Actively Recruiting
A Study to Learn More About the Long-Term Safety of Tofersen (Qalsody) in Chinese Participants With SOD-1 Amyotrophic Lateral Sclerosis (ALS)
Led by Biogen · Updated on 2026-01-20
12
Participants Needed
1
Research Sites
105 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 people with a certain type of amyotrophic lateral sclerosis, also known as ALS. This type is in people who have a mutation in the superoxide dismutase 1 gene, also known as SOD-1. This is known as a "postmarketing" study. In this kind of the study, the goal is to learn more about how a drug works after it has been approved for use in the general public. Tofersen was approved in China in September 2024. The main goal of this study is to collect long-term safety information in Chinese participants with SOD-1 ALS. The main question researchers want to answer in this study is: • How many participants have adverse events (AEs) and serious adverse events (SAEs)? An AE is a health problem that may or may not be caused by a drug during the study. An AE 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 the body processes tofersen. * How much tofersen is found in the cerebrospinal fluid (CSF), or the fluid that surrounds the brain and the spinal cord. This study will be done as follows: * Participants will be screened to check if they can join the study. The screening period will be up to 4 weeks. * After joining the study, participants will receive the first 3 doses of 100 milligrams (mg) of tofersen about 14 days apart. This will be given through an intrathecal (IT) injection. This means it will be given into the fluid surrounding the spine. * After that, participants will receive 10 more doses every 28 days through IT injections. Participants will have up to 13 total doses of tofersen in this study. * Participants will have up to 15 visits to their study research center. Each participant will be in the study for up to 52 weeks (1 year).
CONDITIONS
Official Title
A Study to Learn More About the Long-Term Safety of Tofersen (Qalsody) in Chinese Participants With SOD-1 Amyotrophic Lateral Sclerosis (ALS)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 18 years or older at the time of informed consent
- Diagnosis of SOD1-associated amyotrophic lateral sclerosis (ALS)
- Stable dose of riluzole for at least 30 days prior to Day 1 if taking riluzole
- Started edaravone treatment at least 60 days before Day 1 if taking edaravone
- Women of childbearing potential must use effective contraception during the study
You will not qualify if you...
- Allergy or hypersensitivity to tofersen or its ingredients
- Previous use of tofersen injection commercially or in a clinical study
- Prior or current treatment with small interfering RNA, stem cell therapy, or gene therapy
- Treatment with another investigational drug, biological agent, or device within 1 month or 5 half-lives before baseline visit
- Pregnant, breastfeeding, or planning pregnancy during the study
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Peking University Third Hospital
Beijing, Beijing Municipality, China, 100083
Actively Recruiting
Research Team
U
US Biogen Clinical Trial Center
CONTACT
G
Global Biogen Clinical Trial Center
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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