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New ALS drug QRL-201 enters human safety testing

NCT ID NCT05633459

Disease control Sponsor: QurAlis Corporation 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 study tests whether a new drug called QRL-201 is safe and tolerable for people with ALS (Lou Gehrig's disease). About 69 adults aged 18-80 with recent ALS symptoms will receive either the drug or a placebo. The main goal is to check for side effects, not to cure the disease.

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

The first testing in people. Mainly checks safety and dose, usually in a small group.

Participants

69 people

The number who actually took part.

Started

Dec 2022

Expected to finish

Nov 2027

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 to 80 years

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 (Double-Blind Periods): * Male or female participants aged 18 to 80 years diagnosed with ALS * ALS symptom onset within 24 months of Screening * Slow vital capacity \>50% * Clinical or electrodiagnostic evidence of lower motor neuron involvement * Not pregnant and not nursing * Willing and able to practice effective contraception * Able to tolerate lumbar puncture * If on approved therapies for the treatment of ALS during the course of the study, must be on a stable dose (at the Sponsor's discretion) Exclusion Criteria (Double Blind Periods): * Pathogenic variant, likely pathogenic variant, or variant of uncertain significance in the superoxide dismutase 1 (SOD1) and/or fused in sarcoma (FUS) genes * Currently enrolled in any other clinical study involving either an investigational product (IP) or off-label use of a drug or device * Prior exposure to stem cell or gene therapy products * Any contraindication to intrathecal drug administration * Abnormal laboratory values deemed clinically significant by the Investigator * Significant infection or known inflammatory process * Any sign and/or history of neurological conditions and other neuromuscular disorders that could affect the electrophysiological recordings. * An EEG that shows signs of abnormal electrical activity (e.g., epilepsy) Inclusion Criteria (Open-Label Extended Dosing Period): * Male or female participants in the sporadic ALS and C9orf72 ALS Cohorts of QRL-201-01 who have completed through at least Day 253 of safety follow up in the double-blind study period OR have completed Cohort 1 of Cohort 2 * Not pregnant and not nursing * Willing and able to practice effective contraception * Can visit study site for required visits * Able to tolerate lumbar puncture * If on approved therapies for the treatment of ALS during the course of the study, must be on a stable dose (at the Sponsor's discretion) Exclusion Criteria (Open-Label Extended Dosing Period): * Currently enrolled in any other clinical study involving either an investigational product (IP) or off label use of a drug or device * Off-label use of drugs or devices that are being used as disease-modifying therapies in ALS * Participated in another clinical study within 30 days, or 5 half-lives of the previous investigational product, whichever is greater, prior to the first dose * Prior exposure to stem cell or gene therapy products * Is not suitable for inclusion in the Open-Label Extended Dosing period of the study, in the opinion of the Investigator * An EEG that shows signs of abnormal electrical activity (NOTE: Cohort 1 and Cohort 2 participants only)

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Conditions

The condition(s) this trial relates to.

amyotrophic lateral sclerosis

As listed by the trial registrant

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

Amyotrophic lateral sclerosis

Contacts and locations

Locations

  • CHUM - Hopital Notre-Dame

    Montreal, Quebec, H2L 4M1, Canada

  • Charité Research Organisation

    Berlin, 10117, Germany

  • Deutsches Zentrum für Neurodegenerative Erkrankungen e. V. (DZNE)

    Bonn, North Rhine-Westphalia, 53127, Germany

  • Kings College Hospital NHS Foundation Trust

    London, SE5 9RS, United Kingdom

  • Montreal Neurological Institute-Hospital

    Montreal, Quebec, H3A 2B4, Canada

  • National Hospital for Neurology and Neurosurgery

    London, WC1N 3BG, United Kingdom

  • St James's Hospital

    Dublin, D08 A978, Ireland

  • Sunnybrook Health Science Centre

    Toronto, Ontario, M4N 3M5, Canada

  • The University of Sheffield, Royal Hallamshire Hospital

    Sheffield, United Kingdom, S10 2JF, United Kingdom

  • Universitair Medisch Centrum Utrecht

    Utrecht, Netherlands

  • Universitaire Ziekenhuizen Leuven (UZ Leuven)

    Leuven, B-3000, Belgium

  • University Hospital Schleswig-Holstein (UKSH) Campus Lübeck, Department for Neurology/ Precision Neurology

    Lübeck, Germany

  • University of Alberta

    Edmonton, Alberta, T6G 2G3, Canada

  • University of Calgary

    Calgary, Alberta, T2N 1N4, Canada

  • Universitätsklinikum Ulm

    Ulm, 89081, Germany

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