Umbilical Cord Mesenchymal Stem Cells for Patients With Primary Biliary Cirrhosis
|ClinicalTrials.gov Identifier: NCT01662973|
Recruitment Status : Unknown
Verified May 2013 by Fu-Sheng Wang, Beijing 302 Hospital.
Recruitment status was: Recruiting
First Posted : August 13, 2012
Last Update Posted : May 31, 2013
|Condition or disease||Intervention/treatment||Phase|
|Primary Biliary Cirrhosis||Other: conventional plus UC-MSC treatment Other: Conventional plus placebo treatment||Phase 1 Phase 2|
Primary biliary cirrhosis (PBC) is a slowly progressive cholestatic disease associated with the development of cirrhosis and liver failure that may justify liver transplantation. Ursodeoxycholic acid (UDCA) is currently the only drug approved specifically for the treatment of PBC. However, one out of three patients does not adequately respond to UDCA therapy and many need additional medical therapy or liver transplantation, or both.
The potential for stem cells to differentiate into biliary epithelial cells was recently confirmed. In particular, bone marrow-derived mesenchymal stem cell (BM-MSC) transplantation has been applicated in the clinic for treat several human disease such as GVHD, cardiac injury and brain injury, and displayed good tolerance and efficiency. Recently, umbilical cord-derived MSCs (UC-MSC) has also been used to treat severe autoimmune diseases, such as therapy-resistant rheumatoid arthritis and multiple sclerosis.
The purpose of this study is to learn whether and how UC-MSC can improve the disease condition in patients with primary biliary cirrhosis. This study will also look at how well UC-MSC is tolerated and its safety in PBC patients
Participants in the study will be randomly assigned to one of two treatment arms:
Arm A: Participants will receive 12 weeks of UC-MSC treatment plus UDCA. Arm B: Participants will receive 12 weeks of placebo plus UDCA. UC-MSC will be prepared according to standard procedures and is collected in plastic bags containing anticoagulant. UC-MSCs are given via i.v. under sonography monitoring. After cell therapy, patients are followed up at week 4,8,12,24,36 and 48. The evaluation of some clinical parameters such as the level of serum alkaline phosphatase (ALP), alanine aminotransferase(ALT) aspartate aminotransferase (AST) and total bilirubin (TB), prothrombin time(PT), albumin(ALB), prealbumin(PA), are detected at these time points. Mayo risk score, portal hypertension, Liver histology, MELD score and clinical symptoms were also observed simultaneously.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||100 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||None (Open Label)|
|Official Title:||Phase 1/2 Study of UC-MSC Treatment for Evaluation the Efficacy and Safety in Patients With Primary Biliary Cirrhosis|
|Study Start Date :||October 2011|
|Estimated Primary Completion Date :||October 2013|
|Estimated Study Completion Date :||October 2013|
Experimental: Conventional plus UC-MSC treatment
Participants will receive conventional treatment plus a dose of UC-MSC from day 0 through the week 12 study visit.
Participants will then be followed until the week 48 study visit.
Other: conventional plus UC-MSC treatment
Received conventional treatment and taken i.v., once per 4 week, at a dose of 1*10E6 UC-MSC/kg body weight for 12 weeks.
Placebo Comparator: Conventional plus placebo treatment
Participants will receive conventional plus placebo treatment from day 0 through the week 12 study visit. Participants will then be followed until the week 48 study visit.
Other: Conventional plus placebo treatment
Received conventional treatment and taken i.v., once per 4 week, at 50 ml saline for 12 weeks
- Serum alkaline phosphatase (ALP) [ Time Frame: 0, 4, 8,12, 24, 36,48 weeks after treatment ]
- Histological changes in liver biopsies [ Time Frame: baseline and 48 weeks ]
- Serum Bilirubin [ Time Frame: At base line and at week 4,8,12,24,36 and 48 ]
- Serum AST [ Time Frame: At base line and at week 4,8,12,24,36 and 48 ]
- Mayo risk score [ Time Frame: At base line and at week 4,8,12,24,36 and 48 ]
- Number of patients with Portal Hypertension after 12 weeks treatment [ Time Frame: At base line and at week 12,24,36 and 48 ]
- MELD score [ Time Frame: At base line and at week 4,8,12,24,36 and 48 ]
- Number of participants with improvement of clinical symptoms [ Time Frame: At base line and at week 4,8,12,24,36 and 48 ]clinical symptoms including fatigue (Fatigue Impact Score, FIS) and pruritus ( Visual Analog Scale ,VAS)
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01662973
|Contact: Fu-Sheng Wang, professor||86-10-63879735 ext firstname.lastname@example.org|
|Contact: Zheng Zhang, Doctor||86-10-63879735 ext 2015.12||Zhangzheng1975@yahoo.com.cn|
|Beijing 302 Hospital||Recruiting|
|Beijing, Beijing, China, 100039|
|Contact: Fu-Sheng Wang, professor 86-10-63879735 ext 2015.12 email@example.com|
|Contact: Lifeng Wang, Doctor 86-10-63879735 ext 2015.12 firstname.lastname@example.org|
|Principal Investigator: Fu-Sheng Wang, Professor|
|Principal Investigator:||Fu-Sheng Wang, professor||Beijing 302 Hospital|