FUNCtion ALS: Aiming to Restore UNC13A Function in People Living With ALS

NCT07674667 | Not Yet Recruiting Phase 1 FDA Drug

• 3 other identifiers: TRCN-1023-ALS-1012025-523111-11174619
• Study Type: interventional
• Target: 30
• Locations: 2 countries
• Sites: 2

Brief Summary

The FUNCtion Amyotrophic Lateral Sclerosis (ALS) trial is a randomized, double-blind, placebo-controlled Phase 1/2 trial to evaluate the safety and tolerability of TRCN-1023 in adults living with ALS. TRCN-1023 is an investigational medicine given as a single injection into the fluid surrounding the spine (intrathecal injection). The trial will also assess how the body processes the drug and whether it shows early signs of benefit over 24 weeks.

Conditions

Amyotrophic Lateral Sclerosis

Outcome Measures

Primary Outcomes (1)

• Frequency of Adverse Events

  Number and frequency of adverse events assessed over 24 weeks following a single intrathecal injection of TRCN-1023 or placebo

  24 Weeks

Secondary Outcomes (5)

• Plasma Concentration of TRCN-1023

  Day 1 (pre-dose, 1, 2, 6, and 24 hours post-dose), Weeks 4, 10, 16, and 24

• Cerebrospinal Fluid (CSF) Concentration of TRCN-1023

  Day 1 (pre-dose and 24 hours post-dose), Weeks 4, 16, and 24 (up to 4 lumbar punctures post-dose)

• Time to Peak Plasma Concentration (Tmax)

  Day 1 (pre-dose, 1, 2, 6, and 24 hours post-dose), Weeks 4, 10, 16, and 24

• Peak Plasma Concentration (Cmax) of TRCN-1023

  Day 1 (pre-dose, 1, 2, 6, and 24 hours post-dose), Weeks 4, 10, 16, and 24

• Area Under the Plasma Concentration-Time Curve to Last Measurable Concentration (AUClast) of TRCN-1023

  Day 1 (pre-dose, 1, 2, 6, and 24 hours post-dose), Weeks 4, 10, 16, and 24

Study Arms (2)

TRCN-1023

EXPERIMENTAL

Antisense oligonucleotide (ASO) administered by intrathecal injection

Drug: TRCN-1023

Placebo

PLACEBO COMPARATOR

Matching Placebo Comparator

Other: Placebo

Interventions

TRCN-1023 DRUG

TRCN-1023 administered as an intrathecal injection.

TRCN-1023

Placebo OTHER

Intrathecal injection of artificial cerebrospinal fluid (aCSF).

Placebo

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Trace Neuroscience, Inc.: lead

Study Sites (2)

TUM Klinikum Rechts der Isar

Munich, Germany

Location

University Medical Center Utrecht

Utrecht, 3508 GA, Netherlands

Location

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Approximately 30 adults with Amyotrophic Lateral Sclerosis (ALS) will be randomized to receive a single intrathecal injection of either TRCN-1023 or placebo and followed for 24 weeks.

Study Record Dates

• First Submitted: June 21, 2026
• First Posted: June 29, 2026
• Study Start: July 1, 2026
• Primary Completion (Estimated): September 1, 2027
• Study Completion (Estimated): September 1, 2027
• Last Updated: July 20, 2026

Record last verified: 2026-06

Locations

NL (1); DE (1)