Experimental drug TPN-101 targets rare inflammatory brain disease
NCT ID NCT05613868
First seen Apr 25, 2026 · Last updated Jun 11, 2026 · Updated 10 times
Summary
This study tested an experimental drug called TPN-101 in people with Aicardi-Goutières syndrome, a rare genetic disorder that causes severe brain inflammation. The goal was to see if the drug could lower abnormal immune activity and check for side effects. Only 4 participants were enrolled before the study was stopped early.
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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.
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Contacts and locations
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Locations
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Istituto Neurologico Casimiro Mondino
Pavia, 27100, Italy
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Laboratory of Neurogenetics and Neuroinflammation Imagine Institute - INSERM U1163
Paris, 75015, France
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Presidio Ospedale dei Bambini [Children's Hospital]
Brescia, 25123, Italy
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Royal Hospital for Children and Young People
Edinburgh, EH9 1LF, United Kingdom
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SST Fatebenefratelli Sacco
Milan, 20154, Italy
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.