Primary Biliary Cirrhosis
Conditions
Keywords
Primary Biliary Cirrhosis (now termed as primary biliary cholangitis), mesenchymal stem cell,
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.
Interventions
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
Sponsors
Xijing Hospital of Digestive Diseases
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Masking description
Randomization will be blinded to participant, care provider, investigator and outcome assessors.
Eligibility
Sex/Gender
ALL
Age
18 Years to 70 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change of alkaline phosphatase (ALP) | 1 year | The absolute value change of ALP after 1 year of the initial stem cell treatment |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| 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 |
Countries
China
Contacts
Primary ContactYing Han, MD
Outcome results
None listed