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Umbilical Cord Mesenchymal Stem Cells for Patients With Primary Biliary Cirrhosis

NCT ID: NCT01662973

Last Updated: 2013-05-31

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Clinical Phase

PHASE1/PHASE2

Total Enrollment

100 participants

Study Classification

INTERVENTIONAL

Study Start Date

2011-10-31

Study Completion Date

2013-10-31

Brief Summary

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Primary biliary cirrhosis (PBC) is a slowly progressive disease that causes substantial loss of intrahepatic bile ducts, ultimately resulting in cholestasis, advanced fibrosis, cirrhosis, liver failure and even hepatocellular carcinoma. Histologically, the disease is characterized by chronic portal inflammation with infiltration, destruction and loss of the epithelial cells in the small-sized and medium-sized bile ducts. Currently, Ursodeoxycholic acid (UDCA) in a dose of 13-15mg/kg/day is recommended as therapeutic drugs for PBC by AASLD and is approved for this indication by the U.S. Food and Drug Administration (FDA). Treatment with UDCA may delay disease progression and prolong survival free of liver transplantation. However, one out of three patients does not adequately respond to UDCA therapy and many need additional medical therapy or liver transplantation, or both. UC-MSC has been application for the treatment of several severe autoimmune diseases, such as immune thrombocytopenia, systemic lupus erythematosus, and therapy-resistant rheumatoid arthritis. In this study, the safety and efficacy of UC-MSC transplantation for PBC patients will be evaluated.

Detailed Description

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Primary biliary cirrhosis (PBC) is a slowly progressive cholestatic disease associated with the development of cirrhosis and liver failure that may justify liver transplantation. Ursodeoxycholic acid (UDCA) is currently the only drug approved specifically for the treatment of PBC. However, one out of three patients does not adequately respond to UDCA therapy and many need additional medical therapy or liver transplantation, or both.

The potential for stem cells to differentiate into biliary epithelial cells was recently confirmed. In particular, bone marrow-derived mesenchymal stem cell (BM-MSC) transplantation has been applicated in the clinic for treat several human disease such as GVHD, cardiac injury and brain injury, and displayed good tolerance and efficiency. Recently, umbilical cord-derived MSCs (UC-MSC) has also been used to treat severe autoimmune diseases, such as therapy-resistant rheumatoid arthritis and multiple sclerosis.

The purpose of this study is to learn whether and how UC-MSC can improve the disease condition in patients with primary biliary cirrhosis. This study will also look at how well UC-MSC is tolerated and its safety in PBC patients

Participants in the study will be randomly assigned to one of two treatment arms:

Arm A: Participants will receive 12 weeks of UC-MSC treatment plus UDCA. Arm B: Participants will receive 12 weeks of placebo plus UDCA. UC-MSC will be prepared according to standard procedures and is collected in plastic bags containing anticoagulant. UC-MSCs are given via i.v. under sonography monitoring. After cell therapy, patients are followed up at week 4,8,12,24,36 and 48. The evaluation of some clinical parameters such as the level of serum alkaline phosphatase (ALP), alanine aminotransferase(ALT) aspartate aminotransferase (AST) and total bilirubin (TB), prothrombin time(PT), albumin(ALB), prealbumin(PA), are detected at these time points. Mayo risk score, portal hypertension, Liver histology, MELD score and clinical symptoms were also observed simultaneously.

Conditions

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Primary Biliary Cirrhosis

Keywords

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Primary Biliary Cirrhosis Mesenchymal Stem Cells Serum alkaline phosphatase Serum Bilirubin

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Conventional plus UC-MSC treatment

Participants will receive conventional treatment plus a dose of UC-MSC from day 0 through the week 12 study visit.

Participants will then be followed until the week 48 study visit.

Group Type EXPERIMENTAL

conventional plus UC-MSC treatment

Intervention Type OTHER

Received conventional treatment and taken i.v., once per 4 week, at a dose of 1\*10E6 UC-MSC/kg body weight for 12 weeks.

Conventional plus placebo treatment

Participants will receive conventional plus placebo treatment from day 0 through the week 12 study visit. Participants will then be followed until the week 48 study visit.

Group Type PLACEBO_COMPARATOR

Conventional plus placebo treatment

Intervention Type OTHER

Received conventional treatment and taken i.v., once per 4 week, at 50 ml saline for 12 weeks

Interventions

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conventional plus UC-MSC treatment

Received conventional treatment and taken i.v., once per 4 week, at a dose of 1\*10E6 UC-MSC/kg body weight for 12 weeks.

Intervention Type OTHER

Conventional plus placebo treatment

Received conventional treatment and taken i.v., once per 4 week, at 50 ml saline for 12 weeks

Intervention Type OTHER

Eligibility Criteria

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Inclusion Criteria

1. Written informed consent
2. Primary Biliary Cirrhosis (according to the criteria defined by AASLD practice guidelines , Hepatology, 2009;50:291-308 )
3. Negative pregnancy test (female patients in fertile age)

Exclusion Criteria

1. Hepatocellular carcinoma or other Malignancies
2. Pregnant or lactating women
3. Viral Hepatitis ( HAB, HBV, HCV, et al )
4. Vital organs failure (Cardiac, Renal or Respiratory, et al)
5. Sepsis
6. Active thrombosis in the portal or hepatic veins
Minimum Eligible Age

18 Years

Maximum Eligible Age

65 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Beijing 302 Hospital

OTHER

Sponsor Role lead

Responsible Party

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Fu-Sheng Wang

Director of both the Research Center for Biological Therapy and the Beijing Institute of Translational Hepatology

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Fu-Sheng Wang, professor

Role: PRINCIPAL_INVESTIGATOR

Beijing 302 Hospital

Locations

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Beijing 302 Hospital

Beijing, Beijing Municipality, China

Site Status RECRUITING

Countries

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China

Central Contacts

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Fu-Sheng Wang, professor

Role: CONTACT

Phone: 86-10-63879735

Email: [email protected]

Zheng Zhang, Doctor

Role: CONTACT

Phone: 86-10-63879735

Email: [email protected]

Facility Contacts

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Fu-Sheng Wang, professor

Role: primary

Lifeng Wang, Doctor

Role: backup

References

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Poupon RE, Lindor KD, Cauch-Dudek K, Dickson ER, Poupon R, Heathcote EJ. Combined analysis of randomized controlled trials of ursodeoxycholic acid in primary biliary cirrhosis. Gastroenterology. 1997 Sep;113(3):884-90. doi: 10.1016/s0016-5085(97)70183-5.

Reference Type BACKGROUND
PMID: 9287980 (View on PubMed)

Kuiper EM, Hansen BE, de Vries RA, den Ouden-Muller JW, van Ditzhuijsen TJ, Haagsma EB, Houben MH, Witteman BJ, van Erpecum KJ, van Buuren HR; Dutch PBC Study Group. Improved prognosis of patients with primary biliary cirrhosis that have a biochemical response to ursodeoxycholic acid. Gastroenterology. 2009 Apr;136(4):1281-7. doi: 10.1053/j.gastro.2009.01.003. Epub 2009 Jan 14.

Reference Type BACKGROUND
PMID: 19208346 (View on PubMed)

Poupon R. Primary biliary cirrhosis: a 2010 update. J Hepatol. 2010 May;52(5):745-58. doi: 10.1016/j.jhep.2009.11.027. Epub 2010 Feb 18.

Reference Type BACKGROUND
PMID: 20347176 (View on PubMed)

Silveira MG, Brunt EM, Heathcote J, Gores GJ, Lindor KD, Mayo MJ. American Association for the Study of Liver Diseases endpoints conference: design and endpoints for clinical trials in primary biliary cirrhosis. Hepatology. 2010 Jul;52(1):349-59. doi: 10.1002/hep.23637. No abstract available.

Reference Type BACKGROUND
PMID: 20578151 (View on PubMed)

Other Identifiers

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Beijing302-005

Identifier Type: -

Identifier Source: org_study_id