An Observational Registry-Based Study to Evaluate the Long-Term Safety of Tofersen in People With SOD1-ALS

Study identification

• EU PAS number:
  EUPAS1000000365
• Study ID:
  1000000365
• Official title and acronym:
  An Observational Registry-Based Study to Evaluate the Long-Term Safety of Tofersen in People With SOD1-ALS
• DARWIN EU® study:
  No
• Study countries:
  Belgium

  Ireland

  Switzerland

  United States
• Study description:
  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.
• Study status:
  Planned

Research institutions and networks

Institutions

Biogen

Study timelines

Date when funding contract was signed

Planned:19/01/2026

Study start date

Planned:19/01/2026

Data analysis start date

Planned:30/01/2026

Date of final study report

Planned:30/07/2034

Sources of funding

Pharmaceutical company and other private sector 

More details on funding

Biogen-100%

Regulatory

• Was the study required by a regulatory body?:
  Yes
• Is the study required by a Risk Management Plan (RMP)?:
  EU RMP category 2 (specific obligation of marketing authorisation)