First-in-Human gene therapy trial targets fatal heart damage in rare disease
NCT ID NCT05302271
Summary
This early-stage study is testing the safety of a new gene therapy for the serious heart muscle damage that occurs in people with Friedreich's ataxia. The therapy, called AAVrh.10hFXN, is given through an IV and aims to deliver a working copy of the frataxin gene directly to the heart. Researchers will enroll 25 adults with the condition to find a safe dose and look for early signs that it might help control the heart disease.
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 CARDIOMYOPATHIES 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
-
Weill Cornell Medicine
RECRUITINGNew York, New York, 10021, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
Conditions
Explore the condition pages connected to this study.