Cesca Therapeutics has submitted an investigational device exemption (IDE) supplement to the US Food and Drug Administration (FDA) for its previously approved pivotal trial.
The study is designed to demonstrate the safety and effectiveness of the Company’s point-of-care, SurgWerks system, in the treatment of late stage, “no-option” and “poor-option”, CLI patients. The Company expects the FDA to submit its response to the IDE supplement within 30 days from submission.
“This IDE supplement is the result of extensive review of current literature, as well as discussions with independent advisors, key opinion leaders and the FDA.
“After taking everything into consideration, from scientific to clinical to reimbursement, we believe that amputation free survival, a hard clinical end-point, gives us the best chance for a successful pivotal CLI trial outcome, pre-marketing approval (PMA), and path to commercialization,” said Dr. Xiaochun “Chris” Xu, Cesca’s Interim Chief Executive Officer.
“We remain dedicated to the development of safe and effective point-of-care autologous cell therapies, with CLI being just one of Cesca’s many opportunities in the space. We have several other vascular and orthopedic cellular therapies in our pipeline and are committed to opening up multiple clinical avenues to unlock the immense value and drive the success of the Company.”
Designed to demonstrate the safety and efficacy of the Company’s point-of-care SurgWerks™ system, the revised Phase III trial design will focus on the difference in AFS measured over 12 months between a treatment arm and a sham control arm.
The general design of the study will include 1:1 randomization with a 12 month follow-up program. The estimated sample size needed to successfully demonstrate safety and a statistically powered efficacy result is 362 patients (181 patients in the treatment arm and in the sham control arm, factoring in an estimated dropout rate of 10%).
The supplement also details changes to the protocol designed to improve patient enrollment. These include expanding the cohort to include both “no option” and “poor option” Rutherford Category 4 and 5 CLI patients, using a sham control arm vs placebo, restricting severe diabetic R5 subjects, and stream-lining follow-up procedures.
The trial also includes clinically significant secondary endpoints, such as wound healing and improvement in rest pain, designed to show quality-of-life improvements, which are relevant to receiving reimbursement approval from The Centers for Medicare and Medicaid Services (CMS).
Cesca Therapeutics is engaged in the research, development, and commercialization of cellular therapies and delivery systems for use in regenerative medicine.
The Company is a leader in the development and manufacture of automated blood and bone marrow processing systems that enable the separation, processing and preservation of cell and tissue therapeutics.