Ambroxol for the Treatment of Patients With Parkinson Disease With and Without Glucocerebrosidase Gene Mutations: A Nonrandomized, Noncontrolled Trial.

ABSTRACT:

Importance

Mutations of the glucocerebrosidase gene, GBA1 (OMIM 606463), are the most important risk factor for Parkinson disease (PD). In vitro and in vivo studies have reported that ambroxol increases β-glucocerebrosidase (GCase) enzyme activity and reduces α-synuclein levels. These observations support a potential role for ambroxol therapy in modifying a relevant pathogenetic pathway in PD.

Objective

To assess safety, tolerability, cerebrospinal fluid (CSF) penetration, and target engagement of ambroxol therapy with GCase in patients with PD with and without GBA1 mutations.

Interventions

An escalating dose of oral ambroxol to 1.26 g per day.

Design, setting, and participants

This single-center open-label noncontrolled clinical trial was conducted between Jan

SUBMITTER: Mullin S 

PROVIDER: S-EPMC6990847 | biostudies-literature | 2020 Apr

REPOSITORIES: biostudies-literature