LTX-002: LTX-002 is an antisense oligonucleotide (ASO) targeting SPTLC1 messenger RNA (mRNA). Several dose levels will be tested.
Placebo (aCSF): Sterile aCSF solution formulation intended for intrathecal administration.
Study summary
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.
Eligibility
Sex
ALL
Min age
18 Years
Max age
75 Years
Healthy volunteers
No
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
Primary outcome measure(s)
Safety and Tolerability (Adverse Events) — Screening to Day 169 Incidence and severity of adverse events (AEs), treatment-emergent adverse events (TEAEs) and serious adverse events
Safety and Tolerability (Clinical Laboratory Tests) — Screening to Day 169 Clinical laboratory tests including serum chemistry and hematology; urinalysis
Safety and Tolerability (Vital Signs) — Screening to Day 169 Vital signs will be collected, including blood pressure (mm Hg), heart rate (beats per minute), respiratory rate (breaths per minute), body temperature (°C or °F), total body weight (kilograms), and height (centimeters). Body Mass Index (BMI) will be calculated using the values of total body weight and height, with the formula BMI = weight in kg/(height in cm)\^2.
Safety and Tolerability (Physical and Neurological exams) — Screening to Day 169 Physical and neurological exams will be performed periodically to ensure participant safety. Height (centimeters or inches), weight (kilograms or pounds) and body mass index (BMI; kilograms/meters\^2) will be measured.
Safety and Tolerability (ECGs - heart rate) — Screening to Day 169 Cardiovascular safety will be monitored by performing electrocardiogram (ECG) assessments, including heart rate
Safety and Tolerability (ECGs - QRS) — Time Frame: Screening to Day 169 Cardiovascular safety will be monitored by performing electrocardiogram (ECG) assessments, including the QRS complex (normally 70-100 ms)
Safety and Tolerability (ECGs - QT) — Screening to Day 169 Description: Cardiovascular safety will be monitored by performing electrocardiogram (ECG) assessments, including the QT interval measure
Safety and Tolerability (ECGs - QTc) — Screening to Day 169 Cardiovascular safety will be monitored by performing electrocardiogram (ECG) assessments, including QTc (heart rate-corrected QT interval)
Safety and Tolerability (ECGs - PR intervals) — Screening to Day 169 Cardiovascular safety will be monitored by performing electrocardiogram (ECG) assessments, including PR intervals (normally 120-200 ms)
Trial sites (5)
Facility
City
Region
Status
University Hospital Schleswig-Holstein, Campus Lübeck
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
We use cookies to analyse site traffic and improve your experience. With your consent, we may also use cookies for advertising. You can change your choice at any time.