Treatment Trials

27 Clinical Trials for Various Conditions

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RECRUITING
Predicting Outcomes in Nonalcoholic Steatohepatitis With Advanced Fibrosis
Description

Nonalcoholic Steatohepatitis (NASH) is a condition with increased amount of fat, inflammation and scarring in the liver. In compensated cirrhosis, the liver is coping with this damage and maintaining its important functions. Decompensation occurs when the liver becomes unable to perform all of its functions adequately. Variceal hemorrhage (bleeding from abnormal vessels in the liver called varices), Ascites (abnormal build-up of fluid in the abdomen), and Encephalopathy (brain confusion as a result of the liver not being able to get rid of toxic substances) are three symptoms of liver decompensation. The purpose of this research study is to investigate better ways to routinely monitor the condition of patients with NASH with compensated cirrhosis and to better pinpoint the development of decompensation in the livers of these patients.

WITHDRAWN
Natural History of Non Alcoholic Fatty Liver Disease and Predictors of Advanced Fibrosis
Description

The prevalence of non-alcoholic fatty liver disease (NAFLD ) in the American population is approximately 30% in adults and 10% in children, making it the most common. Cause of chronic liver disease in the United States. Although the majority of patients with NAFLD have a benign clinical course, the development of non-alcoholic steatohepatitis (NASH ), with necro-inflammation and progressive fibrosis, increases the risk for development of cirrhosis and its complications. Among patients with NASH, approximately 28% develop cirrhosis over an 8-year follow-up period. NASH and advanced fibrosis is associated with increased morbidity and mortality among those patients with advanced histologic severity such as NASH and fibrosis the gold standard for diagnosing and staging NAFLD is liver biopsy. Liver biopsy is associated with costs and risks that make it impractical for generalized use in a condition that affects such a high portion of the population. Furthermore, liver biopsy is also limited by significant sampling error in NAFLD. Thus, there is a pressing need for accurate non-invasive predictors of NAFLD that would also allow differentiation of those subjects at higher risk of disease progression. At present, in the clinical setting, some demographic factors, blood tests, and imaging studies can be used to predict a higher risk of disease in patients being evaluated for NAFLD. These predictors, however, are of limited sensitivity and specificity compared with liver biopsy. The development and validation of accurate predictors and scoring systems to identify patients at higher risk for NASH and fibrosis would allow identification of subjects who would benefit the most from liver biopsy and potentially help monitor disease

ACTIVE_NOT_RECRUITING
Prevalence of NAFLD and Advanced Fibrosis in Patients With Type 1 Diabetes
Description

The aim of this study is to assess the prevalence of nonalcoholic fatty liver disease (NAFLD) in patients with type 1 diabetes receiving care at Joslin clinic using noninvasive imaging and serum-based methods with the goal of identifying high-risk patients with advanced fibrosis who should be prioritized for specialty referral

COMPLETED
Hepatic Impairment, Cholestatic Liver Disease, & NASH with Advanced Fibrosis & Normal Hepatic Function
Description

This will be a Phase 1, Open-label Study of Participants with Hepatic Impairment, Cholestatic Liver Disease, and NASH with Advanced Fibrosis and Normal Hepatic Function

COMPLETED
Clinical Trial to Evaluate Efficacy of GR-MD-02 for Treatment of Liver Fibrosis in Patients With NASH With Advanced Fibrosis
Description

A Randomized, Controlled, Double-blind, Parallel Group, Single Center Phase 2 Clinical Trial to Evaluate Multiple Non-Invasive Liver Fibrosis Imaging Methods in the Assessment of the Efficacy of GR-MD-02 for the Treatment of Liver Fibrosis in Patients with NASH with Advanced Fibrosis

COMPLETED
Phase 1 Study to Evaluate Safety of GR-MD-02 in Subjects With Non-Alcoholic Steatohepatitis (NASH) and Advanced Fibrosis
Description

The primary objective of this study is to characterize the safety, tolerability and dose-limiting toxicities (DLTs) for GR-MD-02 when administered intravenously to subjects with biopsy-proven NASH with advanced liver fibrosis.

COMPLETED
A Study to Evaluate Liver Stiffness With Shear Wave Elastography
Description

The purpose of the study is to assess the diagnostic accuracy of Endoscopic Ultrasound (EUS) shear wave elastography in liver fibrosis staging in both normal subjects and subjects with advanced liver fibrosis/cirrhosis

COMPLETED
Study of Long-term Peg Intron vs. Colchicine in Non-responders.
Description

In this study Peg-Intron will be tested to see if it will give better results than Colchicine. At this time, there is currently no recommended maintenance treatment for patients who have failed to respond to Interferon/Rebetron/Peg Intron and have advanced fibrosis. The purpose of this study is to compare two treatments to slow down the progression of liver disease and to prevent liver failure and liver cancer. The treatment will not cure Hepatitis C, but is being evaluated to see if it can slow down disease progression.

TERMINATED
Impact of Fructose on Metabolism, Energy Homeostasis and Magnetic Resonance Biomarkers in Nonalcoholic Fatty Liver Disease
Description

This study will advance several goals of the NIH Action Plan: 1) establish a multidisciplinary team to develop quantitative methodologies and imaging protocols for liver, 2) validate diagnostic criteria and methodologies for imaging in liver in both a cross-sectional and a longitudinal dietary intervention study of patients with Nonalcoholic Fatty Liver Disease (NAFLD), 3) create a liver tissue bank with correlative imaging data, 4) develop reliable non-invasive MR markers to distinguish simple steatosis from Nonalcoholic Steatohepatitis (NASH), and 5) define the dynamic changes in metabolism, energy homeostasis, and MR biomarkers as they relate to fructose-related liver injury.

RECRUITING
LIVERAGE™: A Study to Test Whether Survodutide Helps People With a Liver Disease Called NASH/MASH Who Have Moderate or Advanced Liver Fibrosis
Description

This study is open to adults who are at least 18 years old and have: * a confirmed liver disease called non-alcoholic steatohepatitis (NASH)/metabolic-associated steatohepatitis (MASH) and * moderate or advanced liver fibrosis People with a history of acute or chronic liver diseases other than MASH or chronic alcohol intake cannot take part in this study. The purpose of this study is to find out whether a medicine called survodutide helps people with MASH and moderate or advanced liver fibrosis improve their liver function. This study has 2 parts. The purpose of the first part of this study is to find out the effect of survodutide on MASH and liver fibrosis. The purpose of the second part is to find out how safe and effective survodutide is in improving liver function. Participants are put into 2 groups randomly, which means by chance. 1 group gets survodutide and 1 group gets placebo. Placebo looks like survodutide but does not contain any medicine. Each participant has twice the chance of getting survodutide. Participants and doctors do not know who is in which group. Participants inject survodutide or placebo under their skin once a week. The survodutide doses are slowly increased until the target dose is reached. All participants receive counselling to make changes to their diet and to exercise regularly. Participants are in the study for up to 7 years. During this time, they regularly visit the study site or have remote visits by video call. For about the first year of the study, participants have these visits every 2 weeks, increasing to every 4 weeks and then every 6 weeks. After being in the study for a little over a year participants will then alternate between visiting the study site or having a remote visit every 3 months until the end of the study. The doctors check participants' health and take note of any unwanted effects. The participants' body weight and effects on the stomach and intestines are regularly measured. At some visits the liver is measured using different imaging methods. At 2 or 3 visits doctors take a small sample of liver tissue (biopsy). The participants also fill in questionnaires about their symptoms and quality of life. The results are compared between the groups to see whether the treatment works.

COMPLETED
A Clinical Trial of ALE.F02 in Patients With Advanced Liver Fibrosis and/or With Mild Cirrhosis
Description

The purpose of this study is to evaluate how a human body processes ALE.F02 (pharmacokinetics profile) in patients with impaired liver function.

ACTIVE_NOT_RECRUITING
A Study to Evaluate the Efficacy and Safety of Rencofilstat in Subjects With NASH and Advanced Liver Fibrosis
Description

This is a randomized, double-blind, placebo-controlled, parallel-dosing, multi-center study to evaluate the efficacy and safety of Rencofilstat as evidenced by histopathological improvements in fibrosis in adult NASH subjects with F2 or F3 fibrosis (NASH CRN system). Antifibrotic biomarker activity will be evaluated on an exploratory basis.

COMPLETED
A Study of Experimental Medication BMS-986263 in Adults With Advanced Hepatic Fibrosis After Cure of Hepatitis C
Description

This is a study of experimental medication BMS-986263 in adult patients with advanced hepatic fibrosis (scar tissue in the liver caused by inflammation that is far on in progress) after the patient is cured of hepatitis C (an infection caused by a virus that attacks the liver and leads to inflammation).

TERMINATED
Safety and Efficacy of Simtuzumab (SIM, GS-6624) in Adults With Advanced Liver Fibrosis But Not Cirrhosis Secondary to Non-Alcoholic Steatohepatitis (NASH)
Description

The primary objective of this study is to evaluate whether SIM (formerly referred to as GS-6624) is effective at preventing the histologic progression of liver fibrosis and the clinical progression to cirrhosis in participants with NASH. It will consist of 2 phases: * Randomized Double-Blind Phase * Open-Label Phase (optional)

COMPLETED
Trial of Pentoxifylline in Patients With Functional Disability Caused by Radiation-Induced Advanced Regional Fibrosis
Description

After initial assessment of their condition by specified clinical and laboratory parameters, each of the patients will be treated for 8 weeks at the standard pentoxifylline dose (400 mg po TID). Objective and subjective response parameters will be re-assessed at the end of the treatment and 8 weeks later for possible decay of response.

COMPLETED
A Study to Investigate the Pharmacokinetics of AZD2389 When Administered Alone and in Combination With Itraconazole in Healthy Participants
Description

The purpose of this study is to assess the pharmacokinetics (PK) of AZD2389 when administered alone and in combination with itraconazole in healthy participants.

RECRUITING
A Study to Assess the Safety, Pharmacokinetics, and Activity of RO7790121 in Participants With Advanced MASH Liver Fibrosis
Description

The purpose of this study is to assess the safety, pharmacokinetics (PK), pharmacodynamics (PD), immunogenicity and activity of RO7790121 in participants with advanced metabolic dysfunction-associated steatohepatitis (MASH) fibrosis.

Conditions
RECRUITING
Advanced Imaging for Pulmonary Fibrosis
Description

The purpose of this study is to determine if measurements of active collagen deposition using \[68Ga\]CBP8 positron emission tomography (PET) and tissue injury using dynamic contrast-enhanced magnetic resonance imaging (DCE-MRI) can predict an individual patient's pace of disease progression in non-idiopathic pulmonary fibrosis interstitial lung disease (non-IPF ILD) and identify which individuals will develop progressive pulmonary fibrosis.

COMPLETED
Efficacy and Safety of Nintedanib Co-administered With Sildenafil in Idiopathic Pulmonary Fibrosis Patients With Advanced Lung Function Impairment
Description

To assess efficacy and safety of concomitant treatment with nintedanib and sildenafil in Idiopathic Pulmonary Fibrosis (IPF) patients with advanced lung function impairment.

COMPLETED
Study to Evaluate Lumacaftor and Ivacaftor Combination Therapy in Subjects 12 Years and Older With Advanced Lung Disease
Description

The purpose of this study is to evaluate the safety and tolerability of LUM/IVA combination therapy in subjects 12 years and older with CF and advanced lung disease and who are homozygous for the F508del CFTR mutation

COMPLETED
The NIH Exercise Therapy for Advanced Lung Disease Trials: Response and Adaptation to Aerobic Exercise in Patients With Interstitial Lung Disease
Description

Interstitial lung disease (ILD) is the result of over 200 etiological pathways arising from several different insults to the lung parenchyma: inhaled substances, drug side effects, connective tissue disease, infection, and malignancy. The disease can also be of idiopathic origin. If prolonged, the resulting inflammation causes permanent and progressive fibrotic reorganization of the parenchyma and small airways, which reduces the distensibility of the lung and impedes O2 and carbon dioxide (CO2) exchange. This study is a randomized controlled trial to determine the safety and efficacy of aerobic exercise for patients who have interstitial lung disease (ILD) uncomplicated by pulmonary hypertension. In an uncontrolled study, we observed more efficient cardiorespiratory function, increased physical work capacity, and improved health-related quality of life following aerobic exercise in this study population. Serious adverse events resulting from aerobic exercise training were not observed and our work to date has established plausibility for the efficacy of aerobic exercise training and its safety for patients with ILD.

COMPLETED
Assessment of Pulmonary Specialty Physicians' Approach to Advanced Care Planning in Patients With Chronic Pulmonary Diseases
Description

To understand current practices of pulmonary physicians in relation to Advanced Care Planning (ACP) in order to develop future disease-specific tools that will improve patient-physician communication about ACP.

RECRUITING
Assessing Changes in Myocardial Tissue and Blood in Patients With Advanced Heart Disease
Description

Hypothesis: Tissue and serum samples collected from end-stage heart failure patients receiving left ventricular assist device implantation (LVAD) or heart transplantation will provide information regarding the basic science of heart disease. Tissue and serum samples collected from a limited numbers of "healthy controls" (donor grafts that were not utilized for heart transplantation) will serve as a comparator in research database projects. Design: This is a registry project; there are no investigational treatments, drug or procedures associated with participation in registry activities. This project is an organized functional data and tissue data gathering and storing (database) endeavor with specific focus on the functional, structural, and molecular aspects of heart failure. Data collection will not immediately influence the course of treatment for any patient.

RECRUITING
A Study to Investigate the Pharmacokinetics of AZD2389 in Healthy Participants When Administered Alone and in Combination With Quinidine
Description

The purpose of this study is to assess the pharmacokinetics (PK) and safety of AZD2389 when administered alone and in combination with quinidine in healthy participants.

RECRUITING
A Study to Investigate How Multiple Oral Doses of AZD2389 Affect the Pharmacokinetics of Midazolam, Caffeine, and Bupropion in Healthy Participants
Description

The purpose of this study is to measure the effect of multiple doses of AZD2389 on the pharmacokinetics (PK) of midazolam, caffeine, and bupropion in healthy participants.

RECRUITING
Lung Transplant READY CF 2: CARING CF Ancillary RCT
Description

Lung transplant is an option for treating end-stage lung disease in cystic fibrosis (CF). In the United States, more people with CF and low lung function die each year than undergo lung transplant. More than half of people with CF who die without a lung transplant were never referred for consideration. Patient preference not to undergo lung transplant may account for 25-40% of decisions to defer referral. Patients' health discussion networks function to support individuals in health related matters and may provide critical support during the lung transplant journey. Increasing awareness of lung transplant, and promoting the process of deliberation and utilization of social support, could reduce the number of people with CF who die without lung transplant. Additionally, the most common patient-endorsed barrier to lung transplant discussions is a worry about being a burden on family and friends after lung transplant. For lung transplant recipients with complex post-operative courses, low social support is associated with increased mortality. Additionally, adequate social support is a requirement at all lung transplant programs in the US. Investigators are interested in understanding how caregivers may benefit from using lung transplant educational resources and how caregivers prepare for having discussions with their loved ones and/or helping them make decisions about lung transplant as a treatment option for advanced CF. The purpose of this study is to test whether an investigator-designed research website compared to no caregiver intervention reduces caregiver burden (assessed with the Brief Assessment Scale for Caregivers, BASC), caregiver preparedness for lung transplant discussions, and caregiver lung transplant knowledge as an ancillary study in a multicenter RCT. Further, investigators will assess patient perceptions of caregiver support as measured by the Social Support Effectiveness Questionnaire (SSE-Q) and evaluate caregivers' willingness to provide support through semi-structured interviews in patient-caregiver dyads. Study involvement will span 6 months and study activities will involve the following: * Three Zoom research sessions (15-90 minutes each) * Survey assessments and an interview * Access to a research website that contains educational resources about lung transplant

Conditions
RECRUITING
Lung Transplant READY CF 2: A Multi-site RCT
Description

Lung transplant is an option for treating end-stage lung disease in cystic fibrosis (CF). In the United States, more people with CF and low lung function die each year than undergo lung transplant. More than half of people with CF who die without a lung transplant were never referred for consideration. Patient preference not to undergo lung transplant may account for 25-40% of decisions to defer referral. Rates of death without transplant are higher for people with CF who are members of marginalized communities, including those with Black race, Hispanic ethnicity, or low socioeconomic status. Increasing awareness of lung transplant among people with CF, and promoting understanding of the risks and benefits of transplant, can potentially reduce the number of people with CF who die without a lung transplant. The CF Foundation (CFF) lung transplant referral guidelines were developed to optimize the timing of referral for lung transplant. These guidelines recommend annual conversations with people with CF once their forced expiratory volume in one second (FEV1) is \<50% predicted. Considering lung transplant as a treatment option before it is medically needed will allow more time to learn about lung transplant and address any barriers to lung transplant that may exist. Investigators are interested in understanding how people with CF use lung transplant educational resources and how one prepares for having discussions and/or making decisions about lung transplant as a treatment option for advanced CF. The purpose of this study is to test whether a research website improves patient preparedness for discussions about lung transplant. Investigators also aim to understand whether there are unique factors that affect people with CF from communities with decreased access to transplant ("communities of concern"). Study involvement will span 6 months and study activities will involve the following: * Four Zoom research sessions (15-30 minutes each) * Survey assessments * Access to a research website that contains educational resources about lung transplant * Audio recording of a routine CF clinic visit to determine if and how lung transplant is discussed between a participant and his/her/their CF doctor

Conditions