Mesenchymal Stem Cell Transplantation for Refractory Primary Biliary Cholangitis

Primary Biliary Cirrhosis Clinical Trial

Official title:

Mesenchymal Stem Cell Transplantation for Refractory Primary Biliary Cholangitis, a Randomized Double-blind Placebo Controlled Trial

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT03668145
• Other study ID #: KY20172050-1
• Status: Recruiting
• Phase: N/A
• Start date: November 8, 2017
• Est. completion date: December 31, 2020

Study information

• Verified date: September 2018
• Source: Xijing Hospital of Digestive Diseases
• Contact: Ying Han, MD
• Phone: 862984771539
• Email: guochc[@]fmmu.edu.cn
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 140
• Est. completion date: December 31, 2020
• Est. primary completion date: December 31, 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 70 Years

Eligibility

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

Related Conditions & MeSH terms

• Cholangitis
• Fibrosis
• Liver Cirrhosis, Biliary
• Primary Biliary Cirrhosis

Intervention

Procedure:
• 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

Locations

Country	Name	City	State
China	Changcun Guo	Xi'an	Shaanxi

Sponsors (1)

Lead Sponsor	Collaborator
Xijing Hospital of Digestive Diseases

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change of alkaline phosphatase (ALP)	The absolute value change of ALP after 1 year of the initial stem cell treatment	1 year
Secondary	Change of other liver function indices	The absolute value change of total bilirubin, ALT, AST, GGT after initial stem cell treatment	1 year
Secondary	Change of liver histology	histological scores assessed by liver biopsy at baseline and after treatment	1 year