Clinical Trial

PROFIL Study to Investigate the Effect of GPB on NfL Levels in Patients With Corticobasal Syndrome (CBS)

Study acronym: PROFIL
Active, Not Recruiting Phase 2
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Record status
This record was last updated March 18, 2026 (before its estimated March 30, 2026 completion). Its status may not reflect the trial's current state.
Summary
Corticobasal syndrome (CBS) is a rapidly progressive neurodegenerative disorder with an average survival time of about 6-8 years after the first clinical manifestation. No potent symptomatic treatment is currently available. A disease-modifying therapy does not exist either. Neuroinflammation is key to the pathogenesis in neurodegenerative diseases with Tau- and/or AD-pathology. There is strong evidence that phenylbutyrate can modulate microglial function by enhancing their phagocytic activity, most likely by epigenetic mechanisms. So the main goal of this clinical trial is to study a potential disease-modifying effect of treatment with glycerol phenylbutyrate (GPB), which is a prodrug of phenylbutyric acid, for 26 weeks assessed by the levels of the biomarker neurofilament light chain (NfL) indicating disease progression in CBS. Given the aggressive nature of CBS, it is feasible to study effects of GPB on plasma NfL levels.
Protocol Amendment History 4 amendments
This ClinicalTrials.gov record has been amended 4 times since 2023-08-01; most recent amendment 2026-03-17.
Status change: Recruiting → Active, Not Recruiting 2026-03-17
Status change: Not Yet Recruiting → Recruiting 2024-01-02
Trial Details
NCT Number NCT05983588
Lead Sponsor Technical University of Munich
Conditions Corticobasal Syndrome (CBS)
Enrollment 32 participants
Start Date 2023-12-12
Primary Completion 2026-03-30 (estimated)
Study Completion 2026-03-30 (estimated)
Updated on ClinicalTrials.gov 2026-03-18