What is the drug thought to do in the body?

Ambroxol is a drug that is used in some cough medicines to treat respiratory diseases and break down the production of excess mucus. It is now being tested as a potential treatment for MND, as it also restores lipid metabolism, which has been found to be faulty in people with MND. Lipids are fatty compounds which play an important role in the body, such as storing energy and controlling what goes in and out of cells. Previous research has shown that ambroxol helps to improve motor neurone function, muscle strength and protect motor neurones.

Phase 2 - AMBALS

Current status: recruiting

Primary objective: To investigate if Ambroxol effects how long it takes for a person to experience an event (such as death, need for tracheostomy, gastrostomy feeding tube or non-invasive ventilation)

As Ambroxol is already known to be safe and well tolerated, a phase 1 study into the use for MND was not needed. The Phase 2 trial, called AMBALS, is being conducted to look at the long-term safety and effectiveness of the drug for people with MND. This trial recruiting 50 people with MND at 5 sites across Australia, who will be randomised to receive either the drug or a placebo for 24 weeks. Participants will then be offered the opportunity to continue into an open label phase where everyone will receive the drug for a further 6 months. You can find out more about the trial here.

This trial is recruiting in Australia only. 

Last updated: 21/08/2024