Safety and Efficacy of Human Mesenchymal Stem Cells for Treatment of Liver Failure

Phase 1

• Conditions: Liver Failure; Mesenchymal Stem Cells
• Interventions: Drug: Conventional plus MSC treatment; Drug: Conventional plus pacebo treatment

• Registration Number: NCT01218464
• Lead Sponsor: Beijing 302 Hospital

Brief Summary

Liver failure (LF) is a dramatic clinical syndrome with massive necrosis of liver cells. and liver transplantation is the only available therapeutic option for patients suffering with this condition. However, lack of donors, surgical complications, rejection, and high cost are serious problems. Previous study showed that bone marrow derived mesenchymal stem cells (BM-MSCs) replace hepatocytes in injured liver, and effectively rescue experimental liver failure and contribute to liver regeneration. In this study, the patients with LF will undergo administration of human umbilical cord mesenchymal stem cells (UC-MSCs) via peripheral vein transfusion to evaluate the safty and efficacy of UC-MSCs treatment for these patients.

Detailed Description

Liver failure (LF) is a severe life-threatening condition, and is a dramatic clinical syndrome with massive necrosis of liver cells, and liver transplantation is the only available therapeutic option for patients suffering with this condition. However, lack of donors, surgical complications, rejection, and high cost are serious problems. Since current therapeutic options for LF that is usually with extremely poor prognosis are still limited, recent studies indicate that mesenchymal stem cells (MSCs), due to their function in immune modulation and liver-damage repair, are of great therapeutic potential for this disease. Previous study showed that bone marrow derived mesenchymal stem cells (BM-MSCs) replace hepatocytes in injured liver, and effectively rescue experimental liver failure and contribute to liver regeneration.The purpose of this study is to investigate the safety and initial efficacy of human umbilical cord MSC (UC-MSCs) treatment for patients with LF. In this study, MSCs were isolated from umbilical cord and generated in appropriate growth medium. 50 LF patients with LF received i.v. transfusion of 0.5-1.0×106 cells/kg of MSCs as the treated group and other 20 LF patients with LF were transfused with placebo without MSCs as control group. All 70 of them received the routine management for liver failure. During the 2-year follow up, the evaluation of safty and efficacy will be undergone to help to establish innovative cell-based therapies for the treatment of diseases.

Study Timeline

• Study Start: 2009-03
• Study First Submitted: 2010-10-08
• Study First Submitted QC: 2010-10-08
• Study First Posted: 2010-10-11
• Status Verified: 2013-05
• Last Update Submitted: 2013-05-30
• Last Update Posted: 2013-05-31
• Primary Completion: 2014-03
• Study Completion: 2014-03

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 70

Inclusion Criteria

1. Aged 18-70 years
2. Liver failure
3. Negative pregnancy test (female patients in fertile age)
4. Written consent

Exclusion Criteria

1. Hepatocellular carcinoma or other malignancies
2. Severe problems in other vital organs(e.g.the heart,renal or lungs)
3. Pregnant or lactating women
4. Severe bacteria infection
5. Anticipated with difficulty of follow-up observation
6. Other candidates who are judged to be not applicable to this study by doctors

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Conventional plus MSC treatment	Conventional plus MSC treatment	Participants will receive conventional treatment plus a dose of MSC from day 0 through the week 12 study visit. Participants will then be followed until 2 years study visit
Conventional plus pacebo treatment	Conventional plus pacebo treatment	Participants will receive conventional plus placebo treatment from day 0 through the week 12 study visit. Participants will then be followed until 2 years study visit

Primary Outcome Measures

Name	Time	Method
The levels of serum Total Protein and Albumin	2 years after treatment

Secondary Outcome Measures

Name	Time	Method
Survival rate and time	2 years after treatment
The level of alpha-fetoprotein (AFP)	2 years after treatment
The content of ascites	2 years after treatment
The levels of serum Total Bilirubin and Direct Bilirubin	2 years after treatment
The levels of serum Alanine Aminotransferase (ALT), Aspartate Aminotransferase (AST) and Cholinesterase (CHE)	2 years after treatment
Body temperature, tetter and allergy	Between 0 to 24 hours after UC-MSCs transfusion
The levels of Prothrombin Activity (PA) and Prothrombin Time (PT)	2 years after treatment
The score for Model for End-Stage Liver Disease	2 years after treatment

Trial Locations

Locations (1)

Beijing 302 Hospital; 🇨🇳 Beijing, Beijing, China