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

Sponsor
Leal Therapeutics, Inc
Study ID
NCT07660614
Phase
PHASE1/PHASE2
Status
Recruiting
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Conditions

Eligibility Criteria

Sex
ALL
Age
18 Years - 75 Years
Healthy Volunteers
Not accepted

Interventions

  • LTX-002 — DRUG
    LTX-002 is an antisense oligonucleotide (ASO) targeting SPTLC1 messenger RNA (mRNA). Several dose levels will be tested.
  • Placebo (aCSF) — OTHER
    Sterile aCSF solution formulation intended for intrathecal administration.

Study Details

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.

Key Dates

Start date
Apr 29, 2026
Status verified
Jun 2026
Primary completion
Jun 30, 2031
Completion
Jun 30, 2031

Study Design

Enrollment
56 participants (estimated)
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: LTX-002
    LTX-002 is an antisense oligonucleotide (ASO) targeting SPTLC1 messenger RNA (mRNA). Several dose levels will be tested.
  • Placebo Comparator: Placebo (aCSF)
    Sterile aCSF solution formulation intended for intrathecal administration.

Primary Outcome Measure

Safety and Tolerability (Adverse Events) [ Time Frame: Screening to Day 169 ]

Central Contacts

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