NCT03381729View on ClinicalTrials.gov

Study of Intrathecal Administration of Onasemnogene Abeparvovec-xioi for Spinal Muscular Atrophy

PHASE1TerminatedINTERVENTIONAL
Enrollment

32

Participants

Timeline

Start Date

December 14, 2017

Primary Completion Date

November 18, 2021

Study Completion Date

November 18, 2021

Conditions
Spinal Muscular Atrophy
Interventions
BIOLOGICAL

Onasemnogene Abeparvovec-xioi

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

Trial Locations (11)

19104

Children's Hospital of Philadelphia, Philadelphia

21287

Johns Hopkins, Baltimore

32827

Nemours Children's Hospital, Orlando

43205

Nationwide Children's Hospital, Columbus

60611

Ann & Robert H. Lurie Children's Hospital, Chicago

63130

Washington University, St Louis

75390

UT Southwestern, Dallas

84112

University of Utah, Salt Lake City

90095

UCLA, Los Angeles

94305

Stanford University, Stanford

02115

Boston Children's Hospital, Boston

Sponsors
All Listed Sponsors
lead

Novartis Gene Therapies

INDUSTRY

NCT03381729 - Study of Intrathecal Administration of Onasemnogene Abeparvovec-xioi for Spinal Muscular Atrophy | Biotech Hunter | Biotech Hunter