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New gene therapy for ALS enters first human safety trial

NCT ID NCT07401121

Disease control Sponsor: Celosia Therapeutics Pty Ltd Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
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
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 a single dose of a gene therapy called CTx1000 in 15 people with ALS. The main goal is to see if it is safe and tolerable. Participants must have had ALS for 2 years or less and be on stable standard medications. This is not a cure; it aims to control 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

About 15 people

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

Started

Jan 2026

Expected to finish

Dec 2030

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: * Diagnosis of ALS in accordance with the revised El Escorial criteria and TRICALS risk score * An overall disease duration of ≤ 2 years after the participant's first symptoms * No or low circulating anti-AAV9 antibodies (titre ≤ 1:50 * Stable dosing with a standard of care ALS medication (eg, riluzole and edaravone) and other prescription medications for 30 days prior to Screening * Not pregnant or breastfeeding, or willing to cease breastfeeding * All participants must use a barrier method of contraception Exclusion Criteria: * Any participants with genetic forms of ALS, including C9ORF72 repeat carriers, except for TARDBP gene variants, as confirmed by previous clinical history genetic testing * Any history of myocardial infarction or stroke within 6 months prior to Screening, or uncontrolled diabetes (HbA1C \> 9%) * Positive test for cytomegalovirus, hepatitis C antibody (HCV), hepatitis B surface antigen (HBsAg), human immunodeficiency virus (HIV) antibody. * Inadequate organ function * Any participant with a current open tracheostomy

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Conditions

The condition(s) this trial relates to.

amyotrophic lateral sclerosis motor neuron disorder

As listed by the trial registrant

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

ALS (amyotrophic lateral sclerosis)

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    1 site. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Macquarie University Hospital

    RECRUITING

    Sydney, New South Wales, 2109, Australia

More trials for these conditions

Other studies related to the condition(s) this trial covers.