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Cough medicine repurposed for rare brain disease in kids

NCT ID NCT07285369

Disease control Sponsor: Agyany Pharma LTD Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
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 This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Aug 25, 2026 · Updated 2 times

Summary

This study tested high doses of Ambroxol, a common cough medicine, in 12 children aged 3 to 18 with Type 3 Gaucher disease, a rare genetic disorder affecting the brain and body. The goal was to see if it is safe and can improve movement problems like ataxia. The trial took place in Lahore, Pakistan, and included children who were either new to treatment or already on enzyme therapy.

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.

Phase

Not a phased trial

Phase numbers describe drug development. The registry uses this when they do not apply, as it does for trials of devices, procedures or behaviour changes, and for observational studies.

Participants

12 people

The number who actually took part.

Started

Mar 2025

Finished

Mar 2026

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

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

Ages

3 to 18 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: * Pediatric patients aged 3 to 18 years * Genetically confirmed Type III Gaucher Disease (GD3) * Treatment naïve or receiving enzyme replacement therapy (ERT) * SARA score ≥ 8 * Sexually active females must agree to use contraception * All participants must not be pregnant or breastfeeding Exclusion Criteria: * Life-threatening visceral disease (related or unrelated to Gaucher Disease) * Blood transfusion dependency * Clinically significant cardiovascular, gastrointestinal, pulmonary, neurologic, endocrine, or psychiatric conditions * Serious swallowing difficulties * Renal insufficiency (eGFR \< 30 mL/min/1.73 m²) * Recent chaperone therapy or investigational treatment within the last 6 months * Pregnancy or lactation * History of cancer, drug or alcohol abuse, major organ transplant, or inability to adhere to study requirements

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Conditions

The condition(s) this trial relates to.

Ataxia Gaucher disease Gaucher disease type III lysosomal storage disease Myoclonus Neurologic Manifestations

As listed by the trial registrant

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

Gaucher disease, type 3

Contacts and locations

Locations

  • The Children's Hospital

    Lahore, Pakistan

More trials for these conditions

Other studies related to the condition(s) this trial covers.