Single-Patient trial offers hope for untreatable fatal brain disease
NCT ID NCT07272525
Summary
This is a compassionate use study for a single patient with Cree Leukoencephalopathy, a rare and fatal brain disease with no approved treatments. The study will provide access to an experimental drug called Fosigotifator (FGT) to see if it can slow or stop the progression of the disease. The main goal is to see if the patient survives or avoids needing a ventilator for two years after starting treatment.
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 VANISHING WHITE MATTER 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
-
McGill University Health Centre
Montreal, Quebec, H4A3J1, Canada
Conditions
Explore the condition pages connected to this study.