LY4256984 for people with sporadic ALS.

A Multiple Ascending Dose Phase 1 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered LY4256984 in Participants With Sporadic Amyotrophic Lateral Sclerosis

This trial will test LY4256984 given into the spine to see how safe it is and how it behaves in the body for people with sporadic ALS.

Quick facts

• Phase: Phase 1
• Study type: Interventional
• Enrollment: 32 (estimated)
• Ages: 18 Years to 75 Years
• Sex: All
• Sponsor: Eli Lilly and Company (Industry-sponsored)
• Locations: 12 sites (Leuven and 11 other locations)
• Trial ID: NCT07100119 on ClinicalTrials.gov

What this trial studies

This is a Phase 1 interventional study testing intrathecal (spine) administration of LY4256984 versus placebo to characterize tolerability and side effects in people with sporadic ALS. The protocol includes blood sampling to measure how much drug reaches the bloodstream and how long it stays in the body. Eligible participants have symptom onset within 24 months and meet basic BMI and laboratory criteria, while those with significant comorbidities or major liver or kidney abnormalities are excluded. The trial is sponsored by Eli Lilly and is being conducted at centers in Belgium and Canada.

Who should consider this trial

Good fit: Ideal candidates are adults with a physician-confirmed sporadic ALS diagnosis whose symptoms began within the last 24 months, with BMI 18–35 kg/m² and no significant comorbid medical, hepatic, or renal problems.

Not a fit: People with other major medical illnesses, another neurodegenerative disease, severe cognitive impairment, marked liver enzyme elevation, or significant renal impairment are unlikely to benefit from participation.

Why it matters

Potential benefit: If shown to be safe and well tolerated, LY4256984 could enable further testing as a potential new treatment approach for ALS.

How similar studies have performed: Intrathecal delivery of other experimental ALS agents has produced early safety data in Phase 1 studies, but clear evidence of clinical benefit from this delivery approach remains unproven.

Eligibility criteria

Inclusion Criteria:

* Have a definite, possible, or probable diagnosis of sporadic amyotrophic lateral sclerosis (ALS) made by a physician experienced with the management of ALS
* ALS symptom onset as determined by the Investigator within 24 months of Screening
* Have a body mass index (BMI) within the range of greater than or equal to 18.0 and less than or equal to 35.0 kilogram per square meter (kg/m²) (inclusive)

Exclusion Criteria:

* Have a history or presence of medical illness including, but not limited to, any cardiovascular, renal, hepatic, gastrointestinal, respiratory, hematological, endocrine, psychiatric, or neurological disease, convulsions, or any clinically significant laboratory abnormality
* Have a history of another neurodegenerative disease or significant dementia/severe cognitive problems
* Have serum alanine aminotransferase (ALT), aspartate transferase (AST), or total bilirubin levels greater than 2 x upper limit of normal.
* Have a significant renal impairment (estimated glomerular filtration rate \<60 milliliters per minute \[mL/min\]/1.73 m²).
* Have a 12-lead electrocardiogram (ECG) abnormality at screening, in the opinion of the investigator, that increases the risks associated with participating in the study
* Show clinically significant abnormalities in lumbar spine previously known or determined by screening lumbar X-ray or fluoroscopy (if performed)

Where this trial is running

Leuven and 11 other locations

• UZ Leuven — Leuven, Belgium (Recruiting)
• Heritage Medical Research Clinic — Calgary, Canada (Not_yet_recruiting)
• Walter Mackenzie Health Sciences Centre — Edmonton, Canada (Not_yet_recruiting)
• London Health Sciences Centre — London, Canada (Not_yet_recruiting)
• Montreal Neurological Institute and Hospital — Montreal, Canada (Recruiting)
• Sunnybrook Research Institute — Toronto, Canada (Recruiting)
• Universitätsklinikum Schleswig-Holstein — Lübeck, Germany (Recruiting)
• Universitätsmedizin Rostock Sektion für Translationale Neurodegeneration "Albrecht Kossel" Klinik und Poliklinik für Neurologie — Rostock, Germany (Not_yet_recruiting)
• Universitätsklinikum Ulm — Ulm, Germany (Not_yet_recruiting)
• Universitair Medisch Centrum Utrecht — Utrecht, Netherlands (Recruiting)
• Hospital Universitario de Bellvitge — L'Hospitalet de Llobregat, Spain (Recruiting)
• Hospital Universitari i Politecnic La Fe — Valencia, Spain (Recruiting)

Study contacts

• Study coordinator: Trial questions or participation questions: 1-877-CTLILLY (1-877-285-4559) or

Conditions ALS