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

Phase Ⅰ/Ⅱ Study of Human Umbilical Cord Derived Mesenchymal Stem Cells (UC-MSCs) for Treatment of Liver Failure

Sponsors

Lead Sponsor: Beijing 302 Hospital

Source 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.

Overall Status Unknown status
Start Date March 2009
Completion Date March 2014
Primary Completion Date March 2014
Phase Phase 1/Phase 2
Study Type Interventional
Primary Outcome
Measure Time Frame
The levels of serum Total Protein and Albumin 2 years after treatment
Secondary Outcome
Measure Time Frame
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
The level of alpha-fetoprotein (AFP) 2 years after treatment
The content of ascites 2 years after treatment
Survival rate and time 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
Enrollment 70
Condition
Intervention

Intervention Type: Drug

Intervention Name: Conventional plus MSC treatment

Description: Participants received conventional treatment and taken i.v., once per 4 week, at a dose of 0.5*10E6 MSC/kg body for 12 weeks.

Arm Group Label: Conventional plus MSC treatment

Intervention Type: Drug

Intervention Name: Conventional plus pacebo treatment

Description: Participants received conventional treatment and taken i.v., once per 4 week, at 50 ml saline for 12 weeks.

Arm Group Label: Conventional plus pacebo treatment

Eligibility

Criteria:

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

Gender: All

Minimum Age: 18 Years

Maximum Age: 70 Years

Healthy Volunteers: No

Overall Official
Last Name Role Affiliation
Fu-Sheng Wang, Professor Principal Investigator Beijing 302 Hospital
Overall Contact

Last Name: Fu-Sheng Wang, Professor

Phone: 86-10-63879735

Phone Ext.: 2015.12

Email: [email protected]

Location
Facility: Status: Contact: Beijing 302 Hospital Ming Shi, Professor 86-10-63879735 2015.12 [email protected]
Location Countries

China

Verification Date

May 2013

Responsible Party

Type: Principal Investigator

Investigator Affiliation: Beijing 302 Hospital

Investigator Full Name: Fu-Sheng Wang

Investigator Title: Director

Keywords
Has Expanded Access No
Condition Browse
Number Of Arms 2
Arm Group

Label: Conventional plus MSC treatment

Type: Experimental

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

Label: Conventional plus pacebo treatment

Type: Experimental

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

Study Design Info

Allocation: Randomized

Intervention Model: Parallel Assignment

Primary Purpose: Treatment

Masking: Double (Participant, Care Provider)

Source: ClinicalTrials.gov