The main goal of this study is to evaluate how well taking oral elafibranor 80 mg daily works, compared to a placebo, in reducing or preventing the occurrence of death, liver transplant, worsening of liver disease, and liver disease-related complications in adults with PBC.
The Alpha-1 Foundation Therapeutic Development Network (TDN) aims to make it easier to design and carry out clinical trials that enhance the treatment of patients with Alpha-1 Antitrypsin Deficiency (AATD). To achieve this, the TDN will establish a network of clinical trial centers that have enough patients to gather a comprehensive database of clinical and genetic information. This data will be crucial in determining the criteria for including or excluding participants in the trials and in recruiting suitable subjects.
Specifically, this study will enroll participants by in person or remote consent who will allow collection of medical records to be entered into an Alpha-1 TDN database. Participants will then be invited to future clinical trials.
In this study, we will recruit cirrhotic patients who are undergoing endocscopic procedures as part of their standard of care. Their endoscopies will reveal whether they have portal hypertensive gastropathy. After the procedure, we will ask the patients to provide us with a stool sample, which we will assess for occult GI bleeding. For those patients who DO NOT have occult GI bleeding, they will be contacted every 6 months for 2 years to check whether they have developed GI bleeding.
The study is designed to evaluate the effects of the combination of zibotentan and
dapagliflozin and dapagliflozin monotherapy versus placebo on the hepatic venous pressure gradient (HVPG) response in participants with cirrhosis with features of portal hypertension at 6 weeks of treatment.
Part A is to evaluate the absolute
change in HVPG at 6 weeks from baseline in participants treated with 2.5 mg zibotentan combined with 10 mg dapagliflozin versus placebo. The primary efficacy objective for Part B is to evaluate the proportion of participants treated with 1, 2.5, or 5 mg zibotentan combined with 10 mg dapagliflozin and 10 mg dapagliflozin monotherapy versus placebo achieving a ≥ 20% decrease in HVPG or a reduction to or below 12 mmHg in HVPG
The purpose of this study is to create a de-identified, public use,
repository of data of Chronic Obstructive Pulmonary Disease (COPD)
patients with by Alpha-1 antitrypsin deficiency (AATD), a rare genetic
condition that can cause COPD and emphysema.
The primary purpose of this registry is to evaluate the feasibility and clinical validation of LiverCare in liver transplant recipients, as part of post-transplant surveillance. LiverCare is an investigational panel test that includes 6 components: 1. AlloSure Liver 2. AlloMap Liver 3. AlloHeme Liver 4. iBox Liver 5. HistoMap Liver 6. AlloID. AlloSure Liver is a research test used to measure donor-derived cell free DNA in Liver transplant recipients. AlloMap Liver is a research gene expression profile test using peripheral blood to establish immune activity and is currently undergoing clinical evaluation and development. iBox Liver is an analytic platform that predicts organ outcomes after transplant using a software algorithm based on information from your medical records and is currently undergoing clinical evaluation and development. AlloHeme Liver is a diagnostic test to measure donor and recipient DNA in the blood. HistoMap Liver is a tissue-based gene expression test using tissue collected from standard of care biopsies to establish immune profiles within the organ and is currently undergoing clinical evaluation and development. AlloID is a blood test that will quantify the presence of more than 100 pathogens including standard post-transplant infectious disease screening such as Cytomegalovirus (CMV), Epstein-Barr virus (EBV), adenovirus, Human Herpesvirus 6 (HHV-6) and viral hepatitis.
The purpose of the MUSC Pulmonary Biorepository is to collect and store samples linked to medical and other information from individuals with pulmonary disease as well as healthy controls.
In combination with the clinical data and other approved research studies (that may recruit for and/or utilize samples of the biorepository) this sample repository will provide for uniform, longitudinal, complete and accurate data that can be organized and clinically correlated at the time of sample donation, with longitudinal testing possible as part of future study. Samples will be linked to each participant's unique ID, though will be deidentified and coded for use in future research and subsequent publications with pulmonary disease and control patients.