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.

Disclaimer Read more

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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Conditions

The condition(s) this trial relates to.

Corticobasal Degeneration corticobasal syndrome progressive supranuclear palsy progressive supranuclear palsy-corticobasal syndrome

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • 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

  • 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