International SMA gene therapy trials to start in 2018

AveXis is expanding its spinal muscular atrophy (SMA) clinical development programme for its gene therapy drug, AVXS-101. The drug utilises a harmless adeno-associated virus to deliver a healthy copy of the SMN1 gene into the body.

AveXis is currently testing AVXS-101 in two US-based clinical trials, STR1VE and STRONG. These are assessing the safety and efficacy of the drug in individuals with SMA Type 1 (STR1VE) and Type 2 (STRONG). The company has recently announced plans for three new trials to further evaluate AVXS-101:

STR1VE EU: is a European trial that is intended to provide evidence for marketing approval by the European Medicines Agency (EMA). This is known as a pivotal trial. The trial is designed so all participants will receive the gene therapy and natural history data will be used as a comparator. It is expected to enrol 30 babies with SMA Type 1, who are less than six months old at the time of treatment. In addition to safety, the efficacy of the drug after a single intravenous infusion will be assessed. Outcome measures include the achievement of developmental milestones and the time from birth to an “event”, which is defined as either the requirement of permanent ventilation or death.

SPRINT: is a multi-national trial that will evaluate AVXS-101 in pre-symptomatic babies with SMA Type 1, 2, or 3. The trial is expected to enrol 44 babies with either two, three or four copies of SMN2 and who are less than six weeks of age at the time of the gene therapy treatment. The study will evaluate safety, participant survival and motor milestone development after a single intravenous infusion of AXVS-101.

REACH: is a multi-national trial for participants who do not qualify for other AVXS-101 trials. It is expected to enrol 50 participants between six months and 18 years of age, with either SMA Type 1, 2, or 3. Participants will receive a single intrathecal (spinal) injection of AVXS-101.

AveXis plans to initiate STR1VE EU and SPRINT in the first half of 2018, and REACH in late Q4 2018 or early 2019. The company will provide further information at the time of initiation.



Further information


Read AveXis’ press release

Visit our Clinical Trials Information Service

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