Single-Dose Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1

Key facts

Sponsor

Novartis Gene Therapies

Principal Investigator

AveXis Medinfo

Sponsor GmbH

Intervention

Onasemnogene Abeparvovec-xioi(biological)

Enrollment

33 enrolled

Eligibility

All sexes

Timeline

2018 – 2020

Study locations (10)

• University Hospital Ghent Neuromuscular reference center, Ghent, Belgium
• Neuropédiatrie - Centre de Référence des Maladies Neuromusculaires, Liège, Belgium
• Hôpital Armand Trousseau, Paris, France
• Istituto Gianninia Gaslini, Genova, Italy
• Policlinico "G. Martino", Messina, Italy
• Carlo Besta Neurological Research Institute, Milan, Italy
• University of Milan, Milan, Italy
• Policlinico Gemelli, Rome, Italy
• Great Ormond Street Hospital for Children, London, United Kingdom
• The John Walton Muscular Dystrophy Research Centre MRC Centre for Neuromuscular Diseases at Newcastle, Newcastle upon Tyne, United Kingdom

Primary source

Recruitment status, site addresses, contacts, and full eligibility criteria can change between syncs. Always verify with the trial team before planning travel or treatment.

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