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New ALS drug targets genetic cause in early human trial

NCT ID NCT07660614

Disease control Sponsor: Leal Therapeutics, Inc 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Aug 19, 2026 · Updated 2 times

Summary

This study tests an investigational drug called LTX-002 in people with amyotrophic lateral sclerosis (ALS). The drug is designed to target a specific genetic message linked to the disease. The main goal is to check the drug's safety and tolerability, and to see how it moves through the body and affects disease markers. About 56 adults with ALS will receive either the drug or a placebo via injection into the spinal fluid.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
LTX-002 (an antisense oligonucleotide)
What this could lead to
If safe and effective, this could lead to a new treatment that slows or stops the progression of ALS by targeting a genetic cause.
What could go wrong
This is a very early, small study primarily testing safety. The drug may not work or could cause unexpected side effects. Results may not apply to all ALS patients.

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 56 people

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

Started

Apr 2026

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 to 75 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 per Gold Coast criteria * ALS symptom onset less than 36 months prior to Screening * Slow vital capacity ≥ 50% of predicted value * Body mass index ≥18 and ≤40 kg/m2 Exclusion Criteria: * Current evidence or history of a clinically significant medical condition that, in the Investigator's judgement, would impact the participant's safety, interpretation of study results, or place the participant at high risk of poor treatment compliance or of not completing the study * History of brain or spinal abnormalities on magnetic imaging (MRI) or computed tomography (CT) that might interfere with the lumbar puncture (LP), cerebrospinal fluid (CSF) circulation or safety assessments * Prior treatment with antisense oligonucleotide (ASO), small interfering RNA, stem cell therapy, or gene therapy for any indication * Tracheostomy * HIV, Hepatitis B or C infection (acute or chronic) * Presence of implanted shunt (CSF) or vascular device * Risk for uncontrolled bleeding * Pregnant or breastfeeding

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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

How to take part

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

  1. The places running it

    5 sites in 4 countries. 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

  • AOU Città della Salute e della Scienza di Torino

    RECRUITING

    Torino, Italy

  • Istituto Neurologico "Carlo Besta"

    NOT_YET_RECRUITING

    Milan, Italy

  • Karolinska Universitetssjukhuset

    RECRUITING

    Stockholm, Sweden

  • Universitair Medisch Centrum Utrecht

    RECRUITING

    Utrecht, Netherlands

  • University Hospital Schleswig-Holstein, Campus Lübeck

    RECRUITING

    Lübeck, Germany

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