Recruiting

LY4256984 for Sporadic Amyotrophic Lateral Sclerosis

0 criteria met from your profileSee at a glance how your profile meets each eligibility criteria.
What is being tested

LY4256984

+ Placebo
Drug
Who is being recruted

Amyotrophic Lateral Sclerosis
+8

+ Central Nervous System Diseases
+ Metabolic Diseases
From 18 to 75 Years
+9 Eligibility Criteria
See all eligibility criteria
How is the trial designed

Basic Science Study

Placebo-Controlled
Phase 1
Interventional
Study Start: August 2025
See protocol details

Summary

Principal SponsorEli Lilly and Company
Study ContactTrial questions or participation questions: 1-877-CTLILLY (1-877-285-4559) orMore contacts
Last updated: December 25, 2025
Sourced from a government-validated database.Claim as a partner
Study start date: August 5, 2025Actual date on which the first participant was enrolled.

This study focuses on understanding how a new drug, LY4256984, works in people with a condition called sporadic amyotrophic lateral sclerosis (ALS). ALS is a progressive nervous system disease that affects nerve cells in the brain and spinal cord, causing loss of muscle control. The main goal is to see how well people tolerate this drug and identify any side effects. This research is important because it may lead to better treatment options for those living with ALS, who currently have limited therapeutic options. Participants in the study receive LY4256984 through an injection into the spine, a method called intrathecal administration. Blood tests are conducted to determine how much of the drug enters the bloodstream and how quickly the body processes and eliminates it. This helps researchers understand the drug's behavior in the body. While the study aims to uncover potential benefits, it also carefully monitors for any side effects to ensure participant safety.

Official TitleA 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 
NCT07100119
Principal SponsorEli Lilly and Company
Study ContactTrial questions or participation questions: 1-877-CTLILLY (1-877-285-4559) orMore contacts
Last updated: December 25, 2025
Sourced from a government-validated database.Claim as a partner

Protocol

This section provides details of the study plan, including how the study is designed and what the study is measuring.
Design Details
32 patients to be enrolledTotal number of participants that the clinical trial aims to recruit.
Basic Science Study
Basic science studies help researchers understand how the body works or how a disease develops. They don't test treatments, but they build the foundation for future therapies.

How participants are assigned to different groups/arms
In this clinical study, participants are placed into groups randomly, like flipping a coin. This ensures that the study is fair and unbiased, making the results more reliable. By assigning participants by chance, researchers can better compare treatments without external influences.

Other Ways to Assign Participants
Non-randomized allocation: Participants are assigned based on specific factors, such as their medical condition or a doctor's decision.
None (Single-arm trial): If the study has only one group, all participants receive the same treatment, and no allocation is needed.

How treatments are given to participants
Participants are divided into different groups, each receiving a specific treatment at the same time. This helps researchers compare how well different treatments work against each other.

Other Ways to Assign Treatments
Single-group assignment: Everyone gets the same treatment.
Cross-over assignment: Participants switch between treatments during the study.
Factorial assignment: Participants receive different combinations of treatments.
Sequential assignment: Participants receive treatments one after another in a specific order, possibly based on individual responses.
Other assignment: Treatment assignment does not follow a standard or predefined design.

How the effectiveness of the treatment is controlled
In a placebo-controlled study, some participants receive the experimental treatment, while others receive an inert substance (placebo) to compare outcomes. This method helps to isolate the effect of the treatment from the psychological effects of receiving any treatment at all.

Other Options
Non-placebo-controlled: No placebo is used. All participants receive the actual treatment or alternative interventions (often the Standard of Care), and comparisons are made between these treatments.

How the interventions assigned to participants is kept confidential
Neither participants nor researchers know who is receiving which treatment. This is the most rigorous way to reduce bias, ensuring that expectations do not influence the results.

Other Ways to Mask Information
Open-label: Everyone knows which treatment is being given.
Single-blind: Participants do not know which treatment they are receiving, but researchers do.
Triple-blind: Participants, researchers, and outcome assessors do not know which treatment is given.
Quadruple-blind: Participants, researchers, outcome assessors, and care providers all do not know which treatment is given.

Eligibility

Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.
Conditions
Criteria
Any sexBiological sex of participants that are eligible to enroll.
From 18 to 75 YearsRange of ages for which participants are eligible to join.
Healthy volunteers not allowedIf individuals who are healthy and do not have the condition being studied can participate.
Conditions
Pathology
Amyotrophic Lateral Sclerosis
Central Nervous System Diseases
Metabolic Diseases
Nervous System Diseases
Neuromuscular Diseases
Nutritional and Metabolic Diseases
Spinal Cord Diseases
Motor Neuron Disease
Neurodegenerative Diseases
Proteostasis Deficiencies
TDP-43 Proteinopathies
Criteria
3 inclusion criteria required to participate
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)
6 exclusion criteria prevent from participating
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).

Show More Criteria

Study Plan

Find out more about all the medication administered in this study, their detailed description and what they involve.
Treatment Groups
Study Objectives
2 intervention groups 

are designated in this study

50% chance 

of being blinded to the placebo group

Treatment Groups
Group I
Experimental
Multiple ascending doses (MAD) of LY4256984 administered intrathecally (IT)

Administered IT
Group II
Placebo
Administered IT

Administered IT
Study Objectives
Primary Objectives

A summary of TEAEs, SAEs, and AEs regardless of causality, will be reported in the Reported Adverse Events module
Secondary Objectives

PK: Plasma AUC of LY4256984

PK: Plasma Cmax of LY4256984

PK: CSF Concentrations of LY4256984

Study Centers

These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.
This study has 12 locations
Recruiting
Montreal Neurological Institute and HospitalMontreal, CanadaSee the location
Recruiting
Sunnybrook Research InstituteToronto, Canada
Recruiting soon
UZ LeuvenLeuven, Belgium
Recruiting soon
Heritage Medical Research ClinicCalgary, Canada
Recruiting
12 Study Centers
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