A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Participants With Later-onset Spinal Muscular Atrophy (SMA)
Completed · Phase 3 · Has a placebo group
Conditions studied: Spinal Muscular Atrophy
In brief
The primary objective of this study is to examine the clinical efficacy of nusinersen (ISIS 396443) administered intrathecally to participants with later-onset Spinal Muscular Atrophy (SMA). The secondary objective is to examine the safety and tolerability of nusinersen administered intrathecally to participants with later-onset SMA.
Key facts
- Study ID
- NCT02292537
- Run by
- Biogen
- People needed
- 126
- Starts
- 2014-11-24
- Expected to finish
- 2017-02-20
- Last updated by the study team
- 2021-02-17
Who can join
Age: 2 and older, up to 12. Sex: any. Healthy volunteers: not accepted.
Where it is running
- UCLA Clinical and Translational Research Center — Los Angeles, California, United States
- Lucile Packard Children's Hospital at Stanford — Palo Alto, 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
- Oregon Health & Science University — Portland, Oregon, United States
- Children's Hospital of Philadelphia - Neurology — Philadelphia, Pennsylvania, United States
- Children's Medical Center — Dallas, Texas, United States
- Children's Hospital - London Health Sciences Centre — London, Ontario, Canada
- McGill University Health Centre-Glen Site-CIM — Montreal, Quebec, Canada
- Armand Trousseau Hospital, I-Motion, Clinical Trials Platform — Paris, France
- Universitatsklinikum Essen — Essen, Germany
- University Hospital Freiberg, Center for Paediatrics and Adolescent Medicine, Department of Neuropaediatrics and Muscular Disease — Freiburg im Breisgau, Germany
- The University of Hong Kong, Queen Mary Hospital, Department of Paediatrics and Adolescent Medicine — Hong Kong, Hong Kong SAR, Hong Kong
- AOU Policlinico G. Martino Dipartimento di Neuroscienze e Centro Clinico Nemo Sud — Messina, Italy
- Fondazione Policlinico Universitario Agostino Gemelli-Universita Cattolica de Sacro Cuore-UOC Neuropsichiatre Infantile — Rome, Italy
- Hyogo College of Medicine — Nishinomya-shi, Hyōgo, Japan
- Tokyo Women's Medical University — Shinjuku-ku, Tokyo, Japan
- Seoul National University Children's Hospital — Seoul, Korea, South Korea
- Hospital Sant Joan de Deu — Barcelona, Spain
- University of Gothenburg, The Queen Silvia Children's Hospital — Gothenburg, Sweden
Full record on ClinicalTrials.gov
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