Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
NCT01494701 · ClinicalTrials.gov registry record · Phase 1
An Open-label Safety, Tolerability, and Dose-range Finding Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA)
A Phase 1 study, sponsored by Biogen.
- Completed
- Registry status
- Phase 1
- Development phase
- 28
- Enrollment target
NCT01494701: Completed Phase 1 study, sponsored by Biogen.
NCT01494701 is a Phase 1 study that has completed, run by Biogen. The registered enrollment target is 28 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (53% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01494701, a Phase 1 study, has completed, sponsored by Biogen.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 28 participants
- Enrollment target
Study 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).
Interventions
- DRUG nusinersen
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 28 participants |
| Start Date | 2011-11-30 |
| Est. Completion | 2013-01-31 |
| Phase | Phase 1 |
What the finished NCT01494701 record still lists
NCT01494701 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 28 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (53% lower).
The record links to 0 conditions, and to 1 intervention - of which nusinersen is the first listed.
NCT01494701 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT01494701 about?
NCT01494701 is a clinical study titled "An Open-label Safety, Tolerability, and Dose-range Finding Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA)". 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).
What is the current status of trial NCT01494701?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 28 participants. The study started on 2011-11-30. Estimated completion is 2013-01-31.
What interventions are being tested in trial NCT01494701?
The interventions under investigation include: nusinersen (DRUG).
Who is sponsoring clinical trial NCT01494701?
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 NCT01494701's enrollment target sits among peer trials
28 1447th of 2000 higher than 538 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
Nationwide trials with similar profiles
Cross-condition peers matched on enrollment target and registry start date, not the same-condition list above.
Similar enrollment target
- NCT03471117 · 28 participants · Phase 4
Pioglitazone to Reduce Sympathetic Overactivity in CKD Patients
- NCT03523975 · 28 participants · Phase 1
Venetoclax, Lenalidomide and Rituximab in Patients With Previously Untreated Mantle Cell Lymphoma
- NCT03608631 · 28 participants · Phase 1
iExosomes in Treating Participants With Metastatic Pancreas Cancer With KrasG12D Mutation
- NCT03810651 · 28 participants · Early Phase 1
Pencil Beam Scanning in Patients With Renal Tumors
Similar registry start date
- NCT05962346 · started 2026-12 · NA
Fetal Endoscopic Tracheal Occlusion for Congenital Diaphragmatic Hernia
- NCT07125183 · started 2026-12 · Phase 2
Study on Efficacy and Tolerability of Weekly Doxorubicin in Elderly Patients With Advanced or Metastatic Leiomyosarcoma
- NCT07292298 · started 2026-11 · Phase 2
Phase 2 Single-Arm Rectal Cancer Brachytherapy for Patients With Low-Lying Residual Adenocarcinoma After Total Neoadjuvant Therapy to Improve Organ Preservation Rates
- NCT04263285 · started 2026-10 · NA
Treatment of Depression Post-SCI