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Mesenchymal Stem Cell Transplantation for Refractory Primary Biliary Cholangitis

Primary Purpose

Primary Biliary Cirrhosis

Status
Unknown status
Phase
Not Applicable
Locations
China
Study Type
Interventional
Intervention
mesenchymal stem cell transplantation or placebo infusion via peripheral vein
Sponsored by
Xijing Hospital of Digestive Diseases
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Primary Biliary Cirrhosis focused on measuring Primary Biliary Cirrhosis (now termed as primary biliary cholangitis), mesenchymal stem cell,

Eligibility Criteria

18 Years - 70 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  1. informed consent
  2. Age between 18-70 years old
  3. BMI between 17-28
  4. Meeting at least 2 of the 3 criteria: (1) positive for anti-mitochondial antibody (AMA)；(2) Elevated ALP or GGT；(3) PBC diagnosis confirmed by liver biopsy
  5. Serum ALP >/=1.67 ULN after at leat 6 months treatment with UDCA 1(3-17mg/kg/day) -

Exclusion Criteria:

  1. Pregnancy, breast-feeding females
  2. Use of liver-toxic drugs over 2 week within 6 months prior to recruitment；
  3. refractory variceal bleeding, uncontrolled hepatoencephalopathy or uncontrolled ascites.
  4. Acute of chronic kidney failure.
  5. Commodities with other liver diseases such as viral hepatitis, alcoholic liver disease, auto-immune hepatitis or liver cancer
  6. Severe cardiovascular disease；
  7. liver transplantation listed patients；
  8. ALT/AST over 5xULN，or total bilirubin >85umol/l
  9. anticipated need for liver transplantation within 1 year according to mayo risk score
  10. Other candidates who are judged to be not applicable to this study by doctors

Sites / Locations

  • Changcun GuoRecruiting

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

MSC group

control

Arm Description

mesenchymal stem cell transplantation via peripheral vein: 0.1-1x10E6 MSCs/kg body weight administered via peripheral vein at week 0, 4, 8 plus UDCA.

placebo infusions (placebo infusion differs from the experimental infusion in only that placebo has no mesenchymal stem cells) will be administered via peripheral vein at week 0, 4, 8 plus UDCA.

Outcomes

Primary Outcome Measures

Change of alkaline phosphatase (ALP)
The absolute value change of ALP after 1 year of the initial stem cell treatment

Secondary Outcome Measures

Change of other liver function indices
The absolute value change of total bilirubin, ALT, AST, GGT after initial stem cell treatment
Change of liver histology
histological scores assessed by liver biopsy at baseline and after treatment

Full Information

First Posted
September 10, 2018
Last Updated
September 10, 2018
Sponsor
Xijing Hospital of Digestive Diseases
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1. Study Identification

Unique Protocol Identification Number
NCT03668145
Brief Title
Mesenchymal Stem Cell Transplantation for Refractory Primary Biliary Cholangitis
Official Title
Mesenchymal Stem Cell Transplantation for Refractory Primary Biliary Cholangitis, a Randomized Double-blind Placebo Controlled Trial
Study Type
Interventional

2. Study Status

Record Verification Date
September 2018
Overall Recruitment Status
Unknown status
Study Start Date
November 8, 2017 (Actual)
Primary Completion Date
December 31, 2019 (Anticipated)
Study Completion Date
December 31, 2020 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Xijing Hospital of Digestive Diseases

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

Brief Summary
Ursodeoxycholic acid is the mainstay treatment medicine for primary biliary cholangitis(PBC). About 1/3 of the patients do not respond to UDCA, which is defined as refractory PBC. Mesenchymal stem cells (MSC) has been reported to improve the outcomes of PBC patients. Randomization controlled studies are needed to confirm the long term effect of MSC treatment for refractory PBC. This study aimed to investigate the safety and efficacy of mesenchymal stem cells in PBC patients that do not respond to UDCA treatment. This study is an double-blind multicenter randomized and placebo-controlled study. Patients with with refractory PBC will be randomly assigned to receive MSC treatment plus UDCA or UDCA alone (control). Three times of MSC infusion (0.1-1x10E6 cells/kg body weight) via peripheral vein will be given to the experimental group (once in 4 weeks). The primary outcome is absolute change in alkaline phosphatase. Secondary outcomes are changes of other liver function indices such as ALT TBIL AST GGT, improve of symptoms and liver histology.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Primary Biliary Cirrhosis
Keywords
Primary Biliary Cirrhosis (now termed as primary biliary cholangitis), mesenchymal stem cell,

7. Study Design

Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Masking Description
Randomization will be blinded to participant, care provider, investigator and outcome assessors.
Allocation
Randomized
Enrollment
140 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
MSC group
Arm Type
Experimental
Arm Description
mesenchymal stem cell transplantation via peripheral vein: 0.1-1x10E6 MSCs/kg body weight administered via peripheral vein at week 0, 4, 8 plus UDCA.
Arm Title
control
Arm Type
Placebo Comparator
Arm Description
placebo infusions (placebo infusion differs from the experimental infusion in only that placebo has no mesenchymal stem cells) will be administered via peripheral vein at week 0, 4, 8 plus UDCA.
Intervention Type
Procedure
Intervention Name(s)
mesenchymal stem cell transplantation or placebo infusion via peripheral vein
Intervention Description
0.1-1x10E6 MSCs/kg body weight will be administered via peripheral vein for 3 times at week 0, 4 and 8 mesenchymal stem cell mesenchymal stem cell infusion produced by Cell products of National Engineering Research Center(short for CPNERC), Tianjin, China For control group, placebo infusions will be given to the participants at the same time points
Primary Outcome Measure Information:
Title
Change of alkaline phosphatase (ALP)
Description
The absolute value change of ALP after 1 year of the initial stem cell treatment
Time Frame
1 year
Secondary Outcome Measure Information:
Title
Change of other liver function indices
Description
The absolute value change of total bilirubin, ALT, AST, GGT after initial stem cell treatment
Time Frame
1 year
Title
Change of liver histology
Description
histological scores assessed by liver biopsy at baseline and after treatment
Time Frame
1 year

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
70 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: informed consent Age between 18-70 years old BMI between 17-28 Meeting at least 2 of the 3 criteria: (1) positive for anti-mitochondial antibody (AMA)；(2) Elevated ALP or GGT；(3) PBC diagnosis confirmed by liver biopsy Serum ALP >/=1.67 ULN after at leat 6 months treatment with UDCA 1(3-17mg/kg/day) - Exclusion Criteria: Pregnancy, breast-feeding females Use of liver-toxic drugs over 2 week within 6 months prior to recruitment； refractory variceal bleeding, uncontrolled hepatoencephalopathy or uncontrolled ascites. Acute of chronic kidney failure. Commodities with other liver diseases such as viral hepatitis, alcoholic liver disease, auto-immune hepatitis or liver cancer Severe cardiovascular disease； liver transplantation listed patients； ALT/AST over 5xULN，or total bilirubin >85umol/l anticipated need for liver transplantation within 1 year according to mayo risk score Other candidates who are judged to be not applicable to this study by doctors
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Ying Han, MD
Phone
862984771539
Email
guochc@fmmu.edu.cn
Facility Information:
Facility Name
Changcun Guo
City
Xi'an
State/Province
Shaanxi
ZIP/Postal Code
710032
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Changcun Guo
Phone
13991900180
Email
guochc@sina.com

12. IPD Sharing Statement

Plan to Share IPD
Yes
IPD Sharing Time Frame
The data will be available after the completion of the study and before publications for 1 year

Learn more about this trial

Mesenchymal Stem Cell Transplantation for Refractory Primary Biliary Cholangitis

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