Umbilical Cord Mesenchymal Stem Cells in Primary Sclerosing Cholangitis

Study Overview
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Brief Summary
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Primary sclerosing cholangitis (PSC) is an idiopathic condition with intrahepatic cholangitis and fibrosis, leading to multifocal bile duct stricture. Its main clinical manifestations are chronic cholestatic lesions and is deemed as autoimmune liver disease. PSC are immune abnormalities that occurs in patients with genetic susceptibility. No other pathogenesis is revealed yet. Ursodeoxycholic acid is used as an empirical treatment, and there is no approved drug or a acceptable treatment regimen. The disease often progresses to liver decompensation and requires liver transplantation. In recent years, the clinical application of stem cell therapy has seen many important advances. Stem cells are characterized with properties of multiple differentiation, repair of damaged tissue and immuno-modulation. This study aims to employ UCMSCs to treat PSC patients and observe its efficacy and safety, and to explore the possible therapeutic mechanisms.

Official Title
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Intra-arterial Injection of Umbilical Cord Mesenchymal Stem Cells in Primary Sclerosing Cholangitis

Conditions
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Primary Sclerosing Cholangitis

Intervention / Treatment
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* Drug: UCMSC
* Drug: UDCA


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: progressive PSC willing to give consent Exclusion Criteria: decompensated liver cirrhosis total serum bilirubin >5ULN refractory ascites serum creatinine >1.5mg/dL Cirrhotic nodules with malignant tendencies primary biliary cholangitis IgG4-associated sclerosing cholangitis non-PSC induced bile duct stones biliary tract trauma recurrent suppurative cholangitis neoplastic disease pancreatic disease

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 65 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: UCMSC<br>infusion of aUCMSC and Ursodeoxycholic acid therapy | Drug: UCMSC<br>* infusion of aUCMSC at day 0, 7, 14, and 21<br>Drug: UDCA<br>* oral administration of ursodeoxycholic acid (UDCA) 15mg/kg/d<br>|
| Active Comparator: UDCA<br>Ursodeoxycholic acid therapy 15mg/kg/d | Drug: UDCA<br>* oral administration of ursodeoxycholic acid (UDCA) 15mg/kg/d<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| adverse effects regarding UCMSC infusion |  | 1 year | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Pathological score of liver inflammation |  | 1 year | 
| Changes of biliary lesions in magnetic resonance image | softening in stiffness of bile duct | 1 year | 
| alanine aminotransferase |  | 1 year |