NCT06351592

Brief 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". 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

Trial Health

83
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
42

participants targeted

Target at P50-P75 for phase_1

Timeline
61mo left

Started Aug 2024

Longer than P75 for phase_1

Geographic Reach
6 countries

16 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress25%
Aug 2024Jun 2031

First Submitted

Initial submission to the registry

April 2, 2024

Completed
6 days until next milestone

First Posted

Study publicly available on registry

April 8, 2024

Completed
5 months until next milestone

Study Start

First participant enrolled

August 28, 2024

Completed
6.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 5, 2031

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

June 5, 2031

Last Updated

February 27, 2026

Status Verified

February 1, 2026

Enrollment Period

6.8 years

First QC Date

April 2, 2024

Last Update Submit

February 24, 2026

Conditions

Amyotrophic Lateral Sclerosis (ALS)Mutation in the Superoxide Dismutase-1 (SOD1) Gene

Keywords

SymptomaticKnown pathogenic mutationPredicted pathogenic mutation

Outcome Measures

Primary Outcomes (2)

  • Incidence of Treatment-Emergent Adverse Event (TEAEs) in participants treated with ALN-SOD

    At week 4 and through week 228

  • Severity of TEAEs in participants treated with ALN-SOD

    At week 4 and through week 228

Secondary Outcomes (11)

  • Concentration of Neurofilament Light chain (NfL) in plasma over time

    Up to approximately week 228

  • Change in concentration of NfL in plasma over time

    Up to approximately week 228

  • Concentration of SOD1 protein in Cerebrospinal Fluid (CSF) over time

    Up to approximately week 228

  • Change in concentration of SOD1 protein in CSF over time

    Up to approximately week 228

  • Concentration of NfL in CSF over time

    Up to approximately week 228

  • +6 more secondary outcomes

Study Arms (4)

Cohort 1 - Low

EXPERIMENTAL

Placebo during 4-Week double-blind treatment period

Drug: ALN-SODOther: DiluentDrug: Placebo (PB)

Cohort 2 - Mid-Dose

EXPERIMENTAL

Placebo during 4-Week double-blind treatment period

Drug: ALN-SODOther: DiluentDrug: Placebo (PB)

Cohort 3 - High-Dose

EXPERIMENTAL

Placebo during 4-Week double-blind treatment period

Drug: ALN-SODOther: DiluentDrug: Placebo (PB)

Cohort 4 (Optional) - ≤ High Dose

EXPERIMENTAL

Placebo during 4-Week double-blind treatment period

Drug: ALN-SODOther: DiluentDrug: Placebo (PB)

Interventions

ALN-SODDRUG

Administered per the protocol

Cohort 1 - LowCohort 2 - Mid-DoseCohort 3 - High-DoseCohort 4 (Optional) - ≤ High Dose
DiluentOTHER

Administered per the protocol

Cohort 1 - LowCohort 2 - Mid-DoseCohort 3 - High-DoseCohort 4 (Optional) - ≤ High Dose
Placebo (PB)DRUG

Administered per the protocol

Cohort 1 - LowCohort 2 - Mid-DoseCohort 3 - High-DoseCohort 4 (Optional) - ≤ High Dose

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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
  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (16)

Concord Repatriation General Hospital

Concord, New South Wales, 2139, Australia

RECRUITING

Macquarie University

Sydney, New South Wales, 2109, Australia

RECRUITING

Sunshine Coast University Hospital

Birtinya, Queensland, 4575, Australia

RECRUITING

KU Leuven

Leuven, Vlaams-Brabant, 3000, Belgium

RECRUITING

University of Alberta Hospital, Edmonton, Division of Neurology

Edmonton, Alberta, T6G 2G3, Canada

RECRUITING

University Hospital - London Health Sciences Centre

London, Ontario, N6A 5A5, Canada

RECRUITING

Sunnybrook Research Institute

Toronto, Ontario, M4N 3M5, Canada

RECRUITING

Montreal Neurological Institute and Hospital

Montreal, Quebec, H3A 2B4, Canada

RECRUITING

Hokkaido University Hospital

Sapporo, Hokkaido, 060-8648, Japan

RECRUITING

Tokushima University Hospital

Tokushima, Tokushima, 770-0042, Japan

RECRUITING

Toho University Omori Medical Center

Ōta-ku, Tokyo, 143-8541, Japan

RECRUITING

Kyoto University Hospital

Kyoto, 606-8507, Japan

RECRUITING

Hanyang University Seoul Hospital

Seoul, 04763, South Korea

RECRUITING

Seoul National University Hospital

Seoul, 3080, South Korea

RECRUITING

Kaohsiung Chang Gung Memorial Hospital

Kaohsiung City, 83301, Taiwan

RECRUITING

Taipei Veterans General Hospital

Taipei, 11211, Taiwan

RECRUITING

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Condition Hierarchy (Ancestors)

Spinal Cord DiseasesCentral Nervous System DiseasesNervous System DiseasesMotor Neuron DiseaseNeurodegenerative DiseasesTDP-43 ProteinopathiesNeuromuscular DiseasesProteostasis DeficienciesMetabolic DiseasesNutritional and Metabolic Diseases

Study Officials

  • Clinical Trial Management

    Regeneron Pharmaceuticals

    STUDY DIRECTOR

Central Study Contacts

Clinical Trials Administrator

CONTACT

844-734-6643clinicaltrials@regeneron.com

Study Design

Study Type
interventional
Phase
phase 1
Allocation
RANDOMIZED
Masking
NONE
Masking Details
Phase 1b: Dose Escalation - Double-blind, randomized, and single crossover Phase 2: Cohort Expansion - Open-label
Purpose
TREATMENT
Intervention Model
SEQUENTIAL
Model Details: Phase 1b: Dose Escalation Phase 2: Cohort Expansion
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

April 2, 2024

First Posted

April 8, 2024

Study Start

August 28, 2024

Primary Completion (Estimated)

June 5, 2031

Study Completion (Estimated)

June 5, 2031

Last Updated

February 27, 2026

Record last verified: 2026-02

Data Sharing

IPD Sharing
Will share

All Individual Patient Data (IPD) that underlie publicly available results will be considered for sharing.

Shared Documents
STUDY PROTOCOL, SAP, ICF, CSR, ANALYTIC CODE
Time Frame
When Regeneron has: * received marketing authorization from major health authorities (e.g., FDA, European Medicines Agency (EMA), Pharmaceuticals and Medical Devices Agency (PMDA), etc.) for the product and indication or has globally discontinued development of the product for all indications on or after April 2020 and has no plans for future development * made the study results publicly available (e.g., scientific publication, scientific conference, clinical trial registry) * the legal authority to share the data, and * ensured the ability to protect participant privacy
Access Criteria
Qualified researchers can submit a proposal for access to individual patient or aggregate level data from a Regeneron-sponsored clinical trial through Vivli. Regeneron's Independent Research Request Evaluation Criteria can be found at: https://www.regeneron.com/sites/default/files/Regeneron-External-Data-Sharing-Policy-and-Independent-Research-Request-Evaluation-Criteria.pdf
More information

Locations

BE(1)CA(4)KR(2)TW(2)JP(4)AU(3)