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Trial record 22 of 806 for:    Interventional Studies | mesenchymal

Mesenchymal Stem Cell Transplantation for Refractory Primary Biliary Cholangitis

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ClinicalTrials.gov Identifier: NCT03668145
Recruitment Status : Recruiting
First Posted : September 12, 2018
Last Update Posted : September 12, 2018
Sponsor:
Information provided by (Responsible Party):
Han Ying, Xijing Hospital of Digestive Diseases

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.

Condition or disease Intervention/treatment Phase
Primary Biliary Cirrhosis Procedure: mesenchymal stem cell transplantation or placebo infusion via peripheral vein Not Applicable

Study Type : Interventional
Estimated Enrollment : 140 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking Description: Randomization will be blinded to participant, care provider, investigator and outcome assessors.
Primary Purpose: Treatment
Official Title: Mesenchymal Stem Cell Transplantation for Refractory Primary Biliary Cholangitis, a Randomized Double-blind Placebo Controlled Trial
Actual Study Start Date : November 8, 2017
Estimated Primary Completion Date : December 31, 2019
Estimated Study Completion Date : December 31, 2020


Arm Intervention/treatment
Experimental: 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.
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

Placebo Comparator: 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.
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




Primary Outcome Measures :
  1. Change of alkaline phosphatase (ALP) [ Time Frame: 1 year ]
    The absolute value change of ALP after 1 year of the initial stem cell treatment


Secondary Outcome Measures :
  1. Change of other liver function indices [ Time Frame: 1 year ]
    The absolute value change of total bilirubin, ALT, AST, GGT after initial stem cell treatment

  2. Change of liver histology [ Time Frame: 1 year ]
    histological scores assessed by liver biopsy at baseline and after treatment



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Ages Eligible for Study:   18 Years to 70 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03668145


Contacts
Contact: Ying Han, MD 862984771539 guochc@fmmu.edu.cn

Locations
China, Shaanxi
Changcun Guo Recruiting
Xi'an, Shaanxi, China, 710032
Contact: Changcun Guo    13991900180    guochc@sina.com   
Sponsors and Collaborators
Xijing Hospital of Digestive Diseases

Responsible Party: Han Ying, Prof. Dr., Xijing Hospital of Digestive Diseases
ClinicalTrials.gov Identifier: NCT03668145     History of Changes
Other Study ID Numbers: KY20172050-1
First Posted: September 12, 2018    Key Record Dates
Last Update Posted: September 12, 2018
Last Verified: September 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Supporting Materials: Study Protocol
Time Frame: The data will be available after the completion of the study and before publications for 1 year

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Han Ying, Xijing Hospital of Digestive Diseases:
Primary Biliary Cirrhosis (now termed as primary biliary cholangitis), mesenchymal stem cell,

Additional relevant MeSH terms:
Fibrosis
Cholangitis
Liver Cirrhosis, Biliary
Pathologic Processes
Bile Duct Diseases
Biliary Tract Diseases
Digestive System Diseases
Cholestasis, Intrahepatic
Cholestasis
Liver Diseases
Liver Cirrhosis