Pharr

Researchers are evaluating AZD0780, an oral PCSK9 inhibitor, in a phase 3, randomized, placebo-controlled study. This trial focuses on patients with established atherosclerotic cardiovascular disease ASCVD or those at high risk for a first ASCVD event. The study aims to assess how AZD0780 compares to placebo in reducing the risk of major adverse cardiovascular events, also known as MACE-PLUS, over the course of the trial. Participants are randomly assigned to receive either oral AZD0780 once daily or a matching placebo once daily. The study continues until a primary analysis censoring date, which may be up to approximately 54 months from randomization. After this, a study closure visit will be conducted as the final visit for each participant. During the study, participants will be regularly monitored for cardiovascular events including heart attacks, strokes, urgent coronary revascularizations, and other related outcomes. Researchers will track the time to first occurrence of these events as the primary outcome. Safety and other secondary outcomes like all-cause mortality will also be assessed. The total participation time can last up to about 54 months, with ongoing evaluations throughout this period.

Researchers are studying an investigational drug called ALN-HSD in adults with Metabolic Dysfunction-Associated SteatoHepatitis MASH, a liver condition caused by fat buildup that damages liver cells and causes inflammation and scarring. This condition can worsen to cirrhosis and liver failure. The study aims to evaluate how ALN-HSD affects liver scarring related to MASH and to understand its impact on liver function and inflammation, as well as potential side effects and how the drug is processed in the body. Participants will be randomly assigned to receive either ALN-HSD or a placebo in a double-blind setup. The study involves a 52-week treatment period during which the effects of ALN-HSD on liver fibrosis and other liver-related biomarkers will be assessed. The trial includes genetic risk factor screening for enrollment and collects data on drug levels and metabolites. Treatment is administered according to the study protocol, with monitoring continuing through week 84 for adverse events. Throughout the study, participants will undergo liver biopsies and various laboratory tests to measure liver fibrosis, enzyme levels, and other biomarkers related to MASH. Researchers will track changes from baseline to week 52 in liver fibrosis and inflammation, along with monitoring adverse events until week 84. Participants are involved in regular assessments to evaluate the study drugs impact on their liver health over the course of the trial.

Researchers are evaluating efruxifermin EFX in a phase 3, randomized, double-blind, placebo-controlled study involving adults with compensated cirrhosis caused by NASH Nonalcoholic Steatohepatitis or MASH Metabolic Dysfunction-Associated Steatohepatitis. This study aims to assess the safety and effectiveness of EFX in preventing significant clinical events such as disease progression and liver decompensation over a period of up to 5 years. Participants are randomly assigned to receive either efruxifermin 50 mg or a placebo, both given by subcutaneous injection. The study includes two cohorts one with biopsy-proven compensated cirrhosis and specific metabolic scores, and another with biopsy-proven or non-invasive diagnosis of compensated cirrhosis. The study treatment and monitoring extend up to 5 years, with evaluations at 96 weeks and long-term follow-up to track liver fibrosis, markers of liver injury, insulin sensitivity, glycemic control, body weight, and safety outcomes. During the trial, participants undergo regular assessments including laboratory tests, ECGs, ultrasounds, and vital sign monitoring. Researchers will measure changes in liver fibrosis, steatohepatitis resolution, and metabolic markers throughout the study. Safety and tolerability are closely tracked by documenting adverse events and exposure duration. The study duration allows for long-term observation of treatment effects and disease progression, with participant involvement lasting up to 5 years.

Researchers are evaluating the immune response and safety of GlaxoSmithKlines investigational chickenpox vaccine and a marketed measles, mumps, and rubella MMR vaccine when given to healthy children aged 12 to 15 months. The study compares these vaccines administered by muscle injection to Mercks chickenpox vaccine given just under the skin. It also assesses the immune response and safety when the GSK vaccines are given together with other routine childhood vaccines. Participants are randomly assigned to receive either the candidate varicella vaccine intramuscularly along with MMR, hepatitis A virus HAV vaccine, and a pneumococcal conjugate vaccine PCV, or the marketed varicella vaccine subcutaneously with the same additional vaccines. The PCV given may be PCV 13, Vaxneuvance, or PCV 20 depending on availability and national recommendations. All vaccines are given on Day 1. Throughout the study, children are monitored for immune responses by measuring antibody levels against varicella zoster virus and MMR antigens at Day 43. Safety is evaluated by tracking any local and systemic reactions in the days following vaccination, as well as any adverse events up to six months after the dose. The study aims to understand both the immune response and safety profile over this period in healthy young children receiving these vaccines.

Researchers are evaluating maridebart cafraglutide in adults living with overweight or obesity and elevated liver fat to determine if it is better than a placebo in reducing liver fat content and body weight. This Phase 2b randomized, double-blind, placebo-controlled trial aims to assess the drugs efficacy, safety, and tolerability when combined with a reduced-calorie diet and increased physical activity. Participants include adults aged 18 to 99 with specific body mass index and liver fat requirements. Participants will be randomly assigned to receive either maridebart cafraglutide or a placebo as subcutaneous injections every 4 weeks for 52 weeks. Both groups will follow a reduced-calorie diet and increase physical activity during the study. The trial includes two groups one receiving the experimental drug and the other receiving placebo, allowing comparison of their effects on liver fat and body weight. During the study, participants will undergo various assessments including MRI scans to measure liver fat content and body composition, blood tests for liver enzymes and glucose control markers, and weight measurements at baseline, 24 weeks, and 52 weeks. Researchers will monitor treatment-emergent adverse events and serious adverse events throughout the study, which lasts approximately 69 weeks from screening to completion. This monitoring ensures participant safety and collects data on the drugs effects over time.

Researchers are evaluating the long-term effects of maridebart cafraglutide in adults with obesity or overweight. This extension study follows participants from a previous trial to assess the medications ongoing efficacy, safety, and tolerability over an extended period. The trial is designed as a phase 3 randomized and double-blind study to provide comprehensive information on treatment outcomes. Participants will receive different doses of maridebart cafraglutide administered by subcutaneous injection at varying intervals, including once every 4, 8, or 12 weeks. Some participants who received placebo or lower doses in the previous trial will undergo dose escalation or re-randomization to different dose groups or placebo. The study includes a dose-escalation phase for certain participants before initiating the assigned high dose. During the study, participants will be regularly monitored for changes in body weight compared to the original trial baseline, treatment-emergent adverse events, and serious adverse events. Additional assessments will track waist circumference, quality of life related to weight, and maintenance of weight loss. Participants are expected to complete visits and evaluations over approximately 48 weeks, with safety data collected up to 60 weeks from the start of the extension trial.