Tests LTX-002 in adults with amyotrophic lateral sclerosis
Tests the investigational drug LTX-002, given into the spinal fluid, in adults with ALS.
Plain-language title generated from the registry record
Registry title: A Study of LTX-002 in Adult Participants With Amyotrophic Lateral Sclerosis
Key facts
- Where
- Milan and Turin, ItalySee the 2 sites
- Phase
- Phase 1/2
Could this be for you?
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?
Source: NCT07660614, who can take part
Where in Italy
- IRCCS Foundation Istituto Neurologico Carlo BestaMilan, ItalyTaking people
- Azienda Ospedaliera Universitaria Citta Della Salute E Della Scienza Di TorinoTurin, ItalyTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Leal Therapeutics, Inc
- Trial code
- NCT07660614EU 2025-522039-33-00
- Record updated
- 18 August 2026
- Original description
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.Read full original descriptionShow less
A First-in-Human, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study of Intrathecally Administered LTX-002 in Adult Participants With Amyotrophic Lateral Sclerosis
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.
Inclusion: 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: 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.