New Lambton

Researchers are evaluating different treatment strategies for patients hospitalized with Gram-negative bloodstream infections (GN BSIs) through the BALANCE+ trial. This adaptive platform trial uses an open-label, pragmatic design embedded in routine care to address important questions in managing GN BSIs, including antibiotic treatment duration, antibiotic de-escalation, oral antibiotic options, central line management, specific pathogen treatment, and follow-up blood cultures. The study builds on previous research and aims to improve patient outcomes and reduce antimicrobial resistance, a growing global health concern. The trial includes multiple treatment comparisons, such as de-escalation versus no de-escalation of antibiotics, oral beta-lactams versus non-beta-lactams, central vascular catheter retention versus replacement, cephalosporin versus carbapenem for low-risk AmpC organisms, and routine follow-up blood cultures versus no routine follow-up. Treatments are tailored based on blood culture results and clinical decisions, with specific protocols for antibiotic switching and catheter management. The trial uses Bayesian methods with interim analyses after every 1000 patients initially, then every 200 patients, and stops domains based on predefined criteria or sample sizes. Participants are patients admitted to hospitals with confirmed Gram-negative bacteremia who meet eligibility criteria for each domain. Assessments include monitoring for death, reinfection, readmission, and new antimicrobial resistance over 90 days, measured by the Desirability of Outcome Ranking (DOOR) scale. The trial incorporates detailed inclusion and exclusion criteria and collects data through routine clinical care, ensuring ongoing evaluation of treatment effectiveness and safety throughout the study period.

Researchers are evaluating the safety and effectiveness of two doses of inhaled pirfenidone (called AP01) compared to a placebo in people with progressive pulmonary fibrosis (PPF). This Phase 2b study is randomized, double-blind, and placebo-controlled, involving up to 300 participants who will continue their standard care during the 52-week trial. The goal is to see how well AP01 works and how safe it is when added to usual treatments for PPF. Participants will be randomly assigned to one of three groups: high-dose AP01, low-dose AP01, or placebo. All treatments are given as an oral inhalation solution twice daily. The study will last for 52 weeks, during which researchers will monitor and compare the effects of these treatments on lung function and disease progression. During the study, participants will undergo various assessments including lung function tests and clinical evaluations to track their respiratory health. Researchers will check for changes in lung capacity and symptoms and monitor safety throughout the treatment period. The main outcome measured is the impact of AP01 doses compared to placebo after 52 weeks of treatment.

Researchers are evaluating the effects of TX000045 in patients with pulmonary hypertension caused by heart failure with preserved ejection fraction (PH-HFpEF). This Phase 2, double-blind, randomized, placebo-controlled proof-of-concept study aims to assess two dosing regimens of TX000045 over a 24-week treatment period to understand its impact on pulmonary vascular resistance and safety profile. Participants will be randomly assigned to one of three groups: a placebo group receiving subcutaneous injections every two weeks, a group receiving Dose A of TX000045 subcutaneously every two weeks, and a group receiving Dose B of TX000045 subcutaneously every four weeks alternating with placebo every two weeks. The treatment period lasts for 24 weeks. Throughout the study, participants will undergo assessments including pulmonary vascular resistance measurements, physical examinations, laboratory tests, and monitoring for adverse events from baseline up to 30 weeks after the first dose. Safety evaluations focus on treatment-related side effects and changes in lab values. The study plans to enroll about 180 participants between 18 and 83 years old with specific heart and lung function criteria.

Researchers are evaluating the effectiveness of satralizumab compared to a placebo in treating Myelin Oligodendrocyte Glycoprotein Antibody-Associated Disease (MOGAD). This Phase III study focuses on the time from randomization to the first confirmed MOGAD relapse during the double-blind treatment period. Participants who experience a relapse or complete the double-blind phase may continue into an open-label extension period, with additional adolescents possibly joining the extension after the primary clinical cutoff. The study involves administering satralizumab or placebo by subcutaneous injection in the abdominal or femoral area during site visits after completing other study procedures. Treatment is given as either monotherapy or alongside baseline therapy. Participants remain in the double-blind phase until relapse or study completion, after which they may enter the open-label extension to continue receiving satralizumab. During the study, participants undergo regular assessments to monitor for relapses, including evaluations by an adjudication committee. Researchers measure the time to first MOGAD relapse while also monitoring safety, pharmacokinetics, and pharmacodynamics. The study includes ongoing follow-up and safety monitoring for up to approximately 44 months from randomization, ensuring comprehensive evaluation of the treatment's impact.

Researchers are evaluating the safety and effectiveness of subcutaneous anifrolumab compared with placebo in adults with moderate to severe Idiopathic Inflammatory Myopathies (IIM), specifically polymyositis (PM) or dermatomyositis (DM). This multicenter, randomized, double-blind, placebo-controlled Phase III study adds anifrolumab or placebo to participants' standard of care treatment to assess overall disease activity. Participants will receive weekly subcutaneous injections of either anifrolumab or placebo for 52 weeks. After this period, all participants will receive open-label anifrolumab injections once weekly for an additional 52 weeks. This design allows researchers to evaluate initial treatment effects and longer-term outcomes with anifrolumab. During the study, participants will be monitored regularly for disease activity and safety. The main outcome measured is the Total Improvement Score (TIS) with a response defined as a score of 40 or higher at 52 weeks. The total study participation lasts up to 104 weeks, including the double-blind and open-label extension periods, ensuring comprehensive assessment of the treatment's impact and participant safety.

Researchers are conducting a Phase 3, multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety and effectiveness of tezepelumab in adults aged 40 to 80 years with moderate to very severe chronic obstructive pulmonary disease (COPD). Participants must have experienced at least two moderate or one severe COPD exacerbations in the year before joining and be receiving inhaled maintenance therapy. The study focuses on adults who continue to experience symptoms despite current treatments and aims to assess the impact of tezepelumab on COPD exacerbations. Participants will be randomly assigned to receive monthly subcutaneous injections of either one of two doses of tezepelumab or a placebo. Treatment will last for a minimum of 52 weeks and may extend up to 76 weeks. After the treatment period, there will be a 12-week safety follow-up phase to monitor participants after stopping the study drug. The study compares tezepelumab to placebo to determine its efficacy and safety over this extended period. During the study, participants will undergo regular assessments to monitor their COPD status and any exacerbations. The main outcome measured is the annual rate of moderate or severe COPD exacerbations from the start of treatment through up to 76 weeks. Safety and tolerability will also be closely monitored throughout the treatment and follow-up periods. This long-term involvement ensures comprehensive data on how tezepelumab affects COPD progression and exacerbation frequency.

Researchers are evaluating the effects of felzartamab in adults with Immunoglobulin A nephropathy (IgAN), a kidney disease caused by the buildup of abnormal IgA antibodies in the kidneys. This buildup leads to inflammation and damage, causing protein to appear in the urine. The study aims to understand how felzartamab influences proteinuria and kidney function, while also assessing the safety and how the body processes this treatment. This is a Phase 3, randomized, double-blind, placebo-controlled study focusing on adults with IgAN. Participants will be randomly assigned to receive either felzartamab or a placebo through intravenous (IV) infusions. Neither the participants nor the researchers will know which treatment is given. The treatment period lasts 24 weeks followed by an 80-week follow-up period. In total, participants will attend 17 study visits over about 2 years to receive infusions and participate in study activities. During the study, participants will undergo assessments including urine tests to measure protein levels, kidney function evaluations, and safety monitoring. Researchers will track changes in proteinuria from the start of the study to Week 36 as the main outcome. Additional measurements will include kidney function, clinical endpoints, and the study of how felzartamab is processed by the body. Participant safety and long-term effects will be monitored throughout the study and follow-up periods.

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.

Researchers are evaluating the safety of a new insulin pump called the MiniMed2 NMX8-AID System combined with a continuous glucose monitoring sensor named Disposable Sensor 5/Simplera Sync in people living with diabetes. The study includes participants with Type 1 diabetes aged 7 to 85 years and those with Type 2 diabetes aged 18 to 85 years. Participants will first continue using their current diabetes therapy while wearing the sensor for up to 40 days, during which they will keep a meal and exercise log. After this initial period, participants will be randomly assigned to one of three groups to use the NMX8 pump for 90 days, with differing meal bolusing instructions depending on their group. Those who wish to continue using the pump after the study may join a Continued Access Period. During the treatment phase, participants will use the MiniMed2 NMX8-AID System with the DS5 continuous glucose monitor. The study involves three randomized groups, where participants will either bolus insulin for meals or not, while continuing to log meals and exercise. The initial phase involves wearing the sensor with current therapy for up to 40 days, followed by the randomized 90-day pump usage period. The pump data will be uploaded via an app or computer, and participants must use one of several specified insulins. Participants will be closely monitored throughout approximately 90 days for the percent of time their blood glucose stays within the target range of 70-180 mg/dL and the percent of time spent in hypoglycemia (below 70 mg/dL). They will complete meal and exercise logs and upload pump data regularly. Safety assessments include retinal eye exams and monitoring for adverse events. The study emphasizes adherence to wearing the system continuously and maintaining stable insulin dosing during participation. Overall, participant involvement spans an initial sensor-wearing phase, a randomized pump treatment phase, and possible extended access to the pump.