FUNCtion ALS trial
Sponsored by Trace Neuroscience
The FUNCtion ALS trial investigates the safety and tolerability of the drug TRCN-1023 in different doses.
The FUNCtion ALS trial investigates whether the investigational drug TRCN-1023 is safe and how well it is tolerated by people with ALS. The drug is given by an injection into the lower back.
The investigational compound TRCN-1023
Most people with ALS (97%) have changes in a protein called TDP-43. This pathology results in the faulty processing of hundreds of genes, including a well-known ALS gene: UNC13A. TRCN-1023, the study drug, has been designed to correct errors in how the gene UNC13A is processed in nerve cells, so that the correct protein can be produced and function properly. TRCN-1023 will be administered by an injection into the lower back. Due to the long durability of effect seen in animals, TRCN-1023 or placebo (fake compound) will be administered only once proceeded by an approximate 24-week Follow-up Period in this trial.
Trial design
This trial is a Phase 1/2 trial. The main goal of a Phase 1/2 trial is to see whether a new drug is safe and how well it is tolerated. The trial is designed as a randomized, double-blind, placebo-controlled trial. A placebo is a substance that looks like TRCN-1023, but has no active ingredients. Researchers use a placebo to see if a research medicine works better or is safer than not taking anything at all. Double-blind means you will be randomized (by chance, like flipping a coin) to either the trial drug TRCN-1023 or placebo, where neither you nor your doctor know that you receive medication or placebo.
The chances of receiving TRCN-1023 are dependent on the order of participants. As the trial investigates the drug in different doses, each dose will start with 2 participants having a 1 to 1 chance of receiving the drug or the placebo. Afterwards, the participants in the same dose-group, have a 75-80% chance of getting TRCN-1023.
The trial aims to enroll approximately 30 participants with ALS.
The trial consists of different phases: the Screening Period followed by a Run-in Period, during which participants perform at-home digital health assessments, then the Treatment and a Follow-up Period. During the trial, the participants will receive TRCN-1023 or placebo at the beginning of the Treatment Period and will then need to visit the hospital 3 specific times during the Follow-up Period, to assess safety and tolerability. Assessments during the Follow-up Period may include blood and CSF collections (using lumbar puncture for the latter), brain MRI and nerve stimulation tests.
After the main trial, the participants may have the possibility to extend the use of TRCN-1023 (no placebo anymore). This extension is dependent on regulatory approval.
By participating, you make an important contribution to scientific research on new medicines for ALS. Your participation will help us gather information on the safety of the experimental compound TRCN-1023. If you take part in the study, it is not certain that your ALS symptoms will improve. These symptoms may remain the same or may worsen during the study. You should also be aware that you may not receive any benefit if you are assigned to the placebo group.
Can I participate in the trial?
The main criteria to participate in this trial are:
- Age between 18 and 75 years
- Onset of first ALS related symptoms less than 24 months ago
- Lung function ≥ 60% of normal
- Willing and able to undergo lumbar punctures and MRIs
- Willing to have at least one overnight stay after the treatment
- No confirmed SOD1 or FUS gene mutations
Note: This list is not exhaustive, but these are the main criteria.
Registration and more information
To learn more about participating in the FUNCtion ALS trial, please contact one of the participating TRICALS centres below (contact details can be found by clicking on ‘More about this centre’). Make use of the contact form on each Centre detail page, or when contacting the centre of your choice by email or telephone, please provide your full name, your date of birth and the date of onset of symptoms. The centre can provide you with more information about the trial and/or inclusion criteria and answer any questions you may have. If you experience difficulty contacting a centre, you may reach out directly to the TRICALS operational team at info@tricals.org for support in this process.
More information about the trial can be found at clinicaltrials.gov
