Mesenchymal Stem Cell Transplantation for Refractory Primary Biliary Cholangitis

NCT03668145 | Unknown

• 1 other identifier: KY20172050-1
• Study Type: interventional
• Target: 140
• Locations: 1 country
• Sites: 1

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

Keywords

Primary Biliary Cirrhosis (now termed as primary biliary cholangitis), mesenchymal stem cell,

Outcome Measures

Primary Outcomes (1)

• Change of alkaline phosphatase (ALP)

  The absolute value change of ALP after 1 year of the initial stem cell treatment

  1 year

Secondary Outcomes (2)

• Change of other liver function indices

  1 year

• Change of liver histology

  1 year

Study Arms (2)

MSC group

EXPERIMENTAL

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.

Procedure: mesenchymal stem cell transplantation or placebo infusion via peripheral vein

control

PLACEBO COMPARATOR

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.

Procedure: mesenchymal stem cell transplantation or placebo infusion via peripheral vein

Interventions

mesenchymal stem cell transplantation or placebo infusion via peripheral vein 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

MSC group; control

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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) -

You may not qualify if:

• 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

Sponsors & Collaborators

• Xijing Hospital of Digestive Diseases: lead

Study Sites (1)

Changcun Guo

Xi'an, Shaanxi, 710032, China

RECRUITING

MeSH Terms

Conditions

Liver Cirrhosis, Biliary

Interventions

Mesenchymal Stem Cell Transplantation

Condition Hierarchy (Ancestors)

Cholestasis, Intrahepatic; Cholestasis; Bile Duct Diseases; Biliary Tract Diseases; Digestive System Diseases; Liver Diseases; Liver Cirrhosis; Fibrosis; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Stem Cell Transplantation; Cell Transplantation; Cell- and Tissue-Based Therapy; Biological Therapy; Therapeutics; Transplantation; Surgical Procedures, Operative

Central Study Contacts

Ying Han, MD

CONTACT

862984771539; guochc@fmmu.edu.cn

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Randomization will be blinded to participant, care provider, investigator and outcome assessors.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Prof. Dr.

Study Record Dates

• First Submitted: September 10, 2018
• First Posted: September 12, 2018
• Study Start: November 8, 2017
• Primary Completion: December 31, 2019
• Study Completion: December 31, 2020
• Last Updated: September 12, 2018

Record last verified: 2018-09

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL
• Time Frame: The data will be available after the completion of the study and before publications for 1 year

Locations

CN (1)