B/BE/18/BVW6 - Summary Notification Information Format (SNIF) Public information presented according to the provisions of Directive 2001/18/EC
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
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
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