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

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
› Author Affiliations
Further Information

Publication History

Publication Date:
11 September 2019 (online)

 
 

    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


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    No conflict of interest has been declared by the author(s).