Athersys' MultiStem Cell Therapy for Ischemic Stroke to Be Studied in MASTERS-2
July 31, 2018—Athersys, Inc. announced that the first patient has been enrolled in its phase 3 MASTERS-2 study evaluating the administration of the company's MultiStem cell therapy for stroke treatment and enhanced recovery.
The randomized, double-blind, placebo-controlled MASTERS-2 clinical trial is designed to enroll 300 patients who have experienced moderate to moderate-severe ischemic stroke. Patients will be enrolled in North America and Europe.
According to the company, the enrolled patients will receive either a single intravenous dose of MultiStem cell therapy or placebo, administered within 18 to 36 hours of the occurrence of the stroke, in addition to the standard of care.
The study's primary endpoint will evaluate disability using modified Rankin Scale (mRS) scores at 3 months, comparing the distribution, or the “shift,” between the MultiStem treatment and placebo groups. The mRS shift analysis considers disability across the full spectrum, enabling recognition of large and small improvements in disability and differences in mortality and other serious outcomes among strokes of different severities, explained Athersys.
The study will also assess "Excellent Outcome" (mRS ≤ 1, National Institutes of Health Stroke Scale score ≤ 1, and Barthel Index ≥ 95) at 3 months and 1 year as key secondary endpoints. Additionally, the study will consider other measures of functional recovery, biomarker data, and clinical outcomes, including hospitalization, mortality, life-threatening adverse events, and poststroke complications such as infection.
The MASTERS-2 study has received FDA authorization under a special protocol assessment (SPA) for the design and planned analysis of this pivotal clinical trial. 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 of MultiStem cell therapy as a product for treating ischemic stroke patients. Additionally, the FDA has awarded fast track designation for the MultiStem stroke program.
In addition to the SPA and Fast Track designations from the FDA, the MASTERS-2 trial also obtained a final scientific advice positive opinion from the European Medicines Agency and received the regenerative medicine advanced therapy (RMAT) designation from the FDA.
RMAT designation is for eligible cell therapy and other regenerative medicine and advanced therapies when the FDA agrees that preliminary clinical evidence indicates that the therapy has demonstrated the potential to address unmet medical needs for a serious or life-threatening disease or condition. The designation enables sponsors to discuss with the FDA multidisciplinary strategic development plans, including expediting manufacturing development plans for commercialization to support priority review and accelerated approval, advised Athersys.