A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Participants With Later-onset Spinal Muscular Atrophy (SMA)

PHASE3CompletedINTERVENTIONAL
Enrollment

126

Participants

Timeline

Start Date

November 24, 2014

Primary Completion Date

February 20, 2017

Study Completion Date

February 20, 2017

Conditions
Spinal Muscular Atrophy
Interventions
DRUG

Nusinersen

Administered by intrathecal (IT) lumbar puncture (LP) injection

PROCEDURE

Sham procedure

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

Trial Locations (24)

10032

Columbia University Medical Center, New York

19104

Children's Hospital of Philadelphia - Neurology, Philadelphia

32827

Nemours Children's Hospital, Orlando

60611

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

63110

Washington University School of Medicine, St Louis

75235

Children's Medical Center, Dallas

80045

Children's Hospital Colorado, Aurora

90095

UCLA Clinical and Translational Research Center, Los Angeles

94304

Lucile Packard Children's Hospital at Stanford, Palo Alto

97239

Oregon Health & Science University, Portland

02115

Boston Children's Hospital, Boston

N6A 5W9

Children's Hospital - London Health Sciences Centre, London

H4A 3JI

McGill University Health Centre-Glen Site-CIM, Montreal

Unknown

Armand Trousseau Hospital, I-Motion, Clinical Trials Platform, Paris

Universitatsklinikum Essen, Essen

University Hospital Freiberg, Center for Paediatrics and Adolescent Medicine, Department of Neuropaediatrics and Muscular Disease, Freiburg im Breisgau

The University of Hong Kong, Queen Mary Hospital, Department of Paediatrics and Adolescent Medicine, Hong Kong

AOU Policlinico G. Martino Dipartimento di Neuroscienze e Centro Clinico Nemo Sud, Messina

Fondazione Policlinico Universitario Agostino Gemelli-Universita Cattolica de Sacro Cuore-UOC Neuropsichiatre Infantile, Rome

Hyogo College of Medicine, Nishinomya-shi

Tokyo Women's Medical University, Shinjuku-ku

Seoul National University Children's Hospital, Seoul

Hospital Sant Joan de Deu, Barcelona

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

Sponsors

Lead Sponsor

All Listed Sponsors
lead

Biogen

INDUSTRY