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A Study of BIIB067 (Tofersen) Initiated in Clinically Presymptomatic Adults With a Confirmed Superoxide Dismutase 1 Mutation

Research on BIIB067 for adults with a specific genetic mutation.

Recruiting
18 years and older
All
Phase 3

This study is for adults who are carriers of a gene change called *Superoxide Dismutase 1 (SOD1)* mutation, which can lead to ALS, a disease that affects nerves and muscles. The study tests a medicine called *tofersen* to see if it works and is safe when given before symptoms of ALS start. It checks if starting tofersen early helps control the disease better compared to starting it when symptoms appear.

  • **Study Length:** Participants will need to be part of the study for a period determined by the research team.
  • **Visits:** Regular visits to the study site are required to monitor health and effects of the medicine.
  • **Risks:** Participants should be aware of potential side effects and need to discuss them with the study team.

To join, one must not show ALS symptoms and have no history of HIV, Hepatitis B, or C. Also, they must not have certain other health conditions or take specific medications. The study excludes those with certain medical histories or who are on certain drugs. Participants should discuss with their doctor if they meet the criteria and if the study is suitable for them.

Study details
    Amyotrophic Lateral Sclerosis Associated With a SOD1 Gene Mutation

NCT04856982

Biogen

13 December 2024

Step 1 Get in touch with the nearest study center
What happens next?
  • You can expect the study team to contact you via email or phone in the next few days.
  • Sign up as volunteer  to help accelerate the development of new treatments and to get notified about similar trials.

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Learn more about clinical trials

What is a clinical trial?

A clinical trial is a study designed to test specific interventions or treatments' effectiveness and safety, paving the way for new, innovative healthcare solutions.

Why should I take part in a clinical trial?

Participating in a clinical trial provides early access to potentially effective treatments and directly contributes to the healthcare advancements that benefit us all.

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The duration of clinical trials varies. Some trials last weeks, some years, depending on the phase and intention of the trial.

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Compensation varies per trial. Some offer payment or reimbursement for time and travel, while others may not.

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Clinical trials follow strict ethical guidelines and protocols to safeguard participants' health. They are closely monitored and safety reviewed regularly.
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