PBCPhase 3NCT06715319
Obeticholic Acid Versus Placebo, in Combination with Ursodeoxycholic Acid in Patients with Primary Biliary Cirrhosis
A Phase 3 study testing OCA and UDCA in people with PBC, run by Shanghai Fudan-Zhangjiang Bio-Pharmaceutical Co., Ltd. at 1 site in China.
Trial summary
This is a Phase III, randomized, double-blind, multicentre study assessing the efficacy and safety of obecholic acid and Ursodeoxycholic Acid(UDCA) compared with placebo and UDCA in treating of primary biliary cirrhosis (PBC) in adults with an inadequate response to UDCA. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- PBC
- What is being tested?
- 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
- 100 participants
- Where
- 1 siteChina
- Study dates
- 9 Oct 2021to 29 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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 ≥ 2 months) prior to the first dose；
- Patients of childbearing potential must agree to take contraception during the study and for 1 month after the last day of treatment;
- Patients capable of giving written informed consent.
Who cannot join
- History or presence of other concomitant liver diseases including:
- Hepatitis C virus (HCV) infection; participants with active hepatitis B (HBV) infection will be excluded, however, participants who have seroconverted (hepatitis B surface antigen [Hbs Ag] and hepatitis B e antigen [Hbe Ag] negative) may be included after consultation with the medical monitor.
- Primary sclerosing cholangitis (PSC) Alcoholic liver disease Definite autoimmune liver disease or overlap hepatitis Nonalcoholic steatohepatitis (NASH) Gilbert's Syndrome (due to interpretability of bilirubin levels)Child-pugh grade B or C
- Presence of clinical complications of PBC or clinically significant hepatic decompensation, including:
- History of liver transplantation, current placement on a liver transplant list or current Model for End Stage Liver Disease (MELD) score ≥ 15 Portal hypertension with complications, including: known gastric or large esophageal varices, poorly controlled or diuretic resistant ascites, history of variceal bleeds or related therapeutic or prophylactic interventions (for example, beta blockers, insertion of variceal bands or transjugular intrahepatic portosystemic shunt [TIPS]), or hepatic encephalopathy Cirrhosis with complications, including history or presence of: spontaneous bacterial peritonitis, hepatocellular carcinoma, bilirubin > 2x ULN Hepatorenal syndrome (type I or II) or Screening serum creatinine > 2 mg/deciliter dL) (178 micromoles [µmol])/liter [L])
- Participants with severe pruritus or those requiring systemic treatment for pruritus (for example, with bile acid sequestrants [BAS] or rifampicin) within 2 months prior to the first dose
Show all 21Show fewer
- Patients who took obeticholic acid within 3 months prior to the first dose
- Administration of the following drugs within 1 month prior to the first dose: azathioprine, colchicine, cyclosporine, methotrexate, mycophenolate mofetil, pentoxifylline, bicyclol and with S.chinensis ingredients; 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 2 months prior to the first dose: antibodies or immunotherapy against interleukins or other cytokines or chemokines.
- ALT≥ 10 × ULN with/or ALT≥ 10 × ULN;
- Direct bilirubin>3×ULN;
- Serum creatinine (Cr) ≥ 1.5 × ULN and serum creatinine clearance < 60 mL/min;
- History or presence of clinically concerning cardiac arrhythmias likely to affect survival during the trial, or prolongation of Screening (pretreatment) QT or QTc interval;
- Women who are pregnant or lactating;
- Known history of human immunodeficiency virus (HIV) infection
- Presence of any other disease or condition that is interfering with the absorption, distribution, metabolism, or excretion of drugs including bile salt metabolism in the intestine. Participants with inflammatory bowel disease or who have undergone gastric bypass procedures will be excluded (gastric lap band is acceptable)
- Medical conditions that may cause nonhepatic increases in ALP (for example, Paget's disease) or which may diminish life expectancy to < 2 years, including known cancers (except carcinomas in situ or other stable, relatively benign conditions such as chronic lymphatic leukemia)
- Other clinically significant medical conditions that are not well controlled or for which medication needs are anticipated to change during the trial;
- Anticipated changes to current concomitant medications during the trial;
- Mental instability or incompetence;
- Other circumstances that are deemed not appropriate for the study by the investigator.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Double-blind, Randomized Phase 3 Trial Evaluating the Efficacy and Safety of Obecholic Acid Versus Placebo, in Combination with Ursodeoxycholic Acid in Primary Biliary Cirrhosis Patients with an Inadequate Response to Ursodeoxycholic Acid
This is a Phase III, randomized, double-blind, multicentre study assessing the efficacy and safety of obecholic acid and Ursodeoxycholic Acid(UDCA) compared with placebo and UDCA in treating of primary biliary cirrhosis (PBC) in adults with an inadequate response to UDCA.
Read the detailed description
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.
The study included 2 phases: a 6-month randomized, double-blind (DB), placebo-controlled, parallel-group phase, followed by a 6-month DB treatment phase. In the 6-month randomized, DB placebo-controlled phase, patients will receive OCA/placebo with a dosage of 5 mg once daily for the first 3 months. After the first 3 months, for patients who have not achieved an adequate reduction in ALP and/or total bilirubin and who are tolerating OCALIVA/placebo, increase to a maximum dosage of 10 mg once daily.
Participants in the 6-month DB treatment phase were eligible to receive the treatment of OCA, especially those who received placebo, for patients who have not achieved an adequate reduction in ALP and/or total bilirubin, will receive OCA with a dosage of 5 mg once daily for the first 3 months. After the first 3 months, for patients who have not achieved an adequate reduction in ALP and/or total bilirubin and who are tolerating OCALIVA, increase to a maximum dosage of 10 mg once daily.
- Conditions
- PBC
- Being tested
- OCA, UDCA, Placebo
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
OCADrug
Also called Obeticholic Acid
OCA 5 mg once daily in combination with UDCA for 12 weeks and then titrating up to 10 mg based on tolerability and response for remainder of double-blind period.
UDCADrug
13~15 mg/kg/day
PlaceboDrug
Once a day (QD) by mouth (PO)
| Study group | Role | What they receive |
|---|---|---|
| OCA and 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: OCA; Drug: UDCA |
| Placebo and UDCA | Placebo comparator | Placebo once daily. Subjects receiving UDCA continued taking UDCA throughout the trial. Receives: Drug: UDCA; Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Composite Endpoint Alkaline Phosphatase (ALP) And Total Bilirubin
Composite Endpoint : Percentage of participants at Month 12 with ALP < 1.67 x upper limit of normal (ULN) and total bilirubin ≤ ULN and ALP decrease of ≥ 15% from baseline.
Measured: up to 6 months
Secondary outcomes (8)
Percentage of PBC patients reaching the composite endpoint after 12 weeks, 36 weeks and 48 weeks of treatment
Composite Endpoint : Percentage of participants at Month 12 with ALP < 1.67 x upper limit of normal (ULN) and total bilirubin ≤ ULN and ALP decrease of ≥ 15% from baseline.
Measured: up to 12 months
Absolute change and percentage change of ALP from baseline to Month 3, 6, 9 and 12
Blood samples were evaluated for ALP from baseline to Month 3, 6, 9 and 12 are presented.
Measured: up to 12 months
Absolute change and percentage change of total bilirubin from baseline to Month 3, 6, 9 and 12
Blood samples were evaluated for total bilirubin from baseline to Month 3, 6, 9 and 12 are presented.
Measured: up to 12 months
Absolute change and percentage change of direct bilirubin from baseline to Month 3, 6, 9 and 12
Blood samples were evaluated for direct bilirubin from baseline to Month 3, 6, 9 and 12 are presented.
Measured: up to 12 months
Absolute change and percentage change of alanine transaminase(ALT) from baseline to Month 3, 6, 9 and 12
Blood samples were evaluated for ALT from baseline to Month 3, 6, 9 and 12 are presented.
Measured: up to 12 months
Absolute change and percentage change of alkaline phosphatase(AST) from baseline to Month 3, 6, 9 and 12
Blood samples were evaluated for AST from baseline to Month 3, 6, 9 and 12 are presented.
Measured: up to 12 months
Absolute change and percentage change of gamma glutamyl transpeptidase(GGT) from baseline to Month 3, 6, 9 and 12
Blood samples were evaluated for GGT from baseline to Month 3, 6, 9 and 12 are presented.
Measured: up to 12 months
Incidence of Treatment-Emergent Adverse Events
Adverse Events will be evaluated in terms of adverse events (graded by CTCAE version 5.0).
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 posted4 Dec 2024
- Study start9 Oct 2021
- Primary completion16 Oct 2023
- Study completion29 Apr 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for PBCStudies 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 100 participants (actual)
Sponsor and investigators
- Lead sponsor
- Shanghai Fudan-Zhangjiang Bio-Pharmaceutical Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Junqi Niu, Principal investigator, The First Hospital of Jilin University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06715319
- Sponsor's protocol ID
- FDZJ-OCA-202011
- Status last verified
- Nov 2024
- First submitted
- 27 Nov 2024
- Last updated
- 4 Dec 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 Dec 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Double-blind, Randomized Phase 3 Trial Evaluating the Efficacy and Safety of Obecholic Acid Versus Placebo, in Combination with Ursodeoxycholic Acid in Primary Biliary Cirrhosis Patients with an Inadequate Response to Ursodeoxycholic Acid
- ClinicalTrials.gov ID
- NCT06715319
- Acronym / sponsor ID
- FDZJ-OCA-202011
- Conditions
- PBC
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 100 participants (actual)
- Lead sponsor
- Shanghai Fudan-Zhangjiang Bio-Pharmaceutical Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 9 Oct 2021 (actual)
- Primary completion
- 16 Oct 2023 (actual)
- Study completion
- 29 Apr 2024 (actual)
- First posted
- 4 Dec 2024 (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 is a Phase III, randomized, double-blind, multicentre study assessing the efficacy and safety of obecholic acid and Ursodeoxycholic Acid(UDCA) compared with placebo and UDCA in treating of primary biliary cirrhosis (PBC) in adults with an inadequate response to UDCA.
Detailed description
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.
The study included 2 phases: a 6-month randomized, double-blind (DB), placebo-controlled, parallel-group phase, followed by a 6-month DB treatment phase. In the 6-month randomized, DB placebo-controlled phase, patients will receive OCA/placebo with a dosage of 5 mg once daily for the first 3 months. After the first 3 months, for patients who have not achieved an adequate reduction in ALP and/or total bilirubin and who are tolerating OCALIVA/placebo, increase to a maximum dosage of 10 mg once daily.
Participants in the 6-month DB treatment phase were eligible to receive the treatment of OCA, especially those who received placebo, for patients who have not achieved an adequate reduction in ALP and/or total bilirubin, will receive OCA with a dosage of 5 mg once daily for the first 3 months. After the first 3 months, for patients who have not achieved an adequate reduction in ALP and/or total bilirubin and who are tolerating OCALIVA, increase to a maximum dosage of 10 mg once daily.
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 and UDCA | Experimental | Drug: 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 and UDCA | Placebo comparator | Drug: UDCA, Drug: PlaceboPlacebo once daily. Subjects receiving UDCA continued taking UDCA throughout the trial. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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 ≥ 2 months) prior to the first dose；
- Patients of childbearing potential must agree to take contraception during the study and for 1 month after the last day of treatment;
- Patients capable of giving written informed consent.
Key exclusion criteria (21 listed)
- History or presence of other concomitant liver diseases including:
- Hepatitis C virus (HCV) infection; participants with active hepatitis B (HBV) infection will be excluded, however, participants who have seroconverted (hepatitis B surface antigen [Hbs Ag] and hepatitis B e antigen [Hbe Ag] negative) may be included after consultation with the medical monitor.
- Primary sclerosing cholangitis (PSC) Alcoholic liver disease Definite autoimmune liver disease or overlap hepatitis Nonalcoholic steatohepatitis (NASH) Gilbert's Syndrome (due to interpretability of bilirubin levels)Child-pugh grade B or C
- Presence of clinical complications of PBC or clinically significant hepatic decompensation, including:
- History of liver transplantation, current placement on a liver transplant list or current Model for End Stage Liver Disease (MELD) score ≥ 15 Portal hypertension with complications, including: known gastric or large esophageal varices, poorly controlled or diuretic resistant ascites, history of variceal bleeds or related therapeutic or prophylactic interventions (for example, beta blockers, insertion of variceal bands or transjugular intrahepatic portosystemic shunt [TIPS]), or hepatic encephalopathy Cirrhosis with complications, including history or presence of: spontaneous bacterial peritonitis, hepatocellular carcinoma, bilirubin > 2x ULN Hepatorenal syndrome (type I or II) or Screening serum creatinine > 2 mg/deciliter dL) (178 micromoles [µmol])/liter [L])
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 |
|---|---|---|
| OCAAlso: Obeticholic AcidOCA 5 mg once daily in combination with UDCA for 12 weeks and then titrating up to 10 mg based on tolerability and response for remainder of double-blind period. | Drug | OCA and UDCA |
| UDCA13~15 mg/kg/day | Drug | OCA and UDCA, Placebo and UDCA |
| PlaceboOnce a day (QD) by mouth (PO) | Drug | Placebo and UDCA |
Primary outcome
Composite Endpoint Alkaline Phosphatase (ALP) And Total Bilirubin
Time frame: up to 6 months
Composite Endpoint : Percentage of participants at Month 12 with ALP < 1.67 x upper limit of normal (ULN) and total bilirubin ≤ ULN and ALP decrease of ≥ 15% from baseline.
8 secondary outcomes
Percentage of PBC patients reaching the composite endpoint after 12 weeks, 36 weeks and 48 weeks of treatment
Time frame: up to 12 months
Absolute change and percentage change of ALP from baseline to Month 3, 6, 9 and 12
Time frame: up to 12 months
Absolute change and percentage change of total bilirubin from baseline to Month 3, 6, 9 and 12
Time frame: up to 12 months
Absolute change and percentage change of direct bilirubin from baseline to Month 3, 6, 9 and 12
Time frame: up to 12 months
Absolute change and percentage change of alanine transaminase(ALT) from baseline to Month 3, 6, 9 and 12
Time frame: up to 12 months
Absolute change and percentage change of alkaline phosphatase(AST) from baseline to Month 3, 6, 9 and 12
Time frame: up to 12 months
Absolute change and percentage change of gamma glutamyl transpeptidase(GGT) from baseline to Month 3, 6, 9 and 12
Time frame: up to 12 months
Incidence of Treatment-Emergent Adverse Events
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 4 Dec 2024.
Related trials for PBC
Active, not recruitingPhase 3
Study of Elafibranor in Patients With Primary Biliary Cholangitis (PBC)
Primary Biliary Cirrhosis
RecruitingPhase 3
Seladelpar in Subjects With Primary Biliary Cholangitis (PBC) and Compensated Cirrhosis
Primary Biliary Cholangitis
RecruitingPhase 3
FIREFLY Trial: Fenofibrate Intervention---Randomized Evaluation in First-Line PBC Therapy
Primary Biliary Cholangitis (PBC)
RecruitingPhase 3
Fenofibrate for Patients With Primary Biliary Cirrhosis Who Had An Inadequate Response to Ursodeoxycholic Acid
Primary Biliary Cirrhosis
RecruitingPhase 2/3
Fenofibrate in Patients With Primary Biliary Cholangitis (PBC)
Primary Biliary Cholangitis
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.
Junqi Niu principal investigator
The First Hospital of Jilin University
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 OCA and UDCA in people with PBC, run by Shanghai Fudan-Zhangjiang Bio-Pharmaceutical Co., Ltd. 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 21 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 4 Dec 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?
Composite Endpoint Alkaline Phosphatase (ALP) And Total Bilirubin (time frame: up to 6 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 4 Dec 2024.