Long-Term Evaluation of BIIB067 (Tofersen)
Фаза 3
Завершено
NCT03070119
| Заболевание | ALS Caused by Superoxide Dismutase 1 Mutation |
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| Фаза | Фаза 3 |
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| Спонсор | Biogen |
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| Статус | Завершено |
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| Участники | 139 участн. |
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| Вмешательство | Tofersen |
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| Дата начала | 8.03.2017 |
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| Дата завершения | 12.08.2024 |
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| Публикация результатов | 29.08.2025 |
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Кратко
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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