Messina

Researchers are evaluating a digital support system called Evira designed to help treat childhood obesity, a growing global health concern linked to serious health problems like type 2 diabetes and high blood pressure. The study aims to see if adding Evira to the usual lifestyle treatment can improve outcomes for children and adolescents aged 4 to 17 years with obesity. Evira offers daily weight monitoring at home using a special scale connected to a mobile app, allowing parents and clinicians to closely track weight changes and communicate easily. Participants will be randomly assigned to one of two groups: the intervention group receives Evira support alongside standard lifestyle treatment, while the control group receives only the standard lifestyle care without restrictions on visits or clinical support. The 12-month study begins with information and training on using Evira, including installing scales and apps for parents and possibly the child. The intervention includes personalized weight loss targets for the first three months, with continuous monitoring and communication through the app and website. Throughout the study, all children will undergo clinical exams assessing puberty, heart and lung health, thyroid function, skin, and abdomen, as well as measurements of weight, height, and blood pressure. Blood tests may be done as needed. Participants and parents will complete questionnaires on quality of life, eating behaviors, and treatment satisfaction. Researchers will track weight changes over 12 months and collect safety and background information via electronic forms to evaluate the effectiveness of adding Evira to standard obesity care.

Neurodegenerative diseases (NDGs) such as Amyotrophic Lateral Sclerosis, Alzheimer's Disease, and Parkinson's Disease are serious disorders affecting older adults. Aging brains show changes linked to these diseases, especially in the connection between muscles and nerves at the neuromuscular junction (NMJ). This study, called the M-Brain project, aims to better understand how muscle and brain interactions relate to aging and NDGs by comparing people with good aging and those with bad aging, focusing on movement and muscle-nerve communication. The study will observe and analyze clinical and biological data from participants aged 60 and older, divided into groups based on their aging status and neurological health. It will assess muscle and neurological health through phenotyping and measure biological markers including biomarkers, microRNAs, and extracellular vesicles. The project aims to identify factors that influence muscle-brain communication and to understand how these relate to aging and neurodegenerative diseases. Participants will be evaluated at the start and after six months using the Edmonton Frail Scale to measure changes in frailty. The study will collect clinical information, cognitive tests, and muscle-related data to track how participants' health changes over time. This approach may help identify early indicators of neurodegenerative diseases and inform future clinical strategies to reduce risk and improve outcomes related to muscle and brain health in aging individuals.

Researchers are studying the long-term safety of TEGSEDI (inotersen) in adults with Hereditary Transthyretin Amyloidosis with Polyneuropathy (hATTR-PN), a rare inherited disease where abnormal proteins build up in nerves and organs. This disease progresses over time and can be fatal. The study aims to observe patients in real-world settings to better understand how safe TEGSEDI is over a period of 10 years. The study is non-interventional and includes participants from Europe, the US, and Canada who receive care at specialized centers. The study does not require any mandatory visits, tests, or treatments beyond participants' usual clinical care. Data are collected when patients enroll and during their regular follow-up visits at their clinical sites. There are two groups: those who have recently taken TEGSEDI within 25 weeks before joining the study, and those who have not taken TEGSEDI recently but are eligible for it and may be receiving other treatments for hATTR-PN. Participants will be monitored over a long period to track safety and treatment outcomes. Data collection aligns with routine clinical visits without added procedures. The main outcome is to further understand the long-term safety of TEGSEDI under normal care conditions, with follow-up planned for up to 10 years. Patients provide informed consent before joining, and their health status is observed as part of their standard care.

Researchers are evaluating the use of benralizumab in adults with severe eosinophilic asthma to understand its effectiveness in achieving partial and complete clinical remission. This multicenter, observational, prospective study called the ATHENA study aims to add real-world evidence on benralizumab's role in clinical practice. The study also seeks to explore the immunological effects of benralizumab to better understand asthma biology and to collect long-term safety data. Participants will receive benralizumab, administered as a 30mg subcutaneous injection following the approved prescribing information. The study focuses on patients who have either recently started benralizumab treatment within 7 days before enrollment or plan to start within 7 days after enrollment. Treatment and monitoring will follow routine clinical practice without additional interventions. During the study, researchers will track the number and percentage of patients who achieve clinical remission according to the SANI definition over 24 months. Participants will be closely monitored for treatment effectiveness, immunological changes, and safety outcomes throughout this period. The study involves collecting relevant clinical data and ensuring participants adhere to treatment and follow-up visits as per standard care.

Researchers are evaluating the effect of ziltivekimab on reducing atherosclerotic plaque in the blood vessels of the heart in people who have had a heart attack. This phase 3 study aims to determine if ziltivekimab can reduce plaque compared to a placebo. Ziltivekimab is a new medicine not yet approved anywhere in the world, and the study will last about 15 months. Participants will be randomly assigned to receive either ziltivekimab or a placebo, both given by injection under the skin. The study includes imaging of the coronary arteries using intravascular ultrasound and other techniques to measure changes in plaque over 52 weeks. Treatment starts as soon as possible, within 48 hours after a heart procedure called percutaneous coronary intervention (PCI). During the study, participants will undergo imaging tests to assess plaque changes in the heart vessels, blood tests, and safety monitoring. The main outcome measured is the change in percent atheroma volume in the arteries from the start of treatment to 52 weeks. Participants will be followed for about one year to track the effects and safety of the treatment.

Researchers are evaluating how well the approved weekly injectable insulin icodec controls blood sugar levels compared to daily injectable basal insulins in adults with type 2 diabetes. This Phase 4 study focuses on people who need to start basal insulin treatment and have had type 2 diabetes for at least 180 days. The goal is to understand the effectiveness of once-weekly insulin icodec against standard daily basal insulins in real-world clinical practice over about 13 months. Participants will receive either insulin icodec once a week or one of the daily basal insulin analogues, such as insulin glargine, insulin detemir, or insulin degludec. Both treatments are given by subcutaneous injection. The choice between weekly or daily insulin is based on current treatment standards for type 2 diabetes. The study lasts approximately 52 weeks, during which participants maintain their assigned insulin regimen. During the study, researchers will monitor changes in participants' blood sugar control using the glycated hemoglobin (HbA1c) test from the start until week 52. Participants will have their HbA1c measured within 90 days before starting the treatment. Safety and any reactions to the insulin will also be tracked. The study aims to assess how well the weekly insulin icodec works compared to daily basal insulins in managing blood sugar over a year.

Researchers are evaluating efruxifermin (EFX) in adults aged 18 to 80 who have compensated cirrhosis caused by nonalcoholic steatohepatitis (NASH) or metabolic dysfunction-associated steatohepatitis (MASH). This Phase 3, randomized, double-blind, placebo-controlled study aims to assess the safety and effectiveness of EFX in improving liver health and delaying disease progression in this population. The study focuses on subjects with advanced liver fibrosis (stage 4) but without liver decompensation. Participants are randomly assigned to receive either efruxifermin or a placebo, both administered by subcutaneous injection. The study includes two cohorts: Cohort 1 requires biopsy confirmation of liver fibrosis and specific metabolic features, while Cohort 2 allows biopsy or non-invasive diagnosis. Treatment and observation continue over an extended period to evaluate changes in liver fibrosis and clinical events. During the study, researchers will monitor the time until significant clinical events such as disease progression or liver decompensation occur, with a follow-up of up to five years. For Cohort 1, the proportion of participants showing improvement in fibrosis without worsening steatohepatitis will be assessed at 96 weeks. Participants will undergo regular evaluations including clinical assessments and laboratory tests to track liver function and safety throughout the study period.

Researchers are investigating the safety and effectiveness of efruxifermin in people with non-cirrhotic nonalcoholic steatohepatitis (NASH) or metabolic dysfunction-associated steatohepatitis (MASH) who have moderate to advanced liver fibrosis (stage 2 or 3). This Phase 3 study is randomized, double-blind, and placebo-controlled, enrolling a total of 1650 participants in two groups to evaluate treatment outcomes. Participants will receive either efruxifermin or a placebo by subcutaneous injection. The study involves two cohorts, with Cohort 1 including patients who have biopsy-confirmed NASH or MASH and specific liver fibrosis and activity scores. The treatment period and detailed dosing schedules are not provided but the study compares the effects of the active drug against placebo. During the study, participants will be monitored for improvement in liver disease status, including resolution of NASH/MASH and at least a one-stage improvement in liver fibrosis after 52 weeks for Cohort 1. Long-term outcomes such as event-free survival will be observed over 240 weeks. Safety and efficacy assessments will be conducted throughout the study period, including evaluations of liver histology and metabolic health.

Researchers are studying whether baricitinib can help preserve beta-cell function in children and adults newly diagnosed with type 1 diabetes. This Phase 3 trial focuses on participants aged 1 to less than 36 years who have recently been diagnosed with this condition. The goal is to understand if baricitinib, compared to a placebo, can maintain insulin-producing cell activity. Participants will be randomly assigned to receive either baricitinib or a placebo, both given orally. The study is double-blind, meaning neither participants nor researchers know who receives the active drug or placebo. Treatment and observation will continue for about 60 weeks. During the study, participants will undergo evaluations including measuring C-peptide levels to assess beta-cell function at the start and after 52 weeks. Researchers will monitor health status, collect laboratory tests, and track any side effects or changes in diabetes-related markers to determine the effects of baricitinib over the study period.

Researchers are evaluating the effectiveness and safety of a combination treatment called triple therapy, which includes bempedoic acid, ezetimibe, and either atorvastatin or rosuvastatin. This study focuses on patients with primary hypercholesterolemia or mixed dyslipidemia who are at high or very high cardiovascular risk. The goal is to understand how well this combination lowers LDL cholesterol (LDL-C) in a real-world clinical setting. The study observes patients who have already started triple therapy within the last four weeks. No drugs are administered as part of this study; instead, it monitors the ongoing treatment with bempedoic acid combined with ezetimibe and either rosuvastatin or atorvastatin. The study measures LDL-C changes from baseline to eight weeks after starting triple therapy and continues follow-up for one year to assess lipid goal achievement, adherence to therapy, treatment changes, laboratory value shifts, and occurrence of cardiovascular events. Participants will have their LDL-C levels and other lab values assessed at baseline, eight weeks, and one year after starting triple therapy. Researchers will collect data on adverse events, adherence to treatment, and cardiovascular outcomes such as heart attack, stroke, death from cardiovascular causes, and coronary procedures during the follow-up year. The study also tracks treatment pathways and changes over this period to better understand real-world use and effectiveness of this triple therapy approach.