A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy

PHASE3TerminatedINTERVENTIONAL
Enrollment

122

Participants

Timeline

Start Date

August 19, 2014

Primary Completion Date

November 21, 2016

Study Completion Date

November 21, 2016

Conditions
Spinal Muscular Atrophy
Interventions
DRUG

nusinersen

Administered by intrathecal (IT) injection as specified in the treatment arm.

PROCEDURE

Sham procedure

Small needle prick on the lower back at the location where the IT injection is normally made

Trial Locations (31)

2031

Sydney Children's Hospital, Sydney

3052

Royal Children's Hospital, Children's Neuroscience Centre, Parkville

10032

Columbia University Medical Center, New York

16148

Istituto Giannina Gaslini, Centro Traslazionale di Miologia e Patologie Neurodegenerative, Genova

19104

Children's Hospital of Philadelphia - Neurology, Philadelphia

27710

Duke Children's Hospital, Durham

28046

Hospital Universitario La Paz, Pediatric Neurology Department, Madrid

32827

Nemours Children's Hospital, Orlando

45147

Universitatsklinikum Essen, Essen

60611

Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago

63110

Washington University School of Medicine, St Louis

75012

Institut de Myologie, Paris

75235

UT Southwestern Medical Center/Children's Medical Center Dallas, Dallas

79106

Universtatsklinikum Freiburg, Zentrum fur Kinder-und Jugendmedizin , Abteilung Neuropadiatrie und Muskelerkrankungen, Freiburg im Breisgau

80045

Children's Hospital Colorado, Aurora

84112

Primary Children's Medical Center (University of Utah), Salt Lake City

90095

UCLA Medical Center, Los Angeles

97239

Doernbecher Children's Hospital, Portland

02115

Boston Children's Hospital, Boston

15 - 1020

Hôpital Universitaire des Enfants Reine FABIOLA (HUDERF), Brussels

V6H 3N1

British Columbia Children's Hospital/UBC, Vancouver

M5G 1X8

Hospital for Sick Children, Toronto

00153

Pediatric Neurology Unit, Catholic University, Rome

663-8131

Hyogo College of Medicine, Nishinomiya

162-8666

Tokyo Women's Medical University, Tokyo

Unknown

Seoul National University Hospital, Seoul

University of Gothenburg, The Queen Silvia Children's Hospital, Gothenburg

08035

Hospital Universitario Vall d'Hebron, Barcelona

06230

Hacettepe Children's Hospital, Ankara

WC1N 1EH

UCL Institute of Child Health/Great Ormond Street, London

NE1 3BZ

MRC Centre for Neuromuscular Diseases at Newcastle, Institute of Genetic Medicine Newcastle University, Newcastle

Sponsors

Lead Sponsor

All Listed Sponsors
lead

Biogen

INDUSTRY

NCT02193074 - A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy | Biotech Hunter | Biotech Hunter