McGowan Institute for Regenerative Medicine
affiliated faculty member David Okonkwo, MD, PhD (pictured), assistant professor of Neurological Surgery, clinical director, Brain Trauma Research Center, director, Neurotrauma Program, and director, Spinal Deformity Program, Department of Neurological Surgery, is the University of Pittsburgh Medical Center (UPMC) principal investigator of the BHR Pharma, LLC-initiated, global Phase 3, multi-center trial to evaluate the effectiveness of its proprietary BHR-100 intravenous progesterone infusion product as a neuroprotective agent for treating severe TBI patients. The trial started last year and its first patient was enrolled at UPMC.
Every 19 seconds someone is treated in an emergency room for a traumatic brain injury (TBI). Every 10 minutes one of those patients dies. Despite the fact that emergency departments treat TBIs more frequently than heart attack and stroke patients, there are currently no approved medications to improve outcomes following a TBI. Although most TBI victims will survive, the consequences of the injury are unpredictable. The SyNAPSe trial will enroll approximately 1,200 patients with severe TBI (Glasgow Coma Scale scores of 4-8), and who have sustained a closed-head trauma, at more than 100 trauma centers in the U.S., Europe, Israel, and additional countries. In the trial, the BHR-100 therapy must be delivered within 8 hours of sustaining a TBI.
Previous clinical trials, conducted in the U.S. and China, suggest that progesterone can improve outcomes for TBI victims. Both studies showed about a 50 percent lower mortality in the progesterone-treated group as compared to placebo. The Chinese study also showed a statistically-significant functional improvement and the U.S. study showed a similar trend. The SyNAPSe trial is designed to build on these promising results with the intent of bringing an approved treatment to market. Moreover, SyNAPSe is the only pivotal trial with progesterone for TBI designed for global regulatory approval.
The U.S. Food and Drug Administration (FDA) granted orphan drug status to BHR-100 and placed the drug on a Fast Track Development Program designed to accelerate its potential approval. They also agreed to approve BHR-100 based solely on the outcome of SyNAPSe if it meets certain objectives, something very uncommon.
“Traumatic brain injuries are the leading cause of death and disability among young Americans in the United States and have become known as the ‘signature wound’ of the wars in Iraq and Afghanistan,” said Congressman Todd Platts (PA-19), Co-Chair of the Congressional Brain Injury Task Force. “Making progress on this critical issue will require concerted efforts in Congress to expand access to treatments in a fiscally responsible manner, as well as expanded research in the private sector to help find treatments for this debilitating condition.”
To date, top enrolling facilities include UPMC; Medical University of South Carolina, led by Dr. Samir Fakhry; West Virginia University, led by Dr. Julian Bailes; and Vall d`Hebron University Hospital, Barcelona, Spain, led by Dr. Juan Sahuquillo.
Illustration: University of Pittsburgh.
PR Web (03/07/11)
BHR Pharma, LLC-initiated, global Phase 3, multi-center trial: SyNAPSe
Bio: Dr. David Okonkwo