Following the allowance of the IND by the FDA in August 2014, the open-label VC01-101 STEP ONE* clinical trial launched in September 2014 and has continued through Year 2 of the Strategic Partnership, with the primary goals of demonstrating safety and tolerability, and developing administration procedures to establish effective cell engraftment. This Phase 1/2 clinical trial, designed to evaluate the VC-01 product candidate directly in patients with type 1 diabetes (T1D), was initially launched at the UC San Diego Health System, with the support of the UC San Diego Sanford Stem Cell Clinical Center, under the direction of Principal Investigator Robert Henry, MD. In January 2015 the team received a No Objection Letter from Health Canada providing clearance to proceed with sites in Canada and in July 2015, a second clinical site was opened at the University of Alberta Hospitals in Edmonton, Alberta under the direction of Principal Investigator James Shapiro, MD PhD. Dr. Shapiro is credited with developing effective pancreatic islet transplant methods as a treatment for T1D, and is a valuable addition to the team.
Over the first year of the trial, preparation of the VC-01 test article for the trial has been successful and without issue: all VC-01 lots have been produced on time, with no contaminations, and no meaningful quality control issues. All have been delivered to the clinical sites for administration on time and without issue.
As of October 2015, ten T1D patients have been enrolled serially at the two sites in Cohort 1 and implanted with the VC-01 product candidate at a sub-therapeutic dose of cells. The goals of Cohort 1 are to evaluate the safety of the product candidate, and to develop and optimize surgical procedures and post-surgical care required for the successful engraftment of the cells. The number of patients to be studied in this first phase of the trial is data-driven and based primarily on histological examination of sentinels. When the procedures are successfully optimized, Cohort 1 results will be compiled and reported to the Data Safety Monitoring Board (DSMB). The DSMB will make a recommendation regarding continuation to Cohort 2, which will evaluate safety and efficacy of the VC-01 product candidate, administered at what is calculated to be a therapeutic dose of cells.
* Safety, Tolerability, and Efficacy of the VC-01 Combination Product in Type One Diabetes