Amyotrophic Lateral Sclerosis (ALS)Phase 1/2GOALSNCT06351592
First in Human (FIH) Study of ALN-SOD in Adult Participants With Amyotrophic Lateral Sclerosis Associated With Mutation in the SOD1 Gene (SOD1-ALS)
A Phase 1/2 study testing ALN-SOD and Diluent in people with Amyotrophic Lateral Sclerosis (ALS) and Mutation in the Superoxide Dismutase-1 (SOD1) Gene, run by Regeneron Pharmaceuticals at 22 sites in 9 countries.
Trial summary
This study is researching an experimental drug called ALN-SOD (called "study drug"). This study is focused on people with Amyotrophic Lateral Sclerosis (ALS) caused by a change in a gene called the Superoxide Dismutase-1 (SOD1) gene. This type of ALS is known as "SOD1-ALS". Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Amyotrophic Lateral Sclerosis (ALS), Mutation in the Superoxide Dismutase-1 (SOD1) Gene
- What is being tested?
- ALN-SOD and Diluent
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 54 participants
- Where
- 22 sitesin 9 countries
- Study dates
- 28 Aug 2024to 21 Mar 2032
- Phase
- Phase 1/2
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Weakness attributable to ALS and a SOD1 variant that has been previously described as associated with ALS or is considered likely to cause ALS, as defined in the protocol
- Slow Vital Capacity (SVC) ≥50% predicted value based on age, gender and height, measured in upright position
- Body Mass Index (BMI) ≤35 kg/m2 at time of screening
- If participants are taking riluzole or edaravone, they must be on a stable dose for at least 4 weeks prior to initial dosing visit and are expected to remain at that dose until the end of the study
- Platelet count >50,000/microliter
- Has normal blood pressure readings, as defined in the protocol
Who cannot join
- Concurrent participation in another interventional clinical trial
- Has had a tracheostomy
- Has dementia, as assessed by the investigator
- Has uncontrolled psychiatric disease, including psychosis, active or recent suicidal ideation, untreated major depression, in the past 30 days
- Has a medical history of brain or spinal disease/injury that would interfere with the Lumbar Puncture (LP) process, CSF circulation or safety assessment, as defined in the protocol
- Presence of an implanted shunt for the drainage of CSF or an implanted Central Nervous System (CNS) catheter
Show all 10Show fewer
- Presents any concern to the study investigator that might confound the results of the study or poses an additional risk to the participant by their participation in the study
- Was hospitalized (ie, >24 hours) for any reason other than ALS within 30 days of screening
- Has received treatment with tofersen within 6 months prior to screening
- NOTE: Other protocol defined inclusion / exclusion criteria apply
Trial locations and contacts
22 sites in 9 countries. Contact the study team or a site near you to ask about taking part.
22 sites in 9 countries
Canada4 sites, 4 recruiting
- Recruiting
University of Alberta Hospital, Edmonton, Division of Neurology
Edmonton, Alberta, T6G 2G3
- Recruiting
University Hospital - London Health Sciences Centre
London, Ontario, N6A 5A5
- Recruiting
Montreal Neurological Institute and Hospital
Montreal, Quebec, H3A 2B4
- Recruiting
Sunnybrook Research Institute
Toronto, Ontario, M4N 3M5
Japan4 sites, 4 recruiting
- Recruiting
Kyoto University Hospital
Kyoto, 606-8507
- Recruiting
Toho University Omori Medical Center
Ōta-ku, Tokyo, 143-8541
- Recruiting
Hokkaido University Hospital
Sapporo, Hokkaido, 060-8648
- Recruiting
Tokushima University Hospital
Tokushima, Tokushima, 770-0042
Australia3 sites, 3 recruiting
- Recruiting
Sunshine Coast University Hospital
Birtinya, Queensland, 4575
- Recruiting
Concord Repatriation General Hospital
Concord, New South Wales, 2139
- Recruiting
Macquarie University
Sydney, New South Wales, 2109
Poland3 sites, 3 recruiting
- Recruiting
Szpital Uniwersytecki w Krakowie, Oddzial Kliniczny Neurologii
Krakow, Lesser Poland Voivodeship, 30-688
- Recruiting
MTZ Clinical Research powered by Pratia
Warsaw, 02-172
- Recruiting
Medical University of Silesia
Zabrze, Silesian Voivodeship, 41-800
South Korea2 sites, 2 recruiting
- Recruiting
Hanyang University Seoul Hospital
Seoul, 04763
- Recruiting
Seoul National University Hospital
Seoul, 3080
Sweden2 sites, 2 recruiting
- Recruiting
Karolinska University Hospital, Karolinska Institutet-ALS Center-Department of Clinical Neuroscience
Huddinge, Stockholm County, 141 57
- Recruiting
Noorlands University Hospital-Department of Neurology
Umeå, Noorland, 907 37
Taiwan2 sites, 2 recruiting
- Recruiting
Kaohsiung Chang Gung Memorial Hospital
Kaohsiung City, 83301
- Recruiting
Taipei Veterans General Hospital
Taipei, 11211
Germany1 site, 1 recruiting
- Recruiting
Universitaetsklinikum Ulm AoR
Ulm, Baden-Wurttemberg, 89081
About this study
Official title: First in Human Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple Ascending Doses of Intrathecally Administered ALN-SOD in Participants With Amyotrophic Lateral Sclerosis and SOD1 Mutations
This study is researching an experimental drug called ALN-SOD (called "study drug"). This study is focused on people with Amyotrophic Lateral Sclerosis (ALS) caused by a change in a gene called the Superoxide Dismutase-1 (SOD1) gene. This type of ALS is known as "SOD1-ALS". This is the first time that ALN-SOD will be given to people.
The aim of the study is to see how safe and tolerable the study drug is.
Read more
The study is looking at several other research questions, including:
- The effect the study drug has on specific biomarkers, which are substances in the blood or in the fluid that surrounds the brain and spinal cord, known as Cerebrospinal Fluid (CSF)
- How much study drug is in the blood and in the CSF, at different times
- Whether the body makes antibodies against the study drug (which could make the drug less effective or could lead to side effects)
- What effects the study drug has on ALS symptoms
- Being tested
- ALN-SOD, Diluent, Placebo (PB)
- Keywords
- Symptomatic, Known pathogenic mutation, Predicted pathogenic mutation
- 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.
ALN-SODDrug
Administered per the protocol
DiluentOther
Administered per the protocol
Placebo (PB)Drug
Administered per the protocol
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 - Dose Level 1 | Experimental | Placebo during 4-Week double-blind treatment period Receives: Drug: ALN-SOD; Other: Diluent; Drug: Placebo (PB) |
| Cohort 2 - Dose Level 2 | Experimental | Placebo during 4-Week double-blind treatment period Receives: Drug: ALN-SOD; Other: Diluent; Drug: Placebo (PB) |
| Cohort 3 - Dose Level 3 | Experimental | Placebo during 4-Week double-blind treatment period Receives: Drug: ALN-SOD; Other: Diluent; Drug: Placebo (PB) |
| Cohort 4 - Dose Level 4 | Experimental | Placebo during 4-Week double-blind treatment period Receives: Drug: ALN-SOD; Other: Diluent; Drug: Placebo (PB) |
| Cohort 5 (Optional) - ≤ Dose Level 5 | Experimental | Placebo during 4-Week double-blind treatment period Receives: Drug: ALN-SOD; Other: Diluent; Drug: Placebo (PB) |
| Selected Dose Expansion Cohort(s) | Experimental | Open Label Treatment (OLT) at selected dose levels. EU Participants can only be included in the Dose Expansion portion of study (Phase 2), not in Dose Escalation (Phase 1) Receives: Drug: ALN-SOD |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Treatment-Emergent Adverse Event (TEAEs) in participants treated with ALN-SOD
Measured: At week 4 and through week 228
Severity of TEAEs in participants treated with ALN-SOD
Measured: At week 4 and through week 228
Secondary outcomes (11)
Concentration of Neurofilament Light chain (NfL) in plasma over time
Measured: Up to approximately week 228
Change in concentration of NfL in plasma over time
Measured: Up to approximately week 228
Concentration of SOD1 protein in Cerebrospinal Fluid (CSF) over time
Measured: Up to approximately week 228
Change in concentration of SOD1 protein in CSF over time
Measured: Up to approximately week 228
Concentration of NfL in CSF over time
Measured: Up to approximately week 228
Change in concentration of NfL in CSF over time
Measured: Up to approximately week 228
Change in Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) over time
Measured: Up to approximately week 228
Concentration of ALN-SOD in plasma over time
Measured: Up to approximately week 228
Concentration of ALN-SOD in CSF over time
Measured: Up to approximately week 228
Incidence of Anti-Drug Antibodies (ADAs) to ALN-SOD in serum over time
Measured: Up to approximately week 228
Titer of ADAs to ALN-SOD in serum over time
Measured: Up to approximately week 228
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Apr 2024
- Study start28 Aug 2024
- Primary completion21 Mar 2032 (estimated)
- Study completion21 Mar 2032 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyotrophic Lateral Sclerosis (ALS)Studies of the same condition, open and under way.
- More trials run by Regeneron PharmaceuticalsThe 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 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Model description
- Phase 1b: Dose Escalation Phase 2: Cohort Expansion
- Masking
- None (open label)
- Enrolment
- 54 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Regeneron Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Trial Management, Study director, Regeneron Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06351592
- Sponsor's protocol ID
- ALN-SOD-ALS-2351
- Other IDs
- 2023-510344-20-00, 2023
- Acronym
- GOALS
- Status last verified
- Sep 2026
- First submitted
- 2 Apr 2024
- Last updated
- 11 Sep 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: All Individual Patient Data (IPD) that underlie publicly available results will be considered for sharing.
Study at a glance
From the registry record, last updated by the sponsor on 11 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- First in Human Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple Ascending Doses of Intrathecally Administered ALN-SOD in Participants With Amyotrophic Lateral Sclerosis and SOD1 Mutations
- ClinicalTrials.gov ID
- NCT06351592
- Acronym / sponsor ID
- GOALS / ALN-SOD-ALS-2351
- Keywords
- Symptomatic, Known pathogenic mutation, Predicted pathogenic mutation
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 54 participants (estimated)
- Lead sponsor
- Regeneron PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 28 Aug 2024 (actual)
- Primary completion
- 21 Mar 2032 (estimated)
- Study completion
- 21 Mar 2032 (estimated)
- First posted
- 8 Apr 2024 (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.
This study is researching an experimental drug called ALN-SOD (called "study drug"). This study is focused on people with Amyotrophic Lateral Sclerosis (ALS) caused by a change in a gene called the Superoxide Dismutase-1 (SOD1) gene. This type of ALS is known as "SOD1-ALS". This is the first time that ALN-SOD will be given to people.
The aim of the study is to see how safe and tolerable the study drug is.
The study is looking at several other research questions, including:
- The effect the study drug has on specific biomarkers, which are substances in the blood or in the fluid that surrounds the brain and spinal cord, known as Cerebrospinal Fluid (CSF)
- How much study drug is in the blood and in the CSF, at different times
- Whether the body makes antibodies against the study drug (which could make the drug less effective or could lead to side effects)
- What effects the study drug has on ALS symptoms
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 6
- Model description
- Phase 1b: Dose Escalation Phase 2: Cohort Expansion
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 - Dose Level 1 | Experimental | Drug: ALN-SOD, Other: Diluent, Drug: Placebo (PB)Placebo during 4-Week double-blind treatment period |
| Cohort 2 - Dose Level 2 | Experimental | Drug: ALN-SOD, Other: Diluent, Drug: Placebo (PB)Placebo during 4-Week double-blind treatment period |
| Cohort 3 - Dose Level 3 | Experimental | Drug: ALN-SOD, Other: Diluent, Drug: Placebo (PB)Placebo during 4-Week double-blind treatment period |
| Cohort 4 - Dose Level 4 | Experimental | Drug: ALN-SOD, Other: Diluent, Drug: Placebo (PB)Placebo during 4-Week double-blind treatment period |
| Cohort 5 (Optional) - ≤ Dose Level 5 | Experimental | Drug: ALN-SOD, Other: Diluent, Drug: Placebo (PB)Placebo during 4-Week double-blind treatment period |
| Selected Dose Expansion Cohort(s) | Experimental | Drug: ALN-SODOpen Label Treatment (OLT) at selected dose levels. EU Participants can only be included in the Dose Expansion portion of study (Phase 2), not in Dose Escalation (Phase 1) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Weakness attributable to ALS and a SOD1 variant that has been previously described as associated with ALS or is considered likely to cause ALS, as defined in the protocol
- Slow Vital Capacity (SVC) ≥50% predicted value based on age, gender and height, measured in upright position
- Body Mass Index (BMI) ≤35 kg/m2 at time of screening
- If participants are taking riluzole or edaravone, they must be on a stable dose for at least 4 weeks prior to initial dosing visit and are expected to remain at that dose until the end of the study
- Platelet count >50,000/microliter
Key exclusion criteria (10 listed)
- Concurrent participation in another interventional clinical trial
- Has had a tracheostomy
- Has dementia, as assessed by the investigator
- Has uncontrolled psychiatric disease, including psychosis, active or recent suicidal ideation, untreated major depression, in the past 30 days
- Has a medical history of brain or spinal disease/injury that would interfere with the Lumbar Puncture (LP) process, CSF circulation or safety assessment, as defined in the protocol
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 |
|---|---|---|
| ALN-SODAdministered per the protocol | Drug | Cohort 1 - Dose Level 1, Cohort 2 - Dose Level 2, Cohort 3 - Dose Level 3, Cohort 4 - Dose Level 4, Cohort 5 (Optional) - ≤ Dose Level 5, Selected Dose Expansion Cohort(s) |
| DiluentAdministered per the protocol | Other | Cohort 1 - Dose Level 1, Cohort 2 - Dose Level 2, Cohort 3 - Dose Level 3, Cohort 4 - Dose Level 4, Cohort 5 (Optional) - ≤ Dose Level 5 |
| Placebo (PB)Administered per the protocol | Drug | Cohort 1 - Dose Level 1, Cohort 2 - Dose Level 2, Cohort 3 - Dose Level 3, Cohort 4 - Dose Level 4, Cohort 5 (Optional) - ≤ Dose Level 5 |
Primary outcome
Incidence of Treatment-Emergent Adverse Event (TEAEs) in participants treated with ALN-SOD
Time frame: At week 4 and through week 228
Severity of TEAEs in participants treated with ALN-SOD
Time frame: At week 4 and through week 228
11 secondary outcomes
Concentration of Neurofilament Light chain (NfL) in plasma over time
Time frame: Up to approximately week 228
Change in concentration of NfL in plasma over time
Time frame: Up to approximately week 228
Concentration of SOD1 protein in Cerebrospinal Fluid (CSF) over time
Time frame: Up to approximately week 228
Change in concentration of SOD1 protein in CSF over time
Time frame: Up to approximately week 228
Concentration of NfL in CSF over time
Time frame: Up to approximately week 228
Change in concentration of NfL in CSF over time
Time frame: Up to approximately week 228
Change in Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) over time
Time frame: Up to approximately week 228
Concentration of ALN-SOD in plasma over time
Time frame: Up to approximately week 228
Concentration of ALN-SOD in CSF over time
Time frame: Up to approximately week 228
Incidence of Anti-Drug Antibodies (ADAs) to ALN-SOD in serum over time
Time frame: Up to approximately week 228
Titer of ADAs to ALN-SOD in serum over time
Time frame: Up to approximately week 228
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 11 Sep 2026.
- Individual participant dataSharing planned: Yes. All Individual Patient Data (IPD) that underlie publicly available results will be considered for sharing.
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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.
Clinical Trial Management study director
Regeneron Pharmaceuticals
22 sites in 9 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing ALN-SOD and Diluent in people with Amyotrophic Lateral Sclerosis (ALS) and Mutation in the Superoxide Dismutase-1 (SOD1) Gene, run by Regeneron Pharmaceuticals at 22 sites in 9 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 6 inclusion and 10 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 11 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 22 sites in Canada, Japan, Australia, Poland, South Korea and 4 more countries. 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined 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.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 Clinical Trials Administrator (844-734-6643, clinicaltrials@regeneron.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of Treatment-Emergent Adverse Event (TEAEs) in participants treated with ALN-SOD (time frame: At week 4 and through week 228). The study lists 2 primary outcomes in all. 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 11 Sep 2026.