The US Food and Drug Administration has granted San Diego-based regenerative-medicine company Cytori Therapeutics, Inc. conditional approval for an Investigational Device Exemption or IDE for a central study dubbed, STAR. The investigation will evaluate Cytori Cell Therapy‘s efficacy as a new solution for impaired hand function in patients with scleroderma. Over 90% of scleroderma patients experience hand affectations that result in chronic pain, diminished blood flow, and severe disability.
Scleroderma, which literally translates to “hard skin,” is a rare autoimmune disease characterized by overproduction of collagen, which causes fibrotic changes in the skin, blood vessels, and internal organs, resulting in a reduction in quality of life, and fatal organ failure. The CDC estimates this disease to affect at least 50,000 in the United States alone.
The STAR trial is a pivotal randomized, double blind, placebo-controlled study that will enroll 80 patients across 12 study sites in the United States. It aims to assess the safety and efficacy of cell therapy as processed by the Celution® System, an FDA-regulated Class III medical device that produces an autologous cell product from adipose tissue called, adipose-derived regenerative cells (ADRCs™). Results will be graded using the Cochin hand score 6 months after treatment with a single dose of Cytori Cell Therapy or a placebo. Participants on placebo will receive special consideration to crossover to the active arm of the study once all patients have been examined at 12 months. The STAR trial is expected to open enrollment next year, 2015. Cytori will be disclosing additional information and updates once an agreement has been reached with the FDA.
“The STAR trial is a direct result of our new strategy to focus on identifying innovative, later stage therapeutic applications and moving the most promising into the clinic. We are hopeful that this trial will show data consistent with the efficacy signal observed in the recently published trial,” said Dr. Marc Hedrick, President and CEO of Cytori. “Additionally, the rare nature of scleroderma-associated hand involvement represents a potential straightforward and targeted path to market and reimbursement following the pivotal clinical trial.”
The company’s management team will be holding an informational meeting on the STAR trial, featuring an open forum for questions, during the upcoming J.P. Morgan Healthcare Conference on January 12-15, 2015 in San Francisco. We will be reporting more details on this later in January.