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

NCT07660614 · Status: RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 56

Last updated 2026-08-18

No results posted yet for this 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.

Conditions

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.

Sponsors & Collaborators

  • Leal Therapeutics, Inc

    lead INDUSTRY

Principal Investigators

  • Medical Monitor · Leal Therapeutics, Inc

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
18 Years
Max Age
75 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-04-29
Primary Completion
2031-06-30
Completion
2031-06-30
FDA Drug
Yes

Countries

  • Germany
  • Italy
  • Netherlands
  • Sweden

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07660614 on ClinicalTrials.gov