Trials / Completed
CompletedNCT01494701
An Open-label Safety, Tolerability, and Dose-range Finding Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA)
An Open-label, Escalating Dose Study to Assess the Safety, Tolerability and Dose-range Finding of a Single Intrathecal Dose of ISIS 396443 in Patients With Spinal Muscular Atrophy
- Status
- Completed
- Phase
- Phase 1
- Study type
- Interventional
- Enrollment
- 28 (actual)
- Sponsor
- Biogen · Industry
- Sex
- All
- Age
- 2 Years – 14 Years
- Healthy volunteers
- Not accepted
Summary
This objectives of this study are to evaluate the safety, tolerability, and pharmacokinetics of a single dose of nusinersen (ISIS 396443) administered intrathecally to participants with Spinal Muscular Atrophy (SMA).
Detailed description
This study was conducted and the protocol was registered by Ionis Pharmaceuticals, Inc. In August 2016, sponsorship of the trial was transferred to Biogen.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | nusinersen | Administered by intrathecal (IT) injection |
Timeline
- Start date
- 2011-11-30
- Primary completion
- 2013-01-31
- Completion
- 2013-01-31
- First posted
- 2011-12-19
- Last updated
- 2021-02-18
Locations
4 sites across 1 country: United States
Source: ClinicalTrials.gov record NCT01494701. Inclusion in this directory is not an endorsement.