A Study of LTX-002 in Adult Participants With Amyotrophic Lateral Sclerosis
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: LTX-002, Placebo (aCSF).
- Who it may be relevant to
- Registry conditions: Amyotrophic Lateral Sclerosis. Basic parameters: 18 years — 75 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Germany, Italy, Netherlands, Sweden
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
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
Overview
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.
Detailed description
Eligible participants will be asked to visit the study site 9 times and stay overnight once. In addition, there will be telephone or video calls with the study staff on 8 separate occasions. Procedures will include:
* A review of medical history and medications * Physical exams, including a neurologic (nervous system) assessment * Electrocardiograms (a measure of the heart's electrical activity) * Blood, urine and cerebrospinal fluid (CSF) collection * Measures of lung function * Measures of speech * Measures of muscle strength * Scales to assess ALS symptoms and overall health, and feelings about suicide * Administration of the study drug (or placebo) by injection into the CSF through a lumbar (lower spinal area) injection
Interventions
- Drug LTX-002
LTX-002 is an antisense oligonucleotide (ASO) targeting SPTLC1 messenger RNA (mRNA). Several dose levels will be tested. - Other Placebo (aCSF)
Sterile aCSF solution formulation intended for intrathecal administration.
Primary outcome measures
- Safety and Tolerability (Adverse Events) [Time frame: Screening to Day 169]
- Safety and Tolerability (Clinical Laboratory Tests) [Time frame: Screening to Day 169]
- Safety and Tolerability (Vital Signs) [Time frame: Screening to Day 169]
- Safety and Tolerability (Physical and Neurological exams) [Time frame: Screening to Day 169]
- Safety and Tolerability (ECGs - heart rate) [Time frame: Screening to Day 169]
- Safety and Tolerability (ECGs - QRS) [Time frame: Time Frame: Screening to Day 169]
- Safety and Tolerability (ECGs - QT) [Time frame: Screening to Day 169]
- Safety and Tolerability (ECGs - QTc) [Time frame: Screening to Day 169]
- Safety and Tolerability (ECGs - PR intervals) [Time frame: Screening to Day 169]
Secondary outcome measures (10)
- Pharmacokinetics of LTX-002 (CSF and plasma levels) [Time frame: Day 1 to Day 169]
- Pharmacokinetics of LTX-002 (Cmax) [Time frame: Day 1 to Day 169]
- Pharmacokinetics of LTX-002 (Tmax) [Time frame: Day 1 through Day 169]
- Pharmacokinetics of LTX-002 (AUC0-∞) [Time frame: Day 1 to Day 169]
- Pharmacokinetics of LTX-002 (AUC0-t) [Time frame: Day 1 to Day 169]
- Pharmacokinetics of LTX-002 (Tlag) [Time frame: Day 1 to Day 169]
- Pharmacokinetics of LTX-002 (Vd/F) [Time frame: Day 1 to Day 169]
- Pharmacokinetics of LTX-002 (λz) [Time frame: Day 1 to Day 169]
- Pharmacokinetics of LTX-002 (T1/2) [Time frame: Day 1 to Day 169]
- Pharmacokinetics of LTX-002 (CL/F) [Time frame: Day 1 to Day 169]
Eligibility criteria
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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Randomized
- Model
- Parallel assignment
- Masking
- Quadruple blind
- Primary purpose
- Treatment
Study locations
Italy · 2 centers
- Istituto Neurologico "Carlo Besta" — Milan
- AOU Città della Salute e della Scienza di Torino — Torino
Germany · 1 center
- University Hospital Schleswig-Holstein, Campus Lübeck — Lübeck
Netherlands · 1 center
- Universitair Medisch Centrum Utrecht — Utrecht
Sweden · 1 center
- Karolinska Universitetssjukhuset — Stockholm
Identifiers
NCT: NCT07660614 · LTX-002-101 · 2025-522039-33-00