A Randomized Phase 1/2 Trial Aiming to Restore UNC13A Function in people with ALS: FUNCtion ALS
Research type
Research Study
Full title
A Randomized, Double-Blind, Placebo-Controlled, Phase 1 / 2 Trial Evaluating the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of Single Ascending Doses of TRCN-1023 Administered by Intrathecal Injections to Adult People Living with Amyotrophic Lateral Sclerosis
IRAS ID
1012578
Contact name
Irina Antonijevic
Contact email
Sponsor organisation
Trace Neuroscience, Inc.
Eudract number
2025-523111-11
Clinicaltrials.gov Identifier
Research summary
Rationale
Amyotrophic Lateral Sclerosis (ALS) is a rare, progressive condition that makes a person’s muscles weaker over time. People living with ALS (PlwALS) will continue to decline and may experience respiratory failure and death. Current treatments for pALS are riluzole and edaravone for a broader population, and tofersen for a small sub-population that have a known gene mutation called superoxide dismutase 1 (SOD1). The current treatments are not available in all countries and some treatments may not benefit those suffering from ALS, leaving an unmet need. This trial will test TRCN-1023, an unapproved treatment, to evaluate its safety and potential for treating adult PlwALS.Objective
This trial is designed to assess how safe TRCN-1023 is for PlwALS, whether it causes side effects, how it works in pALS, and how the body responds to the treatments. Also, the clinical trial is designed to assess whether TRCN-1023 can help PlwALS with their condition.Main trial measures
To assess safety and any side effects, the clinical trial will monitor and evaluate the number and severity of adverse events (unintended, harmful, and/or undesirable outcomes) over a 24-week period.Secondary trial measures
Blood and cerebral spinal fluid collected from participants in the clinical trial will be evaluated to determine how long it stays in the body after it is injected. The cerebral spinal fluid will also be evaluated to determine how TRCN-1023 works with the human body.Trial design
This is a randomized, double-blinded, placebo-controlled trial. A placebo-controlled trial means TRCN-1023 will be compared against a placebo (nonactive substance). Participants will be randomly selected, by a computer, to be treated with either TRCN-1023 or placebo. A double-blind trial means that neither the participants nor the trial staff know who is receiving TRCN-1023 or placebo.REC name
London - City & East Research Ethics Committee
REC reference
26/LO/0267
Date of REC Opinion
30 Apr 2026
REC opinion
Further Information Favourable Opinion