Pharr

Researchers are evaluating AZD0780, an oral PCSK9 inhibitor, in a phase 3, randomized, placebo-controlled study to see if it can reduce the risk of major adverse cardiovascular events (MACE-PLUS) in adults with established atherosclerotic cardiovascular disease (ASCVD) or those at high risk for a first ASCVD event. The study compares AZD0780 to a placebo and monitors participants from randomization until the primary analysis censoring date, followed by a final study closure visit. Participants will be randomly assigned to receive either oral AZD0780 or an oral placebo once daily. The treatment period lasts until the primary analysis censoring date, after which a study closure visit will occur. The study is event-driven and designed to assess the time to the first major cardiovascular event during treatment. During the study, participants will be closely monitored with various assessments to evaluate cardiovascular outcomes and safety over approximately 54 months. Researchers will track the time to first event of any component of MACE-PLUS and collect data to assess the effect of AZD0780 compared to placebo. The study includes regular visits and evaluations to ensure participant safety and adherence to treatment.

Researchers are evaluating an investigational drug called ALN-HSD for adults with Metabolic dysfunction-Associated SteatoHepatitis (MASH), a type of liver disease where fat buildup causes liver cell damage, inflammation, and scarring. This condition can lead to serious complications like cirrhosis and liver failure. The study aims to assess how ALN-HSD affects liver scarring associated with MASH and to explore its impact on liver function, inflammation, side effects, and how the drug and its breakdown products appear in the blood. Participants will receive either ALN-HSD or a placebo according to the study protocol in this Phase 2, randomized, double-blind, placebo-controlled trial. The treatment is given based on the protocol's schedule, but specific dosing details are not provided. The study focuses on adults with specific genetic risk factors for MASH and with certain disease stages, ensuring a targeted precision medicine approach. During the study, participants will be monitored for changes in quantitative liver fibrosis from the start of the study to week 52. Researchers will evaluate liver scarring, liver function, inflammation, drug levels in the blood, and any side effects. The study includes genetic testing and specific liver assessments like FibroScan and FAST scores. Participants will be followed closely to understand the drug's effects and safety over the one-year period.

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 evaluating the immune response and safety of an investigational chickenpox vaccine and a marketed measles, mumps, and rubella (MMR) vaccine when given to healthy children aged 12 to 15 months. This Phase 3a study compares the investigational varicella vaccine given as a muscle injection to Merck's chickenpox vaccine administered just under the skin. The study also looks at the immune response and safety of giving these GSK vaccines together with other routine childhood vaccines via muscle injection. Participants receive either the investigational varicella vaccine by intramuscular injection or the marketed varicella vaccine given subcutaneously. The MMR vaccine is administered either subcutaneously or intramuscularly. Other vaccines such as Hepatitis A vaccine, 13-valent, 15-valent (Vaxneuvance), or 20-valent pneumococcal conjugate vaccines may be co-administered intramuscularly depending on country recommendations and availability. During the study, researchers will measure immune responses by assessing antibodies to varicella zoster virus and MMR antigens at Day 43 after vaccination. They will also monitor safety and tolerability throughout the study period. Parents or legal representatives complete diaries and return for follow-up visits to support ongoing safety and immunogenicity assessments. Overall, the study aims to understand how well the vaccines work and how safe they are when given to young children in this age group.

This trial investigates whether maridebart cafraglutide is better than a placebo at reducing liver fat and body weight in adults who are overweight or obese and have elevated liver fat. Participants must be adults with a body mass index between 27 and 40 kg/m2, including those with type 2 diabetes under specific treatment conditions. The study is designed as a Phase 2b randomized, double-blind, placebo-controlled trial to assess the drug's effectiveness, safety, and tolerability. Participants will receive either maridebart cafraglutide or a placebo through subcutaneous injections, alongside a reduced-calorie diet and increased physical activity. The treatment period and dosing schedule are detailed within the study protocol, with injections administered regularly. The study includes careful monitoring and comparison between the drug and placebo groups to determine the effects on liver fat content and weight. During the trial, participants will undergo assessments including MRI scans to measure liver fat content and body weight evaluations at the start and at week 52. Other evaluations will monitor safety and tolerability throughout the study. The total participation period includes baseline assessments and a follow-up at 52 weeks, with detailed monitoring of liver fat and weight changes as the primary outcomes.