Cytori Therapeutics, Inc., a late stage cell therapy company developing autologous cell therapies from adipose tissue to treat a variety of medical conditions, recently announced the beginning of its U.S. phase III/pivotal STAR clinical trial in the United States that will assess the efficacy of the experimental ECCS-50 therapy. ECCS-50 is cellular therapeutic that company believes can improve the processing of an autologous graft consisting of adipose derived regenerative cells (ADRCs) in the treatment of hand dysfunction due to scleroderma.
The announcement of initiation of enrollment comes after a key follow-on U.S. FDA investigational device approval in July that Cytori received for recent updates to the Celution® System, which is being used in the STAR trial. The updates include recent technologic advances such as enhanced software code that substantially improves the efficiency of the bedside therapeutic manufacturing process for ECCS-50.
Three testing centers for the STAR trial in the United States have been initiated, and the company is expecting that by October the majority of investigational sites will be active. Patient screening for the study has begun in the US and the first patients are expected to be treated with ECCS-50 this month.
A variety of promotional activities for the STAR trial will begin after Labor Day; however, the STAR microsite is currently active at: www.startrial-us.com. The company expects that patient enrollment will last for about a year.
“Hand involvement creates one of the most significant quality-of-life issues faced by people living with scleroderma, causing pain and significant disability,” said Robert Riggs, Chief Executive Officer of the Scleroderma Foundation, Inc., the leading patient advocacy and research nonprofit organization for the scleroderma community. “It’s exciting to see a therapy advance through clinical trials that is intended to address this debilitating and disfiguring manifestation of scleroderma.”
The STAR Trial is a prospective, randomized, multi-center device trial intended to assess safety and efficacy of subcutaneous administration of Celution processed ADRCs into fingers of 80 patients with hand dysfunction due to scleroderma.
Eligible patients will undergo pre-operative testing. Subjects will then undergo fat harvesting through small volume liposuction under local anesthesia. Lipoaspirate will be processed in the Celution System to isolate and concentrate ADRCs for immediate subcutaneous administration under local anesthesia.
Subjects will be randomly assigned to receive ADRCs (40 million cells) or a visually-matched placebo in a 1:1 ratio. All subjects will receive subcutaneous administration of test substance (ADRC or placebo) into all fingers of both hands. Following completion of all 48 week visits and database lock, placebo treated subjects will be offered treatment with their ADRCs cells should they continue to qualify according to the inclusion/exclusion criteria and should they wish to be treated with ADRCs and agree to the fat harvest procedure.
The design of the STAR trial was based on promising data from the European phase II/pilot trial named SCLERADEC, which was published in 2014 (Granel et al. Annals of Rheumatic Diseases). Results from this study demonstrated the safety of the autologous SVF cells injection in the hands of patients with SSc. However, the preliminary assessments at 6 months suggested potential efficacy needing confirmation in a randomized placebo-controlled trial on a larger population.
Later follow up of the SCLERADEC patients at 12 months showed that the beneficial effect of Cytori’s ECCS-50 therapy in scleroderma continued beyond 12 months and that data has been accepted for publication and is currently in press.