RAVICTI 1.1 g/ml oral liquid

DRUG2 trials

Sponsors

Assistance Publique Hopitaux De Paris, Klinikum der Technischen Universitaet Muenchen (TUM Klinikum)

Conditions

Corticobasal Syndrome (CBS)Pyruvate dehydrogenase deficiency

Phase 2

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)

Active, not recruitingCTIS2024-516897-31-00

Klinikum der Technischen Universitaet Muenchen (TUM Klinikum)Corticobasal Syndrome (CBS)

Start: 2023-12-01Target: 32Updated: 2025-09-18

PDH-RAVICTI - A PHASE II, MULTICENTRIC, PROSPECTIVE, NON-COMPARATIVE CLINICAL TRIAL TO ASSESS THE EFFICACY AND SAFETY OF THE TREATMENT OF PYRUVATE DEHYDROGENASE DEFICIENCY (PDH) PATIENTS WITH GLYCEROL PHENYLBUTYRATE (RAVICTI®)

RecruitingCTIS2024-516410-38-00

Assistance Publique Hopitaux De ParisPyruvate dehydrogenase deficiency

Start: 2025-10-01Target: 15Updated: 2025-12-29

Related Papers

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)

Neurological Research and Practice2026-07-03