Mallinckrodt achieved target enrollment of 300 participants for a phase 3 clinical study designed to evaluate the safety and efficacy of terlipressin for patients with hepatorenal syndrome type 1, according to a press release.
“Currently, there are no treatments approved in the U.S. and Canada for the treatment of [hepatorenal syndrome type 1 (HRS-1)], which is a serious disease that has a high mortality rate,” Arun Sanyal, MD, lead investigator from the Virginia Commonwealth University, said in the release. “Reaching this critical milestone of full enrollment in the CONFIRM study is an important step forward for both Mallinckrodt and the hepatology community as there is a significant unmet medical need for these patients in the U.S. and Canada.”
Terlipressin is a potent, vasopressin analogue selective for V1a receptors that has been approved for use outside the U.S. and Canada.
The randomized control trial includes adults with liver cirrhosis and ascites with rapidly worsening renal function who have shown no response to diuretics and albumin. The primary endpoint was designed to evaluate renal function improvement, avoidance of dialysis, and short-term survival.
“We are proud to have reached full patient enrollment in the CONFIRM trial, which will support the regulatory submissions for the potential approval of terlipressin as a treatment for HRS-1 in the U.S and Canada,” Steven Romano, MD, executive vice president and chief scientific officer of Mallinckrodt, said in the release. “The ongoing CONFIRM trial and development of terlipressin are testaments to Mallinckrodt's commitment to advancing therapies for patients with rare and severe illnesses.”
The company expects top-line data to be available by the end of 2019 and anticipates submitting a new drug application in early 2020 if results are supportive.