A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy
Stopped early · Phase 3 · Has a placebo group
Conditions studied: Spinal Muscular Atrophy
In brief
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.
Key facts
- Study ID
- NCT02193074
- Run by
- Biogen
- People needed
- 122
- Starts
- 2014-08-19
- Expected to finish
- 2016-11-21
- Last updated by the study team
- 2021-02-17
Who can join
Age: any, up to 1. Sex: any. Healthy volunteers: not accepted.
Where it is running
- UCLA Medical Center — Los Angeles, California, United States
- Children's Hospital Colorado — Aurora, Colorado, United States
- Nemours Children's Hospital — Orlando, Florida, United States
- Ann and Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois, United States
- Boston Children's Hospital — Boston, Massachusetts, United States
- Washington University School of Medicine — St Louis, Missouri, United States
- Columbia University Medical Center — New York, New York, United States
- Duke Children's Hospital — Durham, North Carolina, United States
- Doernbecher Children's Hospital — Portland, Oregon, United States
- Children's Hospital of Philadelphia - Neurology — Philadelphia, Pennsylvania, United States
- UT Southwestern Medical Center/Children's Medical Center Dallas — Dallas, Texas, United States
- Primary Children's Medical Center (University of Utah) — Salt Lake City, Utah, United States
- Sydney Children's Hospital — Sydney, New South Wales, Australia
- Royal Children's Hospital, Children's Neuroscience Centre — Parkville, Victoria, Australia
- Hôpital Universitaire des Enfants Reine FABIOLA (HUDERF) — Brussels, Belgium
- British Columbia Children's Hospital/UBC — Vancouver, British Columbia, Canada
- Hospital for Sick Children — Toronto, Ontario, Canada
- Institut de Myologie — Paris, France
- Universitatsklinikum Essen — Essen, Germany
- Universtatsklinikum Freiburg, Zentrum fur Kinder-und Jugendmedizin , Abteilung Neuropadiatrie und Muskelerkrankungen — Freiburg im Breisgau, Germany
- Istituto Giannina Gaslini, Centro Traslazionale di Miologia e Patologie Neurodegenerative — Genova, Italy
- Pediatric Neurology Unit, Catholic University — Rome, Italy
- Hyogo College of Medicine — Nishinomiya, Hyōgo, Japan
- Tokyo Women's Medical University — Tokyo, Japan
- Seoul National University Hospital — Seoul, South Korea
Full record on ClinicalTrials.gov
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