Trial results
AI generated results summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI
According to the results reported on ClinicalTrials.gov, this trial enrolled 16 people in total, split into two groups of 8 — one group received a 15mg dose and the other received a 30mg dose of an investigational treatment called 3K3A-APC, which was being studied in the context of ALS (also known as motor neurone disease). All 16 participants completed the study. The trial was primarily measuring two things: whether participants experienced any serious unwanted health events (called adverse events) during the study, and whether there were any changes in a brain scan measure called the PERSI score, which tracks a type of brain cell activity (microglial activation) in the motor cortex — the part of the brain involved in movement. The reported data shows that zero participants in either the 15mg or the 30mg group experienced any serious adverse events, or any adverse events rated as more severe than "moderate." Regarding the brain scan measure, the reported data shows the PERSI score changed by −0.14% in the 15mg group and +0.53% in the 30mg group, meaning a very small decrease was recorded in one group and a very small increase in the other. It is important to note that these numbers alone do not tell us what a meaningful change would look like. For the secondary outcome measures — which included MRI brain scans, blood cell activity, and levels of certain proteins in blood and spinal fluid — the reported data shows that no numerical results were submitted to ClinicalTrials.gov for any of these measures. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.
View full results on ClinicalTrials.gov ↗
These are the results as reported to ClinicalTrials.gov, not medical advice. Verify independently with the trial site and discuss what they mean for you with your doctor.
Phase 2 Motor Neurone Disease Trial, Completed
Who may be able to join
AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI
Who might be able to join this trial:
- You have been officially diagnosed with ALS (a specific type confirmed by medical criteria called the Awaji Criteria)
- You are between 18 and 74 years old at the time of joining the study
- Your ALS symptoms first started less than 36 months (3 years) before the screening visit
- You were diagnosed with ALS less than 24 months (2 years) before the screening visit
- Your doctors have confirmed signs that the upper part of your nervous system (brain and upper spinal cord) is affected by ALS
Who may not be able to join:
- You are currently taking blood-thinning medications (such as warfarin, heparin, or similar drugs) that could make a spinal fluid procedure unsafe
- You have a back condition, bleeding disorder, or low platelet count that would make a spinal fluid procedure unsafe
- You have taken part in another drug or device trial within the last 60 days (everyday supplements like coenzyme Q10 are generally fine)
- Your blood clotting test results are more than twice the normal upper limit
- You have severely high or severely low blood pressure
- Your kidneys are not functioning well enough (confirm with trial site)
- Your lung capacity is below 50% of what is considered normal for someone your age and size
- You have previously been given any form of a substance called APC (confirm with trial site)
- You are unable to lie flat for medical procedures such as MRI, PET scan, or spinal fluid collection
- You are currently pregnant or breastfeeding
Important: Always verify eligibility with the trial site directly before applying.
Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.
Contact this trial
Principal Investigator: Dominic Rowe, PhD, MD, Macquarie University, Australia
Australian sites
Contact details sourced from ClinicalTrials.gov. Verify directly with the trial site before attending.
GP referral letter
Print a one-page summary to share with your doctor.
Trial details
Where this trial is recruiting
1 site(s) in Australia. Confirm current status and contact details directly with the trial site.
Primary endpoints
Number of Participants Who Had Any Serious Adverse Events or Any Adverse Events With Severity Higher Than "Moderate".; Percentage of Change in PERSI Score in the Motor Cortex Before and After Dosing
Can't join this trial?
Data last synced from ClinicalTrials.gov: 28 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.