InVivo Therapeutics Holdings (NVIV) announced that it has received approval of a protocol amendment from the US Food and Drug Administration (FDA) that will convert its ongoing pilot study into a pivotal probable benefit study. Prior to this announcement, the approval was conditional solely upon a minor change to the informed consent form.
With this transition, the study will be known formally as "The INSPIRE Study: InVivo Study of Probable Benefit of the Neuro-Spinal Scaffold for Safety and Neurologic Recovery in Subjects with Complete Thoracic AIS A Spinal Cord Injury." The INSPIRE Study is designed to enroll 20 implanted patients, inclusive of the five patients already enrolled in the study.
The INSPIRE study is currently approved to enroll up to 12 patients, but InVivo expects that the FDA will approve the full 20 patients following the review of the complete 6-month data package for the first five patients. The company plans to submit these data in the second quarter of 2016.
An objective performance criterion (OPC) for the study to support probable benefit using historical benchmarks is under discussion with the FDA. In large European and US databases, the published rates of spontaneous improvement of at least one AIS grade in complete (AIS A), thoracic SCI patients at 6 months are less than 16%. An additional study protocol amendment may be required to establish the OPC.
"Receiving full approval to convert our pilot study into a pivotal probable benefit study is a significant step forward for InVivo," said Mark Perrin, Chief Executive Officer and Chairman. "It is extraordinary to think that just over a year after enrolling the first patient in our study, we are now running a pivotal trial with planned international expansion and the intent to file an application for HDE approval in just two years."
Following an acute spinal cord injury, the biodegradable Neuro-Spinal Scaffold is surgically implanted at the epicenter of the wound and is designed to act as a physical substrate for nerve sprouting. Appositional healing to spare spinal cord tissue, decreased post-traumatic cyst formation, and decreased spinal cord tissue pressure have been demonstrated in preclinical models of spinal cord contusion injury.
The Neuro-Spinal Scaffold, an investigational device, has received a Humanitarian Use Device (HUD) designation and is currently being studied in an Investigational Device Exemption (IDE) study for the treatment of patients with complete (AIS A) traumatic acute spinal cord injury.