RecruitingPhase 2ACTRN12624001204538

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

Ambroxol therapy for ALS trial: An Open Label Extension of the 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

20 participants

Start Date

Sep 16, 2024

Study Type

Interventional

Conditions

Amyotrophic Lateral Sclerosis

Summary

Ambroxol is a simple cough medicine that is predicted to slow ALS disease progression. This study is the open label extension (OLE) of the parent study, which aims to investigate if ambroxol in high doses is effective in treating ALS. This OLE study will be carried out across 5 research sites in Australia (2 NSW, 1 VIC, 1 SA and 1 TAS), where ALS patients whole have successfully completed the randomised phase of the parent study, will be asked to participate in the OLE phase. Participation will be over a 52-week period, where they will come in for a an OLE baseline, followed by 48-week treatment, and 4-week end of study safety follow-up period. All participants will active drug that they will take three times a day, 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 (ALS), also known as motor neurone disease, is a devastating progressive illness that affects the nerve cells controlling movement, speech, swallowing, and breathing. There are currently very few effective treatments. This study is an open-label extension of a clinical trial testing whether high-dose ambroxol — a simple, inexpensive cough medicine — can slow the progression of ALS. Participants who completed the original randomised double-blind trial will be invited to continue on active ambroxol treatment for an additional 52 weeks. Unlike the blinded phase, everyone in this extension will receive the real medication. Disease progression will be tracked using standard ALS rating scales, physical tests, questionnaires, and blood biomarkers. You may be eligible if you successfully completed the main AMBALS trial without significant compliance issues and are able to give written consent. People with major compliance problems during the original trial, or who are pregnant or planning to become pregnant, are not eligible. This extension study gives participants who may have previously been on placebo their first opportunity to receive the active treatment, while continuing to generate valuable safety data.

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 who have successfully completed the randomised phase of the parent AMBALS study, will be invited to participate in the Open Label Extension (OLE). The OLE will involve participants taking 420mg capsules of ambroxol, orally, three times a day (total dose 1260mg per day). Participants will complete an OLE baseline visit, for consenting and OLE eligibility assessments. They will then start their OLE dosing. This will then be followed 48 weeks of follow-up (4x in clinic follow-up visits - one every 12 weeks). These 48 weeks will be the drug administration period, meaning that the total duration of drug administration is 48 weeks. Following this drug administration and follow-up period, there will be an End of Study safety-follow up visit that will occur 4 weeks after the final follow-up visit (52 weeks from OLE baseline). Participants will return IP bottles at each of the 4 in-clinic follow-up visits, to monitor IP compliance.