NCT03505099 - Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2 | Biotech Hunter

Enrollment

Participants

Timeline

Start Date

April 2, 2018

Primary Completion Date

June 15, 2021

Study Completion Date

June 15, 2021

Conditions

Spinal Muscular Atrophy

Interventions

BIOLOGICAL

onasemnogene abeparvovec-xioi

A non-replicating recombinant AAV9 containing the complimentary deoxyribonucleic acid (cDNA) of the human SMN gene under the control of the cytomegalovirus (CMV) enhancer/chicken-β-actin-hybrid promoter (CB). The AAV inverted terminal repeat (ITR) has been modified to promote intramolecular annealing of the transgene, thus forming a double-stranded transgene ready for transcription.

Trial Locations (16)

2145

Sydney Children's Hospital, Randwick

4000

Centre Hospitalier Régional Hôpital La Citadelle, Liège

10032

Columbia University Medical Center, New York

17579

Clinic for Special Children, Strasburg

32827

Nemours Children's Hospital, Orlando

43205

Nationwide Children's Hospital, Columbus

49503

Helen DeVos Children's Hospital, Grand Rapids

53792

University Hospital and UW Health Clinics, Madison

63110

St. Louis Children's Hospital, St Louis

75235

Children's Medical Center Dallas, Dallas

80045

Children's Hospital Colorado, Aurora

90095

David Geffen School of Medicine at UCLA, Los Angeles

02114

Massachusetts General Hospital, Boston

K1H8L1

Canada Childrens Hospital of Eastern Ontario, Ottawa

Unknown

Tokyo Women's Medical, Tokyo

WC1N 3JH

Great Ormond Street Hospital for Children NHS Foundation Trust, London