World Data on Ambroxol for Patients With GD and GBA Related PD
Recruiting
Observational Study
Gaucher Disease
Parkinson Disease
GBA Gene Mutation
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
No Study Drug
Researchers observe your health over time — no experimental treatment is given.
At a Glance
- Age
- Up to 100
- Sex
- Any
- Study type
- Observational
- Participants needed
- 300 (estimated)
- Sponsor
- Shaare Zedek Medical Center · Other
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About This Trial
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 safe…
Trial Locations
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Eligibility Criteria
Inclusion Criteria:
* patients with Gaucher disease type 1,2 or 3(a,b,c).
* patients with GBA-related Parkinson disease.
Exclusion Criteria:
* None.
Contacts