Penarth

Researchers are evaluating the safety, tolerability, and effectiveness of VX-522 in people aged 18 to 65 with cystic fibrosis who have specific genetic mutations that do not respond to current CFTR modulator therapies. This Phase 1/2 study focuses on participants with stable cystic fibrosis and particular CFTR gene mutations that result in little or no CFTR protein production. Participants will receive VX-522 through oral inhalation using a nebulizer. The study includes dose escalation to assess safety and tolerability. Another drug, IVA, is provided as a tablet for oral use. The study timeline includes an 8-week period during which safety and adverse events are closely monitored. During the study, participants will undergo assessments including lung function tests such as forced expiratory volume in 1 second (FEV1). Safety is measured by recording any adverse events or serious adverse events from day 1 through week 8. Eligibility requires stable health status and specific lung function criteria. Participant weight and body mass index are also considered to ensure suitability for the study.

Researchers are evaluating maridebart cafraglutide, a drug given as an addition to standard care, to see if it reduces heart-related problems and deaths better than a placebo in people with atherosclerotic cardiovascular disease who are overweight or obese. This phase 3 study focuses on cardiovascular events such as heart attacks, strokes, and deaths related to heart conditions, aiming to improve outcomes in this high-risk population. Participants will receive either maridebart cafraglutide or a placebo, both administered by injection under the skin. The study compares these two groups over a period of up to approximately 35 months, monitoring heart-related health events to assess the drug's impact. The placebo group will receive injections that look identical but contain no active drug, ensuring a double-blind study design. During the study, participants will be regularly evaluated for major cardiovascular events, including heart attack, stroke, heart failure, and death. Researchers will track the time until these events occur to measure the drug's effectiveness. Safety and health will be closely monitored throughout the study period, and participants will be followed for up to nearly three years to gather comprehensive data on cardiovascular outcomes and overall survival.

Mycobacterium abscessus (MABS) is a group of rapidly growing, multi-drug resistant bacteria that can cause serious lung infections, especially in people with underlying inflammatory lung conditions. These infections, called MABS pulmonary disease (MABS-PD), can lead to worsened lung function, increased healthcare needs, and reduced quality of life. The trial, called Finding the Optimal Regimen for Mycobacterium Abscessus Treatment (FORMaT), aims to find the best treatment plans to clear MABS infections while minimizing side effects and treatment burdens. It also seeks to develop biomarkers to help guide treatment decisions and measure disease severity. The trial uses an innovative platform design to test and improve different treatment combinations for both children and adults with MABS-PD. Treatments include various intravenous and oral antibiotics such as amikacin, tigecycline, imipenem, cefoxitin, azithromycin, clarithromycin, clofazimine, ethambutol, linezolid, co-trimoxazole, doxycycline, moxifloxacin, bedaquiline, and rifabutin. The study includes phases of intensive intravenous therapy followed by consolidation with oral and/or inhaled antibiotics. Different durations and combinations of therapy are tested, including short and prolonged intensive treatments and consolidation therapies with or without inhaled amikacin. Participants will be involved in the study for up to 62 weeks, depending on their treatment group. Throughout the study, researchers will collect respiratory samples to monitor bacterial clearance and evaluate tolerance to treatments using standardized criteria for side effects. Additional assessments include quality of life measures, gene expression, imaging, and antibiotic resistance studies. The trial also includes an observational cohort for participants not receiving active treatment, allowing transition to the intervention program if eligible. Safety and treatment response will be closely monitored during and after therapy.