Kashihara

Researchers are evaluating the safety, effectiveness, and how the body processes and responds to NXT007 prophylaxis compared with emicizumab prophylaxis in people aged 12 years and older who have severe or moderate congenital hemophilia A without factor VIII FVIII inhibitors, or any severity of hemophilia A with FVIII inhibitors. This phase 3, randomized, open-label study aims to compare these treatments to better understand their impact on bleeding rates and treatment burden. Participants will be randomly assigned to one of two main treatment groups. One group will receive NXT007 prophylaxis administered subcutaneously using an integrated drug-device combination product. The other group will receive emicizumab prophylaxis via subcutaneous injections, starting with weekly loading doses for 4 weeks, then maintenance dosing at various intervals depending on prior treatment status. After the main treatment period, participants from both arms can continue or switch to NXT007 in an open-label extension phase. Throughout the study, participants will be closely monitored with regular assessments, including measuring annualized bleed rates for different types of bleeds, treatment burden questionnaires, and safety evaluations such as adverse event monitoring and laboratory tests. These evaluations will continue throughout approximately 3.5 years of study participation to provide comprehensive data on treatment effects and safety.

Researchers are evaluating the effectiveness, safety, and how the body processes and responds to NXT007 prophylaxis compared to Factor VIII FVIII prophylaxis in people aged 12 years and older with severe or moderate congenital hemophilia A who do not have inhibitors. This phase III study focuses on participants who have previously been treated with FVIII prophylaxis. The goal is to understand how NXT007 performs against the current standard treatment for this condition. Participants will be randomly assigned to receive either NXT007 prophylaxis, given as a subcutaneous injection with an integrated drug-device combination product, or standard Factor VIII prophylaxis according to local dosing and frequency guidelines. After the main six-month treatment period, those receiving NXT007 may continue this treatment in an open-label extension, and those initially on FVIII prophylaxis may switch to NXT007 during this extension phase. During the study, participants will be closely monitored through various assessments, including tracking the annualized bleed rate ABR for treated bleeds over six months, questionnaires evaluating treatment burden and impact on social and recreational activities, and safety evaluations such as adverse events, injection-site reactions, and antibody development against NXT007. The study will continue follow-up for approximately 3.5 years to gather comprehensive data on treatment effects and safety.

Researchers are evaluating the efficacy and safety of rilvegostomig compared to pembrolizumab monotherapy as the first-line treatment for patients with metastatic non-small cell lung cancer mNSCLC whose tumors express high levels of PD-L1. This Phase III, randomized, double-blind, multicenter global study focuses on patients with mNSCLC without certain genetic mutations who are suitable for this treatment approach. Participants are randomly assigned to receive either rilvegostomig or pembrolizumab intravenously on Day 1 of each 21-day cycle. The study compares these two drugs over repeated treatment cycles as first-line therapy. Both treatments are biological agents given by infusion, and the study is designed to monitor their effects over up to approximately five years. During the trial, participants will undergo regular assessments including physical exams, imaging scans such as CT or MRI to measure tumor lesions, and laboratory tests to evaluate organ function. Researchers will closely monitor overall survival, progression-free survival, treatment response, duration of response, and patient-reported outcomes on physical functioning and quality of life. Safety and immunogenicity of rilvegostomig will also be evaluated. Participants are followed and assessed for up to five years to gather comprehensive data on treatment effects and long-term outcomes.

Researchers are evaluating the efficacy and safety of combining durvalumab and domvanalimab compared to durvalumab plus placebo in adults with locally advanced Stage III, unresectable non-small cell lung cancer NSCLC whose disease has not progressed after definitive platinum-based concurrent chemoradiotherapy cCRT. This Phase III, randomized, double-blind, placebo-controlled, international study aims to provide new insights into treatment options for this patient population. Participants will receive either durvalumab and domvanalimab or durvalumab plus placebo as intravenous infusions every four weeks, beginning on Day 1 and continuing for up to 12 months. The study includes two groups one receiving the combination of durvalumab and domvanalimab, and the other receiving durvalumab with a placebo. Both treatments are given through infusion to assess their effects on disease progression and safety. During the trial, participants will undergo regular assessments including monitoring progression-free survival for up to 8 years after randomization. Other measures include overall survival, response rates, duration of response, and various time-to-event outcomes related to disease progression and symptom deterioration. Researchers will also evaluate drug concentrations and immune responses approximately 12 weeks after the last dose. Participants can expect scheduled visits for infusions and evaluations as part of this long-term study.

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.

This research aims to evaluate mezagitamab for adults with primary Immunoglobulin A nephropathy IgAN, a kidney disease caused by immune protein buildup leading to inflammation and potential kidney damage. The study will compare how mezagitamab affects protein levels in urine proteinuria against a placebo, focusing on safety, tolerability, and maintenance of kidney function over time. Participants will be randomly assigned to either receive mezagitamab or a placebo injection subcutaneously over approximately 22 weeks in the main group, with a 21 ratio favoring mezagitamab. An open-label group includes participants with specific proteinuria or kidney filtration levels, including those from a prior related study, all receiving mezagitamab in the same manner. After treatment, participants will be observed for about 1.5 years with regular check-ups. During the study, participants will attend multiple clinic visits for treatment and monitoring. Researchers will measure changes in proteinuria at Week 36 as the primary outcome, as well as kidney filtration rates over one and two years. Safety and long-term kidney function will be closely monitored throughout the 2-year participation period.

Pancreatic cancer is often diagnosed late, when it has spread to other parts of the body, making treatment challenging. This study focuses on adults with metastatic pancreatic cancer who have a specific KRAS G12D gene mutation. Researchers aim to evaluate whether adding setidegrasib to standard chemotherapy improves survival compared to chemotherapy with placebo. The study also explores how setidegrasib affects disease progression, symptoms, how the body processes the drug, and its safety. Participants receive either setidegrasib or placebo once weekly, combined with one of two chemotherapy regimens mFOLFIRINOX or NALIRIFOX. Chemotherapy is given in 28-day cycles through intravenous infusions. The choice of chemotherapy is determined by the doctor, with NALIRIFOX given only after confirming the safety of setidegrasib combined with it in another study. Treatment continues until cancer worsens, side effects become intolerable, other treatments are started, or the participant or doctor decides to stop. During the study, participants are regularly monitored with safety checks, health assessments, and laboratory tests. Researchers measure overall survival up to 3.5 years, along with progression-free survival, pain improvement, quality of life, response to treatment, and side effects. Pharmacokinetics of setidegrasib are studied up to 9 months. The study involves ongoing evaluation of participants wellbeing and medical status throughout the treatment period.

Researchers are evaluating the combined use of vicadrostat and empagliflozin in adults with chronic heart failure who have a reduced left ventricular ejection fraction LVEF below 40%. Participants must have had chronic heart failure diagnosed at least three months before starting the study. The trial aims to find out if this combination helps people with symptomatic heart failure classified as New York Heart Association classes II to IV. Participants are randomly assigned to one of two groups, with an equal chance of receiving either vicadrostat plus empagliflozin tablets or placebo plus empagliflozin tablets. The study medicines are taken once daily for approximately six months up to about 3.5 years. During this time, participants may continue their usual heart failure treatments, excluding certain medications. The trial includes a double-blind design, meaning neither participants nor study staff know who receives the active drug or placebo. Throughout the study, participants visit the study site regularly, with the number of visits depending on how long they stay enrolled. Some visits may occur by phone. They answer questions about their well-being, and doctors monitor health status, record any heart failure worsening, hospitalizations, or deaths. The main outcome is the time until cardiovascular death, heart failure hospitalization, or urgent heart failure visit, which is compared between groups. Safety and side effects are also closely followed during the trial.

Researchers are evaluating the effectiveness and safety of combining Dato-DXd with rilvegostomig as an additional treatment compared to standard care in adults with high-risk muscle invasive urothelial carcinoma MIUC who have undergone radical surgery. This Phase III trial focuses on participants with residual disease at high risk of recurrence after surgery. The study aims to see if the new combination improves disease-free survival compared to current standard treatments. Participants are randomly assigned to one of three groups Arm 1 receives Dato-DXd plus rilvegostomig intravenously every three weeks for up to one year Arm 2 receives Dato-DXd alone on the same schedule Arm 3 receives standard care, which includes nivolumab, durvalumab, or a combination of enfortumab vedotin and pembrolizumab, given intravenously at intervals ranging from every two to four weeks for up to a year. Treatment duration and dosing can be adjusted based on tolerance and investigator assessment. During the trial, participants will have visits approximately every three weeks or as per standard care schedules, depending on their assigned group, for up to 12 months of treatment. Researchers will monitor disease recurrence and survival outcomes for up to 78 months from the first participants enrollment. Assessments include clinical evaluations and disease status monitoring to measure disease-free survival and overall survival. Participants will be followed closely to assess safety and effectiveness of the treatments over this extended period.

Researchers are evaluating whether survodutide can help adults with liver diseases called non-alcoholic steatohepatitis NASH or metabolic-associated steatohepatitis MASH who have cirrhosis and a body mass index BMI of 27 kgm2 or higher 25 kgm2 for Asian participants. The study compares survodutide to a placebo to see if it improves liver function and related health outcomes over time. This is a Phase III trial with participants randomly assigned to groups, and it is double-blind, meaning neither participants nor doctors know who gets the medicine or placebo. Participants receive weekly injections of survodutide or placebo under the skin and get regular counseling on diet and exercise. The study lasts up to four and a half years, with visits either in person or via video call every 2, 4, or 6 weeks for about 17 months, then every 3 months thereafter until the study ends. The study collects health data including body weight, liver imaging results, and symptom questionnaires to assess the treatment effects. During the study, doctors monitor participants health and record any side effects. Liver health is checked using imaging methods at certain visits, and participants fill out questionnaires about their symptoms. The primary outcome measures include time to serious liver-related events and overall survival. Secondary outcomes look at changes in liver fibrosis, body weight, blood sugar control, liver stiffness, and other blood markers. The study aims to provide detailed long-term information on survodutides impact on liver disease and safety.