An Open-label Safety, Tolerability, and Dose-range Finding Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA)

Study Identifier:
ISIS 396443 - CS1
ClinicalTrials.gov Identifier:
NCT01494701
EudraCT Identifier:
N/A
Sponsor:
Biogen
Study Complete

Study Details

Medical Condition
  • All Spinal Muscular Atrophies
Study Drug
  • Drug: nusinersen
Date
Nov 2011 - Jan 2013
Phase 1
Phase 2
Phase 3
Phase 4
Patient Requirements
Sex: Female & Male
Age: 2 - 14 Years
Requirements Information

Protocol Summary

This objectives of this study are to evaluate the safety, tolerability, and pharmacokinetics of a single dose of nusinersen (ISIS 396443) administered intrathecally to participants with Spinal Muscular Atrophy (SMA).

Trial Locations

Location
Status
Location
Children's Hospital Boston
Boston, Massachusetts, United States, 02115
Status
N/A
Location
Columbia University Medical Center
New York, New York, United States, 10032
Status
N/A
Location
UT Southwestern Medical Center - Children's Medical Center Dallas
Dallas, Texas, United States, 75207
Status
N/A
Location
University of Utah School of Medicine
Salt Lake City, Utah, United States, 84132
Status
N/A

Contact Cure SMA

User Information

If you have general questions regarding clinical trials or would like to be added to the Cure SMA mailing list to receive communications regarding future registries, studies, or clinical trials in SMA, please fill out the form below. Fields marked with an asterisk (*) are mandatory.

  • Disability Advocate who does not have SMA
  • I am a healthcare provider
  • I am a researcher
  • I have no direct connection to SMA
  • I have SMA
  • I lost a child to SMA
  • My child has SMA
  • Someone close to me has/had SMA
  • Argentina
  • Australia
  • Belgium
  • Brazil
  • Canada
  • Chile
  • China
  • Colombia
  • Croatia
  • Czech Republic
  • Denmark
  • Estonia
  • France
  • Germany
  • Greece
  • Hong Kong
  • Hungary
  • India
  • Ireland
  • Israel
  • Italy
  • Japan
  • Latvia
  • Lebanon
  • Malaysia
  • Mexico
  • Netherlands
  • Norway
  • Poland
  • Portugal
  • Puerto Rico
  • Qatar
  • Republic of Korea
  • Romania
  • Russian Federation
  • Saudi Arabia
  • Serbia
  • Singapore
  • South Africa
  • Spain
  • Sweden
  • Switzerland
  • Taiwan, Province of China
  • Thailand
  • Turkey
  • Ukraine
  • United Kingdom
  • United States
  • Vietnam
This site is protected by reCAPTCHA and the Google Privacy Policy and Terms of Service apply.