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

> **NCT01494701** · PHASE1 · COMPLETED · sponsor: **Biogen** · enrollment: 28 (actual)

## Conditions studied

- Spinal Muscular Atrophy

## Interventions

- **DRUG:** nusinersen

## Key facts

- **NCT ID:** NCT01494701
- **Lead sponsor:** Biogen
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE1
- **Study type:** INTERVENTIONAL
- **Status:** COMPLETED
- **Start date:** 2011-11-30
- **Primary completion:** 2013-01-31
- **Final completion:** 2013-01-31
- **Target enrollment:** 28 (ACTUAL)
- **Last updated:** 2021-02-18


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT01494701

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT01494701, "An Open-label Safety, Tolerability, and Dose-range Finding Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA)". Retrieved via AI Analytics 2026-06-02 from https://api.ai-analytics.org/clinical/NCT01494701. Licensed CC0.

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*[Clinical trials dataset](/datasets/clinical-trials) · CC0 1.0*