Cough medicine repurposed for rare brain disease in kids

NCT ID NCT07285369

First seen Jun 27, 2026 · Last updated Jun 27, 2026

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.

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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.

More trials for these conditions

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

Contacts and locations

Locations

  • The Children's Hospital

    Lahore, Pakistan