RecruitingPhase 2ACTRN12622001380785

A double-blind randomised, placebo-controlled clinical trial to test the treatment of amyotrophic lateral sclerosis with ambroxol.

Ambroxol therapy for ALS trial: a double-blind, randomised, placebo-controlled Phase 2 clinical trial of ambroxol for ALS

Sponsor

The Florey Institute of Neuroscience and Mental Health (part of the University of Melbourne)

Enrollment

50 participants

Start Date

Jun 14, 2023

Study Type

Interventional

Conditions

Amyotrophic Lateral Sclerosis

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 till 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.

Eligibility

Sex: Both males and femalesMin Age: 18 YearssMax Age: 85 Yearss

Plain Language Summary

Simplified for easier understanding

Amyotrophic lateral sclerosis, or ALS (also known as motor neurone disease), is a progressive neurological condition that gradually weakens the muscles used for movement, speech, and breathing. There are very few effective treatments available. This trial is testing whether ambroxol — a common cough medicine widely available overseas — might slow the progression of ALS when given at higher-than-usual doses. Ambroxol has been identified in research as a drug that may have protective effects on motor neurons, the nerve cells that ALS destroys. The study is randomised and placebo-controlled, meaning some participants will receive ambroxol and others will receive a harmless dummy medicine, and neither participants nor their medical team will know which they are getting. Participants will gradually increase their dose over time until they reach the maximum tolerated level, then continue for 24 weeks. Disease progression will be tracked using standardised tests, questionnaires, and blood markers. You may be eligible if you are aged 18 to 85, have been diagnosed with ALS within the last two years using Gold Coast diagnostic criteria, can swallow liquids, and have lung function above 60% of the predicted normal value. You would not be eligible if you are on a ventilator or feeding tube, have a serious heart, lung, or psychiatric condition, or have taken any other investigational drug in the past three months.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

Participants randomised to the active arm will receive various doses of ambroxol in solution, taken orally, three times a day. Doses will be increased pending a safety review for each participant. Th

Participants randomised to the active arm will receive various doses of ambroxol in solution, taken orally, three times a day. Doses will be increased pending a safety review for each participant. The doses will be 180mg per day, 260mg per day, 540mg per day, 900mg per day, and 1260 mg per day. Each week safety bloods will be performed to assess tolerance to the dose. If the bloods indicate no safety issues, the dose will be increased. If there are any safety issues, dosing will remain the same for another week, before another safety blood review. These safety bloods will continue weekly until the participant is at their highest tolerated does (up to 1260mg per day max). Once at their tolerated dose, participants will remain on this daily dose for a period of up to 19 weeks (possibly less depending on up individual dosing schedule). To monitor drug compliance, participants will return used IP bottles to clinic for reconciliation. 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 a End of Study safety-follow up visit that will occur 4 weeks after the final follow-up visit (28 weeks from baseline).