Long-Term Evaluation of BIIB067 (Tofersen)
Phase 3
Completed
NCT03070119
| Condition | ALS Caused by Superoxide Dismutase 1 Mutation |
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| Phase | Phase 3 |
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| Sponsor | Biogen |
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| Status | Completed |
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| Enrollment | 139 participants |
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| Interventions | Tofersen |
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| Start date | Mar 8, 2017 |
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| Completion date | Aug 12, 2024 |
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| Results posted | Aug 29, 2025 |
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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.
Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious AEs (TESAEs)
From first dose of the study drug in the current study up to end of follow-up period (up to Week 364) · Participants
| 233AS101: Part C (Prior Placebo) | 31 Participants |
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| 233AS101: Part C (Prior BIIB067 100 mg) | 63 Participants |
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| 233AS101: Part A and B (All Doses) | 43 Participants |
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| 233AS101: Part C (Prior Placebo) | 16 Participants |
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| 233AS101: Part C (Prior BIIB067 100 mg) | 33 Participants |
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| 233AS101: Part A and B (All Doses) | 22 Participants |
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Open in BENITIONS
View on ClinicalTrials.gov
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