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Trial record 52 of 55 for:    stem cell arthritis AND stem cell transplantation

Umbilical Cord Mesenchymal Stem Cells for Patients With Primary Biliary Cirrhosis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01662973
Recruitment Status : Unknown
Verified May 2013 by Fu-Sheng Wang, Beijing 302 Hospital.
Recruitment status was:  Recruiting
First Posted : August 13, 2012
Last Update Posted : May 31, 2013
Sponsor:
Information provided by (Responsible Party):
Fu-Sheng Wang, Beijing 302 Hospital

Tracking Information
First Submitted Date  ICMJE August 5, 2012
First Posted Date  ICMJE August 13, 2012
Last Update Posted Date May 31, 2013
Study Start Date  ICMJE October 2011
Estimated Primary Completion Date October 2013   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: August 9, 2012)
Serum alkaline phosphatase (ALP) [ Time Frame: 0, 4, 8,12, 24, 36,48 weeks after treatment ]
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT01662973 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: August 9, 2012)
  • Histological changes in liver biopsies [ Time Frame: baseline and 48 weeks ]
  • Serum Bilirubin [ Time Frame: At base line and at week 4,8,12,24,36 and 48 ]
  • Serum AST [ Time Frame: At base line and at week 4,8,12,24,36 and 48 ]
  • Mayo risk score [ Time Frame: At base line and at week 4,8,12,24,36 and 48 ]
  • Number of patients with Portal Hypertension after 12 weeks treatment [ Time Frame: At base line and at week 12,24,36 and 48 ]
  • MELD score [ Time Frame: At base line and at week 4,8,12,24,36 and 48 ]
  • Number of participants with improvement of clinical symptoms [ Time Frame: At base line and at week 4,8,12,24,36 and 48 ]
    clinical symptoms including fatigue (Fatigue Impact Score, FIS) and pruritus ( Visual Analog Scale ,VAS)
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Umbilical Cord Mesenchymal Stem Cells for Patients With Primary Biliary Cirrhosis
Official Title  ICMJE Phase 1/2 Study of UC-MSC Treatment for Evaluation the Efficacy and Safety in Patients With Primary Biliary Cirrhosis
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.
Detailed Description

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.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Primary Biliary Cirrhosis
Intervention  ICMJE
  • Other: 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.
  • Other: Conventional plus placebo treatment
    Received conventional treatment and taken i.v., once per 4 week, at 50 ml saline for 12 weeks
Study Arms  ICMJE
  • Experimental: 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.

    Intervention: Other: conventional plus UC-MSC treatment
  • Placebo Comparator: 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.
    Intervention: Other: Conventional plus placebo treatment
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Unknown status
Estimated Enrollment  ICMJE
 (submitted: August 9, 2012)
100
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE October 2013
Estimated Primary Completion Date October 2013   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 65 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE China
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01662973
Other Study ID Numbers  ICMJE Beijing302-005
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Fu-Sheng Wang, Beijing 302 Hospital
Study Sponsor  ICMJE Beijing 302 Hospital
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Fu-Sheng Wang, professor Beijing 302 Hospital
PRS Account Beijing 302 Hospital
Verification Date May 2013

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP