Primary Biliary CirrhosisNCT03668145
Mesenchymal Stem Cell Transplantation for Refractory Primary Biliary Cholangitis
A study of Primary Biliary Cirrhosis, run by Xijing Hospital of Digestive Diseases at 1 site in China.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Primary Biliary Cirrhosis
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 140 participants
- Where
- 1 siteChina
- Study dates
- 8 Nov 2017to 31 Dec 2020
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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) -
Who cannot join
- 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；
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- 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
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
Changcun Guo
Xi'an, Shaanxi, 710032
Changcun Guo, 13991900180, guochc@sina.com
About this study
Official title: Mesenchymal Stem Cell Transplantation for Refractory Primary Biliary Cholangitis, a Randomized Double-blind Placebo Controlled Trial
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
- Being tested
- mesenchymal stem cell transplantation or placebo infusion via peripheral vein
- Keywords
- Primary Biliary Cirrhosis (now termed as primary biliary cholangitis), mesenchymal stem cell,
- Medical subject headings
- Liver Cirrhosis, Biliary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
mesenchymal stem cell transplantation or placebo infusion via peripheral veinProcedure or surgery
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
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: 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. Receives: Procedure: mesenchymal stem cell transplantation or placebo infusion via peripheral vein |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change of alkaline phosphatase (ALP)
The absolute value change of ALP after 1 year of the initial stem cell treatment
Measured: 1 year
Secondary outcomes (2)
Change of other liver function indices
The absolute value change of total bilirubin, ALT, AST, GGT after initial stem cell treatment
Measured: 1 year
Change of liver histology
histological scores assessed by liver biopsy at baseline and after treatment
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Sep 2018
- Study start8 Nov 2017
- Primary completion31 Dec 2019 (estimated)
- Study completion31 Dec 2020 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Primary Biliary CirrhosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 140 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Xijing Hospital of Digestive Diseases
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Han Ying, Xijing Hospital of Digestive Diseases
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03668145
- Sponsor's protocol ID
- KY20172050-1
- Status last verified
- Sep 2018
- First submitted
- 10 Sep 2018
- Last updated
- 12 Sep 2018
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 12 Sep 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Mesenchymal Stem Cell Transplantation for Refractory Primary Biliary Cholangitis, a Randomized Double-blind Placebo Controlled Trial
- ClinicalTrials.gov ID
- NCT03668145
- Acronym / sponsor ID
- KY20172050-1
- Conditions
- Primary Biliary Cirrhosis
- Keywords
- Primary Biliary Cirrhosis (now termed as primary biliary cholangitis), mesenchymal stem cell,
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 140 participants (estimated)
- Lead sponsor
- Xijing Hospital of Digestive DiseasesUniversity, hospital or other
- Study start
- 8 Nov 2017 (actual)
- Primary completion
- 31 Dec 2019 (estimated)
- Study completion
- 31 Dec 2020 (estimated)
- First posted
- 12 Sep 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| MSC group | Experimental | Procedure: mesenchymal stem cell transplantation or placebo infusion via peripheral veinmesenchymal stem cell transplantation via peripheral vein: 0.1-1x10E6 MSCs/kg body weight administered via peripheral vein at week 0, 4, 8 plus UDCA. |
| control | Placebo comparator | Procedure: mesenchymal stem cell transplantation or placebo infusion via peripheral veinplacebo 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. |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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) -
Key exclusion criteria (10 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| mesenchymal stem cell transplantation or placebo infusion via peripheral vein0.1-1x10E6 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 | Procedure or surgery | MSC group, control |
Primary outcome
Change of alkaline phosphatase (ALP)
Time frame: 1 year
The absolute value change of ALP after 1 year of the initial stem cell treatment
2 secondary outcomes
Change of other liver function indices
Time frame: 1 year
Change of liver histology
Time frame: 1 year
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Sep 2018.
- Individual participant dataSharing planned: Yes
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study of Primary Biliary Cirrhosis, run by Xijing Hospital of Digestive Diseases at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 12 Sep 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Ying Han, MD (862984771539, guochc@fmmu.edu.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change of alkaline phosphatase (ALP) (time frame: 1 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Sep 2018.