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)

Condition(s) studied

Investigational drug(s) / intervention(s)

Tofersen: Administered as specified in the treatment arm.

Study 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.

Eligibility

Primary outcome measure(s)

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

Trial sites (1)

More Biogen trials in the USA

Other trials for the same condition