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World Data on Ambroxol for Patients With GD and GBA Related PD

NCT04388969 · Status: RECRUITING · Type: OBSERVATIONAL · Enrollment: 300

Last updated 2026-04-29

No results posted yet for this study

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).

Conditions

Sponsors & Collaborators

  • Shaare Zedek Medical Center

    lead OTHER

Principal Investigators

  • Ari Zimran, MD · Shaare Zedek Medical Center

Eligibility

Max Age
100 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-05-06
Primary Completion
2030-10-31
Completion
2030-11-30

Countries

  • Israel

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04388969 on ClinicalTrials.gov