# An Open-label Safety, Tolerability and Dose-Range Finding Study of Multiple Doses of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy

> **NCT01703988** · PHASE1,PHASE2 · COMPLETED · sponsor: **Biogen** · enrollment: 34 (actual)

## Conditions studied

- Spinal Muscular Atrophy

## Interventions

- **DRUG:** Nusinersen

## Key facts

- **NCT ID:** NCT01703988
- **Lead sponsor:** Biogen
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE1,PHASE2
- **Study type:** INTERVENTIONAL
- **Status:** COMPLETED
- **Start date:** 2012-10-31
- **Primary completion:** 2015-01-31
- **Final completion:** 2015-01-31
- **Target enrollment:** 34 (ACTUAL)
- **Last updated:** 2021-04-13


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT01703988

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT01703988, "An Open-label Safety, Tolerability and Dose-Range Finding Study of Multiple Doses of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy". Retrieved via AI Analytics 2026-06-25 from https://api.ai-analytics.org/clinical/NCT01703988. Licensed CC0.

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