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First in Human (FIH) Study of ALN-SOD in Adult Participants With Amyotrophic Lateral Sclerosis Associated With Mutation in the SOD1 Gene (SOD1-ALS)

NCT06351592 · Status: RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 42

Last updated 2026-02-27

No results posted yet for this study

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

Conditions

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

Interventions

DRUG

ALN-SOD

Administered per the protocol

OTHER

Diluent

Administered per the protocol

DRUG

Placebo (PB)

Administered per the protocol

Sponsors & Collaborators

Principal Investigators

  • Clinical Trial Management · Regeneron Pharmaceuticals

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-08-28
Primary Completion
2031-06-05
Completion
2031-06-05
FDA Drug
Yes

Countries

  • Australia
  • Belgium
  • Canada
  • Japan
  • South Korea
  • Taiwan

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06351592 on ClinicalTrials.gov