Scientists track patients for 15 years after pioneering gene therapy for rare immune disease
NCT ID NCT06282432
Summary
This study follows 9 patients for up to 15 years after they received an experimental gene therapy for Leukocyte Adhesion Deficiency-I (LAD-I), a rare and serious immune system disorder. Researchers want to understand the long-term safety of the treatment and whether it continues to control the disease by helping the body fight infections. The therapy involved collecting patients' own blood stem cells, correcting their genetic defect in a lab, and returning them to the patient.
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 LEUKOCYTE ADHESION DEFICIENCY 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
Locations
-
Hospital Infantil Universitario Niño Jesús
Madrid, 28009, Spain
-
University College London Great Ormond Street Institute of Child Health (GOSH)
London, WC1N 1EH, United Kingdom
-
University of California, Los Angeles (UCLA)
Los Angeles, California, 90095-1489, United States
Conditions
Explore the condition pages connected to this study.