A Study of Multiple Doses of Nusinersen (ISIS 396443) Delivered to Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy

Study Identifier:
232SM201
ClinicalTrials.gov Identifier:
NCT02386553
EudraCT Identifier:
2014-002098-12
Sponsor:
Biogen
Study Complete

Study Details

Medical Condition
  • All Spinal Muscular Atrophies
Study Drug
  • Drug: Nusinersen
Date
May 2015 - Dec 2024
Phase 1
Phase 2
Phase 3
Phase 4
Patient Requirements
Sex: Female & Male
Age: 6 - 6 Weeks
Requirements Information

Protocol Summary

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.

Trial Locations

Location
Status
Location
David Geffen School of Medicine
Los Angeles, California, United States, 90095
Status
N/A
Location
University of California Davis Health System
Sacramento, California, United States, 95817
Status
N/A
Location
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
Status
N/A
Location
Nemours Children's Hospital, Orlando
Orlando, Florida, United States, 32827
Status
N/A
Location
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611-2605
Status
N/A
Location
The Johns Hopkins Hospital
Baltimore, Maryland, United States, 21205
Status
N/A

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