New ALS drug targets genetic cause in early human trial

NCT ID NCT07660614

Recruiting now Disease control Sponsor: Leal Therapeutics, Inc Source: ClinicalTrials.gov ↗

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

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.

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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

AOU Città della Salute e della Scienza di Torino
NOT_YET_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
NOT_YET_RECRUITING

Lübeck, Germany

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