Phase II Trial of Trans Sodium Crocetinate in Acute Stroke Endorsed by FDA

September 14, 2018

TSC has shown potential to become the first approved therapy for acute stroke, able to be administered in the ambulance.

John L. Gainer, PhD

The FDA has approved the start of a phase II clinical trial of trans sodium crocetinate (TSC) in the treatment of acute stroke.

Announced by the therapy’s developer, Diffusion Pharmaceuticals, the PHAST-TSC trial is set to enroll 160 patients randomized 1:1 to TSC and placebo at 23 sites in urban, suburban, and rural area of Los Angeles and central Virginia. Diffusion stated that it will seek to conclude the trial within 2 years, which it noted as “a critically fast turnaround for this potential improvement in the front-line medical treatment of stroke.”

The trial, which will be led by experts from the University of Virginia and the University of California Los Angeles, with the goal of expanding the treatment window and patient outcomes for stroke. Jeffrey Saver, MD, professor of clinical neurology and director of the stroke unit at the David Geffen School of Medicine at UCLA will be the study chair.

"Dr. Saver and his team at UCLA have been pioneers in on-ambulance therapy, and, along with [Karen C. Johnston, MD, MSc] and her team at the University of Virginia, we have the expertise necessary to test TSC's effectiveness in patients suffering from acute stroke," said John L. Gainer, PhD, Diffusion's chief science officer and the inventor of the TSC molecule, in a statement. "While combining the talents of a pharmaceutical company with those of 2 of our country's leading academic medical centers is, perhaps, a paradigm shift for creating new clinical trials, it is one which will hopefully produce substantial benefits for stroke victims and their families."

The therapy’s original purpose was for use on the field of battle by the US military to aid in the treatment of life-threatening injuries, according to David Kalergis, MBA, JD, the chairman and CEO of Diffusion. This trial, he noted, represented a return to the company’s roots in emergency medicine.

"This FDA approval to begin patient enrollment in PHAST-TSC gives us the opportunity to show that treatment with TSC could make an impact on a different battlefield, one where stroke patients are also in a race against the clock," Kalergis said.

Tudor Jovin, MD, an assistant professor of neurology and neurosurgery at the University of Pittsburgh School of Medicine, co-director of the Center for Endovascular Therapy at the University of Pittsburgh Medical Center, stressed to NeurologyLive previously that the time window for treating patients can always improve.

“We still take way too long, and we still don’t get enough patients to the hospital,” he said. “And when we get them to the hospital, we take way too long to get them to a thrombectomy center. Even within the thrombectomy center, we take too long for them to get to the procedure suite.”

In addition to testing TSC in acute stroke, what makes this trial unique is its study of treatment administration in the ambulance prior to hospital arrival. Johnston, the Harrison Distinguished Professor of Neurology and Associate Vice President for Clinical & Translational Research at UVA, said that both Saver and co-principal investigator Andrew Southerland, MD, are both “leaders in the development of innovative approaches to treating acute stroke patients in the ambulance."

Currently, only tissue plasminogen activator (tPA) is FDA approved for dissolving blood clots but is not able to be used in hemorrhagic stroke. Preclinical data, according to Diffusion, has suggested that patient outcomes could be improved with combination use of tPA with TSC, without increasing risk.

REFERENCE

FDA Approves Start of Innovative On-Ambulance Phase 2 Clinical Trial for Treatment of Acute Stroke by Diffusion Pharmaceuticals [press release]. Charlottesville, VA: Diffusion Pharmaceuticals Inc; Published September 12, 2018. nasdaq.com/press-release/fda-approves-start-of-innovative-onambulance-phase-2-clinical-trial-for-treatment-of-acute-stroke-20180912-00644. Accessed September 13, 2018.