The European gene therapy trial STR1VE EU is an open-label, phase III study to evaluate the efficacy and safety of utilisation of a single, intravenous injection of AVXS-101 (an AAV9 carrying the SMN1 gene), in 30 infants with SMA associated proximal spinal muscular atrophy (SMA) type 1, under 6 months of age.
The study was recently launched in an investigator center in France, at the I-Motion Institute (Trousseau Hospital, Paris).
It is currently being carried out in 4 countries (France, Belgium, Italy, United Kingdom).
Gene therapy in SMA
In 2009 and 2011, the Genethon and the Myology Institute teams published the first successful gene therapy in mice models with SMA: effective in the central nervous system, at the AAV9-SMN1 vector and in its administration routes.
In parallel, the American laboratory, AveXis developed the AVXS-101, made by an AAV9 transporting the SMN1 gene.
An initial AVXS-101 phase I trial with intravenous administration in 15 participants with SMA type 1 demonstrated the product’s well-tolerated profile and improvements in motor function and development. These encouraging results led to the United States launch of the STR1VE trial in September 2017 in 20 infants with SMA type 1.
Of note, it was only in March of 2018 that Genethon granted AveXis (recently acquired by Novartis) licensing including worldwide exclusive rights of AAV9-Gene therapy and its administration routes.
The European STR1VE EU trial, the STR1VE trial as well as other planned trials (STRONG trial, SPR1NT trial, REACH trial) provide supplementary data for those with SMA type 2 or 3 or older, in light of the future commercialization of AVXS-101.
In October 2018, AveXis submitted a marketing authorization request for AVXS-101 to American, European, and Japanese authorities.
On Clinicaltrials.gov : Essai STR1VE EU
