Benoni

Researchers are evaluating the safety and effects of marstacimab, a study medicine, for potential treatment of hemophilia in boys and male children aged 1 to 17 years. The trial focuses on those with severe Hemophilia A or moderately severe to severe Hemophilia B, including participants with or without inhibitors. The study aims to compare participants experiences with marstacimab to their historical bleeding episodes without the medicine. All participants will receive weekly subcutaneous injections of marstacimab. The first dose is administered at the study site by staff, and subsequent doses can be given at home or by study staff during the 12-month treatment period. Enrollment opens sequentially by age groups, starting with adolescents 12-17 years, followed by children 6-11 years, and finally children 1-5 years. The study includes about 1 month of screening, 12 months of treatment, and 1 month of follow-up. Participants will visit the study site at least 10 times during the study, with the option for two visits to occur at home if allowed. They will also receive six phone calls approximately every two months. Researchers will monitor bleeding rates, adverse events, injection site reactions, and immune responses. The overall participation lasts about 14 months, including screening, treatment, and follow-up periods.

Researchers are evaluating the combination of baxdrostat and dapagliflozin in people with chronic kidney disease CKD and high blood pressure hypertension. This Phase III, double-blind, placebo-controlled study aims to assess whether this combination reduces the risk of serious kidney damage, heart failure events, or cardiovascular death compared to dapagliflozin alone. The study includes participants with CKD and hypertension who meet specific kidney function and blood pressure criteria. Participants who are not already taking SGLT2 inhibitors will first complete a 4-week dapagliflozin run-in period. Then, they will be randomly assigned to receive either baxdrostat plus dapagliflozin or a placebo plus dapagliflozin. Baxdrostat dosing may start low and be increased if needed. Study visits will occur at 2, 4, 8, 16, 34, and 52 weeks after randomization, and then approximately every four months until the study ends, which is based on the number of key kidney or heart-related events. Throughout the study, participants will have regular assessments including blood tests to monitor kidney function and potassium levels, blood pressure measurements, and evaluations of heart and kidney health. If participants stop the blinded study drug early, they will continue dapagliflozin if possible and remain in the study for ongoing visits and monitoring. The main outcome is whether the combination treatment reduces the risk of a 50% sustained decline in kidney function, kidney failure, heart failure events, or cardiovascular death over up to 37 months.

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 evaluating the safety and effects of a new medicine called NNC0487-0111 in people who have Heart Failure with preserved Ejection Fraction HFpEF or Heart Failure with mildly reduced Ejection Fraction HFmrEF and excess body weight. This phase 3 clinical trial aims to find out if NNC0487-0111 is safe and effective for treating these conditions compared to a placebo. Participants have HFpEF or HFmrEF and a body mass index of 30 or above. The study is sponsored by Novo Nordisk AS and uses a randomized, quadruple-masked design. Participants will receive either NNC0487-0111 or a matching placebo by injection under the skin once a week. The NNC0487-0111 is given in increasing doses over time. The study is parallel in design, meaning participants are randomly assigned to one of the two groups and receive that treatment throughout the trial. This treatment period extends for up to about 165 weeks. The study evaluates the time to certain heart failure events, hospitalizations, cardiovascular deaths, and other major cardiovascular events. During the study, participants will be monitored regularly to assess heart failure outcomes and kidney function, as well as quality of life using questionnaires like the Kansas City Cardiomyopathy Questionnaire. Safety and effectiveness are assessed through hospital visits, heart failure event tracking, and blood tests including kidney function and blood sugar levels. The total participation spans over three years, with ongoing evaluations to measure the time to heart failure events and cardiovascular outcomes. Participants receive close medical monitoring throughout the study period.

This research aims to evaluate the long-term safety and explore the efficacy of astegolimab in adults aged 40 to 90 years with chronic obstructive pulmonary disease COPD. It focuses on participants who have completed a 52-week placebo-controlled treatment period in previous studies GB43311 or GB44332. The study is a phase 3, open-label extension to gather extended safety information on this drug in COPD patients. Participants from the parent studies who qualify will receive subcutaneous injections of astegolimab every two weeks throughout the study until it ends. This open-label extension allows all participants to receive the active drug without placebo comparison. The study continues treatment beyond the initial 52-week period to monitor long-term effects. During the study, participants will be monitored for adverse events up to 12 weeks after their last dose of astegolimab. Researchers will collect safety data to understand the incidence of any side effects. The study involves regular assessments and follow-ups to ensure participant well-being, with the total duration lasting until July 2034.

Researchers are evaluating the activity and safety of barzolvolimab compared to placebo in adults with cold induced urticaria or symptomatic dermographism who continue to have symptoms despite using H1-antihistamines. This Phase 3, randomized, double-blind, placebo-controlled trial aims to understand how barzolvolimab works in these conditions that cause hives and itching triggered by cold or skin friction. Participants will first go through a screening period of up to 4 weeks to confirm eligibility. The treatment period lasts 52 weeks and has two parts for the first 24 weeks, patients receive either barzolvolimab or placebo by subcutaneous injection every 4 weeks, with an initial barzolvolimab dose of 450mg followed by 150mg doses. For the next 28 weeks, all patients receive 300mg barzolvolimab every 8 weeks. After treatment, there is a 16-week follow-up period during which participants are observed without receiving study drug. During the study, participants will have provocation testing to measure responses at various time points, including weeks 4, 12, and 24. Researchers will assess symptoms like itch and hives and measure thresholds related to cold and friction triggers. Participants will complete daily symptom diaries and attend regular visits for safety monitoring and assessments. The main outcome is a complete response to provocation testing at week 12. Overall participation lasts about 1 year including screening, treatment, and follow-up.

Researchers are evaluating the efficacy and safety of tezepelumab in adults with moderate to very severe chronic obstructive pulmonary disease COPD who are receiving inhaled maintenance therapy. This Phase 3, multicenter, randomized, double-blind, placebo-controlled study focuses on adults aged 40 to 80 years who have experienced multiple COPD exacerbations in the year prior to enrollment. The trial aims to assess tezepelumabs impact compared to placebo on COPD exacerbations and lung function. Participants will receive monthly subcutaneous injections of one of two doses of tezepelumab or a matching placebo. Treatment duration ranges from a minimum of 52 weeks to a maximum of 76 weeks. Following the treatment period, participants will undergo a 12-week off-treatment safety follow-up to monitor any lasting effects. Throughout the study, participants will be regularly assessed for COPD exacerbations, lung function changes measured by forced expiratory volume FEV1, and quality of life using questionnaires such as the St. Georges Respiratory Questionnaire and COPD Assessment Test. Blood samples will be collected to measure drug levels and immune responses. Safety and efficacy will be closely monitored, with total participation lasting up to approximately 88 weeks including follow-up.

Researchers are evaluating the efficacy and safety of tozorakimab delivered under the skin in adults with uncontrolled asthma who are already using medium-to-high doses of inhaled corticosteroids. This phase IIb, double-blind, placebo-controlled study aims to find the optimal dosing range of tozorakimab in this population. The study is sponsored by AstraZeneca and uses a randomized, parallel design to compare different doses and placebo. Participants will receive subcutaneous injections of either tozorakimab at one of two dose levels or a placebo. The study arms include dosing with tozorakimab Dose 1, tozorakimab Dose 2, or placebo, administered under the skin. The treatment period lasts from 26 to 52 weeks, during which participants will be monitored for their asthma symptoms and lung function. Throughout the study, participants will undergo assessments including lung function tests such as forced expiratory volume in 1 second FEV1, asthma control questionnaires ACQ-6, quality of life questionnaires AQLQ12, and measurements of asthma exacerbations. Blood samples will be collected to measure drug levels and immune response. Safety and adherence will be closely monitored, and the primary outcome is the annualized rate of severe asthma exacerbations over the treatment period.

Researchers are evaluating the effects of vicadrostat combined with empagliflozin in adults who have type 2 diabetes, high blood pressure, and cardiovascular disease but no history of heart failure. The study aims to assess whether this combination can help reduce cardiovascular risks compared to a placebo with empagliflozin. This Phase III trial involves adults with these conditions who are already receiving treatment for them. Participants are randomly assigned to one of two groups. One group takes vicadrostat and empagliflozin tablets daily, while the other group takes placebo tablets that look like vicadrostat but have no active medicine, alongside empagliflozin. Treatment lasts from two and a half years up to four years and three months. All participants continue their usual medications for diabetes, blood pressure, and heart disease during the study. Throughout the study, lasting up to four years and three months, participants visit the study site regularly for health checks and blood samples. Doctors monitor cardiovascular events and any side effects experienced. The main outcome measured is the time until the first cardiovascular death or heart failure event. Other health indicators like blood pressure and kidney function are also tracked to understand the effects of the treatment combination.

Researchers are evaluating lunsekimig, a subcutaneous injection, compared with placebo in adults aged 40 to 80 years with inadequately controlled Chronic Obstructive Pulmonary Disease COPD characterized by an eosinophilic phenotype. This Phase 2bPhase 3 parallel study aims to assess the efficacy, safety, and tolerability of lunsekimig in reducing COPD exacerbations and improving lung function and symptoms. Participants are randomly assigned to one of three groups lunsekimig dose regimen A, lunsekimig dose regimen B, or a matching placebo. They will receive subcutaneous injections during a 48-week treatment period. The study also includes a screening period of up to 4 weeks before treatment and an approximately 8-week follow-up period after treatment, totaling up to 60 weeks of participation. During the study, participants will undergo regular assessments including lung function tests such as post- and pre-bronchodilator Forced Expiratory Volume in 1 second FEV1, questionnaires measuring respiratory health and symptoms, and monitoring of COPD exacerbations. Safety will be evaluated through reported adverse events and laboratory tests. Researchers will also monitor blood levels of lunsekimig and the presence of antidrug antibodies. Participants will be followed closely throughout the study duration to assess treatment impact and safety.