NCT01839656View on ClinicalTrials.gov

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

PHASE2CompletedINTERVENTIONAL
Enrollment

21

Participants

Timeline

Start Date

May 8, 2013

Primary Completion Date

August 21, 2017

Study Completion Date

August 21, 2017

Conditions
Spinal Muscular Atrophy
Interventions
DRUG

nusinersen

Administered by intrathecal (IT) injection

Trial Locations (4)

10032

Columbia University Medical Center, New York

32827

Nemours Children's Hospital, Orlando

94305

Stanford University Medical Center, Stanford

M5G 1X8

The Hospital for Sick Children (SickKids), Toronto

Sponsors

Lead Sponsor

Biogen
All Listed Sponsors
lead

Biogen

INDUSTRY

NCT01839656 - A Study to Assess the Efficacy, Safety and Pharmacokinetics of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy (SMA) | Biotech Hunter | Biotech Hunter