Long-term safety monitoring of tofersen (Qalsody) in people with SOD1 ALS
An Observational Registry-Based Study to Evaluate the Long-Term Safety of Tofersen in People With SOD1-ALS
This project will see how safe tofersen (Qalsody) is over time in people with ALS caused by SOD1 mutations by collecting health information without changing their care.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 125 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | Biogen Industry-sponsored |
| Locations | 1 site (Boston, Massachusetts) |
| Trial ID | NCT07259980 on ClinicalTrials.gov |
What this trial studies
This observational effort collects safety and clinical data on people with SOD1-ALS who are captured in two contributing registry networks (Precision-ALS in Europe and the ALS/Motor Neuron Disease Natural History Consortium in the United States). The study focuses on documenting serious adverse events, including neurologic events reported in prior tofersen trials (for example myelitis, radiculitis, aseptic meningitis, raised intracranial pressure, or papilledema), as well as new comorbidities, pregnancy outcomes, and treatment discontinuation. Data already began to accrue in the NHC registry while TRICALS collection is pending. Information from participating centers such as Mass General Hospital will be pooled without changing routine clinical care, and data collected during other interventional trials are excluded.
Who should consider this trial
Good fit: Ideal candidates are people with a confirmed SOD1 mutation and an ALS diagnosis who are followed in the contributing registries and who have taken or are taking tofersen.
Not a fit: People without SOD1 mutations, those not enrolled in the contributing registry networks, or whose data were collected while participating in other interventional trials are unlikely to benefit from this study's findings.
Why it matters
Potential benefit: If successful, the study could clarify long-term risks and rare serious neurologic events linked to tofersen and inform safer prescribing and monitoring for people with SOD1-ALS.
How similar studies have performed: Prior clinical trials of tofersen reported potential clinical effects alongside rare serious neurologic adverse events, and registry-based observational approaches have been used previously to monitor long-term safety of ALS therapies.
Eligibility criteria
Show full inclusion / exclusion criteria
Key Inclusion Criteria: * Participants with an ALS diagnosis and a confirmed SOD1 mutation from contributing registry networks will be considered for inclusion in the study. Key Exclusion Criteria: * Data collected while a person with SOD1-ALS is participating in an interventional clinical trial (with tofersen or any other investigational medicinal product) will be excluded. NOTE: Other protocol- defined Inclusion/Exclusion criteria may apply.
Where this trial is running
Boston, Massachusetts
- Mass General Hospital -MGH — Boston, Massachusetts, United States (Recruiting)
Study contacts
- Study coordinator: Study US Biogen Clinical Trial Center
- Email: clinicaltrials@biogen.com
- Phone: 866-633-4636
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.