Participants Sought for SMA Gene Therapy Study


Researchers are looking for individuals with type 1 spinal muscular atrophy (SMA) to participate in the phase 3 STR1VE clinical trial, sponsored by AveXis, to test efficacy for its SMA gene replacement therapy AVXS-101. If your child is younger than 6 months of age and has received a diagnosis of type 1 SMA, he or she may be eligible to participate.

Administered by intravenous infusion (a needle inserted into a vein), AVXS-101 is designed to deliver a new gene that can produce the Survival Motor Neuron (SMN) protein. SMN is critical to the function of the nerves that control muscles and is missing in children with SMA. Increased production of the protein may lead to improvements in muscle strength and function.

All participants in the open-label STR1VE study will receive treatment with the experimental therapy. Study-related visits, tests, and treatments will be provided to participants at no cost.

To be eligible to participate, individuals must be less than 6 months old, have one or two copies of the SMN2 gene, and meet additional criteria.

Trial sites are located in California, Colorado, Illinois, Maryland, Ohio, Oregon and New York. Travel assistance may be available for families who don’t live near one of the research centers.

To learn more about this trial, go to and enter NCT03306277 into the search box, or visit the STR1VE study website and email the trial coordinator at the site nearest you.