2026 · Updated 24 times
Summary
This study tests a one-time gene therapy called FLT201 for adults with Gaucher disease type 1. Participants currently on regular enzyme replacement therapy will stop that treatment after receiving FLT201. The goal is to see if the gene therapy can keep blood counts stable and control the disease without ongoing medication. About 45 people will take part in this Phase 3 trial.
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.
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
Study contacts
-
Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
-
Duke University Medical Center
RECRUITINGDurham, North Carolina, 27710, United States
-
Lysosomal Rare Disorders Research and Treatment Center
RECRUITINGFairfax, Virginia, 22030-6066, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact Phone: •••-•••-•••• Email: •••••@•••••
-
University of Texas Southwestern
RECRUITINGDallas, Texas, 75390, United States
Conditions
Explore the condition pages connected to this study.