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Taking people in ItalyPhase 1/2

Tests LTX-002 in adults with amyotrophic lateral sclerosis

Tests the investigational drug LTX-002, given into the spinal fluid, in adults with ALS.

Key facts

Who runs it
Leal Therapeutics, Inc
Where
Milan and Turin, ItalySee the 2 sitesAlso in 3 other countries, 5 sites
Registry title
A Study of LTX-002 in Adult Participants With Amyotrophic Lateral Sclerosis
Status
Recruiting
Phase
Phase 1/2
People taking part
56 people, planned
Started
29 Apr 2026
Expected to end
Jun 2031
Type of study
Interventional, 2 groupsOne group gets a placebo (a dummy treatment), for comparison.
Treatments tested
  • LTX-002
  • Placebo (aCSF)
Record updated
18 August 2026

Who can take part

AI summary

From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.

The trial is looking for adults aged 18 to 75 with ALS (amyotrophic lateral sclerosis). It tests the study drug LTX-002 given into the spinal fluid, compared with a placebo.

Age
18 Years to 75 Years
Sex
All sexes can join.
Disease stage
ALS diagnosed by the Gold Coast criteria. Symptoms started less than 36 months before screening. Slow vital capacity (a breathing test) at least 50% of the predicted value.
Earlier treatment
People who ever had antisense oligonucleotide (ASO), small interfering RNA, stem cell therapy, or gene therapy for any condition cannot join.

Main reasons for not taking part

  • Prior ASO, small interfering RNA, stem cell or gene therapy
  • Tracheostomy
  • HIV, or acute or chronic hepatitis B or C infection
  • Implanted CSF shunt or vascular device
  • Pregnant or breastfeeding

The registry criteria, word for word

Show all 12 criteriaShow less

Who can take part

  • 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

Who cannot take part

  • Prior treatment with antisense oligonucleotide (ASO), small interfering RNA, stem cell therapy, or gene therapy for any indication
  • 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
  • HIV, Hepatitis B or C infection (acute or chronic)
  • Risk for uncontrolled bleeding
  • Tracheostomy
  • Presence of implanted shunt (CSF) or vascular device
  • Pregnant or breastfeeding

Check if you might fit

Answer for yourself. This is not a decision: only the study team can say whether you can take part.

  • Are you between 18 and 75 years old?

Where in Italy

Also in 3 other countries, 5 sites

  • IRCCS Foundation Istituto Neurologico Carlo BestaMilan, ItalyTaking people
  • Azienda Ospedaliera Universitaria Citta Della Salute E Della Scienza Di TorinoTurin, ItalyTaking people

About the trial

What the trial wants to find out

This study will examine what happens when patients with amyotrophic lateral sclerosis (ALS) are given an investigational medication (study drug) known as LTX-002. Specifically, the researchers will be looking at safety, tolerability (if someone has any side effects from the drug), pharmacokinetics (what the body does to the study drug) and pharmacodynamics (what the study drug does to the body). The study will also investigate the effect of the drug on indicators of the severity of ALS, such as markers in blood and in the cerebrospinal fluid (the fluid that surrounds the brain and spinal cord, CSF) and on measures of the participant's ability to move, speak, and breathe.

The 2 groups in the trial

  • LTX-002Treatment being tested

    LTX-002 is an antisense oligonucleotide (ASO) targeting SPTLC1 messenger RNA (mRNA). Several dose levels will be tested.

  • Placebo (aCSF)Placebo, for comparison

    Sterile aCSF solution formulation intended for intrathecal administration.

Questions to ask the study team

  1. How often do I need to come in, and for how long each time?
  2. Are travel costs paid?
  3. Can I keep my own doctor?
  4. What happens if I want to stop?

Contact the study team: see the registry record → Contacts and Locations

Print a page for my doctor

Data and sources

Registry record
Listed on ClinicalTrials.gov and on the EU's official list of trials (CTIS).
Official title
A First-in-Human, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study of Intrathecally Administered LTX-002 in Adult Participants With Amyotrophic Lateral Sclerosis
Plain-language title
Tests LTX-002 in adults with amyotrophic lateral sclerosisGenerated from the registry record. The registry title is the official one.
AI summary
From the trial's eligibility criteria. Read by machine on 12 November 2026.
Fit questions
Built from NCT07660614, who can take part
Record updated
18 August 2026