Recruiting

Tofersen

Sponsor:

Biogen

Code:

NCT07259980

Conditions

Amyotrophic Lateral Sclerosis

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Interventions

Tofersen

Study Details

Brief summary:

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

Amyotrophic Lateral Sclerosis

Study ID

NCT07259980

Start date

Jan 2, 2026

Status verified date

Apr, 2026

Completion date

Dec 30, 2033

Anticipated

Primary completion date

Dec 30, 2033

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Key Inclusion Criteria:

  • Participants with an ALS diagnosis and a confirmed SOD1 mutation from contributing registry networks will be considered for inclusion in the study.

Key Exclusion Criteria:

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

NOTE: Other protocol- defined Inclusion/Exclusion criteria may apply.

Study Design

Enrollment

125 participants

Anticipated

Interventions and Outcome Measures

Arms

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.

Interventions

Tofersen

Administered as specified in the treatment arm.

Primary outcome measure

  • Baseline Demographic: Age [ Time Frame: At Baseline ]
  • Baseline Demographic: Participant Sex [ Time Frame: At Baseline ]
  • Baseline Demographic: Race/Ethnicity [ Time Frame: At Baseline ]
  • Baseline Demographic: Weight [ Time Frame: At Baseline ]
  • Baseline Demographic: Height [ Time Frame: At Baseline ]
  • Baseline Demographic: Body Mass Index (BMI) [ Time Frame: At Baseline ]
  • Baseline Demographic: Family History of Amyotrophic Lateral Sclerosis (ALS) [ Time Frame: At Baseline ]
  • Clinical Characteristics: Age at Diagnosis and Symptom Onset [ Time Frame: At Baseline ]
  • Clinical Characteristics: Revised El Escorial Classification [ Time Frame: At Baseline ]
  • Clinical Characteristics: Classification of SOD1-ALS Clinical Phenotypes [ Time Frame: At Baseline ]
  • Clinical Characteristics: SOD1 Mutation Type [ Time Frame: At Baseline ]
  • Clinical Characteristics: Medical History [ Time Frame: At Baseline ]
  • Clinical Characteristics: Concomitant Medications [ Time Frame: At Baseline ]
  • Clinical Characteristics: Disease History [ Time Frame: At Baseline ]
  • Clinical Characteristics: Pregnancy Status [ Time Frame: At Baseline ]
  • Number of Participants With Serious Adverse Events [ Time Frame: Up to 7 years ]

Central Contacts and Locations

Central contacts

Study US Biogen Clinical Trial Center

866-633-4636clinicaltrials@biogen.com

Global Biogen Clinical Trial Center

clinicaltrials@biogen.com

Locations

Mass General Hospital -MGH

Recruiting

Boston, Massachusetts, United States, 02114

Contacts

617-643-2935

Principal Investigator:

Alex Sherman

More Information

Sponsor

Biogen

Last update posted

Apr 9, 2026

Last verified

Apr, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Biogen on 2026-04-09.