Experimental drug TPN-101 studied for rare genetic brain disorder
NCT ID NCT05613868
First seen Apr 25, 2026 · Last updated May 01, 2026 · Updated 1 time
Summary
This early study tested an experimental drug called TPN-101 in people with Aicardi-Goutières syndrome (AGS), a rare genetic disorder that causes severe brain inflammation. The goal was to see if the drug could reduce abnormal immune system activity and check for side effects. Only 4 people took part before the study was stopped early.
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 AICARDI-GOUTIÈRES SYNDROME (AGS) 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
-
Istituto Neurologico Casimiro Mondino
Pavia, 27100, Italy
-
Laboratory of Neurogenetics and Neuroinflammation Imagine Institute - INSERM U1163
Paris, 75015, France
-
Presidio Ospedale dei Bambini [Children's Hospital]
Brescia, 25123, Italy
-
Royal Hospital for Children and Young People
Edinburgh, EH9 1LF, United Kingdom
-
SST Fatebenefratelli Sacco
Milan, 20154, Italy
Conditions
Explore the condition pages connected to this study.