Actively Recruiting

Age: 0 - 100Years
All Genders
NCT04388969

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

Age: 0 - 100Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Patients with Gaucher disease type 1, 2, or 3 (a, b, c)
  • Patients with GBA-related Parkinson disease
Not Eligible

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

Complete this quick 3-step screening to check your eligibility

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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

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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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