World Data on Ambroxol for Patients With GD and GBA Related PD

Ambroxol hydrochloride is an oral mucolytic drug available over-the-counter for many years as cough medicine. In 2009 it was found to also act as a pharmacological chaperone (PC) for mutant glucocerebrosidase, albeit in a several-fold higher dose. Unfortunately, due to its low cost, there have been no pharma-driven clinical trials to establish the use of ambroxol. Thus, data are needed on the safety and efficacy of ambroxol for patients with Gaucher disease (GD).

NCT Number	NCT04388969
Lead Sponsor	Shaare Zedek Medical Center
Conditions	Gaucher Disease, Parkinson Disease, GBA Gene Mutation
Enrollment	300 participants
Start Date	2020-05-06
Primary Completion	2030-10-31 (estimated)
Study Completion	2030-11-30 (estimated)
Updated on ClinicalTrials.gov	2026-04-29