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
Status: Recruiting
Location: See all (3) locations...
Study Type: Observational
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
Participation Requirements
Sex: All
Maximum Age: 100
Healthy Volunteers: f
View:
• patients with Gaucher disease type 1,2 or 3(a,b,c).
• patients with GBA-related Parkinson disease.
Locations
Other Locations
Israel
Shaare Zedek Medical Center
RECRUITING
Jerusalem
Shaare Zedek Medical Center
RECRUITING
Jerusalem
Shaare Zedek Medical Center
RECRUITING
Jerusalem
Contact Information
Primary
Majdolen Istaiti, BS.C
joleenist@szmc.org.il
+972-2-5645681
Backup
Ari Zimran, MD
azimran@gmail.com
+972-2-5645681
Time Frame
Start Date: 2020-05-06
Estimated Completion Date: 2030-11-30
Participants
Target number of participants: 300
Treatments
Gaucher disease patients
Patients with type 1,2,3 Gaucher disease.
GBA carriers with Parkinson disease
Patients with Parkinson disease GBA related (carriers)
Related Therapeutic Areas
Sponsors
Leads: Shaare Zedek Medical Center