A Study of LTX-002 in Adult Participants With Amyotrophic Lateral Sclerosis

NCT07660614 | Recruiting Phase 1 DMC FDA Drug

• 2 other identifiers: LTX-002-1012025-522039-33-00
• Study Type: interventional
• Target: 56
• Locations: 4 countries
• Sites: 5

Brief 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.

Conditions

Amyotrophic Lateral Sclerosis

Keywords

ALS

Outcome Measures

Primary Outcomes (9)

• Safety and Tolerability (Adverse Events)

  Incidence and severity of adverse events (AEs), treatment-emergent adverse events (TEAEs) and serious adverse events

  Screening to Day 169

• Safety and Tolerability (Clinical Laboratory Tests)

  Clinical laboratory tests including serum chemistry and hematology; urinalysis

  Screening to Day 169

• Safety and Tolerability (Vital Signs)

  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.

  Screening to Day 169

• Safety and Tolerability (Physical and Neurological exams)

  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.

  Screening to Day 169

• Safety and Tolerability (ECGs - heart rate)

  Cardiovascular safety will be monitored by performing electrocardiogram (ECG) assessments, including heart rate

  Screening to Day 169

• Safety and Tolerability (ECGs - QRS)

  Cardiovascular safety will be monitored by performing electrocardiogram (ECG) assessments, including the QRS complex (normally 70-100 ms)

  Time Frame: Screening to Day 169

• Safety and Tolerability (ECGs - QT)

  Description: Cardiovascular safety will be monitored by performing electrocardiogram (ECG) assessments, including the QT interval measure

  Screening to Day 169

• Safety and Tolerability (ECGs - QTc)

  Cardiovascular safety will be monitored by performing electrocardiogram (ECG) assessments, including QTc (heart rate-corrected QT interval)

  Screening to Day 169

• Safety and Tolerability (ECGs - PR intervals)

  Cardiovascular safety will be monitored by performing electrocardiogram (ECG) assessments, including PR intervals (normally 120-200 ms)

  Screening to Day 169

Secondary Outcomes (10)

• Pharmacokinetics of LTX-002 (CSF and plasma levels)

  Day 1 to Day 169

• Pharmacokinetics of LTX-002 (Cmax)

  Day 1 to Day 169

• Pharmacokinetics of LTX-002 (Tmax)

  Day 1 through Day 169

• Pharmacokinetics of LTX-002 (AUC0-∞)

  Day 1 to Day 169

• Pharmacokinetics of LTX-002 (AUC0-t)

  Day 1 to Day 169

Other Outcomes (9)

• Incidence of anti-LTX-002 antibodies in plasma

  Day 1 to Day 169

• Change from baseline in SPTLC1 protein concentration in cerebrospinal fluid

  Day 1 to Day 169

• Change from baseline in ceramide and other sphingolipid concentrations in cerebrospinal fluid

  Day 1 to Day 169

Study Arms (2)

LTX-002

EXPERIMENTAL

LTX-002 is an antisense oligonucleotide (ASO) targeting SPTLC1 messenger RNA (mRNA). Several dose levels will be tested.

Drug: LTX-002

Placebo (aCSF)

PLACEBO COMPARATOR

Sterile aCSF solution formulation intended for intrathecal administration.

Other: Placebo (aCSF)

Interventions

LTX-002 DRUG

LTX-002 is an antisense oligonucleotide (ASO) targeting SPTLC1 messenger RNA (mRNA). Several dose levels will be tested.

LTX-002

Placebo (aCSF) OTHER

Sterile aCSF solution formulation intended for intrathecal administration.

Placebo (aCSF)

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Leal Therapeutics, Inc: lead

Study Sites (5)

University Hospital Schleswig-Holstein, Campus Lübeck

Lübeck, Germany

NOT YET RECRUITING

Istituto Neurologico "Carlo Besta"

Milan, Italy

NOT YET RECRUITING

AOU Città della Salute e della Scienza di Torino

Torino, Italy

NOT YET RECRUITING

Universitair Medisch Centrum Utrecht

Utrecht, Netherlands

RECRUITING

Karolinska Universitetssjukhuset

Stockholm, Sweden

RECRUITING

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Medical Monitor

  Leal Therapeutics, Inc

  STUDY DIRECTOR

Central Study Contacts

Clinical Trials Information

CONTACT

267-503-4800; clinical.trials@lealtx.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Participants (8 per cohort, randomized in a 6:2 ratio to receive LTX-002 or placebo) will receive a total of three IT injections of LTX-002 (or placebo) on Days 1, 29, and 85

Study Record Dates

• First Submitted: May 18, 2026
• First Posted: June 22, 2026
• Study Start: April 29, 2026
• Primary Completion (Estimated): June 1, 2031
• Study Completion (Estimated): June 1, 2031
• Last Updated: June 22, 2026

Record last verified: 2026-06

Data Sharing

• IPD Sharing: Will not share

Locations

SE (1); DE (1); NL (1); IT (2)