MND Australia

AMBALS (Ambroxol)  

What does Ambroxol do?

Ambroxol is a simple cough medicine that is predicted to slow ALS disease progression. It is being re-purposed as a possible MND treatment because it also restores lipid metabolism, which has ben founds to be dysfunctional in people with ALS. Lipids are fatty compounds that perform a variety of functions within the body, including controlling what goes in and out of your cells, moving and storing energy, absorbing vitamins and making hormones. 


AMBALS is a safety and efficacy study of a drug that has already been approved and re-purposed for use in MND. This Phase 2 study will investigate whether ambroxol in high doses is effective in treating ALS. This study is taking place across five research sites in Australia, where newly diagnosed ALS patients will be asked to participate. Participation will be over a 32-week period, which includes 4 weeks of screening, a 24 week treatment and a 4 week end safety follow up period. Participants in the study will be randomly assigned to receive either Ambroxol or a placebo. A placebo drug contains no active ingredients. The medication will be taken three times a day with the dose being increased each week to the highest dose tolerated by the patient. Throughout the study the participants' disease progression will be assessed using tests, questionnaires and blood biomarkers.

How can I take part in this trial? 

Trial sites and eligibility criteria are listed below. In order to take part in a clinical trial, you should first speak to your neurologist or MND Clinic. Ambroxol is in solution form and taken orally three times per day. 

Eligibility criteria

  • ALS as diagnosed according to the recently proposed Gold Coast diagnostic criteria.
  • First symptom of ALS less than or equal to 18 months prior to screening.
  • Forced vital capacity (FVC) greater than or equal to 60%
  • Exclusion criteria: Use of non-invasive ventilation (NIV) support for ALS only or gastrostomy tube
NSWConcord HospitalProf Steve Vucic
02 9767 8447
Julie Ryder
02 9767 8475

Brain and Mind Centre,
University of Sydney 
Eleanor Ramsey
02 9114 4258
VICCalvary Health Care Bethlehem Ltd, CaulfieldEmma Windebank
03 9834 9430
SAFlinders Medical Centre, Bedford ParkEdlira Dishnica
08 8204 5168
TASLaunceston General HospitalLauren Giles

Want to know more?

For more information visit the Australian New Zealand Clinical Trials Registry (ANZCTR)