Research Article: Human umbilical cord-derived mesenchymal stem cells for the treatment of decompensated cirrhosis (MSC-DLC-1): a dose-escalation, phase I trial protocol
Abstract:
Introduction There are limited therapeutic options to efficiently treat patients with decompensated liver cirrhosis. This trial aims to explore the efficacy and safety of human umbilical cord-derived mesenchymal stem cells (UC-MSCs) for the treatment of patients with decompensated liver cirrhosis. Methods and analysis This study is an open-label, dose-escalation, one-armed phase I trial. A single injection of UC-MSCs will be administered in a predetermined dose in each cohort (5.0×10 7 , 1.0×10 8 , 1.5×10 8 or 2.0×10 8 cells) according to the ‘3+3’ rule. The primary evaluation measures will include the incidence of adverse events and the change in the Model for End-stage Liver Disease (MELD) score from baseline to the 28th day. Secondary evaluation measures will be evaluated at baseline and at each follow-up point. These measures will include the change in the MELD score from baseline to each follow-up point, the incidence of each complication associated with decompensated cirrhosis, liver transplant-free survival and the incidence of liver failure, among other relevant measures. All patients will be followed up for 24 months. This study will evaluate whether the use of UC-MSCs to treat patients with decompensated liver cirrhosis is safe and tolerable. Ethics and dissemination The study has been approved by the Chinese People’s Liberation Army General Hospital (Approval#: 2018-107-D-4). Once conducted, the results from the study will be published in a peer-reviewed journal. Trial registration number NCT05227846 .
Introduction:
Decompensated liver cirrhosis (DLC) is characterised by severely impaired liver function and other associated complications, such as portal hypertension, ascites, spontaneous peritonitis, coagulation dysfunction, gastrointestinal bleeding, hepatic encephalopathy and hepatorenal syndrome. 1 Current available treatment strategies can help improve the patients’ quality of life and partially reduce the complications of DLC, and include taking diuretics and following a low-sodium diet, but there are no efficient…
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