NCT06740149

Efficacy and Safety of BMSCs (CG-BM1) for ACLF Patients

Efficacy and Safety of Allogeneic Human Bone Marrow Mesenchymal Stem Cells for the Treatment of Patients with Acute-on-chronic Liver Failure

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 90 (estimated)
• Sponsor: Sun Yat-sen University · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The goal of this clinical trial is to learn if CG-BM1 Allogeneic Human Bone Marrow Mesenchymal Stem Cell Injection (hereinafter referred to as CG-BM1) can treat acute-on-chronic liver failure (ACLF) patients. Main purposes of this clinical trial are: * To evaluate the safety and tolerability of CG-BM1 for the treatment of adult patients with ACLF. * To observe the preliminary effectiveness of CG-BM1 in treating adult ACLF patients, and to provide a basis for subsequent clinical trial protocol design.

Detailed description

CG-BM1 is a bone marrow mesenchymal stem cell product independently developed by Guangzhou Cellgenes Biotechnology Co., Ltd. The active ingredient of CG-BM1 is human bone marrow mesenchymal stem cell, which is derived from bone marrow donated by healthy adults and prepared into stem cell injection under aseptic conditions. CG-BM1 is the first bone marrow mesenchymal stem cell (BMSC) therapeutic drug for the treatment of ACLF in China. Preclinical data showed that CG-BM1 has the ability to immunomodulate, inhibit the secretion of pro-inflammatory factors by immune cells, and up-regulate the level of anti-inflammatory factors, which can significantly improve the liver function, reduce the inflammatory response, and reverse hepatic fibrosis in ACLF animal models, and the results of the study suggest that it is safe and effective, supporting its further clinical development. The purpose of this study was to evaluate the safety and tolerability of CG-BM1 for the treatment of patients with ACLF, as well as to evaluate the preliminary efficacy of CG-BM1 for the treatment of patients with ACLF. The study was divided into 2 phases, the first with an open-labeled, dose-escalation design; the second with a multicenter, randomized, double-blind, placebo-controlled design. Phase I: Patients was divided into three dose groups using a traditional "3+3" design. 3-6 subjects were enrolled in each dose. Phase II: Multiple-dose, randomized, double-blind, placebo-controlled trial. Based on the results of the phase I trial, two dose groups were selected for phase II. A total of 90 subjects were enrolled and randomized 1:1:1. The experiment group received CG-BM1 + conventional treatment regimen, and the control group received placebo + conventional treatment regimen.

Conditions

• Acute-On-Chronic Liver Failure

Interventions

Type	Name	Description
DRUG	Bone marrow mesenchymal stem cells (low dose)	Administered intravenously. For phase I: single infusion, 1.0×10\^6 cells/kg. For phase II: 1.0×10\^6 cells /kg once a week for a total of 4 times.
DRUG	Solvent of CG-BM1	Administered intravenously, once a week for a total of 4 doses.
DRUG	Bone marrow mesenchymal stem cells (medium dose)	Administered intravenously. For phase I: single infusion, 2.0×10\^6 cells /kg. For phase II: 2.0×10\^6 cells /kg once a week for a total of 4 times.
DRUG	Bone marrow mesenchymal stem cells (high dose)	Administered intravenously. For phase I: single infusion, 4×10\^6 cells /kg.

Timeline

• Start date: 2023-03-01
• Primary completion: 2025-10-31
• Completion: 2026-01-31
• First posted: 2024-12-18
• Last updated: 2024-12-18

Locations

1 site across 1 country: China