Neuropediatrics 2019; 50(S 02): S1-S55
DOI: 10.1055/s-0039-1698171
Oral Presentations
Neuromuscular Diseases and Varia
Georg Thieme Verlag KG Stuttgart · New York

Study Design of STR1VE-EU, a Phase 3 Trial of AVXS-101 Gene-Replacement Therapy (GRT) in Patients With Spinal Muscular Atrophy Type 1 (SMA1) in Europe

Authors

  • Francesco Muntoni

    1   University College London, Department of Developmental Neuroscience, London, United Kingdom

  • Giovanni Baranello

    2   Fondazione IRCCS Istituto Neurologico Carlo Besta, Developmental Neurology Unit, Milan, Italy

  • Claudio Bruno

    3   Istituto Giannina Gaslini, Translational and Experimental Myology Centre, Genoa, Italy

  • Stefania Corti

    4   Neuroscience Section, Department of Pathophysiology and Transplantation (DEPT), Neurology Unit, University of Milan, Dino Ferrari Centre, IRCCS Foundation Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy

  • Riccardo Masson

    2   Fondazione IRCCS Istituto Neurologico Carlo Besta, Developmental Neurology Unit, Milan, Italy

  • Volker Straub

    5   University of Newcastle, MRC Centre for Neuromuscular Diseases Institute of Genetic Medicine, Newcastle upon Tyne, United Kingdom

  • Giuseppe Vita

    6   Messina University Hospital, Unit of Neurology and Neuromuscular Diseases, Messina, Italy

  • Uwe Ernst

    7   AveXis, Inc., Medical Affairs, Bannockburn, Illinois, United States

  • Elaine Kernbauer

    8   AveXis, Inc., Clinical Operations, Bannockburn, Illinois, United States

  • Susan Williamson

    8   AveXis, Inc., Clinical Operations, Bannockburn, Illinois, United States

  • Haojun Ouyang

    9   AveXis, Inc., Biostatistics, Clinical Development, Bannockburn, Illinois, United States

  • Douglas E. Feltner

    10   AveXis, Inc., Clinical Development, Bannockburn, Illinois, United States

  • Matthew Meriggioli

    10   AveXis, Inc., Clinical Development, Bannockburn, Illinois, United States

  • Arseniy Lavrov

    10   AveXis, Inc., Clinical Development, Bannockburn, Illinois, United States

  • James L’Italien

    11   AveXis, Inc., Regulatory and Quality, Bannockburn, Illinous, United States

  • Douglas M. Sproule

    10   AveXis, Inc., Clinical Development, Bannockburn, Illinois, United States

  • Eugenio Mercuri

    12   Catholic University, Department of Paediatric Neurology and Nemo Clinical Centre, Rome, Italy
Further Information

Publication History

Publication Date:
11 September 2019 (online)

Also available at
 

Research Question: SMA1, a rapidly progressing disease, typically results in death/need for permanent ventilation by 2 years of age. Survival motor neuron 1 gene (SMN1) biallelic deletion/mutation is the genetic root cause of SMA and leads to irreversible motor neuron loss. Onasemnogene abeparvovec (AVXS-101) is an investigational, one-time GRT based on a non-replicating, recombinant adeno-associated virus serotype 9 (AAV9) containing the human SMN gene. AVXS-101 is designed for immediate and sustained SMN protein expression, allowing rapid onset and durable effect in neurons. In a phase 1/2a study (CT.gov: NCT02122952), SMA1 patients treated with AVXS-101 demonstrated exceptional improvements in survival, motor function, bulbar function, and milestone achievement. STR1VE-EU is a phase 3, multicenter, open-label, single-arm, one-time-dose study (EudraCT: 2017-000266-29; CT.gov: NCT03461289) investigating efficacy and safety of AVXS-101 in up to 30 SMA1 patients