Cough medicine repurposed: ambroxol registry launches for rare brain diseases
NCT ID NCT04388969
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study creates a registry to collect real-world information on the safety and effectiveness of ambroxol, a common cough medicine, when used at higher doses for Gaucher disease or GBA-related Parkinson disease. Researchers aim to gather data from 300 patients worldwide who are already taking ambroxol off-label. The goal is to provide a reliable reference for patients, families, and doctors considering this treatment.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
ambroxol
What this could lead to
If successful, this registry could provide real-world evidence to guide doctors and patients considering ambroxol for Gaucher disease or GBA-related Parkinson disease.
What could go wrong
This is an observational registry, not a controlled trial, so it cannot prove cause and effect. Data quality depends on voluntary reporting, and results may be limited by incomplete or biased information.
Disclaimer
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the original study
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Shaare Zedek Medical Center
RECRUITINGJerusalem, Please Select..., 9103102, Israel
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Shaare Zedek Medical Center
RECRUITINGJerusalem, Israel
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Shaare Zedek Medical Center
RECRUITINGJerusalem, Israel
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••