Actively Recruiting

Age: 18Years +
All Genders
NCT07259980

A Study to Learn More About the Long-Term Safety of Tofersen (Qalsody) in Participants With Superoxide Dismutase 1 (SOD-1) Amyotrophic Lateral Sclerosis (ALS)

Led by Biogen · Updated on 2026-04-09

125

Participants Needed

1

Research Sites

417 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

In this study, researchers will learn more about the safety of tofersen, also known as Qalsody®. This is a drug available for doctors to prescribe for participant with a certain type of amyotrophic lateral sclerosis, also known as ALS. This type is in participant who have a mutation in the superoxide dismutase 1 gene, also known as SOD-1. This is known as an "observational" study, which collects health information about study participants without changing their medical care. Participants for this study will be found using 2 different groups of study research centers that help provide clinical care for participant with ALS. These groups are in Europe and the United States and are called: * the Precision-ALS programme * the ALS/Motor Neuron Disease (MND) Natural History Consortium (NHC) The main goal of this study is to collect safety information in participants with SOD-1 ALS who were in either of the groups. The main question researchers want to answer in this study is: * What are the characteristics of the participants in this study? * How many participants had serious adverse events (SAEs), including ones that affect the brain, spinal cord, or nerves? An adverse event is a health problem that may or may not be caused by a drug during the study. An adverse event is considered serious when it results in death, is life-threatening, causes lasting problems, or requires hospital care. Researchers will also learn more about: * How many participants develop other health conditions or become pregnant, including how the pregnancy turned out * Why and when participants stopped treatment This study will be done as follows: * Participants will be screened to check if they can join the study. * Data from the participants' regular visits to their clinic will be collected based on which study research center they are in. * Each participant will be in the study until they decide to leave or until death. Currently, the study is planned to last at least 7 years.

CONDITIONS

Official Title

A Study to Learn More About the Long-Term Safety of Tofersen (Qalsody) in Participants With Superoxide Dismutase 1 (SOD-1) Amyotrophic Lateral Sclerosis (ALS)

Who Can Participate

Age: 18Years +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Participants must have an ALS diagnosis and a confirmed SOD1 mutation from contributing registry networks.
Not Eligible

You will not qualify if you...

  • Data collected while a person with SOD1-ALS is participating in an interventional clinical trial with tofersen or any other investigational drug will be excluded.

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 1 location

1

Mass General Hospital -MGH

Boston, Massachusetts, United States, 02114

Actively Recruiting

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Research Team

S

Study US Biogen Clinical Trial Center

CONTACT

G

Global Biogen Clinical Trial Center

CONTACT

How is the study designed?

Study Type

OBSERVATIONAL

Masking

N/A

Allocation

N/A

Model

N/A

Primary Purpose

N/A

Number of Arms

1

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