NCT02122952View on ClinicalTrials.gov

Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1

PHASE1CompletedINTERVENTIONAL
Enrollment

15

Participants

Timeline

Start Date

May 5, 2014

Primary Completion Date

December 15, 2017

Study Completion Date

December 15, 2017

Conditions
Spinal Muscular Atrophy 1
Interventions
BIOLOGICAL

AVXS-101

Self-complementary AAV9 carrying the SMN gene under the control of a hybrid CMV enhancer/chicken-β-actin promoter

Trial Locations (1)

43205

Nationwide Children's Hospital, Columbus

Sponsors
All Listed Sponsors
lead

Novartis Gene Therapies

INDUSTRY