FUNCtion ALS: Aiming to Restore UNC13A Function in People Living With ALS (FUNCtion ALS)
8. september 2026 opdateret af: Trace Neuroscience, Inc.
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.
Studieoversigt
Status
Rekruttering
Betingelser
Intervention / Behandling
Detaljeret beskrivelse
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.
Undersøgelsestype
Interventionel
Tilmelding (Anslået)
30
Fase
- Fase 2
- Fase 1
Kontakter og lokationer
Dette afsnit indeholder kontaktoplysninger for dem, der udfører undersøgelsen, og oplysninger om, hvor denne undersøgelse udføres.
Studiekontakt
- Navn: Trace Neuroscience Clinical Trials
- Telefonnummer: +1 415-390-9509
- E-mail: Clinicaltrials@traceneuro.com
Studiesteder
-
-
-
Sheffield, Det Forenede Kongerige, S10 2HQ
- Rekruttering
- University of Sheffield
-
Kontakt:
- Prof Jonathan Cooper-Knock
- E-mail: j.cooper-knock@sheffield.ac.uk
-
Ledende efterforsker:
- Prof Jonathan Cooper-Knock
-
-
-
-
-
Utrecht, Holland, 3508 GA
- Rekruttering
- University Medical Center Utrecht
-
Kontakt:
- Henk-Jan Westeneng
- Telefonnummer: +31 (0)88 75 53204
- E-mail: trialals@umcutrecht.nl
-
Ledende efterforsker:
- Henk-Jan Westeneng
-
-
-
-
-
Munich, Tyskland
- Rekruttering
- TUM Klinikum rechts der Isar
-
Kontakt:
- Paul Lingor, MD
- Telefonnummer: +49 89 4140 4606
- E-mail: paul.lingor@mri.tum.de
-
Ledende efterforsker:
- Paul Lingor, MD
-
Rostock, Tyskland, 18147
- Rekruttering
- University Medical Center Rostock
-
Kontakt:
- Sophie Fischer
- Telefonnummer: +49 381 494 149542
- E-mail: Sophie.fischer@med.uni-rostock.de
-
-
Deltagelseskriterier
Forskere leder efter personer, der passer til en bestemt beskrivelse, kaldet berettigelseskriterier. Nogle eksempler på disse kriterier er en persons generelle helbredstilstand eller tidligere behandlinger.
Berettigelseskriterier
Aldre berettiget til at studere
- Voksen
- Ældre voksen
Tager imod sunde frivillige
Ingen
Beskrivelse
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
Studieplan
Dette afsnit indeholder detaljer om studieplanen, herunder hvordan undersøgelsen er designet, og hvad undersøgelsen måler.
Hvordan er undersøgelsen tilrettelagt?
Design detaljer
- Primært formål: Behandling
- Tildeling: Randomiseret
- Interventionel model: Parallel tildeling
- Maskning: Firedobbelt
Våben og indgreb
Deltagergruppe / Arm |
Intervention / Behandling |
|---|---|
|
Eksperimentel: TRCN-1023
Antisense oligonucleotide (ASO) administered by intrathecal injection
|
TRCN-1023 administered as an intrathecal injection.
|
|
Placebo komparator: Placebo
Matching Placebo Comparator
|
Intrathecal injection of artificial cerebrospinal fluid (aCSF).
|
Hvad måler undersøgelsen?
Primære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
|---|---|---|
|
Frequency of Adverse Events
Tidsramme: 24 Weeks
|
Number and frequency of adverse events assessed over 24 weeks following a single intrathecal injection of TRCN-1023 or placebo
|
24 Weeks
|
Sekundære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
|---|---|---|
|
Plasma Concentration of TRCN-1023
Tidsramme: Day 1 (pre-dose, 1, 2, 6, and 24 hours post-dose), Weeks 4, 10, 16, and 24
|
Plasma TRCN-1023 concentrations
|
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
Tidsramme: Day 1 (pre-dose and 24 hours post-dose), Weeks 4, 16, and 24 (up to 4 lumbar punctures post-dose)
|
Cerebrospinal fluid (CSF) TRCN-1023 concentrations
|
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)
Tidsramme: Day 1 (pre-dose, 1, 2, 6, and 24 hours post-dose), Weeks 4, 10, 16, and 24
|
Time of maximum observed plasma concentration (Tmax) of TRCN-1023
|
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
Tidsramme: Day 1 (pre-dose, 1, 2, 6, and 24 hours post-dose), Weeks 4, 10, 16, and 24
|
Maximum observed 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
Tidsramme: 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 from time zero 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
|
Samarbejdspartnere og efterforskere
Det er her, du vil finde personer og organisationer, der er involveret i denne undersøgelse.
Sponsor
Datoer for undersøgelser
Disse datoer sporer fremskridtene for indsendelser af undersøgelsesrekord og resumeresultater til ClinicalTrials.gov. Studieregistreringer og rapporterede resultater gennemgås af National Library of Medicine (NLM) for at sikre, at de opfylder specifikke kvalitetskontrolstandarder, før de offentliggøres på den offentlige hjemmeside.
Studer store datoer
Studiestart (Faktiske)
20. august 2026
Primær færdiggørelse (Anslået)
1. september 2027
Studieafslutning (Anslået)
1. september 2027
Datoer for studieregistrering
Først indsendt
21. juni 2026
Først indsendt, der opfyldte QC-kriterier
24. juni 2026
Først opslået (Faktiske)
29. juni 2026
Opdateringer af undersøgelsesjournaler
Sidste opdatering sendt (Faktiske)
9. september 2026
Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier
8. september 2026
Sidst verificeret
1. juni 2026
Mere information
Begreber relateret til denne undersøgelse
Yderligere relevante MeSH-vilkår
Andre undersøgelses-id-numre
- TRCN-1023-ALS-101
- 2025 (U.S. NIH-bevilling/kontrakt: Faculty of Social Sciences Scientific Grant at the University of Gdańsk)
- 2025-523111-11 (EudraCT nummer)
- 174619 (Anden identifikator: Food And Drug Administration (FDA))
Plan for individuelle deltagerdata (IPD)
Planlægger du at dele individuelle deltagerdata (IPD)?
UBESLUTET
Lægemiddel- og udstyrsoplysninger, undersøgelsesdokumenter
Studerer et amerikansk FDA-reguleret lægemiddelprodukt
Ja
Studerer et amerikansk FDA-reguleret enhedsprodukt
Ingen
Disse oplysninger blev hentet direkte fra webstedet clinicaltrials.gov uden ændringer. Hvis du har nogen anmodninger om at ændre, fjerne eller opdatere dine undersøgelsesoplysninger, bedes du kontakte register@clinicaltrials.gov. Så snart en ændring er implementeret på clinicaltrials.gov, vil denne også blive opdateret automatisk på vores hjemmeside .