Trials / Terminated
TerminatedNCT02193074
A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy
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 Infantile-onset Spinal Muscular Atrophy
- Status
- Terminated
- Phase
- Phase 3
- Study type
- Interventional
- Enrollment
- 122 (actual)
- Sponsor
- Biogen · Industry
- Sex
- All
- Age
- 210 Days
- Healthy volunteers
- Not accepted
Summary
The primary objective of the study is to examine the clinical efficacy of nusinersen (ISIS 396443) administered intrathecally (IT) to participants with infantile-onset with infantile-onset spinal muscular atrophy (SMA). The secondary objective of the study is to examine the safety and tolerability of nusinersen administered intrathecally to participants with infantile-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) injection as specified in the treatment arm. |
| PROCEDURE | Sham procedure | Small needle prick on the lower back at the location where the IT injection is normally made |
Timeline
- Start date
- 2014-08-19
- Primary completion
- 2016-11-21
- Completion
- 2016-11-21
- First posted
- 2014-07-17
- Last updated
- 2021-02-17
- Results posted
- 2017-07-28
Locations
31 sites across 13 countries: United States, Australia, Belgium, Canada, France, Germany, Italy, Japan, South Korea, Spain, Sweden, Turkey (Türkiye), United Kingdom
Source: ClinicalTrials.gov record NCT02193074. Inclusion in this directory is not an endorsement.