NCT07660614RecruitingPhase 1/2
A Study of LTX-002 in Adult Participants With Amyotrophic Lateral Sclerosis
A First-in-Human, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study of Intrathecally Administered LTX-002 in Adult Participants With Amyotrophic Lateral Sclerosis
Source: ClinicalTrials.gov · Last updated 18 Aug 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Recruiting
- Phase (registry)
- Phase 1/2
- Study type
- Interventional
- Enrollment
- 56 (estimated)
- First submitted
- 18 May 2026
- Start date
- 29 Apr 2026
- Primary completion date
- Jun 2031
- Lead sponsor
- Leal Therapeutics, Inc
- Diseases
- ALS (motor neurone disease)
- NCT number
- NCT07660614
- EU CT number
- 2025-522039-33-00
What you commit to
- Study type: Interventional · Phase 1/2
- Planned participants: 56
- From 29 Apr 2026 to the primary completion date, Jun 2031
- 7 sites in 4 countries
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Prior treatment with antisense oligonucleotide (ASO), small interfering RNA, stem cell therapy, or gene therapy for any indication
Other illness
- 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
Other
- Tracheostomy
- Presence of implanted shunt (CSF) or vascular device
- Pregnant or breastfeeding
Who the study is looking for
- 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
Age in the registry: 18 years to 75 years · Sex: All · Does not accept healthy volunteers
Sites (7)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Charite Universitaetsmedizin Berlin KöR Coordinator of this site? Confirm its status | Berlin | Germany | Recruiting |
| Universitaetsklinikum Schleswig-Holstein AöR Coordinator of this site? Confirm its status | Luebeck | Germany | Recruiting |
| IRCCS Foundation Istituto Neurologico Carlo Besta Coordinator of this site? Confirm its status | Milan | Italy | Recruiting |
| Azienda Ospedaliera Universitaria Citta Della Salute E Della Scienza Di Torino Coordinator of this site? Confirm its status | Turin | Italy | Recruiting |
| Universitair Medisch Centrum Utrecht Coordinator of this site? Confirm its status | Utrecht | Netherlands | Recruiting |
| Karolinska University Hospital Coordinator of this site? Confirm its status | Huddinge | Sweden | Recruiting |
| Region Vaesterbotten Coordinator of this site? Confirm its status | Umea | Sweden | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.