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

Recruitingn/aAges any - 100 YearsAllShaare Zedek Medical Center
NCT04388969 on ClinicalTrials.gov ↗
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In plain words

What the trial is, and whether it could suit you

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).

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📋 Eligibility rules (as published)
Inclusion Criteria: * patients with Gaucher disease type 1,2 or 3(a,b,c). * patients with GBA-related Parkinson disease. Exclusion Criteria: * None.
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What it tests

  • Not listed
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Where

3 sites
Shaare Zedek Medical Center
Jerusalem, Israel
Shaare Zedek Medical Center
Jerusalem, Israel
Shaare Zedek Medical Center
Jerusalem, Israel
🤝 Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible — always decide together with your own doctor.
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