One-Shot gene therapy trial offers hope for devastating infant disease
NCT ID NCT04771416
Summary
This study is testing a one-time gene therapy called PBKR03 for babies with early infantile Krabbe disease, a rare and severe neurological disorder. Researchers will give the therapy directly into the fluid around the brain to deliver a working copy of a missing gene. The main goals are to see if the treatment is safe and if it can slow or stabilize the disease by improving nerve function and development.
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 LEUKODYSTROPHY, GLOBOID CELL 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
-
Amsterdam UMC
Amsterdam, Netherlands
-
Ann & Robert Lurie
Chicago, Illinois, 60611, United States
-
Hospital De Clinicas De Porto Alegre
Porto Alegre, Brazil
-
Manchester University
Manchester, United Kingdom
-
Montreal Children's Hospital
Montreal, Canada
-
New York-Presbyterian
New York, New York, 10065, United States
-
Shaare Zadek Medical Center
Jerusalem, Israel
-
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
-
University of Utah School of Medicine
Salt Lake City, Utah, 84132, United States
Conditions
Explore the condition pages connected to this study.