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Cesca Therapeutics Inc. announced that the Company received approval from the U.S. Food and Drug Administration (FDA) for significant revisions to the Company’s pivotal study for treatment of Critical Limb Ischemia (CLI). The CLI clinical trial is designed to demonstrate the safety and efficacy of the Company’s point-of-care SurgWerks™ system for the treatment of CLI patients with limited or no treatment options. The study was first approved by FDA in June 2015, but was not initiated at that time pending funding.
The changes approved by the FDA are intended to increase patient enrollment by expanding the patient pool from Rutherford Category 5 patients only, to also include Rutherford Category 4 patients, or patients with a less severe form of the disease. In addition, the study population has been expanded to include patients who are poor candidates for either surgery or endovascular therapies, as opposed to only those patients with no viable treatment options. Finally, the FDA accepted to change the control arm from a placebo, which was both invasive and carried measureable safety risk, to a much less risky sham procedure.
Although the primary endpoint remains Amputation Free Survival (AFS), a standard in all current CLI drug and biologic trials, the FDA recognized the need to increase the available patient population in order to properly power the clinical trial. Compared to the initial study design, the sample size was increased from 224 to 362 patients, which in turn allowed the superiority margin to be reduced from 20% to 13%. We believe that the reduced superiority margin is both achievable and clinically relevant. Due to the increased patient pool, it is expected that enrollment rates will be better than previous CLI clinical trials.
Other improvements to the study include a reduction in study-related testing, improved statistical methods, and streamlined study oversight, all of which are intended to reduce costs and improve patient recruitment and investigator participation. “We are pleased the FDA approved our IDE supplement. The changes, especially expanding the patient population and the improved statistical methods, allow us to strike an important balance between patient enrollment rates, superiority margin, cost and time”, said Dr. Xiaochun “Chris” Xu, Cesca’s Interim CEO. “With this compelling trial design we look forward to finding strategic partners to help develop and commercialize the SurgWerks™ CLI system. We remain committed to advancing our innovative point-of-care platforms and devices and making Cesca the leading solution provider in the broader cell therapy and healthcare space.
