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NCT01839656 · ClinicalTrials.gov registry record · Phase 2
A Study to Assess the Efficacy, Safety and Pharmacokinetics of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy (SMA)
A Phase 2 study, sponsored by Biogen.
- Completed
- Registry status
- Phase 2
- Development phase
- 21
- Enrollment target
NCT01839656: Completed Phase 2 study, sponsored by Biogen.
NCT01839656 is a Phase 2 study that has completed, run by Biogen. The registered enrollment target is 21 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (84% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01839656, a Phase 2 study, has completed, sponsored by Biogen.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 21 participants
- Enrollment target
Study Summary
The primary objective is to examine the clinical efficacy of multiple doses of nusinersen (ISIS 396443) administered intrathecally to participants with Infantile-Onset Spinal Muscular Atrophy (SMA). The secondary objectives are to examine the safety and tolerability of multiple doses of nusinersen administered intrathecally to participants with infantile-onset SMA and to examine the cerebral spinal fluid (CSF) and plasma Pharmacokinetics (PK) of multiple doses of nusinersen administered intrathecally to participants with infantile-onset SMA.
Primary Outcome
Section 2 of HINE consists of 8 independent milestone categories. Within each of these categories, participants can progress from complete absence of a motor ability (the lowest level in each category) through multiple milestones (2 to 4 levels in each category) to the highest level within the category. Overall, there are a total of 26 milestones that can be achieved across the 8 categories. Improvement was defined as any of the following: 1. An increase from baseline of 2 milestones or more, or
Interventions
- DRUG nusinersen
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 21 participants |
| Start Date | 2013-05-08 |
| Est. Completion | 2017-08-21 |
| Phase | Phase 2 |
What the finished NCT01839656 record still lists
NCT01839656 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 21 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (84% lower).
The record links to 0 conditions, and to 1 intervention - of which nusinersen is the first listed.
NCT01839656 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT01839656 about?
NCT01839656 is a clinical study titled "A Study to Assess the Efficacy, Safety and Pharmacokinetics of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy (SMA)". The primary objective is to examine the clinical efficacy of multiple doses of nusinersen (ISIS 396443) administered intrathecally to participants with Infantile-Onset Spinal Muscular Atrophy (SMA). The secondary objectives are to examine the safety and tolerability of multiple doses of nusinersen a...
What is the current status of trial NCT01839656?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 21 participants. The study started on 2013-05-08. Estimated completion is 2017-08-21.
What interventions are being tested in trial NCT01839656?
The interventions under investigation include: nusinersen (DRUG).
Who is sponsoring clinical trial NCT01839656?
This trial is sponsored by Biogen, which has 334 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT01839656's enrollment target sits among peer trials
21 1794th of 2000 higher than 196 of 2,000 other Phase 2 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 2 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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