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

Studying BIIB067 in adults with early signs of SOD1 mutation.

Recruiting
18 years and older
All
Phase 3

This study tests a drug called **Tofersen** for adults who have a genetic change called **Superoxide Dismutase 1 (SOD1) mutation**. A mutation is a change in a gene. People with this mutation can develop a disease called **amyotrophic lateral sclerosis (ALS)**, which affects muscles. The study will check if Tofersen helps before any symptoms appear. **Neurofilament (NF)** is a protein that can be high in people with nerve damage, and this study checks its levels too.

To join, participants should have a specific SOD1 mutation and a low NF level. They should not have symptoms of ALS.

  • **Length**: The study involves multiple visits but exact length isn't mentioned.
  • **Risks**: Participants must not have certain infections or be on certain medications.
  • **Compensation**: Not mentioned.

Before joining, participants should ensure they fit the criteria and understand the visit schedule and any risks involved. The study will look at Tofersen's safety, how well people tolerate it, and its effects.

Study details
    Amyotrophic Lateral Sclerosis Associated With a SOD1 Gene Mutation

NCT04856982

Biogen

13 December 2024

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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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