LTX-002 in adults with ALS

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This early-phase study investigates the safety, tolerability, pharmacokinetics, and pharmacodynamics of LTX-002 delivered via lumbar puncture in adults with ALS. Participants will undergo multiple clinic visits and occasional remote contacts, with assessments including medical history, neurologic exams, heart monitoring, and multiple samples (blood, urine, CSF) along with tests of lung function, speech, muscle strength, and ALS symptom scales. The study will compare LTX-002 to placebo and monitor effects on biomarkers in blood and CSF and functional outcomes related to movement, speech, and breathing.

Why it matters

Who can join: the full rules

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

Where it's running

• University Hospital Schleswig-Holstein, Campus Lübeck
  Lübeck, Germany Recruiting
• AOU Città della Salute e della Scienza di Torino
  Torino, Italy Recruiting
• Universitair Medisch Centrum Utrecht
  Utrecht, Netherlands Recruiting
• Karolinska Universitetssjukhuset
  Stockholm, Sweden Recruiting
• Istituto Neurologico "Carlo Besta"
  Milan, Italy Not yet recruiting

Study contacts

• Study coordinator: Clinical Trials Information
• Email: clinical.trials@lealtx.com
• Phone: 267-503-4800

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.