ALS (Amyotrophic Lateral Sclerosis)Phase 1NCT07100119
A Study of LY4256984 in Participants With Sporadic Amyotrophic Lateral Sclerosis
A Phase 1 study testing LY4256984 in people with ALS (Amyotrophic Lateral Sclerosis), run by Eli Lilly and Company at 12 sites in 5 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- ALS (Amyotrophic Lateral Sclerosis)
- What is being tested?
- LY4256984
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 36 participants
- Where
- 12 sitesin 5 countries
- Study dates
- 5 Aug 2025to Sep 2027
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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)
Who cannot join
- 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)
Trial locations and contacts
12 sites in 5 countries. Contact the study team or a site near you to ask about taking part.
Trial questions or participation questions: 1-877-CTLILLY (1-877-285-4559) or contact
Physicians interested in becoming principal investigators please contact contact
12 sites in 5 countries
Canada5 sites, 5 recruiting
- Recruiting
Heritage Medical Research Clinic
Calgary, T2N4Z6
403-210-7006
Theodore Mobach
- Not yet recruiting
Walter Mackenzie Health Sciences Centre
Edmonton, T6G 2X8
780.248.1089
Peter Dobrowolski
- Recruiting
London Health Sciences Centre
London, N6A 5A5
519-663-3597
Christen Shoesmith
- Recruiting
Montreal Neurological Institute and Hospital
Montreal, H3A 2B4
514-449-8741
Angela Genge
- Recruiting
Sunnybrook Research Institute
Toronto, M4N 3M5
4134806860
Agessandro Abrahao
Germany3 sites, 3 recruiting
- Recruiting
Universitätsklinikum Schleswig-Holstein
Lübeck, 23538
4936419323426
Julian Grosskreutz
- Not yet recruiting
Universitätsmedizin Rostock Sektion für Translationale Neurodegeneration "Albrecht Kossel" Klinik und Poliklinik für Neurologie
Rostock, 18147
49 (0)381 494-9541
Andreas Hermann
- Recruiting
Universitätsklinikum Ulm
Ulm, 89081
(497315) 006-3099
Jochen Weishaupt
Spain2 sites, 2 recruiting
- Recruiting
Hospital Universitario de Bellvitge
L'Hospitalet de Llobregat, 8907
+34 933359011 ext 8486
Monica Povedano
- Recruiting
Hospital Universitari i Politecnic La Fe
Valencia, 46026
34644490025
Juan Francisco Vazquez Costa
Belgium1 site, 1 recruiting
- Recruiting
UZ Leuven
Leuven, 3000
(321) 634-4280
Philip Van Damme
Netherlands1 site, 1 recruiting
- Recruiting
Universitair Medisch Centrum Utrecht
Utrecht, 3584
31887553204
Leonard van den Berg
About this study
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
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)
- Being tested
- LY4256984, Placebo
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LY4256984Drug
Administered IT
PlaceboDrug
Administered IT
| Study group | Role | What they receive |
|---|---|---|
| LY4256984 (Cohorts 1-3) | Experimental | Multiple ascending doses (MAD) of LY4256984 administered intrathecally (IT) Receives: Drug: LY4256984 |
| Placebo | Placebo comparator | Administered IT Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
A summary of TEAEs, SAEs, and AEs regardless of causality, will be reported in the Reported Adverse Events module
Measured: Baseline up to Approximately Day 253
Secondary outcomes (3)
Pharmacokinetics (PK): Plasma Area Under the Concentration Versus Time Curve (AUC) of LY4256984
PK: Plasma AUC of LY4256984
Measured: Baseline up to Approximately Day 253
PK: Plasma Maximum Concentration (Cmax) of LY4256984
PK: Plasma Cmax of LY4256984
Measured: Baseline up to Approximately Day 253
PK: Cerebrospinal Fluid Concentrations (CSF) of LY4256984
PK: CSF Concentrations of LY4256984
Measured: Baseline up to Approximately Day 253
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Aug 2025
- Study start5 Aug 2025
- Primary completionAug 2027 (estimated)
- Study completionSep 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for ALS (Amyotrophic Lateral Sclerosis)Studies of the same condition, open and under way.
- More trials run by Eli Lilly and CompanyThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Basic science
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 36 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Eli Lilly and Company
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Call 1-877-CTLILLY (1-877-285-4559) or 1-317-615-4559 Mon - Fri 8 AM - 8 PM Eastern time (UTC/GMT - 5 hours, EST), Study director, Eli Lilly and Company
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07100119
- Sponsor's protocol ID
- 27427
- Other IDs
- J6I-MC-OWAA, 2025-521295-6, U1111-1321-6118, 2025
- Status last verified
- Nov 2026
- First submitted
- 31 Jul 2025
- Last updated
- 5 Nov 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Nov 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT07100119
- Acronym / sponsor ID
- 27427
- Conditions
- ALS (Amyotrophic Lateral Sclerosis)
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 36 participants (estimated)
- Lead sponsor
- Eli Lilly and CompanyIndustry (pharma, biotech, devices)
- Study start
- 5 Aug 2025 (actual)
- Primary completion
- Aug 2027 (estimated)
- Study completion
- Sep 2027 (estimated)
- First posted
- 3 Aug 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Basic science
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| LY4256984 (Cohorts 1-3) | Experimental | Drug: LY4256984Multiple ascending doses (MAD) of LY4256984 administered intrathecally (IT) |
| Placebo | Placebo comparator | Drug: PlaceboAdministered IT |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- 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)
Key exclusion criteria (6 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| LY4256984Administered IT | Drug | LY4256984 (Cohorts 1-3) |
| PlaceboAdministered IT | Drug | Placebo |
Primary outcome
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
A summary of TEAEs, SAEs, and AEs regardless of causality, will be reported in the Reported Adverse Events module
3 secondary outcomes
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Nov 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Trial questions or participation questions: 1-877-CTLILLY (1-877-285-4559) or contact
Physicians interested in becoming principal investigators please contact contact
Call 1-877-CTLILLY (1-877-285-4559) or 1-317-615-4559 Mon - Fri 8 AM - 8 PM Eastern time (UTC/GMT - 5 hours, EST) study director
Eli Lilly and Company
12 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing LY4256984 in people with ALS (Amyotrophic Lateral Sclerosis), run by Eli Lilly and Company at 12 sites in 5 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 5 Nov 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in Canada, Germany, Spain, Belgium, Netherlands. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Trial questions or participation questions: 1-877-CTLILLY (1-877-285-4559) or (1-317-615-4559, LillyTrials@Lilly.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the 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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Nov 2026; AzTrials last refreshed its copy on 8 Nov 2026.