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
Information for the public
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Public information presented according to the provisions of Directive 2001/18/EC
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Information related to the decision procedure
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7 January 2019 - The Biosafety Advisory Council issues a positive advice (with conditions) for this trial
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31 January 2019: The Federal Ministers give a final decision (positive) for this trial