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Cough medicine repurposed: ambroxol registry launches for rare brain diseases

NCT ID NCT04388969

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

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Participants

About 300 people

The number the study aims to enrol. It can still change while the study runs.

Started

May 2020

Expected to finish

Nov 2030

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Who is studied

Patients with Gaucher disease type 1,2,3 (a,b,c) and patients with GBA-related Parkinson disease.

Ages

Up to 100 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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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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

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    3 sites. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

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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