A Double-blind Randomised, Placebo-controlled Clinical Trial to Test Ambroxol Treatment in ALS

Summary

Ambroxol is a simple cough medicine that is predicted to slow ALS disease progression. This study aims to investigate if ambroxol in high doses is effective in treating ALS. This study will be carried out across 5 research sites in Australia (2 NSW, 1 VIC, 1 SA and 1 TAS), where newly diagnosed ALS patients will be asked to participate. Participation will be over a 32-week period, where they will come in for a 4-week screening, 24-week treatment, and 4-week end of study safety follow-up period. The participants will receive either the placebo or drug solution that they will take three times a day, up-dosing each week until they reach the maximum dose or highest dose they can tolerate. Throughout the study their disease progression will be assessed using tests, questionnaires, and blood biomarkers.

Conditions

• Amyotrophic Lateral Sclerosis

Interventions

DRUG

Ambroxol

Participants in the study will receive varying doses of ambroxol in solution, 3 times per day. Doses will be increased pending a safety review, up to a maximum of 1260mg/day. Blood tests will be conducted weekly to assess tolerance. Compliance will be monitored by returning used bottles. The study will last 32 weeks, including 24 weeks of drug administration and follow-up visits. After the final follow-up, there will be an end of study safety visit occurring 4 weeks later. The total time of participation will be 32 weeks. This includes a screening visit up to 4 weeks prior to Baseline, then a Baseline visit, followed by 24 weeks of follow-up (3x in clinic follow-up visits). These 24 weeks will be the drug administration period, meaning that the total duration of drug administration is 24 weeks. Following this drug administration and follow-up period, there will be an EoS safety-follow up visit that will occur 4 weeks after the final follow-up visit (28 weeks from baseline).

DRUG

Placebo

Participants randomised to the control arm will receive a placebo for the duration of the study. The placebo will look and taste like ambroxol, but will have no active ingredient. Participants will not be told which arm they have been randomised to. The placebo will primarily be a glucose solution, however it will also have flavouring (e.g. bitters) and colouring, so as to make it look and taste like ambroxol, to maintain blinding.

Sponsors & Collaborators

• Mobius Medical Pty Ltd.

  collaborator INDUSTRY

• The University of Queensland

  collaborator OTHER

• The Florey Institute of Neuroscience and Mental Health

  lead OTHER

Principal Investigators

• Bradley Turner · The Florey Institute of Neuroscience and Mental Health

• Steve Vucic · Concord Repatriation General Hospital

• Matthew Kiernan · Brain and Mind Centre (The University of Sydney)

• Susan Mathers · Calvary Health Care Bethlehem

• David Schultz · Flinders Medical Centre

• Lauren Giles · Launceston General Hospital

Study Design

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Model

PARALLEL

Eligibility

Min Age

18 Years

Max Age

85 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2023-06-13

Primary Completion

2024-06-30

Completion

2024-12-31

Countries

• Australia

Study Locations