FDA Approves IDE for Cytori Cell Therapy Trial

San Diego, Calif.-based Cytori Therapeutics Inc. has gained U.S. Food and Drug Administration (FDA) conditional approval for an investigational device exemption (IDE) for a pivotal clinical trial, named the Star trial, to evaluate Cytori cell therapy as a potential treatment for impaired hand function in scleroderma, a rare autoimmune disease affecting approximately 50,000 patients in the United States.

Scleroderma is a chronic autoimmune disorder associated with fibrosis of the skin, destructive changes in blood vessels and multiple organ systems as the result of a generalized overproduction of collagen. Scleroderma affects women four times more frequently than men and is typically detected between the ages of 30 and 50. Most people suffering from scleroderma have disabling disease involving the hands. The National Institute of Arthritis and Musculoskeletal and Skin Diseases estimates that the number of people in the United States with systemic sclerosis is approximately 50,000. Treatment options are directed at protecting the hands from injury and detrimental environmental conditions as well as the use of vasodilators. When the disease is advanced, immunosuppressive and other medications may be used but are often accompanied by intolerable side effects.

The active element in Cytori cell therapy is a mixture of cells derived from the patient’s own fat tissue termed adipose-derived stem and regenerative cells (ADRCs). ADRCs are obtained through Cytori’s proprietary Celution system, which is a medical device that enables point-of-care access to adult ADRCs by automating and standardizing the extraction, washing, and concentration of a patient’s own ADRCs for present and future clinical use. The Celution system is being used in clinical research and under limited approvals in Europe and Japan, where it is registered as a medical device.

The Star trial is conditionally approved to be a randomized, double blind, placebo-controlled pivotal clinical trial of 80 patients in up to 12 U.S. sites. The trial evaluates the safety and efficacy of a single administration of Cytori Cell Therapy in scleroderma patients affecting the hands and fingers. More than 90 percent of scleroderma patients have hand involvement that is typically progressive and can result in chronic pain, blood flow changes and severe dysfunction. The limited available treatments for scleroderma may provide some benefit but do little to modify disease progression or substantially improve symptoms.

Cytori’s cell therapy processing technology—the Celution system—is regulated as a class III (highest risk) medical device by the FDA and requires a successful pivotal trial and premarket authorization (PMA) before it can be marketed in the United States.

The Star trial is predicated on a completed pilot trial, Scleradec-I. In data presented earlier this year, patients in Scleradec-I showed, on average, 50 percent improvement at six months across four important and validated endpoints used to assess the clinical status in patients with systemic scleroderma with impaired hand function. This data was published in the Annals of the Rheumatic Diseases in May.

“Hand problems from scleroderma result in substantial distress, pain and impaired quality of life. We need more effective therapeutic options for our patients,” said Dinesh Khanna, M.D., director, University of Michigan Scleroderma Program and a principal investigator of the Star trial. I was impressed by the single center French study with Celution processed adipose-derived regenerative cells inpatients with scleroderma hand disability, and although it is limited by a small number of patients, it provides the data to support the larger, well-designed, randomized, controlled trial that we will be conducting.”

It’s planned for the Star trial to use the Cochin hand score, a validated measure of hand function, as the primary endpoint measured at 6 months after a single administration of Cytori cell therapy or placebo. Patients in the placebo group will be eligible for crossover to the active arm of the trial after all patients have completed 12 months of follow up. Additional details about the trial will be disclosed once finalized and agreement is reached with the FDA. The trial is anticipated to begin enrollment in 2015.

“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,” said Marc Hedrick, M.D., president and CEO of Cytori. “We are hopeful that this trial will show data consistent with the efficacy signal observed in the recently published trial. 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.”