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NCT01703988 · ClinicalTrials.gov registry record · Phase 1
An Open-label Safety, Tolerability and Dose-Range Finding Study of Multiple Doses of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy
A Phase 1 study, sponsored by Biogen.
- Completed
- Registry status
- Phase 1
- Development phase
- 34
- Enrollment target
NCT01703988: Completed Phase 1 study, sponsored by Biogen.
NCT01703988 is a Phase 1 study that has completed, run by Biogen. The registered enrollment target is 34 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (43% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01703988, a Phase 1 study, has completed, sponsored by Biogen.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 34 participants
- Enrollment target
Study Summary
This study will test the safety, tolerability, and pharmacokinetics of escalating doses of nusinersen (ISIS 396443) administered into the spinal fluid either two or three times over the duration of the trial, in participants with spinal muscular atrophy (SMA). Four dose levels will be evaluated sequentially. Each dose level will be studied in a cohort of approximately 8 participants, where all participants will receive active drug.
Primary Outcome
An AE is any unfavorable and unintended sign, symptom, or disease temporally associated with the study or use of the investigational drug product, whether or not the AE is considered related to the investigational drug product. An SAE is any AE that, in the view of either the Investigator or Sponsor, meets any of the following criteria: results in death; is life threatening; requires inpatient hospitalization or prolongation of existing hospitalization; results in a persistent or significant inc
Interventions
- DRUG Nusinersen
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 34 participants |
| Start Date | 2012-10-31 |
| Est. Completion | 2015-01-31 |
| Phase | Phase 1 |
What the finished NCT01703988 record still lists
NCT01703988 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 34 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (43% lower).
The record links to 0 conditions, and to 1 intervention - of which Nusinersen is the first listed.
NCT01703988 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT01703988 about?
NCT01703988 is a clinical study titled "An Open-label Safety, Tolerability and Dose-Range Finding Study of Multiple Doses of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy". This study will test the safety, tolerability, and pharmacokinetics of escalating doses of nusinersen (ISIS 396443) administered into the spinal fluid either two or three times over the duration of the trial, in participants with spinal muscular atrophy (SMA). Four dose levels will be evaluated seq...
What is the current status of trial NCT01703988?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 34 participants. The study started on 2012-10-31. Estimated completion is 2015-01-31.
What interventions are being tested in trial NCT01703988?
The interventions under investigation include: Nusinersen (DRUG).
Who is sponsoring clinical trial NCT01703988?
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 NCT01703988's enrollment target sits among peer trials
34 1273rd of 2000 higher than 721 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 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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