A Study of LY4256984 in Participants With Sporadic Amyotrophic Lateral Sclerosis
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: LY4256984, Placebo.
- Who it may be relevant to
- Registry conditions: ALS (Amyotrophic Lateral Sclerosis). Basic parameters: 18 years — 75 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Belgium, Canada, Germany, Netherlands, Spain
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
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
Overview
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.
Interventions
- Drug LY4256984
Administered IT - Drug Placebo
Administered IT
Primary outcome measures
- 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]
Secondary outcome measures (3)
- Pharmacokinetics (PK): Plasma Area Under the Concentration Versus Time Curve (AUC) of LY4256984 [Time frame: Baseline up to Approximately Day 253]
- PK: Plasma Maximum Concentration (Cmax) of LY4256984 [Time frame: Baseline up to Approximately Day 253]
- PK: Cerebrospinal Fluid Concentrations (CSF) of LY4256984 [Time frame: Baseline up to Approximately Day 253]
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)
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Randomized
- Model
- Parallel assignment
- Masking
- Double blind
- Primary purpose
- Basic science
Study locations
Canada · 5 centers
- Heritage Medical Research Clinic — Calgary
- Walter Mackenzie Health Sciences Centre — Edmonton
- London Health Sciences Centre — London
- Montreal Neurological Institute and Hospital — Montreal
- Sunnybrook Research Institute — Toronto
Germany · 3 centers
- Universitätsklinikum Schleswig-Holstein — Lübeck
- Universitätsmedizin Rostock Sektion für Translationale Neurodegeneration "Albrecht Kossel" — Rostock
- Universitätsklinikum Ulm — Ulm
Spain · 2 centers
- Hospital Universitario de Bellvitge — L'Hospitalet de Llobregat
- Hospital Universitari i Politecnic La Fe — Valencia
Belgium · 1 center
- UZ Leuven — Leuven
Netherlands · 1 center
- Universitair Medisch Centrum Utrecht — Utrecht
Identifiers
NCT: NCT07100119 · 27427 · J6I-MC-OWAA · 2025-521295-6 · U1111-1321-6118