Health Trials

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

ConditionAmyotrophic Lateral Sclerosis
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age18-75
SponsorTrace Neuroscience, Inc.

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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

Disqualifiers

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)

Trial design

Design model

Parallel

Treatments tested in this trial

  • TRCN-1023

    Drug

    TRCN-1023 administered as an intrathecal injection.

  • Placebo

    Other intervention

    Intrathecal injection of artificial cerebrospinal fluid (aCSF).

Treatment groups

30 Participants
are divided into 2 treatment groups
Group A: TRCN-1023Experimental treatment 1 intervention
Group B: PlaceboPlacebo comparator 1 intervention

Trial outcomes

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

Time frame
24 Weeks

Secondary outcomes

1

Plasma Concentration of TRCN-1023

Plasma TRCN-1023 concentrations

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

Cerebrospinal Fluid (CSF) Concentration of TRCN-1023

Cerebrospinal fluid (CSF) TRCN-1023 concentrations

Time frame
Day 1 (pre-dose and 24 hours post-dose), Weeks 4, 16, and 24 (up to 4 lumbar punctures post-dose)
3

Time to Peak Plasma Concentration (Tmax)

Time of maximum observed plasma concentration (Tmax) of TRCN-1023

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

Peak Plasma Concentration (Cmax) of TRCN-1023

Maximum observed 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

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Trace Neuroscience, Inc.

Lead sponsor

Source ClinicalTrials.gov
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