A Study of Multiple Doses of Nusinersen (ISIS 396443) Delivered to Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy
Phase 2
Completed
NCT02386553
| Condition | Spinal Muscular Atrophy |
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| Phase | Phase 2 |
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| Sponsor | Biogen |
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| Status | Completed |
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| Enrollment | 25 participants |
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| Interventions | Nusinersen |
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| Start date | May 18, 2015 |
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| Completion date | Dec 17, 2024 |
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| Results posted | Oct 20, 2025 |
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Summary
The primary objective of the study is to examine the efficacy of multiple doses of Nusinersen administered intrathecally in preventing or delaying the need for respiratory intervention or death in infants with genetically diagnosed and presymptomatic spinal muscular atrophy (SMA). Secondary objectives of this study are to examine the effects of Nusinersen in infants with genetically diagnosed and presymptomatic SMA.
Time to Death or Respiratory Intervention
Screening up to Day 2891 · months
| ISIS 396443 2 SMN2 Copies | NA months |
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Open in BENITIONS
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