Cesca Therapeutics (KOOL), an autologous cell-based regenerative medicine company, today announced that it has received a letter from the US Food and Drug Administration responding to the Company's investigational device exemption (IDE) application for its pivotal CLIRST III clinical trial.
The IDE application, filed on November 20, 2014, sought approval to begin a pivotal, randomized, placebo-controlled study of 224 late stage CLI patients at up to 60 clinical sites in the US and India.
In its letter, the FDA noted several deficiencies in the Company’s submission, requesting, among other things, additional information on characterization of the output of the SurgWerks(TM) – CLI and VXP system, a plan for testing device output for conformity to predefined intra-operative release criteria prior to administration, modifications to the management of potential adverse events and a strengthening of the language in the risk clauses in the Informed Consent Document.
"The clarity of the feedback from the FDA is very helpful and we are now in the process of addressing the various points raised in their letter," commented Robin Stracey, Chief Executive Officer of Cesca.
"We anticipate completing that work and submitting our amended IDE application sometime in February 2015," he continued.
In its letter, the FDA noted no deficiencies relating to the Company’s overall trial design, including the patient population size of 224, the 3:1 randomization and the use of a blinded independent committee for determination of amputation decisions.
The xcompany believes that, upon FDA approval, its study will be one of the first CLI trials to use a blinded independent committee review to assess the primary endpoint. Cesca included this element in its trial design specifically to overcome the trial bias observed in previous pivotal CLI trials with major amputation free survival rates as the primary endpoint.
CLI is the most severe form of Peripheral Arterial Disease (PAD) affecting over two million patients in the United States. It is often associated with chronic foot and leg ulcers, leading to approximately two hundred thousand amputations per year. A diagnosis of CLI statistically results in a 25% mortality rate and a 25% amputation rate within one year.