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New gene therapy targets rare, aggressive ALS

NCT ID NCT06100276

Disease control Sponsor: UniQure Biopharma B.V. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This early-stage trial tests a gene therapy called AMT-162 in 20 adults with a specific genetic form of ALS (SOD1-ALS). The treatment is given as a single injection into the spinal fluid. The main goal is to check safety and tolerability, while also looking for early signs that it might slow the disease. Participants must have a confirmed SOD1 gene mutation and still have at least moderate physical function.

Why investors are watching

uniQure is testing AMT-162, a gene therapy given directly into the spinal fluid, in 20 adults with a rare, inherited form of ALS caused by SOD1 mutations. This early-stage trial measures safety and whether the therapy slows the disease. For a small company like uniQure, this readout matters because it could validate their gene therapy platform in a new disease area.

If it works: If the trial shows AMT-162 is safe and hints at slowing ALS progression, uniQure could advance the drug to later-stage testing. That would strengthen its pipeline and attract more attention from partners or investors.

If it fails: Gene therapies for the nervous system carry high risk, and early trials often fail to show clear benefit. A safety problem or lack of effect could set the program back and hurt uniQure's prospects, since the company's value depends heavily on its pipeline succeeding.

AI-written from the trial record. Speculative, and not investment advice.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

About 20 people

The number the study aims to enrol. It can still change while the study runs.

Started

Aug 2024

Expected to finish

Jun 2031

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Confirmed clinical and genetic diagnosis of SOD1-mediated ALS (SOD1-ALS) experiencing signs and/or symptoms of lower motor neuron dysfunction (weakness, atrophy, cramps, fasciculations), with or without upper motor neuron symptoms (weakness, bring reflexes, spasticity). * ALSFRS-R score ≥ 25 at Screening. * Slow vital capacity (SVC) ≥50% of predicted normal value. * Capable of providing informed consent and complying with trial procedures, including: medically able to undergo lumbar puncture and has a responsible caregiver able to attend all clinic visit with the Participant. Exclusion Criteria: * SOD1 pathogenic or likely pathogenic variants in amino acid regions 43-47. * Pathogenic repeat expansion in the C9orf72 gene * Any of the following prior or concomitant treatments: * Any prior SOD1 suppression therapy with viral microRNA mediators * Prior SOD suppression therapy with antisense oligonucleotide (ASO) mediators such as tofersen (QALSODY™). Exception: Patients who previously received tofersen may be enrolled if the last dose of tofersen was received at least 20 weeks prior to the first Screening assessment and if there were no previous tofersen-related SAEs or ongoing tofersen-related adverse events that would increase the risk of receiving AMT-162, per Investigator judgment. * Other ALS medications riluzole (RILUTEK®, TIGLUTIK®), edaravone (RADICAVA®), and sodium phenylbutyrate and taururosdiol combination (RELYVRIO) or bioequivalents are allowed if dose is stable for 30 days prior to immunosuppression. * Any prior administration of an AAV gene therapy. * Participants must be willing to forego new ALS treatments through at least 6 months after infusion of AMT-162. After 6 months, Investigators and participants may decide to add new ALS medications or change existing ALS medications.

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Conditions

The condition(s) this trial relates to.

amyotrophic lateral sclerosis amyotrophic lateral sclerosis type 1

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Amyotrophic lateral sclerosis

Contacts and locations

Locations

  • Barrow Neurological Institute

    Phoenix, Arizona, 85013, United States

  • California Pacific Medical Center

    San Francisco, California, 94109, United States

  • Columbia University Irving Medical Center

    New York, New York, 10032, United States

  • Massachusetts General Hospital, Sean M. Healey and AMG Center for ALS Research

    Boston, Massachusetts, 02114, United States

  • Mayo Clinic Florida

    Jacksonville, Florida, 32224, United States

  • Mayo Clinic Rochester

    Rochester, Minnesota, 55905, United States

  • Norrlands Universitetssjukhus

    Umeå, Vasterbottens Ian, Sweden

  • Northwestern University Feinberg School of Medicine

    Chicago, Illinois, 60611, United States

  • University of California Irvine

    Irvine, California, 92697, United States

  • University of Kansas Medical Center

    Fairway, Kansas, 66205, United States

  • University of Pennsylvania School of Medicine

    Philadelphia, Pennsylvania, 19104, United States

  • Winship Cancer Institute of Emory University

    Atlanta, Georgia, 30322, United States

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