In short
TRCN-1023 is an investigational substance being studied for amyotrophic lateral sclerosis (ALS). One clinical trial is currently authorised in the EU, a Phase 1/2 study testing single doses of TRCN-1023 given by intrathecal injection to 30 participants with ALS. The trial aims to assess safety and tolerability and to restore UNC13A function.
Key points
- TRCN-1023 is an investigational substance being studied in amyotrophic lateral sclerosis (ALS).
- The authorised trial is a Phase 1/2 study with a planned enrollment of 30 participants.
- TRCN-1023 is given by intrathecal injection, directly into the fluid around the spinal cord.
- The trial aims to restore UNC13A function, a protein linked to ALS.
- The primary outcome is the frequency of adverse events over several weeks.
- The trial is currently authorised but not yet recruiting.
What is TRCN-1023?
TRCN-1023 is an investigational substance being developed for the treatment of amyotrophic lateral sclerosis (ALS), a progressive neurodegenerative disease that affects nerve cells in the brain and spinal cord. The substance is also referred to as TRCN-1023 sodium salt in the trial record. TRCN-1023 is not an authorised medicine; it is still in clinical development and has not received marketing approval.
How does TRCN-1023 work?
The trial aims to restore the function of a protein called UNC13A, which is involved in the release of neurotransmitters at nerve cell junctions. In people with ALS, reduced UNC13A function is thought to contribute to the disease process. TRCN-1023 is designed to act on this target, though the exact mechanism is still being investigated.
Which conditions is it studied for?
- Amyotrophic lateral sclerosis (ALS) — a progressive condition in which the nerve cells that control voluntary muscle movement degenerate, leading to weakness and loss of function.
How is TRCN-1023 given?
TRCN-1023 is given by intrathecal injection, which means it is injected directly into the cerebrospinal fluid surrounding the spinal cord. In the authorised trial, single doses of TRCN-1023 are administered this way. The trial also includes a comparison arm using artificial cerebral spinal fluid.
What do the clinical trials show?
One clinical trial of TRCN-1023 is currently authorised in the EU. It is a randomised Phase 1/2 trial titled "A Randomized Phase 1 / 2 Trial Aiming to Restore UNC13A Function in People living with ALS: FUNCtion ALS". The study plans to enroll 30 participants with ALS.
The trial is designed to assess the safety and tolerability of single doses of TRCN-1023 given by intrathecal injection. The primary outcome measure is the frequency of adverse events over several weeks. The trial is currently authorised, meaning it has received regulatory approval to proceed but has not yet started recruiting participants.
The study is interventional and randomised, with participants receiving either TRCN-1023 or artificial cerebral spinal fluid as a comparison. This design helps researchers determine whether any observed effects are due to the substance itself rather than the injection procedure.
What side effects are known?
Because TRCN-1023 is still in early clinical development, the full side effect profile is not yet known. The primary goal of the current trial is to measure the frequency of adverse events, which will provide the first systematic data on side effects in people with ALS.