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

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.

Timeline

Start

2023-12-12

Primary completion

2026-03-30

Completion

2026-03-30

Publications

• Palleis C, Weidinger E, Maass S, Linke M, Brendel M, Stocklein S, Al Tawil A, Mansmann U, Teupser D, Borst T, Heimke-Brinck R, Hemmer B, Hoglinger GU, Bidner H, Simons M, Levin J. Study protocol: double-blind, randomized, prospective, placebo controlled parallel group phase II study to investigate the effect of glycerol phenylbutyrate (GPB) on neurofilament light chain (NfL) levels in patients with corticobasal syndrome (CBS). Neurol Res Pract. 2026 Jul 3;8(1):54. doi: 10.1186/s42466-026-00504-5.

Drugs

Indications