Mesenchymal Stem Cell Transplantation for Refractory Primary Biliary Cholangitis

NCT ID: NCT03668145

Last Updated: 2018-09-12

Basic Information

• Recruitment Status: UNKNOWN
• Clinical Phase: NA
• Total Enrollment: 140 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2017-11-08
• Study Completion Date: 2020-12-31

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.

Conditions

Primary Biliary Cirrhosis

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

MSC group

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.

Group Type: EXPERIMENTAL

mesenchymal stem cell transplantation or placebo infusion via peripheral vein

Intervention Type: PROCEDURE

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

control

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.

Group Type: PLACEBO_COMPARATOR

mesenchymal stem cell transplantation or placebo infusion via peripheral vein

Intervention Type: PROCEDURE

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

Interventions

mesenchymal stem cell transplantation or placebo infusion via peripheral vein

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

Intervention Type: PROCEDURE

Eligibility Criteria

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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 70 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Xijing Hospital of Digestive Diseases

OTHER

Sponsor Role: lead

Responsible Party

Han Ying

Prof. Dr.

Responsibility Role: PRINCIPAL_INVESTIGATOR

Locations

Changcun Guo

Xi'an, Shaanxi, China

Site Status: RECRUITING

Countries

China

Central Contacts

Ying Han, MD

Role: CONTACT

guochc@fmmu.edu.cn

862984771539

Facility Contacts

Changcun Guo

Role: primary

guochc@sina.com

13991900180

Other Identifiers

KY20172050-1

Identifier Type: -

Identifier Source: org_study_id