Primary Biliary CholangitisPhase 4UDCA PBCNCT04650243
Clinical Research of Tapering UDCA in PBC Patients With a Complete Response
A Phase 4 study testing ursodeoxycholic acid in people with Primary Biliary Cholangitis, run by Peking Union Medical College Hospital at 1 site in China.
Trial summary
This study explores the feasibility of the reducing medication regimen for Ursodeoxycholic Acid(UDCA) in the treatment of primary biliary cholangitis. The participants will be distributed randomly into two experimental groups and one control group. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Primary Biliary Cholangitis
- What is being tested?
- ursodeoxycholic acid
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 90 participants
- Where
- 1 siteChina
- Study dates
- 21 Jan 2020to May 2025
- Phase
- Phase 4
- Are there results?
- Not yet: the study is under way
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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Satisfied the diagnostic criteria of PBC by the AASLDin 2000;
- Age≥18 years
- Clinical stage 2 and 3 (i.e. abnormal liver function and symptomatic phase);
- Patients with improved liver biochemical index( ALP and AST≤1.5× upper limit of normal, with a normal bilirubin level) after 6 to 12 months treatment of UDCA;
- Informed consent obtained.
Who cannot join
- Overlapped with other liver diseases (such as HBV, HCV, alcoholic cirrhosis, etc.) or serum ALT, AST more than 2 ULN;
- Decompensation of liver function (Child grade B/C);
- Complicated with important organ failure (such as renal insufficiency), serious infection or other serious complications;
- Pregnancy, preparation for pregnancy or pregnancy Lactation, psychiatric subjects, etc.;
- Participating in other clinical trials or participated in other clinical trials in three months.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Peking Union Medical College Hospital
Beijing
Li Wang, 8613801175089, wangli2221@sina.com
Li Wang
About this study
Official title: Clinical Research of Tapering Ursodeoxycholic Acid in Primary Biliary Cholangitis Patients With A Complete Response
This study explores the feasibility of the reducing medication regimen for Ursodeoxycholic Acid(UDCA) in the treatment of primary biliary cholangitis. The participants will be distributed randomly into two experimental groups and one control group. The two experimental groups will receive reduced dosage of UDCA at different level, while the control group will receive standard dosage of UDCA. The effect of therapy will be evaluated every three months.
Read the detailed description
Primary biliary cholangitis is a chronic, progressive liver disease of autoimmune origin characterized by nonpurulent destruction of intrahepatic ductule, lymphatic infiltration of portal area and long-term intrahepatic cholestasis leading to liver fibrosis and cirrhosis in absence of treatment. The diagnosis is made in the presence of antimitochondrial antibodies (AMA) coupled with an increase in alkaline phosphatase (ALP), a histologic confirmation being mandatory only in seronegative cases or overlap syndrome. Treatment is based on ursodeoxycholic acid (UDCA) and obeticholic acid, which are proved effective in improving biochemical index and preventing disease progression. While obeticholic acid is only approved in USA and Canada, UDCA seem to be the only choice for PBC patients in China. Study has shown that liver function improvement can be expected in six to nine months when patients receive standard dosage( 13 -15mg/kg/d) of UDCA. Recovery of liver function takes two years in 20% of patients ,and five years in 15% to 35% of patients. Lifetime medication is recommended among patients with good respond to UDCA, while the high cost has placed great burden on patients as well as the medical service system. Exploration of the reducing medication regimen of UDCA among stable PBC patients is of great significance under this circumstance. In our study, the 90 recruited PBC patients with a complete response will be distributed randomly into two experimental groups and one control group. The two experimental groups will receive reduced dosage of UDCA at 10mg/Kg and 5mg/Kg respectively, while the control group will receive standard 13-15mg/Kg dosage of UDCA. The effect of therapy will be evaluated every three months, which includes assessment of symptoms, life quality, disease progression, complete blood count, urinalysis, liver biochemical markers (ALT, AST, ALP, GGT, TBIL, DBIL, TP, ALB), blood lipid (CHO, TG, LDL, HDL), immunoglobulins, ESR, AMA, liver morphology and cirrhosis degree, along with peripheral T lymphocyte subpopulations and cytokines test.
- Conditions
- Primary Biliary Cholangitis
- Being tested
- ursodeoxycholic acid
- Keywords
- ursodeoxycholic acid, reducing medication regimen
- 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.
ursodeoxycholic acidDrug
Also called UDCA
The two experimental groups will receive reduced dosage of UDCA at 10mg/Kg and 5mg/Kg everyday respectively, while the control group will receive standard dosage of UDCA.The treatment duration will be at least one year.
| Study group | Role | What they receive |
|---|---|---|
| 10mg/Kg UDCA | Experimental | Patients will receive a reduced dosage of 10mg/Kg UDCA orally everyday. Receives: Drug: ursodeoxycholic acid |
| 5mg/Kg UDCA | Experimental | Patients will receive a reduced dosage of 5mg/Kg UDCA orally everyday. Receives: Drug: ursodeoxycholic acid |
| standard 13-15mg/Kg UDCA | Active comparator | Patients will receive standard dosage of 13-15mg/Kg UDCA everyday. Receives: Drug: ursodeoxycholic acid |
What is being measured
The primary outcome is the main question the study is designed to answer.
Recurrence rate of primary biliary cholangitis
Liver biochemical markers (AST and ALP in U/L, TBIL in umol/L) that restored to normal increase(bilirubin>1xULN,ALP≥1.5X ULN, AST≥1.5XULN) again is considered to be PBC recurrence according to the Paris II criteria. The rate of recurrence will be described in percent.
Measured: change from baseline to month3,6,9,12.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Dec 2020
- Study start21 Jan 2020
- Primary completionMay 2025 (estimated)
- Study completionMay 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Primary Biliary CholangitisStudies of the same condition, open and under way.
- More trials run by Peking Union Medical College HospitalThe sponsor's other registered studies.
- 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 4
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- In our study, the 90 recruited PBC patients with a complete response will be distributed randomly into two experimental groups and one control group. The two experimental groups will receive reduced dosage of UDCA at 10mg/Kg and 5mg/Kg respectively, while the control group will receive standard 13-15mg/Kg UDCA. The effect of therapy will be evaluated every three months.
- Masking
- None (open label)
- Enrolment
- 90 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Peking Union Medical College Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Li Wang, Study director, Peking Union Medical College Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04650243
- Sponsor's protocol ID
- ZS-2237
- Acronym
- UDCA PBC
- Status last verified
- Jan 2025
- First submitted
- 27 Apr 2020
- Last updated
- 1 Apr 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: all IPD that underlie results in a publication
Study at a glance
From the registry record, last updated by the sponsor on 1 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Clinical Research of Tapering Ursodeoxycholic Acid in Primary Biliary Cholangitis Patients With A Complete Response
- ClinicalTrials.gov ID
- NCT04650243
- Acronym / sponsor ID
- UDCA PBC / ZS-2237
- Conditions
- Primary Biliary Cholangitis
- Keywords
- ursodeoxycholic acid, reducing medication regimen
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 90 participants (estimated)
- Lead sponsor
- Peking Union Medical College HospitalUniversity, hospital or other
- Study start
- 21 Jan 2020 (actual)
- Primary completion
- May 2025 (estimated)
- Study completion
- May 2025 (estimated)
- First posted
- 2 Dec 2020 (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.
This study explores the feasibility of the reducing medication regimen for Ursodeoxycholic Acid(UDCA) in the treatment of primary biliary cholangitis. The participants will be distributed randomly into two experimental groups and one control group. The two experimental groups will receive reduced dosage of UDCA at different level, while the control group will receive standard dosage of UDCA. The effect of therapy will be evaluated every three months.
Detailed description
Primary biliary cholangitis is a chronic, progressive liver disease of autoimmune origin characterized by nonpurulent destruction of intrahepatic ductule, lymphatic infiltration of portal area and long-term intrahepatic cholestasis leading to liver fibrosis and cirrhosis in absence of treatment. The diagnosis is made in the presence of antimitochondrial antibodies (AMA) coupled with an increase in alkaline phosphatase (ALP), a histologic confirmation being mandatory only in seronegative cases or overlap syndrome. Treatment is based on ursodeoxycholic acid (UDCA) and obeticholic acid, which are proved effective in improving biochemical index and preventing disease progression. While obeticholic acid is only approved in USA and Canada, UDCA seem to be the only choice for PBC patients in China. Study has shown that liver function improvement can be expected in six to nine months when patients receive standard dosage( 13 -15mg/kg/d) of UDCA. Recovery of liver function takes two years in 20% of patients ,and five years in 15% to 35% of patients. Lifetime medication is recommended among patients with good respond to UDCA, while the high cost has placed great burden on patients as well as the medical service system. Exploration of the reducing medication regimen of UDCA among stable PBC patients is of great significance under this circumstance. In our study, the 90 recruited PBC patients with a complete response will be distributed randomly into two experimental groups and one control group. The two experimental groups will receive reduced dosage of UDCA at 10mg/Kg and 5mg/Kg respectively, while the control group will receive standard 13-15mg/Kg dosage of UDCA. The effect of therapy will be evaluated every three months, which includes assessment of symptoms, life quality, disease progression, complete blood count, urinalysis, liver biochemical markers (ALT, AST, ALP, GGT, TBIL, DBIL, TP, ALB), blood lipid (CHO, TG, LDL, HDL), immunoglobulins, ESR, AMA, liver morphology and cirrhosis degree, along with peripheral T lymphocyte subpopulations and cytokines test.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
- Model description
- In our study, the 90 recruited PBC patients with a complete response will be distributed randomly into two experimental groups and one control group. The two experimental groups will receive reduced dosage of UDCA at 10mg/Kg and 5mg/Kg respectively, while the control group will receive standard 13-15mg/Kg UDCA. The effect of therapy will be evaluated every three months.
| Arm | Type | Receives |
|---|---|---|
| 10mg/Kg UDCA | Experimental | Drug: ursodeoxycholic acidPatients will receive a reduced dosage of 10mg/Kg UDCA orally everyday. |
| 5mg/Kg UDCA | Experimental | Drug: ursodeoxycholic acidPatients will receive a reduced dosage of 5mg/Kg UDCA orally everyday. |
| standard 13-15mg/Kg UDCA | Active comparator | Drug: ursodeoxycholic acidPatients will receive standard dosage of 13-15mg/Kg UDCA everyday. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Satisfied the diagnostic criteria of PBC by the AASLDin 2000;
- Age≥18 years
- Clinical stage 2 and 3 (i.e. abnormal liver function and symptomatic phase);
- Patients with improved liver biochemical index( ALP and AST≤1.5× upper limit of normal, with a normal bilirubin level) after 6 to 12 months treatment of UDCA;
- Informed consent obtained.
Key exclusion criteria (5 listed)
- Overlapped with other liver diseases (such as HBV, HCV, alcoholic cirrhosis, etc.) or serum ALT, AST more than 2 ULN;
- Decompensation of liver function (Child grade B/C);
- Complicated with important organ failure (such as renal insufficiency), serious infection or other serious complications;
- Pregnancy, preparation for pregnancy or pregnancy Lactation, psychiatric subjects, etc.;
- Participating in other clinical trials or participated in other clinical trials in three months.
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 |
|---|---|---|
| ursodeoxycholic acidAlso: UDCAThe two experimental groups will receive reduced dosage of UDCA at 10mg/Kg and 5mg/Kg everyday respectively, while the control group will receive standard dosage of UDCA.The treatment duration will be at least one year. | Drug | 10mg/Kg UDCA, 5mg/Kg UDCA, standard 13-15mg/Kg UDCA |
Primary outcome
Recurrence rate of primary biliary cholangitis
Time frame: change from baseline to month3,6,9,12.
Liver biochemical markers (AST and ALP in U/L, TBIL in umol/L) that restored to normal increase(bilirubin>1xULN,ALP≥1.5X ULN, AST≥1.5XULN) again is considered to be PBC recurrence according to the Paris II criteria. The rate of recurrence will be described in percent.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Apr 2025.
- Individual participant dataSharing planned: Yes. all IPD that underlie results in a publication
Related trials for Primary Biliary Cholangitis
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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.
Li Wang study director
Peking Union Medical College Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing ursodeoxycholic acid in people with Primary Biliary Cholangitis, run by Peking Union Medical College Hospital 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 5 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: looking for participants now, as of the sponsor's last update on 1 Apr 2025. 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Li Wang (8613801175089, wangli2221@sina.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Recurrence rate of primary biliary cholangitis (time frame: change from baseline to month3,6,9,12.). 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 1 Apr 2025.