NEW YORK – Saol Therapeutics and GeneDx have partnered to identify young children with the rare mitochondrial disorder pyruvate dehydrogenase complex deficiency, who might be eligible to participate in a Phase III clinical study of dichloroacetate (DCA).
Beyond identifying potential patients through GeneDx's genetic testing tools, the companies aim to speed recruitment by collaborating to make physicians treating PDCD aware of the pivotal trial. DCA has Orphan Product designation from the US Food and Drug Administration for congenital lactic acidosis, including patients with PDCD.
PDC deficiency underlies the often fatal disorder, which is the most common cause of congenital lactic acidosis.
"There are currently no FDA-approved treatments for patients with PDCD," Peter Stacpoole, the trial's principal investigator, said in a statement. "Despite this, finding and recruiting children appropriate for participation in clinical trials is not easy. With the help of GeneDx, we hope to complete trial recruitment this year."
GeneDx, currently a subsidiary of BioReference Laboratories, is in the process of being acquired by Sema4 in an approximately $623 million deal.