Protocol of ASPro-PD: a phase 3 trial of ambroxol to slow progression in genetically stratified Parkinson's disease.

ABSTRACT:

Introduction

Genetic studies have identified the GBA1 gene as a significant genetic risk factor for Parkinson's disease (PD), with 10-15% of PD patients carrying GBA1 variants. GBA1 variants affect the glucocerebrosidase (GCase) enzyme, often leading to reduced GCase activity and associated altered lysosomal function, implicated in PD pathogenesis. Ambroxol, a small molecule widely used for respiratory diseases, has emerged as a potential therapeutic agent for PD, acting by increasing GCase activity. A phase 2 trial demonstrated ambroxol's safety and efficacy in penetrating cerebrospinal fluid (CSF) and engaging with its target in PD patients, including those with GBA1 variants.

Methods

We present the protocol of the ASPro-PD trial, a phase 3, multicentre, randomised, double

SUBMITTER: Toffoli M 

PROVIDER: S-EPMC12916515 | biostudies-literature | 2026 Feb

REPOSITORIES: biostudies-literature