Primary Biliary CholangitisPhase 3NCT05450887
Efficacy and Safety of Obeticholic Acid in the Treatment of Primary Biliary Cholangitis
A Phase 3 study testing Obeticholic Acid Tablets（OCA） and UDCA in people with Primary Biliary Cholangitis, run by Nanjing Chia-tai Tianqing Pharmaceutical at 1 site in China.
Trial summary
Obecholic acid is a modified bile acid and Farnesoid X receptor (FXR) agonist. FXR is a key regulator of bile acid synthesis and transport. Bile acids are used by the body to help with digestion. Conventional therapy with obecholic acid will improve liver function of patients with (primary biliary cholangitis)PBC. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Primary Biliary Cholangitis
- What is being tested?
- Obeticholic Acid Tablets（OCA） and UDCA
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 108 participants
- Where
- 1 siteChina
- Study dates
- 23 Sep 2021to 9 Apr 2024
- Phase
- Phase 3
- 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 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age ≥18 and ≤75 years；
- Definite PBC diagnosis, as demonstrated by the presence of ≥ 2 of the following 3 diagnostic factors: ① Indicators reflecting cholestasis such as elevated ALP；② Positive antimitochondrial antibody (AMA) or AMA-M2, or positive PBC-specific antibody (anti-GP210 and/or anti-SP100) if AMA negative；③ Liver biopsy consistent with PBC；
- At least 1 of the following qualifying biochemistry values: ① ALP ≥ 1.67x ULN ；② Total bilirubin > ULN but < 2x ULN；
- Taking UDCA for at least 6 months (stable dose for ≥ 3 months) prior to Day 0, or unable to tolerate UDCA (no UDCA for ≥ 3 months) prior to Day 0；
- Understand the study, comply with the study protocol, and voluntarily sign the informed consent form.
Who cannot join
- Patients who took obeticholic acid within 3 months prior to Day 0;
- Known hypersensitivity to obeticholic acid, ursodeoxycholic acid;
- History or presence of other concomitant liver diseases;
- Cirrhosis-related complications or end-stage liver disease manifestations;
- Serum creatinine (Cr) ≥ 1.5 × ULN and serum creatinine clearance < 60 mL/min;
- Patients with severe pruritus or requiring systemic drug therapy within 2 months prior to Day 0;
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- Patients with HIV or syphilis infection;
- Presence of diseases or physiological conditions that interfere with the absorption, distribution, metabolism or excretion of test drugs, such as inflammatory bowel disease and previous gastric bypass surgery;
- Presence of diseases that may cause non-hepatogenic ALP elevation, or diseases that may lead to a life expectancy of less than 2 years;
- Administration of the following drugs within 6 months prior to Day 0: azathioprine, colchicine, cyclosporine, methotrexate, mycophenolate mofetil, pentoxifylline; fenofibrate or other fibrates; budesonide and other systemic corticosteroids; hepatotoxic drugs (including α-methyldopa, sodium valproate, isoniazid, nitrofurantoin, etc.);
- Administration of the following drugs within 12 months prior to Day 0: antibodies or immunotherapy against interleukins or other cytokines or chemokines;
- Patients with serious cardiovascular system, digestive system, respiratory system, urinary system, nervous system, mental illness, immunodeficiency disease, and judge by investigators that they are not suitable for participating in the trial;
- Other conditions that are not considered appropriate by the investigator.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Randomized, Double-blind, Multicenter, Placebo-controlled Phase III Clinical Trial to Evaluate the Efficacy and Safety of Obeticholic Acid in Patients With Primary Biliary Cholangitis
Obecholic acid is a modified bile acid and Farnesoid X receptor (FXR) agonist. FXR is a key regulator of bile acid synthesis and transport. Bile acids are used by the body to help with digestion. Conventional therapy with obecholic acid will improve liver function of patients with (primary biliary cholangitis)PBC.
The main objectives of the study were to assess the effects of Obeticholic Acid (OCA) on serum alkaline phosphatase (ALP) and total bilirubin, together as a composite endpoint and on safety in participants with PBC.
- Conditions
- Primary Biliary Cholangitis
- Being tested
- Obeticholic Acid Tablets（OCA）, UDCA, Placebo
- 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.
Obeticholic Acid Tablets（OCA）Drug
Obeticholic Acid：Once a day (QD) by mouth (PO).
UDCADrug
UDCA：13~15 mg/kg/day
PlaceboDrug
Placebo：Once a day (QD) by mouth (PO).
| Study group | Role | What they receive |
|---|---|---|
| OCA 5 mg titrated to 10 mg ± UDCA | Experimental | OCA 5 mg once daily for 3 months and then titrating up to 10 mg based on tolerability and response.
Subjects receiving UDCA continued taking UDCA throughout the trial. If the subjects could not tolerate UDCA, they were not treated with UDCA. Receives: Drug: Obeticholic Acid Tablets（OCA）; Drug: UDCA |
| Placebo ± UDCA | Placebo comparator | Placebo once daily. Subjects receiving UDCA continued taking UDCA throughout the trial. If the subjects could not tolerate UDCA, they were not treated with UDCA. Receives: Drug: UDCA; Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of PBC patients reaching the compound endpoint after 12 months of treatment (Compound endpoint: alkaline phosphatase (ALP) < 1.67× Upper Limit of Normal(ULN), and ALP decrease ≥ 15% from baseline, and total bilirubin ≤ ULN )
Compound endpoint: ALP < 1.67× ULN, and ALP decrease ≥ 15% from baseline, and total bilirubin ≤ ULN
Measured: up to 12 months
Secondary outcomes (2)
Absolute change and percentage change of ALP, total bilirubin, direct bilirubin, ALT, AST and GGT from baseline to Month 3, 6, 9 and 12
Blood samples were evaluated for ALP, total bilirubin, direct bilirubin, ALT, AST and GGT levels. Absolute change and percentage change of ALP, total bilirubin, direct bilirubin, ALT, AST and GGT from baseline to Month 3, 6, 9 and 12 are presented.
Measured: up to 12 months
Quality of life for PBC measure (PBC-40) score percentage change from baseline to Month 3, 6, 9 and 12
PBC-40 score was evaluated at certain visits. PBC-40 score percentage change from baseline to Month 3, 6, 9 and 12 is presented.
Measured: up to 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Jul 2022
- Study start23 Sep 2021
- Primary completion18 Mar 2024
- Study completion9 Apr 2024
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 Nanjing Chia-tai Tianqing PharmaceuticalThe 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 108 participants (actual)
Sponsor and investigators
- Lead sponsor
- Nanjing Chia-tai Tianqing Pharmaceutical
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05450887
- Sponsor's protocol ID
- ABDSP2021-III
- Status last verified
- Jul 2024
- First submitted
- 4 Jul 2022
- Last updated
- 11 Jul 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Jul 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-blind, Multicenter, Placebo-controlled Phase III Clinical Trial to Evaluate the Efficacy and Safety of Obeticholic Acid in Patients With Primary Biliary Cholangitis
- ClinicalTrials.gov ID
- NCT05450887
- Acronym / sponsor ID
- ABDSP2021-III
- Conditions
- Primary Biliary Cholangitis
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 108 participants (actual)
- Lead sponsor
- Nanjing Chia-tai Tianqing PharmaceuticalIndustry (pharma, biotech, devices)
- Study start
- 23 Sep 2021 (actual)
- Primary completion
- 18 Mar 2024 (actual)
- Study completion
- 9 Apr 2024 (actual)
- First posted
- 11 Jul 2022 (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.
Obecholic acid is a modified bile acid and Farnesoid X receptor (FXR) agonist. FXR is a key regulator of bile acid synthesis and transport. Bile acids are used by the body to help with digestion. Conventional therapy with obecholic acid will improve liver function of patients with (primary biliary cholangitis)PBC.
The main objectives of the study were to assess the effects of Obeticholic Acid (OCA) on serum alkaline phosphatase (ALP) and total bilirubin, together as a composite endpoint and on safety in participants with PBC.
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 |
|---|---|---|
| OCA 5 mg titrated to 10 mg ± UDCA | Experimental | Drug: Obeticholic Acid Tablets（OCA）, Drug: UDCAOCA 5 mg once daily for 3 months and then titrating up to 10 mg based on tolerability and response. Subjects receiving UDCA continued taking UDCA throughout the trial. If the subjects could not tolerate UDCA, they were not treated with UDCA. |
| Placebo ± UDCA | Placebo comparator | Drug: UDCA, Drug: PlaceboPlacebo once daily. Subjects receiving UDCA continued taking UDCA throughout the trial. If the subjects could not tolerate UDCA, they were not treated with UDCA. |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Age ≥18 and ≤75 years；
- Definite PBC diagnosis, as demonstrated by the presence of ≥ 2 of the following 3 diagnostic factors: ① Indicators reflecting cholestasis such as elevated ALP；② Positive antimitochondrial antibody (AMA) or AMA-M2, or positive PBC-specific antibody (anti-GP210 and/or anti-SP100) if AMA negative；③ Liver biopsy consistent with PBC；
- At least 1 of the following qualifying biochemistry values: ① ALP ≥ 1.67x ULN ；② Total bilirubin > ULN but < 2x ULN；
- Taking UDCA for at least 6 months (stable dose for ≥ 3 months) prior to Day 0, or unable to tolerate UDCA (no UDCA for ≥ 3 months) prior to Day 0；
- Understand the study, comply with the study protocol, and voluntarily sign the informed consent form.
Key exclusion criteria (13 listed)
- Patients who took obeticholic acid within 3 months prior to Day 0;
- Known hypersensitivity to obeticholic acid, ursodeoxycholic acid;
- History or presence of other concomitant liver diseases;
- Cirrhosis-related complications or end-stage liver disease manifestations;
- Serum creatinine (Cr) ≥ 1.5 × ULN and serum creatinine clearance < 60 mL/min;
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 |
|---|---|---|
| Obeticholic Acid Tablets（OCA）Obeticholic Acid：Once a day (QD) by mouth (PO). | Drug | OCA 5 mg titrated to 10 mg ± UDCA |
| UDCAUDCA：13~15 mg/kg/day | Drug | OCA 5 mg titrated to 10 mg ± UDCA, Placebo ± UDCA |
| PlaceboPlacebo：Once a day (QD) by mouth (PO). | Drug | Placebo ± UDCA |
Primary outcome
Percentage of PBC patients reaching the compound endpoint after 12 months of treatment (Compound endpoint: alkaline phosphatase (ALP) < 1.67× Upper Limit of Normal(ULN), and ALP decrease ≥ 15% from baseline, and total bilirubin ≤ ULN )
Time frame: up to 12 months
Compound endpoint: ALP < 1.67× ULN, and ALP decrease ≥ 15% from baseline, and total bilirubin ≤ ULN
2 secondary outcomes
Absolute change and percentage change of ALP, total bilirubin, direct bilirubin, ALT, AST and GGT from baseline to Month 3, 6, 9 and 12
Time frame: up to 12 months
Quality of life for PBC measure (PBC-40) score percentage change from baseline to Month 3, 6, 9 and 12
Time frame: up to 12 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Jul 2024.
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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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Obeticholic Acid Tablets（OCA） and UDCA in people with Primary Biliary Cholangitis, run by Nanjing Chia-tai Tianqing Pharmaceutical 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 13 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: finished, as of the sponsor's last update on 11 Jul 2024. 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.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percentage of PBC patients reaching the compound endpoint after 12 months of treatment (Compound endpoint: alkaline phosphatase (ALP) < 1.67× Upper Limit of Normal(ULN), and ALP decrease ≥ 15% from baseline, and total bilirubin ≤ ULN ) (time frame: up to 12 months). 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 11 Jul 2024.