RecruitingRecruiting
A Study of LTX-002 in Adult Participants With Amyotrophic Lateral Sclerosis
NCT07660614 · Leal Therapeutics, Inc
In plain English
Click the button to translate this study into plain language — what it is, who qualifies, and what participation looks like.
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
About this study
Eligible participants will be asked to visit the study site 9 times and stay overnight once. In addition, there will be telephone or video calls with the study staff on 8 separate occasions. Procedures will include:
* A review of medical history and medications
* Physical exams, including a neurologic (nervous system) assessment
* Electrocardiograms (a measure of the heart's electrical activity)
* Blood, urine and cerebrospinal fluid (CSF) collection
* Measures of lung function
* Measures of speech
* Measures of muscle strength
* Scales to assess ALS symptoms and overall health, and feelings about suicide
* Administration of the study drug (or placebo) by injection into the CSF through a lumbar (lower spinal area) injection
Eligibility criteria
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
Study design
Enrollment target: 56 participants
Allocation: randomized
Masking: quadruple
Age groups: adult, older_adult
Timeline
Starts: 2026-04-29
Estimated completion: 2031-06
Last updated: 2026-06-22
Interventions
Drug: LTX-002Other: Placebo (aCSF)
Primary outcomes
- • Safety and Tolerability (Adverse Events) (Screening to Day 169)
- • Safety and Tolerability (Clinical Laboratory Tests) (Screening to Day 169)
- • Safety and Tolerability (Vital Signs) (Screening to Day 169)
Sponsor
Leal Therapeutics, Inc · industry
Contacts & investigators
ContactClinical Trials Information · contact · clinical.trials@lealtx.com · 267-503-4800
InvestigatorMedical Monitor · study_director, Leal Therapeutics, Inc
All locations (5)
University Hospital Schleswig-Holstein, Campus LübeckNot Yet Recruiting
Lübeck, Germany
Istituto Neurologico "Carlo Besta"Not Yet Recruiting
Milan, Italy
AOU Città della Salute e della Scienza di TorinoNot Yet Recruiting
Torino, Italy
Universitair Medisch Centrum UtrechtRecruiting
Utrecht, Netherlands
Karolinska UniversitetssjukhusetRecruiting
Stockholm, Sweden