Collects real-world data on ambroxol for Gaucher disease and GBA-linked Parkinson disease
Records safety and effects of ambroxol in people with Gaucher disease or Parkinson disease.
Plain-language title generated from the registry record
Registry title: World Data on Ambroxol for Patients With GD and GBA Related PD
Key facts
- Where
- Jerusalem, IsraelSee the site
Where in Israel
- Shaare Zedek Medical CenterJerusalem, IsraelTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Shaare Zedek Medical Center
- Trial code
- NCT04388969
- Record updated
- 29 April 2026
- Original description
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).Read full original descriptionShow less
IIR REGISTRY for the Collection of Real World Data on the Safety and Efficacy of Ambroxol for Patients With Gaucher Disease or GBA Carriers With Parkinson Disease
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).
Inclusion: patients with Gaucher disease type 1,2 or 3(a,b,c); patients with GBA-related Parkinson disease.
Exclusion: None.