Actively Recruiting
A Multicenter, Open-Label, Postmarketing Study of Tofersen (BIIB067) in Adults With SOD1-Associated Amyotrophic Lateral Sclerosis (ALS) in China
Led by Biogen · Updated on 2026-01-20
12
Participants Needed
1
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are studying the long-term safety of the drug tofersen, also known as Qalsody®, in adults with amyotrophic lateral sclerosis (ALS) caused by a mutation in the superoxide dismutase 1 (SOD1) gene. This postmarketing study aims to collect safety information in Chinese participants since tofersen was approved there in September 2024. The main focus is on counting how many participants experience adverse events or serious adverse events during the study. Researchers will also examine how the drug is processed in the body and how much is present in the cerebrospinal fluid surrounding the brain and spinal cord. Participants will receive up to 13 doses of tofersen over about 44 weeks. The first three doses, called loading doses, are 100 milligrams each and given about two weeks apart through injections into the fluid around the spine (intrathecal injection). Then, participants receive 10 more maintenance doses approximately every 28 days by the same method. This study includes a screening period of up to 4 weeks before treatment begins. During the study, participants will visit the research center up to 15 times over a total duration of about one year. Researchers will monitor safety by tracking any health problems or serious issues that occur. They will also perform tests to measure the drug levels in the blood and cerebrospinal fluid. The study will help better understand tofersen’s safety and how it behaves in the body over time in people with SOD1-ALS.
CONDITIONS
Brief 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 amyotrophic lateral sclerosis associated with a SOD1 gene mutation
- If taking riluzole, stable dose for at least 30 days prior to Day 1 and expected to remain stable through the study
- If taking edaravone, started treatment at least 60 days prior to Day 1 and expected to remain stable through the study unless medically discontinued
- Women of childbearing potential must use effective contraception during the study
You will not qualify if you...
- Allergy or hypersensitivity to tofersen or any ingredients in the injection
- Previous treatment with tofersen in any setting
- Prior or current treatment with small interfering RNA, stem cell therapy, or gene therapy
- Use of 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 period
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Duration - Up to 44 weeks
Participants receive tofersen 100 mg by intrathecal injection as 3 loading doses once every 2 weeks followed by 10 maintenance doses once every 4 weeks, for a maximum of 44 weeks.
13 dosing visits
Duration - Up to 8 weeks after treatment
Participants are monitored for safety and pharmacokinetics after treatment completion.
Visits as scheduled for safety assessments
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
G
Global Biogen Clinical Trial Center
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
Similar Trials
Frequently Asked Questions
Have more questions? Get in touch with our team for quick support
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here