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)

Part of paid clinical trials in Boston, Massachusetts.

Sponsor
Biogen
Study ID
NCT07259980
Status
Recruiting
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Conditions

Eligibility Criteria

Sex
ALL
Age
18 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • Tofersen — DRUG
    Administered as specified in the treatment arm.

Study Details

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.

Key Dates

First listed
Dec 2, 2025
Start date
Jan 2, 2026
Status verified
Apr 2026
Primary completion
Dec 30, 2033
Completion
Dec 30, 2033

Study Design

Enrollment
125 participants (estimated)

Arms

  • Arm: Tofersen
    Data for participants with SOD1-ALS will be collected via ALS disease registries, the TRICALS network's Precision-ALS programme and ALS/MND NHC.

Primary Outcome Measure

Baseline Demographic: Age [ Time Frame: At Baseline ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
Mass General Hospital -MGHBostonMassachusetts02114
617-643-2935
Alex Sherman (PRINCIPAL_INVESTIGATOR)

Find similar trials in Boston, MA

By research site
Mass General Hospital -MGH· Boston, MA

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