Gene therapy for blood disorder: will patients stay Transfusion-Free?
NCT ID NCT06479616
First seen Oct 31, 2025 · Last updated May 16, 2026 · Updated 28 times
Summary
This study follows 5 people with beta-thalassemia who already received a one-time gene therapy called CS-101. Researchers want to see if the treatment remains safe and effective over many years, including whether patients can stay free from blood transfusions. The study does not offer new treatment but monitors long-term outcomes.
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 BETA-THALASSEMIA 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: •••••@•••••
-
Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
-
Children's Hospital of Fudan University
RECRUITINGShanghai, Shanghai Municipality, 201102, China
Contact Phone: •••-•••-•••• Email: •••••@•••••
Conditions
Explore the condition pages connected to this study.