Actively Recruiting
World Data on Ambroxol for Patients With GD and GBA Related PD
Led by Shaare Zedek Medical Center · Updated on 2026-04-29
300
Participants Needed
3
Research Sites
551 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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).
CONDITIONS
Official Title
World Data on Ambroxol for Patients With GD and GBA Related PD
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patients with Gaucher disease type 1, 2, or 3 (a, b, c)
- Patients with GBA-related Parkinson disease
You will not qualify if you...
History of severe allergic reactions to study medication Currently pregnant or breastfeeding Recent participation in another clinical trial within the last 30 days Presence of uncontrolled medical conditions that could affect safety
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 3 locations
1
Shaare Zedek Medical Center
Jerusalem, Please Select..., Israel, 9103102
Actively Recruiting
2
Shaare Zedek Medical Center
Jerusalem, Israel
Actively Recruiting
3
Shaare Zedek Medical Center
Jerusalem, Israel
Actively Recruiting
Research Team
M
Majdolen Istaiti, BS.C
CONTACT
A
Ari Zimran, MD
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
2
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