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A Study of LTX-002 in Adult Participants With Amyotrophic Lateral Sclerosis
Sponsor: Leal Therapeutics, Inc
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.
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
Key Details
Gender
All
Age Range
18 Years - 75 Years
Study Type
INTERVENTIONAL
Enrollment
56
Start Date
2026-04-29
Completion Date
2031-06
Last Updated
2026-08-18
Healthy Volunteers
No
Conditions
Interventions
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.
Locations (5)
University Hospital Schleswig-Holstein, Campus Lübeck
Lübeck, Germany
Istituto Neurologico "Carlo Besta"
Milan, Italy
AOU Città della Salute e della Scienza di Torino
Torino, Italy
Universitair Medisch Centrum Utrecht
Utrecht, Netherlands
Karolinska Universitetssjukhuset
Stockholm, Sweden