Mesenchymal Stem Cell Transplantation for Refractory Primary Biliary Cholangitis

Not Applicable

• Conditions: Primary Biliary Cirrhosis
• Interventions: Procedure: mesenchymal stem cell transplantation or placebo infusion via peripheral vein

• Registration Number: NCT03668145
• Lead Sponsor: Xijing Hospital of Digestive Diseases

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2017-11-08
• Status Verified: 2018-09
• Study First Submitted: 2018-09-10
• Study First Submitted QC: 2018-09-10
• Last Update Submitted: 2018-09-10
• Study First Posted: 2018-09-12
• Last Update Posted: 2018-09-12
• Primary Completion: 2019-12-31
• Study Completion: 2020-12-31

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 140

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
MSC group	mesenchymal stem cell transplantation or placebo infusion via peripheral vein	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.
control	mesenchymal stem cell transplantation or placebo infusion via peripheral vein	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.

Primary Outcome Measures

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

Secondary Outcome Measures

Name	Time	Method
Change of other liver function indices	1 year	The absolute value change of total bilirubin, ALT, AST, GGT after initial stem cell treatment
Change of liver histology	1 year	histological scores assessed by liver biopsy at baseline and after treatment

Trial Locations

Locations (1)

Changcun Guo; 🇨🇳 Xi'an, Shaanxi, China