Umbilical Cord Mesenchymal Stem Cells for Patients With Primary Biliary Cirrhosis
Phase 1/2 Study of UC-MSC Treatment for Evaluation the Efficacy and Safety in Patients With Primary Biliary Cirrhosis
1 other identifier
interventional
100
1 country
1
Brief Summary
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for phase_1
Started Oct 2011
Typical duration for phase_1
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
October 1, 2011
CompletedFirst Submitted
Initial submission to the registry
August 5, 2012
CompletedFirst Posted
Study publicly available on registry
August 13, 2012
CompletedPrimary Completion
Last participant's last visit for primary outcome
October 1, 2013
CompletedStudy Completion
Last participant's last visit for all outcomes
October 1, 2013
CompletedMay 31, 2013
May 1, 2013
2 years
August 5, 2012
May 30, 2013
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Serum alkaline phosphatase (ALP)
0, 4, 8,12, 24, 36,48 weeks after treatment
Secondary Outcomes (7)
Histological changes in liver biopsies
baseline and 48 weeks
Serum Bilirubin
At base line and at week 4,8,12,24,36 and 48
Serum AST
At base line and at week 4,8,12,24,36 and 48
Mayo risk score
At base line and at week 4,8,12,24,36 and 48
Number of patients with Portal Hypertension after 12 weeks treatment
At base line and at week 12,24,36 and 48
- +2 more secondary outcomes
Study Arms (2)
Conventional plus UC-MSC treatment
EXPERIMENTALParticipants 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.
Conventional plus placebo treatment
PLACEBO COMPARATORParticipants 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.
Interventions
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.
Received conventional treatment and taken i.v., once per 4 week, at 50 ml saline for 12 weeks
Eligibility Criteria
You may qualify if:
- Written informed consent
- Primary Biliary Cirrhosis (according to the criteria defined by AASLD practice guidelines , Hepatology, 2009;50:291-308 )
- Negative pregnancy test (female patients in fertile age)
You may not qualify if:
- Hepatocellular carcinoma or other Malignancies
- Pregnant or lactating women
- Viral Hepatitis ( HAB, HBV, HCV, et al )
- Vital organs failure (Cardiac, Renal or Respiratory, et al)
- Sepsis
- Active thrombosis in the portal or hepatic veins
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (1)
Beijing 302 Hospital
Beijing, Beijing Municipality, 100039, China
Related Publications (4)
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.
PMID: 9287980BACKGROUNDKuiper 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.
PMID: 19208346BACKGROUNDPoupon 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.
PMID: 20347176BACKGROUNDSilveira 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.
PMID: 20578151BACKGROUND
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Fu-Sheng Wang, professor
Beijing 302 Hospital
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- OTHER
- Responsible Party
- PRINCIPAL INVESTIGATOR
- PI Title
- Director of both the Research Center for Biological Therapy and the Beijing Institute of Translational Hepatology
Study Record Dates
First Submitted
August 5, 2012
First Posted
August 13, 2012
Study Start
October 1, 2011
Primary Completion
October 1, 2013
Study Completion
October 1, 2013
Last Updated
May 31, 2013
Record last verified: 2013-05