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67 recruiting in MA
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Endoscopic Ultrasound Shear Wave Elastography in Patients With Non-alcoholic Fatty Liver Disease

Non-Alcoholic Fatty Liver Disease ยท Non-alcoholic Steatohepatitis ยท Obesity

The goal of this observation study is to assess whether endoscopic ultrasound shear wave elastography (EUS-SWE) may be a useful tool for liver fibrosis screening in patients with elevated body mass index and non alcoholic fatty liver disease as compared to other non-invasive screening modalities, which have traditionally had less accurate results in this population. The main questions it aims to answer are: * Determine accuracy of EUS-SWE for liver fibrosis screening compared to other non-invasive scoring systems, such as the FIB-4 score and Fibroscan in patients with elevated body mass index * Establish optimal stiffness (kPa) cutoffs for liver fibrosis grading for EUS-SWE for this patient population in reference to the gold standard liver biopsy, as no standard cutoffs currently exist. Participants will undergo routine endoscopic ultrasound as part of their standard clinical care and indication. Participants are consented for the procedure and undergoing the shear wave elastography. In addition to their standard ultrasound test, it takes on average an extra 2-3 minutes to perform the shear wave elastography. The procedure itself adds no additional risk to the patient and does not expose them to radiation.

Boston, MA18+ yrsAll genders
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A Study to Evaluate the Efficacy and Safety of Pegozafermin in Participants With Compensated Cirrhosis Due to MASH

Metabolic Dysfunction-Associated Steatohepatitis (MASH) / Nonalcoholic Steatohepatitis (NASH) With Compensated Cirrhosis

The study will assess the efficacy and safety of pegozafermin administered in participants with compensated cirrhosis due to MASH (biopsy-confirmed fibrosis stage F4 MASH \[previously known as nonalcoholic steatohepatitis, NASH\]).

Burlington, Framingham, MA18โ€“75 yrsAll genders
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COffee and Metabolites Modulating the Gut MicrobiomE in Colorectal caNCER

Colorectal Cancer ยท Coffee ยท Gastrointestinal Microbiome

This is research study is assessing the effects of 6-g daily use of freeze-dried instant coffee on liver fat and fibrosis and the gut microbiome and metabolome in patients who have completed routine treatment (including surgery, chemotherapy and radiotherapy) for stage I-III colorectal cancer.

Boston, MA18+ yrsAll genders
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Stereotactic Magnetic Resonance Guided Radiation Therapy

Pancreas Cancer ยท Lung Cancer ยท Renal Cancer

This is a master prospective Phase I-II trial evaluating feasibility and efficacy of stereotactic magnetic resonance (MR) guided adaptive radiation therapy (SMART) in patients with cancer. * The phase 1 study will evaluate the feasibility and safety of delivering SMART in patients with cancer. * Phase 2 will evaluate efficacy of SMART with specific reference to tumor control and improvement in patient reported outcome measures

Boston, MA18+ yrsAll genders
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Visugromab, Nivolumab and Lenvatinib Compared to Double Placebo and Lenvatinib in Unresectable or Metastatic Hepatocellular Carcinoma Post Anti-PD-(L)1 Failure

Unresectable or Metastatic Hepatocellular Carcinoma ยท Child-Pugh A Hepatocellular Carcinoma ยท Failure of First-Line Treatment That Included an Approved Anti PD-(L)1 Compound

This is a Phase 2b, randomized, blinded clinical trial investigating the efficacy and safety of visugromab in combination with nivolumab and Lenvatinib compared to double placebo and Lenvatinib in participants with unresectable or metastatic HCC and compensated liver function (Child-Pugh A) after failure of 1L treatment that included an anti-PD-(L)1 compound. The trial consists of 2 Parts: a non-randomized Safety-run-in part (Part 1) and the subsequent randomized part (Part 2) with 2 treatment arms (A and B). Randomization of participants into Treatment Arm A and B will continue until 40 efficacy-evaluable participants are enrolled into each Treatment Arm.

Boston, MA18โ€“100 yrsAll genders
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A Study Evaluating the Efficacy and Safety of Pegozafermin in Participants With MASH and Fibrosis (ENLIGHTEN-Fibrosis)

Metabolic Dysfunction-Associated Steatotic Liver Disease (MASH) / Nonalcoholic Steatohepatitis (NASH) With Fibrosis

The study will assess the efficacy and safety of 2 dose regimens of pegozafermin compared to placebo for the treatment of liver fibrosis stage F2 or F3 in adult participants with MASH.

Burlington, MA18โ€“80 yrsAll genders
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A Study of TSRA-196 in Adults With PiZZ Alpha-1 Antitrypsin Deficiency (AATD)

Alpha-1 Antitrypsin Deficiency (AATD)

This is a Phase 1/2, open-label, multi-center, dose escalation (Part 1), dose expansion (Part 2), and single repeat dose (Part 3) study to evaluate the safety, tolerability, efficacy, and PK/PD parameters of TSRA-196 in adults with the PiZZ genotype who have lung and/or liver disease associated with severe alpha-1 antitrypsin deficiency (AATD)

Boston, MA18โ€“70 yrsAll genders
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A Study of NST-6179 in Subjects With Intestinal Failure-Associated Liver Disease (IFALD).

Intestinal Failure Associated Liver Disease

This is a phase 2a, multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of NST-6179 in subjects with intestinal failure-associated liver disease (IFALD) receiving parenteral nutrition (PN). The study will be conducted in 2 sequential parts. Up to 36 subjects diagnosed with IFALD will be enrolled in the study, of which up to 18 subjects will be enrolled in each of the 2 parts and randomized (2:1) to receive NST-6179 (N=12/part) or matched placebo (N=6/part). Subjects in Part A will receive once daily (QD) oral administration of 800 mg (32 mL solution) NST-6179 or placebo for 4 weeks. The NST-6179 dose for Part B is planned to be 1200 mg QD for 12 weeks. Actual dose, however, will be determined during the safety review meeting.

Boston, MA16+ yrsAll genders
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A Study to Check Liver Health in Boys With XLMTM, a Serious Genetic Muscle Condition

X-Linked Myotubular Myopathy

XLMTM (X-linked myotubular myopathy) is a serious genetic muscle condition. It is caused by changes in the MTM1 gene which stops or slows down normal muscle development, causing severe muscle weakness. There is currently no cure for XLMTM. Ongoing care is needed to manage symptoms and prevent further medical problems from this condition. Recent research shows that individuals with XLMTM often have reduced bile flow which can affect liver and gallbladder health. Bile is a liquid made in the liver that helps digest fat. Ongoing liver health checks may help with the routine care of people with XLMTM. There is a need to understand liver problems that develop in individuals with XLMTM over time. The main aim of the study is to learn how many boys with XLMTM have new cases of liver problems during the study. This study is about collecting information only. This is known as an observational study. The individual's doctor decides on treatment, not the study sponsor (Astellas). In this study, boys under 18 diagnosed with XLMTM will be followed for about 1 year. The health of their liver and gallbladder will be checked about every 6 weeks. This can be done at home, if preferred. A scan called a Fibroscan (also known as transient elastography) will check for signs of scarring in the liver (fibrosis) and the build-up of lipids. It is suggested that each boy will have a Fibroscan when they start the study and another scan when they complete the study. This study will help understand liver, gallbladder, and bile duct issues in individuals with XLMTM over time. The goal is to improve their care and provide information to use in future clinical studies.

Boston, MAUp to 17 yrsMen
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A Study to Assess the Efficacy and Safety of FORE8394 in Participants With Cancer Harboring BRAF Alterations

Cancer Harboring BRAF Alterations ยท HGG ยท LGG

The objective of this Master Protocol is to evaluate the efficacy and safety of plixorafenib in participants with BRAF altered (BRAF V600E or BRAF fusions) locally advanced or metastatic solid tumors, or recurrent or progressive primary central nervous system (CNS) tumors, including rare BRAF V600E-mutated solid tumors, including anaplastic thyroid, ovarian, cholangiocarcinoma or other rare cancers.

Boston +more, MA8+ yrsAll genders
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National Liver Cancer Screening Trial

Carcinoma, Hepatocellular ยท Liver Cancer ยท Liver Cirrhosis

The National Liver Cancer Screening Trial is an adaptive randomized phase IV Trial comparing ultrasound-based versus biomarker-based screening in 5500 patients with cirrhosis from any etiology or patients with chronic hepatitis B infection. Eligible patients will be randomized in a 1:1 fashion to Arm A using semi-annual ultrasound and AFP-based screening or Arm B using semi-annual screening using GALAD alone. Randomization will be stratified by sex, enrolling site, Child Pugh class (A vs. B), and HCC etiology (viral vs. non-viral). Patients will be recruited from 15 sites (mix of tertiary care and large community health systems) over a 3-year period, and the primary endpoint of the phase IV trial, reduction in late-stage HCC, will be assessed after 5.5 years.

Boston, MA18โ€“85 yrsAll genders
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Effect of Endoscopic Sleeve Gastroplasty in Patients With Obesity and MASH: A Randomized Controlled Trial

Obesity ยท Liver Diseases ยท Liver Fibrosis

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.

Boston, MA18+ yrsAll genders
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Safety and Preliminary Anti-Tumor Activity of TYRA-430 in Advanced Hepatocellular Carcinoma and Other Solid Tumors With Activating FGF/FGFR Pathway Aberrations

Metastatic Hepatocellular Carcinoma ยท Solid Tumors ยท Solid Tumor, Adult

A Phase 1 study to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamic (PD), and preliminary antitumor activity of TYRA-430 in cancers with FGF/FGFR pathway aberrations, including locally advanced/metastatic hepatocellular carcinoma and other advanced solid tumors.

Boston, MA18+ yrsAll genders
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Integrated Cancer Repository for Cancer Research

Pancreatic Cancer ยท Thyroid Cancer ยท Lung Cancer

The iCaRe2 is a multi-institutional resource created and maintained by the Fred \& Pamela Buffett Cancer Center to collect and manage standardized, multi-dimensional, longitudinal data and biospecimens on consented adult cancer patients, high-risk individuals, and normal controls. The distinct characteristic of the iCaRe2 is its geographical coverage, with a significant percentage of small and rural hospitals and cancer centers. The iCaRe2 advances comprehensive studies of risk factors of cancer development and progression and enables the design of novel strategies for prevention, screening, early detection and personalized treatment of cancer. Centers with expertise in cancer epidemiology, genetics, biology, early detection, and patient care can collaborate by using the iCaRe2 as a platform for cohort and population studies.

Holyoke, MA19โ€“110 yrsAll genders
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