Could a common drug slow down this rare brain disease?
NCT ID NCT05983588
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This phase 2 trial tests whether glycerol phenylbutyrate (GPB) can slow corticobasal syndrome (CBS), a rare and fast-progressing brain disease. 32 adults with CBS will receive either GPB or a placebo for 26 weeks. The main goal is to see if GPB lowers levels of a nerve damage marker called NfL in the blood.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- glycerol phenylbutyrate (GPB)
- What this could lead to
- If successful, this could point toward a treatment that slows the progression of corticobasal syndrome, a rare and aggressive brain disease.
- What could go wrong
- This is a small early-phase trial with only 32 people, so results may not apply widely. The drug may not reduce NfL levels or improve symptoms, and side effects are possible.
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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Klinikum der Universität München (KUM), Campus Großhadern, Klinik und Poliklinik für Neurologie & German Center for Neurodegenerative Diseases (DZNE), Department of Neurology
Munich, Bavaria, 81377, Germany
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Klinikum rechts der Isar Technische Universität München Klinik und Poliklinik für Neurologie & German Center for Neurodegenerative Diseases (DZNE), Department of Neurology
Munich, Bavaria, 81377, Germany
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