Testing ambroxol treatment for ALS

AMBroxol Therapy for ALS (AMBALS) Trial: a Double-blind, Randomised, Placebo-controlled Phase 2 Clinical Trial of Ambroxol for ALS

This study is testing if ambroxol, a common cough medicine, can help slow down the progression of ALS in newly diagnosed patients.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 50 (estimated)
• Ages: 18 Years to 85 Years
• Sex: All
• Sponsor: The Florey Institute of Neuroscience and Mental Health (Academic / other)
• Locations: 5 sites (Sydney, New South Wales and 4 other locations)
• Trial ID: NCT05959850 on ClinicalTrials.gov

What this trial studies

This clinical trial investigates the effectiveness of ambroxol, a common cough medicine, in slowing the progression of Amyotrophic Lateral Sclerosis (ALS). It is a double-blind, randomized, placebo-controlled phase 2 trial involving newly diagnosed ALS patients across five sites in Australia. Participants will be randomly assigned to receive either ambroxol or a placebo over a 24-week treatment period, with doses adjusted based on safety assessments. The study will monitor disease progression through various tests and questionnaires, aiming to determine the drug's safety and efficacy.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18 to 85 who have been diagnosed with ALS within the last 18 months and have a forced vital capacity of at least 60%.

Not a fit: Patients with advanced ALS symptoms or those who do not meet the eligibility criteria will not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly slow the progression of ALS, improving quality of life for patients.

How similar studies have performed: While ambroxol has been studied in other contexts, this specific application for ALS is novel and has not been extensively tested in previous studies.

Eligibility criteria

Inclusion Criteria:

1. Must have given written informed consent before any study related assessments are performed and must be able to understand purpose of the study, including any possible risks and adverse events.
2. ALS as diagnosed according to the recently proposed Gold Coast diagnostic criteria.
3. First symptom of ALS less than or equal to 18 months prior to screening. The qualifying first symptoms of ALS are limited to manifestations of weakness in extremity, bulbar, or respiratory muscles. Cramps, fasciculations, or fatigue should not be taken in isolation as a first symptom of ALS.
4. Forced vital capacity (FVC) greater than or equal to 60% of predicted value as adjusted for gender, height and age at the Screening Visit.
5. Male or female patients aged 18 years or greater (inclusive) and less than 85 years at the time of ALS diagnosis.
6. Able to swallow liquid.
7. Able to perform reproducible pulmonary function tests
8. Female patients must be post-menopausal or sterilized or must not be breastfeeding, have no intention to become pregnant during the study, and use acceptable methods of contraception or abstain from intercourse.
9. Male patients who have not had a vasectomy and confirmed zero sperm count must agree after receiving the first dose of study drug either to use acceptable methods of contraception or abstain from intercourse.
10. If on riluzole, stable dosing for 30-days prior to screening.
11. Pre-study ALSFRS-R progression between disease onset and screening of greater than or equal to 0.5 points/month (calculated by ALSFRS-R total score decline from 48 divided by the months since onset of ALS symptoms).

Exclusion Criteria:

1. Use of non-invasive ventilation (NIV) support for ALS only or gastrostomy tube at time of screening.
2. Exposure to investigational drug within 12-weeks prior to screening.
3. At screening of any medically significant cardiac, pulmonary, GI, musculoskeletal, or psychiatric illness that might interfere with the patient's ability to comply with study procedures or that might confound the interpretation of clinical safety or data.
4. Patient with a history of significant other major medical conditions based on the Investigator's judgment.
5. Based on the investigator's judgment, patients who may have difficulty complying with the protocol and/or any study procedures.
6. Any person who is an employee or an Investigator or Sponsor, or an immediate relative of an Investigator.

Where this trial is running

Sydney, New South Wales and 4 other locations

• Brain and Mind Centre — Sydney, New South Wales, Australia (Recruiting)
• Concord Repatriation General Hospital — Sydney, New South Wales, Australia (Recruiting)
• Flinders Medical Centre — Adelaide, South Australia, Australia (Recruiting)
• Launceston General Hospital — Launceston, Tasmania, Australia (Recruiting)
• Calvary Health Care Bethlehem — Melbourne, Victoria, Australia (Recruiting)

Study contacts

• Principal investigator: Steve Vucic — Concord Repatriation General Hospital
• Study coordinator: Bradley Turner

Conditions Amyotrophic Lateral Sclerosis