A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy
Public ClinicalTrials.gov record NCT02193074. 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
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
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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.
Study identification
- NCT ID
- NCT02193074
- Recruitment status
- Terminated
- Study type
- Interventional
- Phase
- Phase 3
- Enrollment
- 122 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- Up to 210 Days
- 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
- Aug 18, 2014
- Primary completion
- Nov 20, 2016
- Completion
- Nov 20, 2016
- Last update posted
- Feb 16, 2021
2014 – 2016
United States locations
- U.S. sites
- 12
- U.S. states
- 12
- U.S. cities
- 12
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| UCLA Medical Center | Los Angeles | California | 90095 | — |
| Children's Hospital Colorado | Aurora | Colorado | 80045 | — |
| Nemours Children's Hospital | Orlando | Florida | 32827 | — |
| Ann and Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | 60611 | — |
| Boston Children's Hospital | Boston | Massachusetts | 02115 | — |
| Washington University School of Medicine | St Louis | Missouri | 63110 | — |
| Columbia University Medical Center | New York | New York | 10032 | — |
| Duke Children's Hospital | Durham | North Carolina | 27710 | — |
| Doernbecher Children's Hospital | Portland | Oregon | 97239 | — |
| Children's Hospital of Philadelphia - Neurology | Philadelphia | Pennsylvania | 19104 | — |
| UT Southwestern Medical Center/Children's Medical Center Dallas | Dallas | Texas | 75235 | — |
| Primary Children's Medical Center (University of Utah) | Salt Lake City | Utah | 84112 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 19 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT02193074, 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 16, 2021 · Synced Sep 7, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT02193074 live on ClinicalTrials.gov.