Cough medicine repurposed: ambroxol registry launches for rare brain diseases

NCT ID NCT04388969

Recruiting now Knowledge-focused Sponsor: Shaare Zedek Medical Center Source: ClinicalTrials.gov ↗

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.

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Conditions

The condition(s) this trial relates to.

Gaucher disease Gaucher disease type I Gaucher disease type II Gaucher disease type III GBA1-related Parkinson disease, susceptibility Parkinson disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

GAUCHER DISEASE GBA GENE MUTATION PARKINSON DISEASE

Contacts and locations

Locations

Shaare Zedek Medical Center
RECRUITING

Jerusalem, Please Select..., 9103102, Israel
Shaare Zedek Medical Center
RECRUITING

Jerusalem, Israel
Shaare Zedek Medical Center
RECRUITING

Jerusalem, Israel

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