Clinical Research Directory

Liver Micro Flow - MRI

This study integrates and evaluates a series of novel MRI methods for quantifying blood perfusion and tissue microstructure. The proposed perfusion and microstructure measures may provide biomarkers for fibrosis, cirrhosis, portal hypertension, and response to treatment. The precision of these methods will be evaluated in 110 participants, including healthy volunteers, people with chronic liver disease (CLD), and people with liver fibrosis.

RTX001 Autologous Engineered Macrophages for Liver Cirrhosis

The purpose of this study is to assess the safety and efficacy of RTX001 in patients with end-stage liver disease. This study is the first time RTX001, a macrophage cell therapy engineered to have an anti-inflammatory and anti-fibrotic effect, will be given to humans.

Liver Cancer Prevention Randomized Control Trial

To determine the effectiveness of a behaviorally-based tailored disease management intervention in patients with fibrosis or steatosis and risk factors for cirrhosis.

Implementation of a Digital Clinic for Alcohol-associated Liver Disease and Alcohol Use Disorder (DALC)

The purpose of this study is to determine whether a multidisciplinary digital clinic will improve health outcomes, reduce costs, increase access, and improve provider satisfaction. The primary aim of this study is to improve clinical outcomes in patients with ALD through the implementation of a novel digital health platform for personalized multi-disciplinary treatment of patients with ALD and AUD. Secondary aims include improvement in provider and patient-reported outcomes including satisfaction with AUD treatment.

Development of a New Method for Liver Stiffness Measurement Using FibroScan

This is an European, prospective, interventional, multicenter clinical investigation that will take place in 2 French sites. 153 adults patients will be included. The study objective is to develop a new method for measuring liver stiffness using FibroScan.

Development of a FibroScan Liver Examination Using a Single Probe

This is an exploratory, international, prospective, interventional, multicenter clinical investigation that will take place in 1 Hong Kong site and 3 French sites and 309 adults patients will be included. The study objective is to assess the LSM reproducibility between the FibroScan examination performed with the Single Probe (SP) and the FibroScan examination performed with the reference probes (M and XL).

Everolimus bAsed caLcineurin inhibiTor frEe immunosuppRession oNe Year AfTer lIver transplantatiON (ALTERNATION)

The overall aim of this study is nephroprotection based on a calcineurin inhibitor (CNI)-free therapy beyond year one after orthotopic liver transplantation (OLT) in highly pre-selected patients with low rejection risk.

Normal Liver Stiffness by MR Elastography

Healthy children will be recruited to under Magnetic Resonance Imaging (MRI)along with MR elastography to determine normal liver stiffness values.

Patients With Hepatocellular Carcinoma (HCC)

Hepatocellular carcinoma (HCC) is one of the leading causes of cancer-related mortality worldwide and is frequently diagnosed at an advanced stage, resulting in limited therapeutic options. Despite the advances in immunotherapy, a substantial proportion of patients fail to respond adequately due to mechanisms of immune resistance. The gut microbiota plays a crucial role in modulating the response to immune checkpoint inhibitors (ICIs), and fecal microbiota transplantation (FMT) has demonstrated the ability to enhance their efficacy in other tumors, such as melanoma. In patients with HCC and cirrhosis, intestinal dysbiosis, characterized by a reduction in beneficial bacteria (e.g., Bifidobacterium, Akkermansia) and increased inflammation, is associated with an immunosuppressive profile. Furthermore, a dysbiosis index has been correlated with response to ICIs. In this context, FMT represents a promising strategy to enhance the efficacy of immunotherapy in HCC, although data regarding its efficacy and safety are still limited.

Early Detection of Hepatocellular Carcinoma in a High-risk Prospective Cohort (ELEGANCE)

Hepatocellular carcinoma (HCC) is the 7th most common cause of cancer death globally but only 20% are diagnosed in its early stages where cure is possible. Current standard-of-care surveillance of patients at high risk of developing HCC with 6-monthly serum alpha-fetoprotein (AFP) and ultrasound imaging (US) has a sensitivity of approximately 63% for detecting early HCC. There is an urgent need for a more efficacious and convenient modality of surveillance of high-risk patients to diagnose HCC at an early stage. This prospective study aims to address this unmet clinical need by validating a panel of circulating miRNA biomarkers to develop an in-vitro diagnostic (IVD) kit for the detection of early HCC in a cohort of high-risk patients. Additionally, this study also aims to develop a multi-parametric MRI-based AI algorithm to quantify individual risks of developing HCC and to predict the progression of chronic liver disease in this cohort to enable targeted surveillance. Lastly, by identifying changes in the microbiome and metabolites as HCC develops in this cohort enables the establishment of actionable biomarkers that can prevent and predict the development of HCC.

Primary Sclerosing Cholangitis in Children

Primary sclerosing cholangitis (PSC) is a rare liver disease that damages the liver's bile ducts. Bile ducts are tiny tubes that carry bile from the liver to the small intestine. Bile is a liquid produced by the liver that helps us absorb and use the nutrients in the food we eat. In people with PSC, the bile backs up into the liver and will damage it, causing scarring of the liver. The purposes of this study are to: * Collect medical and other data to learn more about PSC, how it progresses, and identify factors that may cause the disease to progress more quickly. * Ask questions about how PSC symptoms affect your child's life to learn more about its impact on your child's daily functioning * Children with PSC who are seen at one of the participating clinical sites in the Childhood Liver Disease Research Network (ChiLDReN) will be asked to contribute information, DNA, and other specimens. The information and specimens will be available to investigators to carry out approved research aimed at learning more about the possible causes and long-term effects of PSC.

Genetic Collection Protocol

This study involves the one-time collection of whole blood or saliva samples for the extraction and storage of DNA for use in ongoing and future ChiLDReN studies.

Safety and Clinical Activity of QEL-001 in A2-mismatch Liver Transplant Patients

The purpose of this study is to evaluate the safety and tolerability of QEL-001 in the prevention of liver transplant rejection following immunosuppression withdrawal. QEL-001 is a product made from a patients own cells, which are genetically modified and designed to help the transplant recipient's body accept their donated liver and prevent their immune system from rejecting it once immune suppression is withdrawn.

Screening for Alcohol-related Cognitive Impairments in Cirrhotic Patients

80% of patients with alcohol use disorders (AUD) present cognitive impairments, such as memory and executive functions. These disorders may have repercussions in addiction treatment by altering the patient's adherence to care. The level of impairment is dependent on the onset of addiction, and also the duration of abstinence. A complete neuropsychological evaluation is necessary to highlight cognitive impairments. In practice, the evaluation of these disorders by practitioners, is done with the help of tools of screening like the MoCa (Montreal cognitive assesment) and the BEARNI (Brief evaluation of alcohol related neuropsychological impairment). However, none of these tools have been evaluated in patients with alcoholic cirrhosis. Indeed, some studies have suggested that liver disorders including cirrhosis may be a factor aggravating cognitive disorders. The purpose of this study is to evaluate the ability of the BEARNI tool to detect alcohol-related cognitive problems in patients with alcohol-related cirrhosis.

Veteran-Centered Care for Advanced Liver Disease (Vet-CALD)

Advanced liver disease is a serious illness that disproportionately affects Veterans, many of whom hope for curative liver transplantation. However, too few receive a transplant and most continue to suffer from increasing symptoms and hospitalizations. The proposed project uses a whole person, Veteran-centered approach that identifies Veterans with advanced liver disease using a population-based health management system and integrates curative and early supportive care using a telemedicine-based nurse care counselor to (1) discuss patient's understanding of illness severity and prognosis, (2) identify priorities and care preferences and (3) align curative and supportive care options to achieve patient priorities. Study outcomes include changes in (1) rates of consideration for liver transplantation, and (2) completion of serious illness discussions. Findings will inform adaptations to the intervention and facilitators for its dissemination.

Effect of Endoscopic Sleeve Gastroplasty in Patients With Obesity and MASH: A Randomized Controlled Trial

Metabolic dysfunction-associated steatotic liver disease (MASLD) is the most common chronic liver disease globally. While weight loss through lifestyle modification is the standard treatment, most patients regain weight limiting ultimate improvement in liver disease. On the other end of the spectrum, bariatric surgery has shown promise in the treatment of MASLD/metabolic dysfunction-associated steatohepatitis (MASH) due to its efficacy in inducing weight loss. Nevertheless, its adoption has been hindered by the perceived invasiveness of surgery. Over the past decade, endoscopic sleeve gastroplasty (ESG) has gained recognition as a promising minimally-invasive approach to weight loss. The procedure involves utilizing a Food and Drug Administration (FDA)-authorized endoscopic suturing device to reduce the gastric volume by 70%. Studies reveal that ESG is associated with approximately 18.2% weight loss at one year after the procedure, with sustained results for at least 10 years. Nevertheless, the effect of ESG on MASH remains unknown. In this study, the investigators will compare ESG + lifestyle modification versus lifestyle modification alone in treating histologic MASH. The study will randomize patients to one of two different treatment options: ESG + lifestyle modification or lifestyle modification alone.

A Study to Assess the Safety and Pharmacokinetics of INCB123667 When Administered Orally to Adult Participants With Moderate or Severe Hepatic Impairment

This study will be conducted to assess the safety and pharmacokinetics of INCB123667 when administered orally to adult participants with moderate or severe hepatic impairment.

China Metastatic Liver Cancer Clinical Registry Cohort Database

This study is led by the Department of Hepatobiliary Surgery of the First Affiliated Hospital of the University of Science and Technology of China (USTC), in close collaboration with multiple participating centers nationwide. The primary objective is to establish a standardized, large-scale clinical cohort database specifically for metastatic liver cancer. By collecting comprehensive data on patient demographics, primary tumor characteristics, systemic therapies, and local interventions, this registry aims to elucidate the clinical patterns and prognostic factors of secondary liver malignancies. This database will serve as a robust platform for conducting high-quality real-world studies and advancing evidence-based clinical research in the management of metastatic liver cancer.

Comparison of Inflammatory Profiles and Regenerative Potential in Alcoholic Liver Disease

The main objective of this study is the comparison of the profile of the pro-inflammatory cytokines at the patients suffering from an alcoholic hepatitis to that of two groups witnesses: patients suffering from an alcoholic cirrhosis and unhurt patients of chronic liver disease

Restoring Bile Acid Homeostasis Via Lifestyle Adjustments to Prevent the Development of Liver Cancer

This is a prospective, single-arm, non-randomized interventional study nested within the existing ELEGANCE cohort. Patients eligible for the RE-BALANCE study will be selected from the ELEGANCE cohort based on predefined high-risk criteria, specifically a low 12-non-hydroxy/12-hydroxy bile acid ratio indicative of elevated hepatocarcinogenic risk. The study comprises Baseline Assessments (Visit 1), Intervention Visits (Visit 2-8), and Follow-up Assessments (Visit 9-11).

The Treatment of Liver Injury After Liver Resection With Polyene Phosphatidylcholine

A multicenter, open-label, randomized, controlled study. It is proposed to evaluate the treatment plan of receiving 930mg polyene phosphatidylcholine injection one day before the operation, twice a day, and 930mg polyene phosphatidylcholine injection twice a day,combined with 100mg of magnesium isoglycyrrhizinate injection once a day,from the 1st to the 5th day after the operation. Compared with the monotherapy regimen of no liver protection treatment before the operation and receiving magnesium isoglycyrrhizinate injection 100mg once a day from the 1st to the 5th day after the operation.The efficacy and safety of treating postoperative liver function injury in patients undergoing laparoscopic hepatectomy for hepatocellular carcinoma were compared.

Extracellular RNA Markers of Liver Disease and Cancer

The study will examine and evaluate the use of extracellular RNA in blood as markers for the diagnosis of liver disease or cancer, and as markers for prediction of response to treatment or recurrence of cancer after surgery

Impact of Iron Overload on the Incidence of Liver Complications in Long-Term Survivors (≥10 Years) of Allogeneic Hematopoietic Stem-Cell Transplantation.

This single-center, non interventional cohort study investigates whether chronic iron overload influences the incidence of liver complications in adults who are at least 10 years beyond allogeneic hematopoietic stem cell transplantation (allo HSCT). Approximately 400-500 survivors transplanted at Hôpital Saint Louis between January 2004 and December 2014 will be evaluated. Transplant characteristics, prior iron overload therapy, and historical hepatic events will be collected through the Promise database. At the same time, the prospective visit will include laboratory panels and non invasive liver stiffness measurement by FibroScan or shear wave elastography. The study's primary objective is to assess the impact of iron overload on the incidence of hepatic complications in patients more than 10 years after an allogeneic hematopoietic stem cell transplantation. Secondary aims include describing the spectrum and frequency of hepatic complications, determining risk factors (including graft versus host disease, conditioning regimen, and comorbidities), and evaluating the long term effectiveness of previous iron reduction treatments (phlebotomy or chelation). Results will clarify whether monitoring and treating iron overload in long term allo HSCT survivors can prevent late hepatic morbidity.

Assessing Declined Liver Grafts With Normothermic Machine Perfusion to Reduce Transplant Waiting Time

The goal of this study is to find out if quality assessment by normothermic machine perfusion can be used to safely increase the number of usable donor livers, helping more people get transplants faster and with better results. This process keeps a donated liver working outside the body before transplantation, allowing surgeons to assess whether livers previously considered unsuitable can still be used. The main questions this study aims to answer are: * Does this method help patients get a transplant sooner? * Can this method make more livers available for transplant? * Does it improve survival and health after transplant? Participants in this study must be on the waiting list for a liver transplant with a ReMELD-Na-Score of 21 or less (equivalent to MELD ≤25) and must not qualify for certain special exceptions. Participants will be randomly placed into one of two groups: * Experimental group: In addition to regular organ offers, these participants may receive a liver that was initially not considered for transplantation but meets quality standards after at least four hours of machine perfusion. * Control group: These participants will receive a liver through the usual transplant process. The main measure of success is how quickly participants receive a transplant. Researchers will also look at other important factors, such as survival rates, quality of life, hospital stay, and complications after transplant. This study may help improve liver transplantation by making better use of available donor livers, reducing waiting times, and improving patient outcomes.