Efficacy and Safety Study of Allogenic Mesenchymal Stem Cells for Patients With Refractory Primary Biliary Cirrhosis (MSCsTreatPBC)

August 1, 2012 updated by: Robert Chunhua Zhao, MD, PhD

Phase I Clinical Trial, Randomized, Controlled, to Evaluate the Efficacy and Safety of Therapy With Allogenic Mesenchymal Stem Cells From Bone Marrow for Patients With Refractory Primary Biliary Cirrhosis

The study is designed to evaluate the safety and efficacy of intravenous administration of bone marrow derived mesenchymal stem cells for patients with refractory primary biliary cirrhosis (PBC).

Study Overview

Status

Unknown

Conditions

Intervention / Treatment

Detailed Description

Primary biliary cirrhosis (PBC) is an organ-specific inflammatory disease and characterized by immune mediated destruction of intrahepatic bile ducts, then lead to liver cirrhosis and eventually failure.Currently, ursodeoxycholic acid (UDCA) is the only drug approved by the Food and Drug Administration (FDA). Novel treatment is urgently needed for patients who have an incomplete response to UDCA. Mesenchymal stem cells (MSC) represent a promising tool for cell-based therapies of autoimmune diseases. To explore the therapeutic effect of MSCs for PBC, the investigators plan to conduct an open-label, randomized clinical trial. Patients with PBC will be enrolled and randomly divided into two groups which will receive MSCs and UDCA respectively. The investigators will evaluate the efficacy and safety of MSCs for PBC by comparison of symptom improvement, survival rate and side effects in the two groups.

Study Type

Interventional

Enrollment (Anticipated)

20

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 60 years (Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • There must be at least two of the following: a concentration in serum of AMAs at titres of 1:40 or higher; an unexplained rise in the amount of alkaline phosphatase of at least 1•5 times the upper limit of normal for more than 24 weeks; and compatible liver histological findings, specifically non-suppurative cholangitis and interlobular bile duct injury.
  • Incomplete response to UDCA at 13-15 mg/kg/day, Criteria for the group of complete responders is including: concentrations of alkaline phosphatase less than three times the upper limit of normal, aspartate aminotransferase less than twice the upper limit of normal, and bilirubin less than 17 μmol/L;and normalisation of abnormal concentrations of bilirubin, albumin, or both.
  • Liver pathological staging in 2 or3, Histological staging is based on Ludwig's and Scheuer's classifications

Exclusion Criteria:

  • Patients are receiving any other investigational agents within 4 weeks of study entry
  • Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection (defined as invasive fungal infection and progressive CMV viremia), symptomatic congestive heart failure (NYH class III and IV), unstable angina pectoris, or cardiac arrhythmia
  • In pregnancy or lactation
  • Psychiatric illness or mental deficiency making compliance with treatment or informed consent impossible
  • HCVpositive ，HBSAg positive or with other liver diseases
  • Combined with other autoimmune disease
  • Expected survival time is less than one year
  • Decompensation of liver function（Child B or C）
  • Have a history of allergy or Allergic constitution

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: allogenic mesenchymal stem cells (MSCs)
Patients who have primary biliary cirrhosis.
Biological: Biological: mesenchymal stem cell
Mesenchymal stem cells,5-50 million/kg, Intravenous infusion, One dosage，whether to give another dosage depending on patients' condition
Other Names:
  • regenerative medicine:MSCs
Active Comparator: ursodeoxycholic acid (UDCA)
Patients who have primary biliary cirrhosis.
Drug: ursodeoxycholic acid
13-15 mg/kg/day, to the end of the study
Other Names:
  • UDCA

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
serum level of alkaline phosphatase
Time Frame: 24 months after MSCs administration
Serum level of alkaline phosphatase will be measured at entry, 1 months，3 months, 6 months and 24 months after therapy
24 months after MSCs administration

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
histological changes in liver biopsies
Time Frame: 6 months after therapy
Liver biopsy of each patient will be taken before entry into therapeutic trials and at 6 months after therapy.
6 months after therapy
Serum levels of TNF-alpha
Time Frame: 6 months after therapy
serum levels of TNF-alpha will be assessed before entry into therapeutic trials and at 6 months after therapy
6 months after therapy
changes in fatigue
Time Frame: 6 months after theraphy
changes in fatigue will be evaluated before test (baseline), 1 month,3 months and 6 months after theraphy by PBC-40 score.
6 months after theraphy
The occurrence of cirrhosis and its complications
Time Frame: 24 months after therapy
24 months after therapy
Serum levels of Interleukin
Time Frame: 6 months after therapy
serum levels of Interleukin will be assessed before entry into therapeutic trials and at 6 months after therapy
6 months after therapy
changes in pruritus severity
Time Frame: 6 months after therapy
changes in pruritus severity will be evaluated before test (baseline), 1 month,3 months and 6 months after theraphy by VAS score.
6 months after therapy

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Robert Chunhua Zhao, MD, PhD

Collaborators

Peking Union Medical College Hospital

Investigators

  • Principal Investigator: Robert Chunhua Zhao, MD,PhD, Cancer Institute and Hospital, Chinese Academy of Medical Sciences

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

November 1, 2011

Primary Completion (Anticipated)

November 1, 2013

Study Completion (Anticipated)

December 1, 2013

Study Registration Dates

First Submitted

September 20, 2011

First Submitted That Met QC Criteria

September 23, 2011

First Posted (Estimate)

September 26, 2011

Study Record Updates

Last Update Posted (Estimate)

August 3, 2012

Last Update Submitted That Met QC Criteria

August 1, 2012

Last Verified

September 1, 2011

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 2008BAI59B03/2011AA020119

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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