Status:
UNKNOWN
World Data on Ambroxol for Patients With GD and GBA Related PD
Lead Sponsor:
Shaare Zedek Medical Center
Conditions:
Gaucher Disease
Parkinson Disease
Eligibility:
All Genders
Up to 100 years
Brief 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 glucocereb...
Detailed Description
The three decades of success of enzyme replacement therapy (ERT) for patients with type 1 Gaucher disease (GD1) have been just a partial success to those patients with the more severe neuronopathic fo...
Eligibility Criteria
Inclusion
- patients with Gaucher disease type 1,2 or 3(a,b,c).
- patients with GBA-related Parkinson disease.
Exclusion
- None.
Key Trial Info
Start Date :
May 6 2020
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
January 31 2024
Estimated Enrollment :
300 Patients enrolled
Trial Details
Trial ID
NCT04388969
Start Date
May 6 2020
End Date
January 31 2024
Last Update
February 14 2023
Active Locations (3)
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1
Shaare Zedek Medical Center
Jerusalem, Please Select..., Israel, 9103102
2
Shaare Zedek Medical Center
Jerusalem, Israel
3
Shaare Zedek Medical Center
Jerusalem, Israel