Global study of a recombinant adeno-associated virus designed to address the monogenic root cause of Spinal Muscular Atrophy

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Study title: 
Global study of a recombinant adeno-associated virus designed to address the monogenic root cause of Spinal Muscular Atrophy
Long title: 
The dossier concerns two studies: AVXS-101-Cl-302: Phase 3, Open-Label, Single-Arm, Single-Dose Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies Delivering AVXS-101 by Intravenous Infusion AVXS-101-CL-304: European - A Global Study of a Single, One-Time Dose of AVXS-101 Delivered to Infants with Genetically Diagnosed and Pre-symptomatic Spinal Muscular Atrophy with Multiple Copies of SMN2
Date receipt dossier: 
16 oct 2018
EU record number: 
B/BE/18/BVW6
EudraCT number: 
2017-004087-35
Company / Sponsor: 
AveXis
Phase: 
III
Treated organism: 
Humans
Indication category: 
Neuromuscular disorders
Disease: 
Spinal Muscular Atrophy
Therapeutic approach: 
Gene therapy
Genetic modification: 
Recombinant adeno-associated virus serotype 9 (AAV9) containing the cDNA of the human SMN (Survival Motor Neuron) gene
Method of transfer of nucleic acid of interest: 
Non-replicating, non-integrating recombinant self-complementary adeno-associated virus serotype 9 (AAV9)
Administered biological material: 
Recombinant virus
Route of administration: 
Intravenous
Locations in Belgium: 
Centre Hospitalier Régional Hôpital La Citadelle (Liège); UZ Gent
Nr of subjects: 
44
Foreseen duration: 
5 years
Type of procedure: 
Contained use and Deliberate release
Current status: 
Authorized

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