A Study of BIIB067 (Tofersen) Initiated in Clinically Presymptomatic Adults With a Confirmed Superoxide Dismutase 1 Mutation
Public ClinicalTrials.gov record NCT04856982. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
A Phase 3 Randomized, Placebo-Controlled Trial With a Longitudinal Natural History Run-In and Open-Label Extension to Evaluate BIIB067 Initiated in Clinically Presymptomatic Adults With a Confirmed Superoxide Dismutase 1 Mutation
Study identification
- NCT ID
- NCT04856982
- Recruitment status
- Active, not recruiting
- Study type
- Interventional
- Phase
- Phase 3
- Lead sponsor
- Biogen
- Industry
- Enrollment
- 158 participants
Conditions and interventions
Interventions
- Placebo Drug
- Tofersen Drug
Drug
Eligibility (public fields only)
- Age range
- 18 Years and older
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- May 16, 2021
- Primary completion
- Aug 6, 2027
- Completion
- Apr 29, 2028
- Last update posted
- Mar 29, 2026
2021 – 2028
United States locations
- U.S. sites
- 12
- U.S. states
- 10
- U.S. cities
- 12
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| HonorHealth Neurology | Scottsdale | Arizona | 85258 | — |
| University of California San Diego Medical Center | La Jolla | California | 92093-0949 | — |
| California Pacific Medical Center Research Institute | San Francisco | California | 94107 | — |
| Holy Cross Hospital | Fort Lauderdale | Florida | 33308 | — |
| University of Miami School of Medicine | Miami | Florida | 33136 | — |
| The Emory Clinic | Atlanta | Georgia | 30322-4200 | — |
| Northwestern Medicine | Chicago | Illinois | 60611 | — |
| Johns Hopkins Hospital | Baltimore | Maryland | 21287 | — |
| Massachusetts General Hospital, MA | Charlestown | Massachusetts | 02129 | — |
| Washington University School of Medicine | St Louis | Missouri | 63110 | — |
| Columbia University Medical center | New York | New York | 10032 | — |
| Austin Neuromuscular Center | Austin | Texas | 78756 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 20 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT04856982, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Mar 29, 2026 · Synced May 8, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT04856982 live on ClinicalTrials.gov.