NCT04042025 - Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi | Biotech Hunter

Enrollment

Participants

Timeline

Start Date

February 10, 2020

Primary Completion Date

December 31, 2035

Study Completion Date

December 31, 2035

Conditions

Spinal Muscular Atrophy Type ISpinal Muscular Atrophy Type IISpinal Muscular Atrophy Type IIISMA

Interventions

BIOLOGICAL

Onasemnogene Abeparvovec-xioi

Onasemnogene abeparvovec-xioi is a non-replicating recombinant adeno-associated virus serotype 9 containing the human survival motor neuron gene under the control of the cytomegalovirus enhancer/chicken β-actin-hybrid promoter. Onasemnogene abeparvovec-xioi administered as a one-time intravenous (IV) infusion or intrathecal (IT) injection. Dosage determined by participant weight.

Trial Locations (31)

2145

Sydney Children's Hospital, Randwick

9000

Universitair Ziekenhuis Gent, Ghent

10032

Columbia University Medical Center, New York

10048

National Taiwan University Hospital, Taipei

16147

Instituto Gianninia Gaslini, Genova

17579

Clinic for Special Children, Strasburg

19104

Children's Hospital of Philadelphia, Philadelphia

20122

Universita Degli Studi Di Milano, Milan

20133

Istituto Neurologico di Ricerca, Milan

21287

John Hopkins Hospital - David M. Rubenstein Child Health Building, Baltimore

23298

Virginia Commonwealth University, Richmond

23507

Children's Hospital of The King's Daughters, Norfolk

27713

Duke University, Durham

43205

Nationwide Children's Hospital, Columbus

49503

Spectrum Health Hospitals Helen DeVos Children's Hospital, Grand Rapids

53792

University of Wisconsin, Madison, Madison

60611

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

63110

Washington Unviersity School of Medicine in Saint Louis, St Louis

75012

Hôpital Armand Trousseau, Paris

75235

Children's Health Specialty Center Dallas Campus, Dallas

80045

Children's Hospital Colorado, Aurora

84112

University of Utah Health, Salt Lake City

94304

Stanford University Medical Center, Palo Alto

02114

Massachusetts General Hospital, Boston

02115

Boston Children's Hospital, Boston

B-4000

Centre de Référence des Maladies Neuromusculaires, Liège

K1H8L1

Children's Hospital of Eastern Ontario Research Institute, Ottawa

00168

Fondazione Policlinico Universitario Agostino Gemelli, Roma

162-8666

Tokyo Women's Medical University Hospital, Tokyo

WC1N 3JH

Great Ormond Street Hospital for Children NHS Foundation Trust, London

NE1 4LP

The Newcastle Upon Tyne Hospitals NHS Foundation Trust, Newcastle upon Tyne