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

Beijing 302 Hospital

Status and phase: Unknown; Phase 2; Phase 1

Conditions: Liver Failure; Mesenchymal Stem Cells

Treatments: Drug: Conventional plus MSC treatment; Drug: Conventional plus pacebo treatment

Study type: Interventional

Funder types: Other

Identifiers

NCT01218464

Beijing302-003

Details and patient eligibility

About

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.

Full 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.

Enrollment: 70 estimated patients

Sex: All

Ages: 18 to 70 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

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70 participants in 2 patient groups

Conventional plus MSC treatment

Experimental group

Description:

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

Treatment:

Drug: Conventional plus MSC treatment

Conventional plus pacebo treatment

Experimental group

Description:

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

Treatment:

Drug: Conventional plus pacebo treatment