Amyotrophic Lateral SclerosisPhase 1/2FUNCtion ALSNCT07674667
FUNCtion ALS: Aiming to Restore UNC13A Function in People Living With ALS
A Phase 1/2 study testing TRCN-1023 in people with Amyotrophic Lateral Sclerosis, run by Trace Neuroscience, Inc. at 6 sites in 3 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Amyotrophic Lateral Sclerosis
- What is being tested?
- TRCN-1023
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 30 participants
- Where
- 6 sitesin 3 countries
- Study dates
- 20 Aug 2026to Sep 2027
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
6 sites in 3 countries. Contact the study team or a site near you to ask about taking part.
6 sites in 3 countries
Germany3 sites, 3 recruiting
- Recruiting
Universitätsklinikum Schleswig-Holstein
Lübeck, Schleswig-Holstein, 23538
Julian Großkreutz, MD, + 49 451 500 43468, neuro.pnl@uni-luebeck.de
Julian Großkreutz, MD
- Recruiting
TUM Klinikum Rechts der Isar
Munich
Paul Lingor, MD, +49 89 4140 4606, paul.lingor@mri.tum.de
Paul Lingor, MD
- Recruiting
University Medical Center Rostock
Rostock, 18147
Sophie Fischer, +49 381 494 149542, Sophie.fischer@med.uni-rostock.de
United Kingdom2 sites, 2 recruiting
- Recruiting
University College London
London, WC1N 3BG
Central Study Contact Central Study Contact, +1 415-390-9509, clinicaltrials@traceneuro.com
- Recruiting
University of Sheffield
Sheffield, S10 2HQ
Prof Jonathan Cooper-Knock, j.cooper-knock@sheffield.ac.uk
Jonathan Cooper-Knock, MD
Netherlands1 site, 1 recruiting
- Recruiting
University Medical Center Utrecht
Utrecht, 3508 GA
Henk-Jan Westeneng, +31 (0)88 75 53204, trialals@umcutrecht.nl
Henk-Jan Westeneng, MD
About this study
Official title: A Randomized, Double-Blind, Placebo-Controlled, Phase 1 / 2 Trial Evaluating the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of TRCN-1023 Administered by Intrathecal Injections to Adult People Living With Amyotrophic Lateral Sclerosis
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.
Read the detailed description
TRCN-1023 is an investigational medicine designed to restore the function of a protein called UNC13A, which becomes disrupted in most people with Amyotrophic Lateral Sclerosis (ALS) due to a breakdown in how nerve cells process genetic information. This trial is designed to evaluate the safety and tolerability of TRCN-1023 given as a single injection into the fluid surrounding the spine, and to identify the best dose to carry forward into future studies. TRCN-1023 stays in the brain and spinal cord long enough that it may only need to be administered once every 24 weeks. The trial will also assess how the body processes the drug, whether it engages its intended target, and its effects on disease progression, physical function, and quality of life.
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- TRCN-1023, Placebo
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TRCN-1023Drug
TRCN-1023 administered as an intrathecal injection.
PlaceboOther
Intrathecal injection of artificial cerebrospinal fluid (aCSF).
| Study group | Role | What they receive |
|---|---|---|
| TRCN-1023 | Experimental | Antisense oligonucleotide (ASO) administered by intrathecal injection Receives: Drug: TRCN-1023 |
| Placebo | Placebo comparator | Matching Placebo Comparator Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Frequency of Adverse Events
Number and frequency of adverse events assessed over 24 weeks following a single intrathecal injection of TRCN-1023 or placebo
Measured: 24 Weeks
Secondary outcomes (5)
Plasma Concentration of TRCN-1023
Plasma TRCN-1023 concentrations
Measured: 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
Cerebrospinal fluid (CSF) TRCN-1023 concentrations
Measured: 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)
Time of maximum observed plasma concentration (Tmax) of TRCN-1023
Measured: 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
Maximum observed plasma concentration (Cmax) of TRCN-1023
Measured: 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
Area under the plasma concentration-time curve from time zero to last measurable concentration (AUClast) of TRCN-1023
Measured: Day 1 (pre-dose, 1, 2, 6, and 24 hours post-dose), Weeks 4, 10, 16, and 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Jun 2026
- Study start20 Aug 2026
- Primary completionSep 2027 (estimated)
- Study completionSep 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyotrophic Lateral SclerosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- 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.
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Trace Neuroscience, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07674667
- Sponsor's protocol ID
- TRCN-1023-ALS-101
- Other IDs
- 2025-523111-11, 174619, 2025
- Acronym
- FUNCtion ALS
- Status last verified
- Oct 2026
- First submitted
- 21 Jun 2026
- Last updated
- 14 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 14 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-Blind, Placebo-Controlled, Phase 1 / 2 Trial Evaluating the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of TRCN-1023 Administered by Intrathecal Injections to Adult People Living With Amyotrophic Lateral Sclerosis
- ClinicalTrials.gov ID
- NCT07674667
- Acronym / sponsor ID
- FUNCtion ALS / TRCN-1023-ALS-101
- Conditions
- Amyotrophic Lateral Sclerosis
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Trace Neuroscience, Inc.Industry (pharma, biotech, devices)
- Study start
- 20 Aug 2026 (actual)
- Primary completion
- Sep 2027 (estimated)
- Study completion
- Sep 2027 (estimated)
- First posted
- 29 Jun 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
TRCN-1023 is an investigational medicine designed to restore the function of a protein called UNC13A, which becomes disrupted in most people with Amyotrophic Lateral Sclerosis (ALS) due to a breakdown in how nerve cells process genetic information. This trial is designed to evaluate the safety and tolerability of TRCN-1023 given as a single injection into the fluid surrounding the spine, and to identify the best dose to carry forward into future studies. TRCN-1023 stays in the brain and spinal cord long enough that it may only need to be administered once every 24 weeks. The trial will also assess how the body processes the drug, whether it engages its intended target, and its effects on disease progression, physical function, and quality of life.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- 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.
| Arm | Type | Receives |
|---|---|---|
| TRCN-1023 | Experimental | Drug: TRCN-1023Antisense oligonucleotide (ASO) administered by intrathecal injection |
| Placebo | Placebo comparator | Other: PlaceboMatching Placebo Comparator |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- 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
Key exclusion criteria (8 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| TRCN-1023TRCN-1023 administered as an intrathecal injection. | Drug | TRCN-1023 |
| PlaceboIntrathecal injection of artificial cerebrospinal fluid (aCSF). | Other | Placebo |
Primary outcome
Frequency of Adverse Events
Time frame: 24 Weeks
Number and frequency of adverse events assessed over 24 weeks following a single intrathecal injection of TRCN-1023 or placebo
5 secondary outcomes
Plasma Concentration of TRCN-1023
Time frame: 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
Time frame: 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)
Time frame: 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
Time frame: 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
Time frame: Day 1 (pre-dose, 1, 2, 6, and 24 hours post-dose), Weeks 4, 10, 16, and 24
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Oct 2026.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
6 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing TRCN-1023 in people with Amyotrophic Lateral Sclerosis, run by Trace Neuroscience, Inc. at 6 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 14 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in Germany, United Kingdom, Netherlands. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Trace Neuroscience Clinical Trials (+1 415-390-9509, Clinicaltrials@traceneuro.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Frequency of Adverse Events (time frame: 24 Weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Oct 2026.