Actively Recruiting

Phase 1
Age: 18Years - 75Years
All Genders
ID07100119

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

Led by Eli Lilly and Company · Updated on 2026-03-06

32

Participants Needed

12

Research Sites

4 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

Researchers are evaluating the safety and tolerance of LY4256984 in people with sporadic amyotrophic lateral sclerosis (ALS). This Phase 1 study aims to understand what side effects may occur when LY4256984 is given intrathecally (into the spine) and how the drug behaves in the body. The study includes adults aged 18 to 75 with a recent ALS diagnosis and a certain body mass index range. Participants will receive multiple ascending doses of LY4256984 or a placebo administered directly into the spine. The study is randomized and double-blinded, meaning participants receive either the drug or placebo without knowing which one. The treatment period lasts up to approximately 253 days, during which dosing and monitoring occur. Throughout the study, researchers will perform blood tests to measure how much LY4256984 reaches the bloodstream and how long it stays there. They will track side effects and serious adverse events related to the treatment from the start until about day 253. Safety assessments include checking cerebrospinal fluid drug levels, monitoring vital signs, and reviewing lumbar spine health. Participants can expect regular evaluations and laboratory tests during their involvement in the trial, which runs from screening through treatment and follow-up.

CONDITIONS

Brief Title

A Study of LY4256984 in Participants With Sporadic Amyotrophic Lateral Sclerosis

Who Can Participate

Age: 18Years - 75Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • 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/m8) (inclusive)
Not Eligible

You will not qualify if you...

  • 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 m8)
  • 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)

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Your Study Journey

Screening

Duration - 2 to 4 weeks

Participants are screened for eligibility to participate in the trial.

1 visit (in-person)

Treatment

Duration - Up to approximately 253 days

Participants receive multiple ascending doses of LY4256984 or placebo administered intrathecally.

Multiple dosing visits over the treatment period

Trial Site Locations

Total: 12 locations

1

UZ Leuven

Leuven, Belgium, 3000

Actively Recruiting

2

Heritage Medical Research Clinic

Calgary, Canada, T2N4Z6

Not Yet Recruiting

3

Walter Mackenzie Health Sciences Centre

Edmonton, Canada, T6G 2X8

Not Yet Recruiting

4

London Health Sciences Centre

London, Canada, N6A 5A5

Not Yet Recruiting

5

Montreal Neurological Institute and Hospital

Montreal, Canada, H3A 2B4

Actively Recruiting

6

Sunnybrook Research Institute

Toronto, Canada, M4N 3M5

Actively Recruiting

7

Universitätsklinikum Schleswig-Holstein

Lübeck, Germany, 23538

Actively Recruiting

8

Universitätsmedizin Rostock Sektion für Translationale Neurodegeneration "Albrecht Kossel" Klinik und Poliklinik für Neurologie

Rostock, Germany, 18147

Not Yet Recruiting

9

Universitätsklinikum Ulm

Ulm, Germany, 89081

Not Yet Recruiting

10

Universitair Medisch Centrum Utrecht

Utrecht, Netherlands, 3584

Actively Recruiting

11

Hospital Universitario de Bellvitge

L'Hospitalet de Llobregat, Spain, 8907

Actively Recruiting

12

Hospital Universitari i Politecnic La Fe

Valencia, Spain, 46026

Actively Recruiting

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Research Team

T

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

P

Physicians interested in becoming principal investigators please contact

How is the study designed?

Study Type

INTERVENTIONAL

Masking

DOUBLE

Allocation

RANDOMIZED

Model

PARALLEL

Primary Purpose

BASIC_SCIENCE

Number of Arms

2

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