Trials / Completed
CompletedNCT03070119
Long-Term Evaluation of BIIB067 (Tofersen)
An Extension Study to Assess the Long-Term Safety, Tolerability, Pharmacokinetics, and Effect on Disease Progression of BIIB067 Administered to Previously Treated Adults With Amyotrophic Lateral Sclerosis Caused by Superoxide Dismutase 1 Mutation
- Status
- Completed
- Phase
- Phase 3
- Study type
- Interventional
- Enrollment
- 139 (actual)
- Sponsor
- Biogen · Industry
- Sex
- All
- Age
- 18 Years
- Healthy volunteers
- Not accepted
Summary
The primary objective of the study is to evaluate the long-term safety and tolerability of BIIB067 (tofersen) in participants with amyotrophic lateral sclerosis (ALS) and confirmed superoxide dismutase 1 (SOD1) mutation. The secondary objectives are to evaluate the pharmacokinetic (PK), pharmacodynamic (PD), biomarker effects, and efficacy of BIIB067 administered to participants with ALS and a confirmed SOD1 mutation.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | Tofersen | Participants will receive a loading dose regimen followed by maintenance dosing. |
Timeline
- Start date
- 2017-03-08
- Primary completion
- 2024-08-12
- Completion
- 2024-08-12
- First posted
- 2017-03-03
- Last updated
- 2025-08-29
- Results posted
- 2025-08-29
Locations
30 sites across 9 countries: United States, Belgium, Canada, Denmark, France, Germany, Italy, Japan, United Kingdom
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT03070119. Inclusion in this directory is not an endorsement.