PROFIL Study to Investigate the Effect of GPB on NfL Levels in Patients With Corticobasal Syndrome (CBS)
Status:
Recruiting
Trial end date:
2025-03-01
Target enrollment:
Participant gender:
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.