Aims to restore UNC13A function in people with ALS

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a randomized, double-blind, placebo-controlled Phase 1/2 study evaluating a single intrathecal injection of TRCN-1023 in adults with ALS. The goal is to assess safety, tolerability, how the drug moves through the body, and early signals on disease progression, function, and quality of life over 24 weeks. TRCN-1023 is designed to restore UNC13A function in nerve cells affected by ALS and may require infrequent dosing. The trial includes dose-finding to identify the best dose for future studies and will monitor target engagement and clinical outcomes. Participants will undergo brain MRI, lumbar punctures, blood tests, and will wear sensors and complete home-based assessments.

Why it matters

Who can join: the full rules

Key Inclusion Criteria:

* Adults aged 18 to 75 years
* Diagnosis of ALS (clinically definite, clinically probable, or clinically probable laboratory supported)
* ALS symptoms began within the past 24 months
* Able to perform breathing tests: slow vital capacity (SVC) with consistent results, with breathing capacity of at least 60% of the expected value
* Able and willing to meet all study requirements, including travel to the study site, brain magnetic resonance imaging (MRI) scans, lumbar punctures, and blood draws
* Able and willing to use wearable sensors and complete speech assessments at home
* On a stable dose of approved ALS medication for at least 4 weeks prior to screening
* Capable of providing informed consent

Key Exclusion Criteria:

* Carries a confirmed SOD1 or FUS gene mutation
* Has a tracheostomy or requires continuous assisted ventilation more than 22 hours per day during the preceding 3 months before the first Screening Visit
* Has a contraindication to brain MRI (e.g., pacemaker, metal implants)
* Has a contraindication to lumbar puncture or spinal injection (e.g., blood clotting disorders, certain blood thinners, signs of increased pressure in the brain)
* Has significant abnormal liver, kidney, or blood test results
* Is currently enrolled in another clinical trial or has received an investigational treatment within the past 4 weeks
* Has previously received gene therapy, stem cell therapy, or another Antisense oligonucleotide (ASO) treatment
* Has a clinically significant condition other than ALS that could interfere with study participation

Where it's running

• Universitätsklinikum Schleswig-Holstein
  Lübeck, Schleswig-Holstein, Germany Recruiting
• TUM Klinikum Rechts der Isar
  Munich, Germany Recruiting
• University Medical Center Rostock
  Rostock, Germany Recruiting
• University Medical Center Utrecht
  Utrecht, Netherlands Recruiting
• University College London
  London, United Kingdom Recruiting
• University of Sheffield
  Sheffield, United Kingdom Recruiting

Study contacts

• Study coordinator: Trace Neuroscience Clinical Trials
• Email: Clinicaltrials@traceneuro.com
• Phone: +1 415-390-9509

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.