Long-term safety of Qalsody (tofersen) for Chinese people with SOD1 ALS

A Multicenter, Open-Label, Postmarketing Surveillance Study of Tofersen (BIIB067) in Adults With Amyotrophic Lateral Sclerosis (ALS) Associated With a Mutation in the Superoxide Dismutase 1 (SOD1) Gene in China

This will follow Chinese adults with SOD1‑ALS who take Qalsody (tofersen) to see if the medicine is safe over the long term.

Quick facts

• Phase: Phase 4
• Study type: Interventional
• Enrollment: 12 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Biogen (Industry-sponsored)
• Locations: 1 site (Beijing, Beijing Municipality)
• Trial ID: NCT07223723 on ClinicalTrials.gov

What this trial studies

This is a postmarketing, Phase 4 study enrolling adults in China with SOD1‑mutation ALS to collect long‑term safety and pharmacokinetic data on tofersen (Qalsody). Participants who have not previously received tofersen will receive the drug and be monitored for adverse events, serious adverse events, and blood/CSF drug levels over scheduled clinic visits. Key exclusions include prior tofersen use, hypersensitivity to the drug, and recent or current treatment with certain gene‑targeted or stem‑cell therapies. The study is sponsored by Biogen and conducted at Peking University Third Hospital in Beijing.

Who should consider this trial

Good fit: Adults (age ≥18) with a confirmed SOD1‑ALS diagnosis who have not previously received tofersen and who can comply with study visits (including contraception requirements for women of childbearing potential) are ideal candidates.

Not a fit: People who already received tofersen, have a known hypersensitivity to the drug, or have recently received conflicting gene‑targeted or stem‑cell therapies are unlikely to benefit from joining this safety study.

Why it matters

Potential benefit: If tofersen is shown to be safe in this population, the results could reassure doctors and patients about long‑term use and help guide treatment decisions for Chinese people with SOD1 ALS.

How similar studies have performed: Previous clinical trials and open‑label extensions of tofersen have demonstrated target engagement and provided safety and biomarker data, though long‑term safety specifically in Chinese patients has not been reported.

Eligibility criteria

Key Inclusion Criteria:

* Age ≥ 18 years at the time of informed consent.
* Must have diagnosis of SOD1-ALS.
* If taking riluzole, participant must be on a stable dose for ≥ 30 days prior to Day 1 and expected to remain at that dose until the final study visit.
* If taking edaravone, participant must have initiated edaravone ≥ 60 days (2 treatment cycles) prior to Day 1 and expected to remain at that dose until the final study visit, unless the Investigator determines that edaravone should be discontinued for medical reasons, in which case it may not be restarted during the study.
* All women of childbearing potential must practice effective contraception during the study.

Key Exclusion Criteria:

* Hypersensitivity to the active substance or any of the excipients of tofersen injection.
* Current or past administration of tofersen injection, in either a commercial or a clinical study setting.
* Prior or current treatment with small interfering ribonucleic acid (RNA), stem cell therapy, or gene therapy.
* Treatment with another investigational drug, biological agent, or device within 1 month or 5 half-lives of study agent, whichever is longer, prior the Baseline Visit.
* Participants who are pregnant or currently breastfeeding, and those intending to become pregnant during the study.

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

Where this trial is running

Beijing, Beijing Municipality

• Peking University Third Hospital — Beijing, Beijing Municipality, China (Recruiting)

Study contacts

• Study coordinator: US Biogen Clinical Trial Center

Conditions Amyotrophic Lateral Sclerosis