The first patient has been enrolled in a phase 3 study, MASTERS-2 (NCT03545607), which is a pivotal study that has received special protocol assessment, fast-track designation, and regenerative medicine advanced therapy designation from the Food and Drug Administration (FDA). The SPA provides formal agreement from the FDA that the protocol design, clinical endpoints, planned conduct and statistical analyses in this Phase 3 study are acceptable to support a regulatory submission for marketing approval. The fast track designation makes the program eligible for accelerated approval and priority review and helps ensure a timely regulatory review. The RMAT designation signifies FDA agreement that the therapy has demonstrated the potential to address unmet medical needs for a serious or life-threatening disease or condition.
The trial is designed to enroll 300 people from North America and Europe who have experienced moderate to moderate-severe ischemic stroke. Patients enrolled in the trial will receive either an intravenous dose of cell therapy (Multistem; Athersys, Cleveland, OH) or a placebo injection, administered within 18-36 hours of stroke occurrence, as an adjunct to current standard-of-care treatment.
The effect of therapy will be measured 3 months after treatment with the modified Rankin Scale (mRS), a measure of disability that allows recognition of both large and small improvements. The study will also assess outcomes 3 and 12 months after treatment as secondary endpoints using multiple rating scales. Other measures will include functional recovery, biomarker data, and clinical outcomes, including hospitalization, mortality, life-threatening adverse events and poststroke complications.
The cell therapy being tested is a patented regenerative medicine product in clinical development that promotes tissue repair and healing. Putative mechanisms of action are production of multiple therapeutic factors produced by the cell treatment in response to signals of inflammation and tissue damage. The therapy represents a unique off-the-shelf stem-cell product that can administered without tissue matching or immunosuppression.Next Story