A Study of Multiple Doses of Nusinersen (ISIS 396443) Delivered to Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy
Completed · Phase 2
Conditions studied: Spinal Muscular Atrophy
In brief
The primary objective of the study is to examine the efficacy of multiple doses of Nusinersen administered intrathecally in preventing or delaying the need for respiratory intervention or death in infants with genetically diagnosed and presymptomatic spinal muscular atrophy (SMA). Secondary objectives of this study are to examine the effects of Nusinersen in infants with genetically diagnosed and presymptomatic SMA.
Key facts
- Study ID
- NCT02386553
- Run by
- Biogen
- People needed
- 25
- Starts
- 2015-05-18
- Expected to finish
- 2024-12-17
- Last updated by the study team
- 2025-10-20
Who can join
Age: any, up to 0. Sex: any. Healthy volunteers: not accepted.
Where it is running
- David Geffen School of Medicine — Los Angeles, California, United States
- University of California Davis Health System — Sacramento, California, United States
- Children's Hospital Colorado — Aurora, Colorado, United States
- Nemours Children's Hospital, Orlando — Orlando, Florida, United States
- Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois, United States
- The Johns Hopkins Hospital — Baltimore, Maryland, United States
- Massachusetts General Hospital — Boston, Massachusetts, United States
- Columbia University — New York, New York, United States
- The Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
- University of Utah — Salt Lake City, Utah, United States
- Seattle Children's Research Institute — Seattle, Washington, United States
- Queensland Children's Hospital — South Brisbane, Queensland, Australia
- Royal Children's Hospital — Parkville, Victoria, Australia
- Universitaetsklinikum Freiburg — Freiburg im Breisgau, Baden-Wurttemberg, Germany
- Ospedale Pediatrico Bambino Gesù — Rome, Lazio, Italy
- Fondazione Serena Onlus - Centro Clinico Nemo — Milan, Italy
- Hamad General Hospital — Doha, Qatar
- Kaohsiung Medical University Chung-Ho Memorial Hospital — Kaohsiung City, Taiwan
- National Taiwan University Hospital — Taipei, Taiwan
- Hacettepe University Medical Faculty — Ankara, Turkey (Türkiye)
- Yeditepe University Medical School Hospital — Istanbul, Turkey (Türkiye)
Full record on ClinicalTrials.gov
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