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NCT04856982 · ClinicalTrials.gov registry record · Phase 3
A Study of BIIB067 (Tofersen) Initiated in Clinically Presymptomatic Adults With a Confirmed Superoxide Dismutase 1 Mutation
A Phase 3 study of Amyotrophic Lateral Sclerosis Associated With a SOD1 Gene Mutation, sponsored by Biogen.
- Active
- Registry status
- Phase 3
- Development phase
- 158
- Enrollment target
- 20
- Study locations
NCT04856982 is a Phase 3 study of Amyotrophic Lateral Sclerosis Associated With a SOD1 Gene Mutation that is active but no longer recruiting, run by Biogen. The registered enrollment target is 158 participants. The trial reports 20 study locations across 18 states.
The verdict
NCT04856982, a Phase 3 study of Amyotrophic Lateral Sclerosis Associated With a SOD1 Gene Mutation, is active but no longer recruiting, sponsored by Biogen.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 3
- Development phase
- 158 participants
- Enrollment target
- 20
- Study locations
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 |
Interested in This Trial?
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, categorized as Phase 3. 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 334 total studies on file at ClinicalTrials.gov.
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.
NCT04856982 reports 20 study locations spanning 18 distinct geographic areas - top geographies include California, Florida, Arizona.
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.
What interventions are being tested in trial NCT04856982?
The interventions under investigation include: Placebo (DRUG), Tofersen (DRUG).
Who is sponsoring clinical trial NCT04856982?
This trial is sponsored by Biogen, which has 334 total clinical trials registered on ClinicalTrials.gov.
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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