NCT01494701View on ClinicalTrials.gov

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

PHASE1CompletedINTERVENTIONAL
Enrollment

28

Participants

Timeline

Start Date

November 30, 2011

Primary Completion Date

January 31, 2013

Study Completion Date

January 31, 2013

Conditions
Spinal Muscular Atrophy
Interventions
DRUG

nusinersen

Administered by intrathecal (IT) injection

Trial Locations (4)

10032

Columbia University Medical Center, New York

75207

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

84132

University of Utah School of Medicine, Salt Lake City

02115

Children's Hospital Boston, Boston

Sponsors

Lead Sponsor

Biogen
All Listed Sponsors
lead

Biogen

INDUSTRY

NCT01494701 - An Open-label Safety, Tolerability, and Dose-range Finding Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA) | Biotech Hunter | Biotech Hunter