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A Study of BIIB067 (Tofersen) Initiated in Clinically Presymptomatic Adults With a Confirmed Superoxide Dismutase 1 Mutation
NCT04856982 · View on ClinicalTrials.gov ↗
Study Summary
The primary objective of this study is to evaluate the efficacy of tofersen in presymptomatic adult carriers of a superoxide dismutase 1 (SOD1) mutation with elevated neurofilament (NF). The secondary objectives of this study are to evaluate the safety and tolerability tofersen and to evaluate the effect of tofersen on pharmacodynamics (PD)/treatment response biomarkers when initiated prior to versus at the time of emergence of clinically manifest amyotrophic lateral sclerosis (ALS).
Conditions Studied
Interventions
- DRUG Placebo
- DRUG Tofersen
Study Locations (20)
California
- University of California San Diego Medical Center — La Jolla
- California Pacific Medical Center Research Institute — San Francisco
Florida
- Holy Cross Hospital Phil Smith Neuroscience Institute — Fort Lauderdale
- University of Miami School of Medicine — Miami
Arizona
- HonorHealth Neurology — Scottsdale
Georgia
- The Emory Clinic — Atlanta
Illinois
- Northwestern Medicine — Chicago
Maryland
- Johns Hopkins Hospital — Baltimore
Massachusetts
- Massachusetts General Hospital — Charlestown
Missouri
- Washington University School of Medicine — St Louis
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 158 participants |
| Start Date | 2021-05-17 |
| Est. Completion | 2027-08-07 |
| Phase | Phase 3 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT04856982
The ClinicalTrials.gov registry entry for NCT04856982 describes a study currently listed as active not recruiting. It is categorized as Phase 3, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 158 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Biogen, which has 74 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 1 condition, with Amyotrophic Lateral Sclerosis Associated With a SOD1 Gene Mutation appearing as the primary indexed condition, and to 2 interventions — of which Placebo is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT04856982 reports 20 study locations spanning 18 distinct geographic areas — top geographies include California, Florida, Arizona. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT04856982 about?
NCT04856982 is a clinical study titled "A Study of BIIB067 (Tofersen) Initiated in Clinically Presymptomatic Adults With a Confirmed Superoxide Dismutase 1 Mutation". The primary objective of this study is to evaluate the efficacy of tofersen in presymptomatic adult carriers of a superoxide dismutase 1 (SOD1) mutation with elevated neurofilament (NF). The secondary objectives of this study are to evaluate the safety and tolerability tofersen and to evaluate the e...
What is the current status of trial NCT04856982?
This trial is currently active not recruiting. It is a Phase 3 study. The enrollment target is 158 participants. The study started on 2021-05-17. Estimated completion is 2027-08-07.
What conditions does trial NCT04856982 study?
This clinical trial studies the following conditions: Amyotrophic Lateral Sclerosis Associated With a SOD1 Gene Mutation. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT04856982?
The interventions under investigation include: Placebo (DRUG), Tofersen (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT04856982?
This trial is sponsored by Biogen, which has 74 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT04856982 being conducted?
This trial has 20 study locations across Arizona, California, Florida, Georgia, Illinois. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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