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
1 other identifier
observational
300
1 country
3
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 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).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for all trials
Started May 2020
Longer than P75 for all trials
3 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
May 6, 2020
CompletedFirst Submitted
Initial submission to the registry
May 10, 2020
CompletedFirst Posted
Study publicly available on registry
May 15, 2020
CompletedPrimary Completion
Last participant's last visit for primary outcome
October 31, 2030
ExpectedStudy Completion
Last participant's last visit for all outcomes
November 30, 2030
April 29, 2026
April 1, 2026
10.5 years
May 10, 2020
April 23, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (2)
Establishing a registry to be used as a reference for patients,parents and physicians, who consider using Ambroxol
With no specific treatment to offer for patients (particularly children and young adults) with nGD, and with all the above-mentioned encouraging preliminary reports, in our current era of rapidly spreading information (in many meetings worldwide, in the internet and via the social media) individual patients in many countries have started to receive off-label Ambroxol by their treating physicians and a few case reports have been published from Korea , Israel and Canada . In order to upscale the level of evidence from anecdotal reports to an observational study, we herein suggest to establish a specific IIR, wherein the "R" stands for a "Registry", that may be used as a reference for patients , parents and physicians, who consider using Ambroxol.
10 years
Safety Outcome
By reporting adverse events
10 years
Study Arms (2)
Gaucher disease patients
Patients with type 1,2,3 Gaucher disease.
GBA carriers with Parkinson disease
Patients with Parkinson disease GBA related (carriers)
Eligibility Criteria
Patients with Gaucher disease type 1,2,3 (a,b,c) and patients with GBA-related Parkinson disease.
You may qualify if:
- patients with Gaucher disease type 1,2 or 3(a,b,c).
- patients with GBA-related Parkinson disease.
You may not qualify if:
- None.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (3)
Shaare Zedek Medical Center
Jerusalem, Please Select..., 9103102, Israel
Shaare Zedek Medical Center
Jerusalem, Israel
Shaare Zedek Medical Center
Jerusalem, Israel
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Ari Zimran, MD
Shaare Zedek Medical Center
Central Study Contacts
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- RETROSPECTIVE
- Sponsor Type
- OTHER
- Responsible Party
- PRINCIPAL INVESTIGATOR
- PI Title
- Principal Investigator
Study Record Dates
First Submitted
May 10, 2020
First Posted
May 15, 2020
Study Start
May 6, 2020
Primary Completion (Estimated)
October 31, 2030
Study Completion (Estimated)
November 30, 2030
Last Updated
April 29, 2026
Record last verified: 2026-04