Experimental drug TPN-101 tested in rare childhood brain disease
NCT ID NCT05613868
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tested a drug called TPN-101 (censavudine) in people with Aicardi-Goutières syndrome, a rare genetic disorder that causes severe brain inflammation. The trial enrolled only 4 participants and aimed to see if the drug could reduce immune system overactivity and check for side effects. The study was terminated early, so results are limited.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- TPN-101 (censavudine)
- What this could lead to
- If it works, this could point toward a treatment that reduces harmful immune system activity in Aicardi-Goutières syndrome.
- What could go wrong
- This was a very small, early-phase trial that was terminated, so results are limited. The drug may not effectively control the disease or could cause side effects.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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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.
Contacts and locations
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
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