Recruiting
Phase 1

LY4256984

Sponsor:

Eli Lilly and Company

Code:

NCT07100119

Conditions

ALS (Amyotrophic Lateral Sclerosis)

Eligibility Criteria

Sex: All

Age: 18 - 70+

Healthy Volunteers: Not accepted

Interventions

LY4256984

Placebo

Study Details

Brief summary:

The purpose of this study is to evaluate how well LY4256984 is tolerated and what side effects may occur in participants with sporadic amyotrophic lateral sclerosis (ALS). The study drug will be administered intrathecally (IT) into the spine. Blood tests will be performed to check how much LY4256984 gets into the bloodstream and how long it takes the body to eliminate it.

Conditions

ALS (Amyotrophic Lateral Sclerosis)

Study ID

NCT07100119

Start date

Aug 5, 2025

Status verified date

Mar, 2026

Completion date

Sep, 2027

Anticipated

Primary completion date

Aug, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18 - 70+

Healthy Volunteers: Not accepted

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)

Study Design

Enrollment

32 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Basic Science

Interventions and Outcome Measures

Arms

experimental: LY4256984 (Cohorts 1-4)

Multiple ascending doses (MAD) of LY4256984 administered intrathecally (IT)

placebo comparator: Placebo

Administered IT

Interventions

LY4256984

Administered IT

Placebo

Administered IT

Primary outcome measure

  • Number of Participants with One or More Treatment Emergent Adverse Events (TEAEs), Serious Adverse Event(s) (SAEs), and Adverse Event(s) (AEs) Considered by the Investigator to be Related to Study Drug Administration [ Time Frame: Baseline up to Approximately Day 253 ]

Central Contacts and Locations

Central contacts

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

1-317-615-4559LillyTrials@Lilly.com

Physicians interested in becoming principal investigators please contact

clinical_inquiry_hub@lilly.com

Locations

Montreal Neurological Institute and Hospital

Recruiting

Montreal, Canada, H3A 2B4

Contacts

514-449-8741

Principal Investigator:

Angela Genge

Sunnybrook Research Institute

Recruiting

Toronto, Canada, M4N 3M5

Contacts

4134806860

Principal Investigator:

Agessandro Abrahao

More Information

Sponsor

Eli Lilly and Company

Last update posted

Mar 6, 2026

Last verified

Mar, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Eli Lilly and Company on 2026-03-06.