What does the drug do?

Ambroxol is a drug that is used in some cough medicines to treat respiratory diseases and break down excess mucus being produced. It is now being tested as a potential treatment for MND to see whether it could help to slow the progression of the disease. Previous research in different animal models of MND has shown that Ambroxol can reprogramme faulty lipid metabolism in diseased neurons which could help to strengthen the connections between the motor neurons and muscles. 

Phase 2 - AMBALS

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 in Australia is recruiting 50 people with MND at 5 sites across the country 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: 18/04/2024