Share
Save
A Double-blind Randomised, Placebo-controlled Clinical Trial to Test Ambroxol Treatment in ALS
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.
Study details:
This study is a double-blind, randomised, placebo-controlled phase 2 clinical trial, to assess the safety, tolerability and efficacy of ambroxol therapy in ALS patients by using electrophysiological and functional measures to detect preservation of motor units. The study design will have participants be randomised to either ambroxol or placebo at a 2:1 ratio (ambroxol (n=34) and placebo (n=16)). 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.
Participants randomised to the control arm will receive a placebo for the duration of the study. Disease progression will be assessed by the following, time to event (death, need for tracheostomy, the need for gastrostomy feeding or non-invasive ventilation support (≥12 hours a day in a 24-hour period), or ≥6-point progression (ALS functional rating score-revised).
Eligibility criteria
Researchers look for people who fit a certain description, called eligibility criteria. See if you qualify.
Inclusion criteria
Exclusion criteria
Eligibility
Age eligible for study : 18 and older
Healthy volunteers accepted : No
Gender eligible for study: All
Things to know
Study dates
Study start: 2023-06-13
Primary completion: 2024-06-01
Study completion finish: 2024-12-01
Study type
TREATMENT
Phase
PHASE2
Trial ID
NCT05959850
Intervention or treatment
DRUG: Ambroxol
DRUG: Placebo
Conditions
- • Amyotrophic Lateral Sclerosis
Find a site
Closest Location:
Concord Repatriation General Hospital
Research sites nearby
Select from list below to view details:
Concord Repatriation General Hospital
Sydney, New South Wales, Australia
Flinders Medical Centre
Adelaide, South Australia, Australia
Launceston General Hospital
Launceston, Tasmania, Australia
Brain and Mind Centre
Sydney, New South Wales, Australia
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Participant Group/Arm | Intervention/Treatment |
---|---|
EXPERIMENTAL: Experimental: Active
| DRUG: Ambroxol
|
PLACEBO_COMPARATOR: Placebo Comparator: Control
| DRUG: Placebo
|
What is the study measuring?
Primary outcome
Primary Outcome Measure | Primary Outcome Description | Primary Outcome Time Frame |
---|---|---|
Time to event | Time to event (death, need for tracheostomy, the need for gastrostomy feeding or non-invasive ventilation (NIV) support (greater than or equal to 12 hours a day in a 24-hour period), or greater than or equal to 6-point progression on the Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS)) This will be measured by patient medical records, and the completion of the ALSFRS by investigators. | Time to event for a maximum of 24 weeks from baseline |
Secondary outcome
Secondary Outcome Measure | Secondary Outcome Description | Secondary Outcome Time Frame |
---|---|---|
ALS functional rating score-revised (ALSFRS-R) | Change in ALSFRS-R Score | 24 weeks from Baseline |
Motor unit number estimation (MUNIX) | Change in MUNIX values | 24 weeks from Baseline |
Split Hand Index (SI) | Change in SI value | 24 weeks from Baseline |
Neurophysiology Index (NPI) | Change in NPI Value | 24 weeks from Baseline |
Kings staging system | Change in Kings stage | 24 weeks from Baseline |
Muscle strength assessment as measured by the Medical Research Council (MRC) Scale for Muscle Strength | Change in Muscle strength, where Grade 0 is no visible contraction and Grade 5 is Normal | 24 weeks from Baseline |
Respiratory function (FVC) as measure by a Spirometer | Change in FVC | 24 weeks from Baseline |
Survival | Overall survival rate | 24 weeks from Baseline |
Serum NFL levels | Change in Serum NFL Levels | 24 weeks from Baseline |
Assessment of Quality of Life (AQoL) | Change in AQoL score | 24 weeks from Baseline |
Frequently Asked Questions
Please note: some questions and answers are submitted by anonymous patients or using AI, and have not been verified by Clinrol
No questions submitted. Be the first to ask a question!