Double-blind, randomised, 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)

Conditions

Corticobasal Syndrome (CBS)

Brief summary

Primary efficacy endpoint: The primary outcome measure will be the longitudinal change of NfL in plasma between V1 and V3 comparing GPB vs. Placebo-treated patients., Safety: Incidence of ARs, Safety laboratory evaluation (basic clinical chemistry), Vital signs (blood pressure, heart rate, temperature), Physical and neurological examination, Survival time and survival rate during the study period, Tolerability: Number of subjects (and % of the intention-to-treat population), who discontinue the clinical trial due to adverse reactions

Detailed description

Longitudinal changes in clinical scales (MDS-UPDRS Part III, PSP-RS, PSP-CDS, CBFS, DATE, MoCA, SEADL, CGI-s, PSP-QoL) between V1 and V3 comparing placebo- vs. verum-treated patients

Carla Palleis, Endy Weidinger, Sylvia Maaß, Melanie Linke, Matthias Brendel et al. (16 authors)

Neurological Research and Practice2026-07-03

10.1186/s42466-026-00504-5

Interventions

DRUGRAVICTI 1.1 g/ml oral liquid

DRUGhydroxypropyl methylcellulose HPMC 4000

DRUGsodium benzoate

DRUGpropylene glycole

DRUGaqua destillata

Eligibility

Sex/Gender

All

Age

18 Years to No maximum

Design outcomes

Primary

Measure	Time frame
Primary efficacy endpoint: The primary outcome measure will be the longitudinal change of NfL in plasma between V1 and V3 comparing GPB vs. Placebo-treated patients., Safety: Incidence of ARs, Safety laboratory evaluation (basic clinical chemistry), Vital signs (blood pressure, heart rate, temperature), Physical and neurological examination, Survival time and survival rate during the study period, Tolerability: Number of subjects (and % of the intention-to-treat population), who discontinue the clinical trial due to adverse reactions	—

Measure

Time frame

Primary efficacy endpoint: The primary outcome measure will be the longitudinal change of NfL in plasma between V1 and V3 comparing GPB vs. Placebo-treated patients., Safety: Incidence of ARs, Safety laboratory evaluation (basic clinical chemistry), Vital signs (blood pressure, heart rate, temperature), Physical and neurological examination, Survival time and survival rate during the study period, Tolerability: Number of subjects (and % of the intention-to-treat population), who discontinue the clinical trial due to adverse reactions

—

Secondary

Measure	Time frame
Longitudinal changes in clinical scales (MDS-UPDRS Part III, PSP-RS, PSP-CDS, CBFS, DATE, MoCA, SEADL, CGI-s, PSP-QoL) between V1 and V3 comparing placebo- vs. verum-treated patients	—

Countries

Germany

Outcome results

None listed