Nonalcoholic Steatohepatitis (NASH)

Researchers are studying chronic liver diseases and tumors, including non-alcoholic steatohepatitis (NASH), non-alcoholic fatty liver disease (NAFLD), cirrhosis, and hepatocellular carcinoma (HCC). These conditions can be caused by viral infections like hepatitis B, C, and D or lifestyle factors such as overeating and lack of exercise. The goal is to develop better understanding and new treatments by analyzing liver tissues and tumors at the single-cell level to identify therapeutic targets and improve patient outcomes. The study involves creating patient-derived preclinical models, such as spheroid cultures and mouse xenograft models, to test new treatment strategies. Blood samples and tissue biopsies are collected during surgeries, biopsies, or diagnostic tests. Researchers use advanced molecular techniques like single-cell RNA sequencing to examine tumor heterogeneity and the liver environment, aiming to discover new biomarkers and treatment predictors. Participants will undergo procedures as part of their care, including hepato-bilio-pancreatic surgery, biopsies, or locoregional treatments. Blood samples are taken during these procedures to support research. Researchers will monitor surgery outcomes over an average of eight years. By studying patient samples and clinical data, the team aims to improve personalized medicine approaches and identify new preventive and therapeutic targets for chronic liver diseases and liver cancer.

Researchers are evaluating the safety and tolerability of Efimosfermin Alfa in adults aged 18 to 75 years who have known or suspected metabolic dysfunction-associated steatohepatitis (MASH) with liver fibrosis at stages F2 or F3. This phase 3, randomized, double-blind, placebo-controlled study aims to better understand how this drug affects participants with this liver condition related to metabolic syndrome components. Participants will be randomly assigned to one of three groups: one receiving Efimosfermin Alfa at dose level 1, another receiving Efimosfermin Alfa at dose level 2, and a third group receiving a placebo. The study will monitor the effects of these treatments over a period of 52 weeks, assessing safety and tolerability as well as changes in liver fibrosis markers and other related health measures. During the trial, participants will undergo regular assessments including laboratory tests, imaging scans such as MRI and elastography to measure liver stiffness and fat content, and blood tests to monitor liver enzymes and metabolic markers. Researchers will track treatment-emergent adverse events and laboratory abnormalities. The study involves continuous monitoring up to week 52 to evaluate changes from baseline and to ensure participant safety throughout the trial.

Researchers are evaluating whether a new blood biomarker can help identify depression and schizophrenia in patients attending psychiatric clinics. The study aims to see if changes in this biomarker can distinguish depressed patients who respond to treatment from those who do not, and differentiate individuals having a first psychotic episode from those at risk of developing schizophrenia. This observational study involves up to 500 participants grouped by diagnosis into Depression (DEP) and Psychosis/Schizophrenia (PSY) arms. Participants will attend three study visits over six months, during which blood samples and standard mental health assessments will be collected. These visits will help researchers analyze the relationship between the biomarker characteristics and the mental health conditions. The study does not involve any treatments but focuses on monitoring and measuring the biomarker and clinical factors over time. During the study, participants will complete standardized mental health questionnaires at each visit, and researchers will collect blood samples to study the biomarker changes. The main outcomes measured include the relationship between depression or psychotic disorders and biomarker characteristics over the six-month period. Researchers will also explore how clinical and demographic factors relate to these biomarker changes. The total participation time spans about six months with three scheduled visits.

Researchers are evaluating QX1206 in an open-label phase 1b trial involving adults with Type 2 Diabetes Mellitus (T2DM) and Non-Alcoholic Fatty Liver Disease (NAFLD). The study aims to determine the recommended dose for future studies and examine the preliminary effects of QX1206 on blood sugar control and other metabolic factors. Participants will take QX1206 orally before bedtime. The trial will last up to 12 weeks, during which laboratory tests and measurements will be taken before the first dose and throughout the study to monitor effects and safety. The study focuses on finding the best dose and assessing changes in blood sugar and insulin resistance. During the study, participants will undergo regular assessments including blood tests for hemoglobin A1c, fasting glucose, insulin levels, and insulin resistance. Researchers will track the recommended phase 2 dose from day 1 through week 12. Patients must comply with study procedures and attend all visits. The total participation period is up to 12 weeks.

Researchers are evaluating the safety and effectiveness of efimosfermin alfa in people with metabolic dysfunction-associated steatohepatitis (MASH) who have biopsy-confirmed liver fibrosis at stage F2 or F3. This Phase 3, randomized, double-blind, placebo-controlled study aims to compare efimosfermin alfa to placebo in improving liver-related outcomes and resolving steatohepatitis over a long-term period. Participants will be randomly assigned to one of three groups: two different dose levels of efimosfermin alfa or a placebo group. The study treatment will be administered under medical supervision, and the trial includes multiple assessments at baseline, Week 52, and Month 48. The study will measure liver fibrosis improvement, steatohepatitis resolution, and liver-related clinical outcomes, among other key health indicators. During the study, participants will undergo liver biopsies, blood tests, imaging scans, and questionnaires to monitor liver health and treatment effects. Researchers will track clinical outcomes for up to 48 months from the start of treatment. Safety will be closely monitored through adverse event reporting and laboratory tests. Participants are expected to attend regular visits for evaluations and follow-up throughout the study duration.

Researchers are evaluating an investigational drug called ALN-HSD for adults with Metabolic Dysfunction-Associated SteatoHepatitis (MASH), a liver condition where fat buildup causes inflammation and scarring. The study aims to see how ALN-HSD affects liver scarring in MASH and explores how the drug works, its side effects, and how the body processes it. This is a phase 2, randomized, double-blind, placebo-controlled clinical trial focusing on participants with genetic risk factors for MASH. Participants will be randomly assigned to receive either ALN-HSD or a placebo in equal groups. The study drug and placebo are given according to the study protocol. The trial includes a treatment period lasting up to 52 weeks, with follow-up monitoring extending to 84 weeks to assess safety and side effects. During the study, participants will undergo evaluations including liver biopsies to measure changes in liver fibrosis, blood tests for liver enzymes and fibrosis biomarkers, and genetic assessments. Researchers will closely monitor side effects and the drug's behavior in the body. The main outcome is the change in quantitative liver fibrosis from baseline to week 52, with additional measures assessing liver function and disease progression. Participants' involvement may last up to 84 weeks to cover treatment and safety follow-up.

This research aims to better understand metabolic dysfunction-associated fatty liver disease (MAFLD), a condition defined by fatty liver with metabolic risks like obesity, diabetes, or metabolic problems. The study also seeks to compare MAFLD with non-alcoholic fatty liver disease (NAFLD), including patients who have liver fat but no metabolic risk, and those with both MAFLD and other liver diseases. The goal is to clarify clinical features and improve diagnosis and outcomes for these patients. The study includes two parts: first, a cross-sectional analysis collecting clinical data from patients with fatty liver to examine their characteristics; second, a long-term follow-up involving collection of plasma, urine, and stool samples to identify biomarkers that help predict disease progression and patient stratification. The trial will observe patients over time to assess these factors. Participants will undergo clinical data collection and sample provision, with researchers monitoring for outcomes such as all-cause death over 10 years. They will also track liver-related complications and other important health issues like cardiovascular disease. The study aims to understand how different metabolic factors relate to disease progression. This long-term observational approach will provide insights to inform better management of MAFLD and NAFLD.

Researchers are evaluating the effectiveness of bariatric surgery compared to standard medical treatment in obese patients with nonalcoholic steatohepatitis (NASH) complicated by advanced liver fibrosis (grades F3 and F4). This study aims to show whether bariatric surgery leads to the disappearance of NASH without worsening fibrosis in patients with a body mass index (BMI) between 30 and 35 kg/m² for F3 fibrosis, and between 30 and 50 kg/m² for F4 fibrosis. The trial is open-label and randomized, designed to compare surgical intervention with lifestyle therapy.

Researchers are evaluating the safety and tolerability of a new medicine called NNC4005-0001 in adults who have increased body weight and liver fat, specifically those with fatty liver disease. This early phase 1 clinical trial aims to understand how the medicine behaves in the body and whether it causes any side effects. The study is sponsored by Novo Nordisk A/S and is designed as a randomized, double-blind, placebo-controlled trial to ensure reliable results. Participants will receive a single injection of either NNC4005-0001 or a matching placebo under the skin. The trial includes up to six groups with increasing doses of the medicine to assess its effects at different levels. Each participant will only receive one dose, and the study will monitor them closely for about 7 to 8 months. During the study, participants will have various assessments to track safety and how the medicine is processed in the body. Researchers will record any side effects from the day of dosing until the end of the study visit on day 169. Blood samples will be taken to measure the concentration of the medicine over time, including the highest level reached and how quickly it is cleared from the body. The study involves regular visits and evaluations to ensure participant safety and gather important information about the treatment.

Researchers are evaluating efruxifermin (EFX) in a randomized, double-blind, placebo-controlled Phase 3 study involving adults aged 18 to 80 with compensated cirrhosis caused by Nonalcoholic Steatohepatitis (NASH) or Metabolic Dysfunction-Associated Steatohepatitis (MASH). The study aims to assess the safety and efficacy of EFX in preventing disease progression and liver complications over a long-term period. Participants receive either efruxifermin or a placebo by subcutaneous injection. The study includes multiple groups, with one cohort requiring biopsy-proven compensated cirrhosis and specific metabolic features, and another allowing biopsy or non-invasive diagnosis. The treatment and follow-up last up to five years, with detailed monitoring at 96 weeks and beyond. During the trial, participants will undergo regular assessments of liver fibrosis, markers of liver injury, lipoproteins, insulin sensitivity, glycemic control, and body weight. Researchers will also monitor adverse events, laboratory tests, ECGs, ultrasounds, and vital signs throughout the study. The primary outcome measures include time to significant clinical events and improvements in fibrosis and steatohepatitis, with extensive safety and efficacy evaluations conducted over five years.