Clinical Research Directory

A Long-Term Study of Elafibranor in Adult Participants With Primary Biliary Cholangitis

The participants of this study will have confirmed Primary Biliary Cholangitis (PBC) and cirrhosis (scarring of the liver). PBC is a slowly progressive disease, characterised by damage to the bile ducts in the liver, leading to a build-up of bile acids which causes further damage. The liver damage in PBC may lead to cirrhosis. PBC may also be associated with multiple symptoms. Many patients with PBC may require liver transplant or may die if the disease progresses and a liver transplant is not done. This study will compare a daily dose of elafibranor (the study drug) to a daily dose of placebo (a dummy treatment) and will last up to 3.5 years for each participant. The main aim of this study is to determine if elafibranor is better than placebo in preventing clinical outcome events showing disease worsening (including progression of disease leading to liver transplant or death). This study will also study the safety of long-term treatment with elafibranor, as well as the impact on symptoms such as itching and tiredness.

CS0159 in Chinese Patients With PBC (Primary Biliary Cholangitis)

A phase II study to evaluate safety, tolerability and efficacy of CS0159 in patients with PBC (Primary Biliary Cholangitis).

A Two-part Study to Investigate the Effects in Adults of Two Doses of Golexanolone in Patients With Primary Biliary Cholangitis (PBC) With Fatigue and Cognitive Dysfunction

The present phase 1b/2 randomised, double-blind, placebo-controlled, two-part study is designed to evaluate the safety, tolerability, pharmacokinetic characteristics and preliminary efficacy of two dose levels of golexanolone compared with placebo among subjects with a history of non-cirrhotic or Child-Pugh class A cirrhotic Primary Biliary Cholangitis (PBC) with clinically significant fatigue and cognitive symptoms on stable background standard of care (SoC) PBC medication. The objectives of this research study are to assess the safety and tolerability as well the pharmacokinetic (PK) characteristics of golexanolone administered 40 mg BID for 5 days in the target population (part A) and to assess the safety and tolerability, the effects of golexanolone on health-related quality of life (HRQoL), including fatigue, day-time sleepiness and cognitive function as well as Investigator's overall impression of treatment effect of 28 days twice per day (BID) treatment with two dose levels of golexanolone versus placebo (part B).

Biochemical Response and Clinical Outcomes in Patients With PBC

This study is a bidirectional cohort study. The investigators conduct a bidirectional cohort study utilizing a database in mainland China, continuously collecting demographics, clinical symptoms, and biochemical characteristics of diagnosed PBC patients. The study aims to analyze the association between varying post-treatment alkaline phosphatase (AKP) levels and complication-free survival rates, with the objective to develop and validate a predictive survival model.

Fenofibrate in Combination With Ursodeoxycholic Acid in Primary Biliary Cholangitis: a Real World Study

An Open Label Long-Term Study to Evaluate the Safety and Tolerability of Fenofibrate in Combination with Ursodeoxycholic Acid in Subjects with Primary Biliary Cholangitis (PBC)

Fenofibrate in Primary Biliary Cholangitis: a Real World Study

An Open Label Long-Term Study to Evaluate the Safety and Tolerability of Fenofibrate in Combination with Ursodeoxycholic Acid in Subjects with Primary Biliary Cholangitis (PBC)

FIREFLY Trial: Fenofibrate Intervention---Randomized Evaluation in First-Line PBC Therapy

The goal of this clinical trial is to learn if the drug Fenofibrate works to treat adults with a liver disease called Primary Biliary Cholangitis (PBC) who have not received previous treatment. It will also learn about the safety of Fenofibrate. The main questions it aims to answer are: Is Fenofibrate better at helping the liver return to normal function (measured by a blood test called ALP) than the standard medication, Ursodeoxycholic What kind of medical problems do participants have when taking Fenofibrate compared to those taking UDCA? Researchers will compare Fenofibrate to the active drug UDCA (the current standard treatment) to see which one works better.\*\* Participants will: Be randomly assigned to take either Fenofibrate plus a UDCA placebo, or UDCA plus a Fenofibrate placebo, every day for 12 months. (Neither they nor their doctor will know which group they are in.) Visit the clinic 5 times over the year (at 1, 3, 6, 9, and 12 months) for check-ups, blood tests, and questionnaires. Undergo a special scan (like FibroScan) to measure liver stiffness at some visits. Be encouraged to have a liver biopsy at the start and end of the study to provide detailed information about liver health (this is optional).

A Multicenter, Randomized, Controlled Trial of Prednisone Combined With Ursodeoxycholic Acid in the Treatment of Primary Biliary Cholangitis With Moderate to Severe Interface Hepatitis Characteristics

About 1/3 of patients with primary biliary cholangitis (PBC) exhibit moderate to severe interface hepatitis, and this group of patients have poor response to UDCA treatment. However, as it is not yet sufficient to diagnose autoimmune hepatitis or overlap syndrome, it is difficult to initiate immunosuppressive therapy such as steroids according to current guidelines. The aim of this study is to explore whether PBC patients with moderate to severe interfacial inflammation can benefit from UDCA combined with prednisone therapy, and its treatment safety.

Early Diagnosis of Primary Biliary Cholangitis (PBC) in Patients With Positive Anti-Mitochondrial Antibodies (AMA) and Normal Alkaline Phosphatase (ALP)

The goal of this observational study is to achieve early diagnosis of Primary Biliary Cholangitis (PBC) in patients with positive Anti-Mitochondrial Antibodies (AMA) and Normal Alkaline Phosphatase (ALP) in adults aged 18-80 years with positive AMA and normal ALP. The main question it aims to answer is: • Are there any predictive factors for histological PBC in patients with positive AMA and normal ALP? Participants will undergo a liver biopsy and specific blood tests if eligible based on inclusion and exclusion criteria.

RELIEF: Multimodal Prehabilitation to Treat Fatigue in Patients With Primary Biliary Cholangitis

The implementation of a non-pharmacological multimodal intervention program-including physical exercise, nutritional counseling, and psychological support-is expected to improve fatigue in patients with primary biliary cholangitis. Consequently, this improvement is anticipated to enhance quality of life and cognitive symptoms, while also positively impacting emotional, social, and occupational aspects. From a pathophysiological perspective, it is hypothesized that chronic cholestasis and/or immune system activation, with the release of pro-inflammatory cytokines, leads to both central and peripheral alterations causing fatigue. At the central level, systemic inflammation may induce neuronal senescence in the basal ganglia, resulting in altered functional connectivity networks dependent on these regions and/or structural and connectivity changes in areas involved in interoception, such as the insula and anterior cingulate cortex. At the peripheral level, the hypothesis is that chronic inflammation mediated by anti-mitochondrial antibodies causes mitochondrial metabolic dysfunction in muscle cells, which would be reflected in changes observed in the gene expression analysis of these cells. Improvement in fatigue following the multimodal intervention program is expected to be associated with normalization of the immunological profile, enhanced functional brain connectivity, and improved mitochondrial metabolism in muscle.

PBC Long-term Outcomes Study

Primary biliary cholangitis (PBC) is an autoimmune liver disease predominantly affecting middle-aged women. While historically it was deemed rare, advancements in specific auto-antibody tests have led to increased recognition of PBC. The long-term survival of PBC patients in China is not yet fully understood. Several studies have investigated the prognosis of PBC in China. While these studies provide valuable insights into the disease characteristics and prognostic factors of PBC in China, they are all single-center studies with limitations. They lack consideration of the impact of symptoms, varying disease stages, and second-line treatments on prognosis. Therefore, there is a pressing need for multicenter and large-scale studies to further elucidate the characteristics and long-term survival of PBC patients in China. Biochemical response to ursodeoxycholic acid (UDCA) is an independent factor associated with long-term survival. Unfortunately, approximately 30-40% of PBC patients demonstrate insufficient biochemical response to UDCA. In our cohort, we reported a higher nonresponse rate, with about 44% according to the Paris I criteria. These patients remain at risk for disease progression to advanced stages and may benefit from additional second-line drug therapies. With significant advancements in the development of new drugs for PBC, a comprehensive understanding of patients with suboptimal responses to UDCA-including long-term prognosis, the distribution of different disease stages, and the prevalence of pruritus-will provide a basis for individualized treatment strategies. Additionally, fatigue and pruritus are prevalent symptoms for PBC patients and fluctuates independently of disease activity or stage, which significantly diminishing health-related quality of life. However, the incidence and impact of fatigue and pruritus on long-term outcomes for PBC patients in China need further clarification. Therefore, we would like to conduct this multicenter study to estimate the prevalence of PBC in China and to evaluate the symptomatic burden, treatment, and long-term outcomes among PBC patients in China.

Fenofibrate in Subjects With Primary Biliary Cholangitis (PBC)

An Open Label Long-Term Study to Evaluate the Safety and Tolerability of Fenofibrate in Subjects with Primary Biliary Cholangitis (PBC)

EMPower Mechanisms

Primary Biliary Cholangitis (PBC) is a chronic liver disease that can cause fatigue, itching, brain fog, and emotional distress, all of which can lower quality of life. While the standard treatment, ursodeoxycholic acid (UDCA), helps slow the disease, it does not relieve these symptoms. Research shows that mind-body practices-such as breathing exercises, meditation, and gentle movement-can help improve mental and physical well-being in people with chronic conditions, but their benefits for PBC are not yet well understood. This study will test a 10-week online wellness program designed for women with PBC. The program will include guided breathing, meditation, and movement exercises, plus optional weekly group sessions and educational videos. Some participants will also receive nutrition guidance on the Mediterranean diet to see if it adds extra benefits. The study will evaluate feasibility via recruitment, adherence, and retention. To assess acceptability, participants will provide feedback through surveys and interviews. The study will also explore early signs of effectiveness by measuring changes in symptoms like anxiety, depression, fatigue, and stress, as well as biological markers, brain activity, physical function, and data from wearable devices. The results will help determine whether an online mind-body program, with or without nutrition support, could be a simple and effective way to help people with PBC manage their symptoms and improve their quality of life.

Spanish Registry of Autoimmune and Cholestatic Liver Diseases (ColHai)

The purpose of the registry is to know the status of primary biliary colgantis, autoimmune hepatitis, primary sclerosing cholagitis and genetic cholestatic diseases in Spain.

PET/CT Scans Using the Tracer 11C-Csar, a Bile Acid Analog, to Depict and Visualize Changes in the Hepatobiliary System in Patients With Primary Biliary Cholangitis Before and After Treatment.

Purpose The primary purpose is to use an imaging diagnostic method to examine how the medication for primary biliary cholangitis (PBC) works. This will be studied using a radioactive tracer that behaves like human bile and can be observed with a PET/CT scanner. The aim is to gain insight into the disease mechanisms and how the medication affects them. The standard treatment for PBC is ursodeoxycholic acid, also known as Ursochol, which stimulates bile flow. If treatment with Ursochol is insufficient, bezafibrate can be added. The effectiveness of treatment is currently monitored through blood tests, which do not necessarily reflect the severity of the liver disease. However, this can be assessed using advanced PET/CT scans with a radioactive tracer, 11C-CSAR, which quickly clears from the body. The purpose of this study is to use the method to show how 11C-CSAR moves through the liver and bile ducts in PBC patients before and after treatment with either Ursochol or a combination of Ursochol and bezafibrate. We aim to: * Observe how the disease affects the liver\'s handling of bile salts and how this changes with medication. * Determine the excretion kinetics of 11C-CSAR, including specific rate constants. * Assess changes in liver blood flow before and after treatment. * Compare routine blood tests with 11C-CSAR PET/CT findings to evaluate how well blood tests reflect actual liver involvement. Study Plan The scientific study involves two examination days. Both days follow the same procedure. Participants must arrive fasting on the examination day. An intravenous catheter will be placed in each arm, a catheter in a wrist vessel, and a liver vein catheter. The liver vein catheter will be inserted by a trained liver doctor through a small sheath in a neck vein, guided by ultrasound, and the final placement will be confirmed with fluoroscopy. The tracer 11C-CSAR and the dye indocyanine green (ICG) will be administered through the intravenous lines. ICG will be given as a constant infusion 90 minutes before the scan to distribute in the tissue. The tracer will be injected just before the scan. Blood samples will be taken from the liver catheter and wrist catheter during the scan, which lasts approximately 45 minutes. After the scan, the catheters will be removed, and the participant can leave shortly afterward. About 250-300 ml of blood will be drawn during the scan, which will have no significant impact on participants. The entire process is expected to take about four hours. Study Participants Our goal is to include 20 newly diagnosed PBC patients and 10 patients who do not respond to standard treatment and are about to begin bezafibrate. Some participants may complete both parts of the study. If newly diagnosed patients do not respond sufficiently to Ursochol and need to start bezafibrate while the study is ongoing, they may participate both before and after starting Ursochol and bezafibrate.