Trials / Completed
CompletedNCT02292537
A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Participants With Later-onset Spinal Muscular Atrophy (SMA)
A Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients With Later-onset Spinal Muscular Atrophy
- Status
- Completed
- Phase
- Phase 3
- Study type
- Interventional
- Enrollment
- 126 (actual)
- Sponsor
- Biogen · Industry
- Sex
- All
- Age
- 2 Years – 12 Years
- Healthy volunteers
- Not accepted
Summary
The primary objective of this study is to examine the clinical efficacy of nusinersen (ISIS 396443) administered intrathecally to participants with later-onset Spinal Muscular Atrophy (SMA). The secondary objective is to examine the safety and tolerability of nusinersen administered intrathecally to participants with later-onset 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) lumbar puncture (LP) injection |
| PROCEDURE | Sham procedure | Small needle prick on the lower back at the location where the IT injection is normally made |
Timeline
- Start date
- 2014-11-24
- Primary completion
- 2017-02-20
- Completion
- 2017-02-20
- First posted
- 2014-11-17
- Last updated
- 2021-02-17
- Results posted
- 2018-02-22
Locations
24 sites across 10 countries: United States, Canada, France, Germany, Hong Kong, Italy, Japan, South Korea, Spain, Sweden
Source: ClinicalTrials.gov record NCT02292537. Inclusion in this directory is not an endorsement.