An Open-label Safety and Tolerability Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in ISIS 396443-CS2 (NCT01703988) or ISIS 396443-CS10 (NCT01780246)
Public ClinicalTrials.gov record NCT02052791. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
An Open-label Study to Assess the Safety and Tolerability of ISIS 396443 in Patients With Spinal Muscular Atrophy Who Previously Participated in 396443-CS2 or 396443-CS10
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
The primary objective of this study is to examine the safety and tolerability of nusinersen (ISIS 396443) administered intrathecally to participants with Spinal Muscular Atrophy (SMA) who previously participated in ISIS 396443-CS2 (NCT01703988) or ISIS 396443-CS10 (NCT01780246). The secondary objective is to examine the plasma and cerebrospinal fluid (CSF) pharmacokinetic(s) (PK) of nusinersen administered intrathecally to participants with SMA who previously participated in ISIS 396443-CS2 or ISIS 396443-CS10.
Study identification
- NCT ID
- NCT02052791
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 1
- Enrollment
- 47 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- Not listed
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Jan 30, 2014
- Primary completion
- Jan 30, 2017
- Completion
- Jan 30, 2017
- Last update posted
- Feb 15, 2021
2014 – 2017
United States locations
- U.S. sites
- 4
- U.S. states
- 4
- U.S. cities
- 4
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Boston Children's Hospital | Boston | Massachusetts | 02115 | — |
| Columbia University Medical Center | New York | New York | 10032 | — |
| UT Southwestern Medical Center - Children's Medical Center Dallas | Dallas | Texas | 75207 | — |
| University of Utah School of Medicine | Salt Lake City | Utah | 84132 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT02052791, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Feb 15, 2021 · Synced Sep 1, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT02052791 live on ClinicalTrials.gov.