World Data on Ambroxol for Patients With GD and GBA Related PD
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
Shaare Zedek Medical Center
300 participants
May 6, 2020
OBSERVATIONAL
Conditions
Summary
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).
Eligibility
Inclusion Criteria2
- patients with Gaucher disease type 1,2 or 3(a,b,c).
- patients with GBA-related Parkinson disease.
Exclusion Criteria1
- None.
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Locations(3)
View Full Details on ClinicalTrials.gov
For the most up-to-date information, visit the official listing.
NCT04388969