Kashihara

Researchers are evaluating the efficacy, safety, pharmacokinetics, and pharmacodynamics of a new treatment called NXT007 compared to emicizumab prophylaxis in people aged 12 years and older with severe or moderate congenital hemophilia A without factor VIII inhibitors, as well as those with hemophilia A of any severity who have factor VIII inhibitors. This phase III clinical trial aims to better understand how well these treatments work and how safe they are for managing hemophilia A. Participants will receive either NXT007 or emicizumab as prophylactic treatments. NXT007 is given by subcutaneous injection using a combined drug-device product, while emicizumab is administered subcutaneously using a vial and syringe. The study compares these two methods of treatment over a main study treatment period starting from the second month until at least seven months after the first dose. During the study, researchers will monitor how often treated bleeding episodes occur, measured as the Annualized Bleed Rate (ABR). Participants' medical histories, including previous treatments and bleeding episodes, will be carefully documented. Safety and treatment effects will be closely observed throughout the study to evaluate the overall impact of both treatments on the participants' condition.

Researchers are evaluating the safety, effectiveness, pharmacokinetics, and pharmacodynamics of NXT007 prophylaxis compared to standard Factor VIII (FVIII) prophylaxis in people aged 12 years and older with severe or moderate congenital hemophilia A who do not have inhibitors. The study focuses on participants previously treated with FVIII prophylaxis and aims to understand how well NXT007 works versus the current standard treatment. Participants will receive either NXT007 administered subcutaneously via a combined drug-device product or Factor VIII prophylaxis given according to local medical guidelines and dosing schedules. This phase III randomized, open-label trial compares these two treatment approaches over a main study treatment period. During the study, researchers will monitor the annualized bleed rate for treated bleeds over six months. Participants will be followed to assess treatment effects, safety, and drug behavior in the body. The study includes detailed tracking of bleeding episodes, treatment adherence, and clinical evaluations to gather comprehensive data on each therapy's impact.

Researchers are evaluating the efficacy and safety of rilvegostomig compared to pembrolizumab as first-line treatments for patients with metastatic non-small cell lung cancer (mNSCLC) whose tumors have high PD-L1 expression. This Phase III, randomized, double-blind, and global study focuses on participants with stage IV mNSCLC who do not have certain genetic mutations or rearrangements and are eligible for systemic therapy. Participants receive either rilvegostomig or pembrolizumab intravenously on Day 1 of each 21-day cycle. The study compares these two biological treatments given as monotherapy. Both groups will be monitored over time to assess treatment impact and safety. Throughout the study, participants undergo evaluations including tumor measurements by CT or MRI, performance status assessments, and organ function tests. Researchers will measure overall survival and progression-free survival for up to approximately five years. Tumor samples are collected before treatment for central testing, and participants’ health and treatment responses are closely followed during the trial period.

Researchers are evaluating the safety and effectiveness 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 worsened after definitive platinum-based concurrent chemoradiation therapy. This Phase III, randomized, double-blind, placebo-controlled, international study involves multiple centers. Participants receive intravenous infusions of durvalumab and domvanalimab or durvalumab and placebo. The treatments are given after patients have completed concurrent platinum-based chemotherapy and radiation therapy with a total radiation dose of approximately 60 Gy. The study monitors patients over time to assess treatment effects and safety. During the study, participants undergo evaluations including tumor tissue analysis for PD-L1 status, performance status assessments, and monitoring of organ and marrow function. The main outcome measured is progression-free survival up to 8 years after randomization. Researchers also monitor for any adverse effects and disease progression throughout the study period.

Researchers are evaluating AZD0780, an oral PCSK9 inhibitor, in a phase 3, randomized, placebo-controlled study to see if it can reduce the risk of major adverse cardiovascular events (MACE-PLUS) in adults with established atherosclerotic cardiovascular disease (ASCVD) or those at high risk for a first ASCVD event. The study compares AZD0780 to a placebo and monitors participants from randomization until the primary analysis censoring date, followed by a final study closure visit. Participants will be randomly assigned to receive either oral AZD0780 or an oral placebo once daily. The treatment period lasts until the primary analysis censoring date, after which a study closure visit will occur. The study is event-driven and designed to assess the time to the first major cardiovascular event during treatment. During the study, participants will be closely monitored with various assessments to evaluate cardiovascular outcomes and safety over approximately 54 months. Researchers will track the time to first event of any component of MACE-PLUS and collect data to assess the effect of AZD0780 compared to placebo. The study includes regular visits and evaluations to ensure participant safety and adherence to treatment.

Immunoglobulin A nephropathy (IgAN) is a kidney disease caused by the build-up of immune protein complexes in the kidneys, leading to inflammation and possible kidney damage. This Phase 3 study is evaluating how well mezagitamab, compared to a placebo, reduces protein levels in the urine (proteinuria) in adults with primary IgAN. It also aims to assess the safety and tolerability of mezagitamab and its ability to maintain kidney function over the long term. Participants will be randomly assigned to one of two groups in the main study: two-thirds will receive mezagitamab injections under the skin, and one-third will receive placebo injections that look identical but have no active medicine. Treatment will occur in two 1-year cycles, each including about six months of dosing and six months of observation with monthly check-ups. An open-label group will include a small number of participants with lower proteinuria or kidney filtering issues, including those who previously received mezagitamab in another study; these participants will receive mezagitamab similarly to the main group. During the study, participants will visit the clinic several times for assessments. Researchers will monitor changes in proteinuria from the start through week 36, along with safety and kidney function. They will also perform regular evaluations and check-ups throughout each treatment and observation period to track participants' health and response to treatment.

Researchers are investigating the effectiveness of Saruparib (AZD5305) combined with a physician's choice of new hormonal agents (NHA) compared to a placebo plus NHA in men with metastatic castration-sensitive prostate cancer (mCSPC). This phase III study aims to demonstrate whether Saruparib plus NHA can improve radiographic progression-free survival (rPFS) in two groups of participants: those with homologous recombination repair mutations (HRRm) and those without (non-HRRm). About 1800 adult male participants with mCSPC will be divided into two cohorts based on their HRRm status. Each cohort will be randomized equally to receive either Saruparib orally with their chosen NHA or a placebo orally with the chosen NHA. The new hormonal agents may include abiraterone acetate, darolutamide, or enzalutamide. Participants will continue their assigned treatment and undergo regular tumor evaluation scans until their disease progresses or treatment is stopped for other reasons. Throughout the study, participants will have tumor tissue and blood samples collected to confirm HRRm status and monitor disease. They will be followed for survival until the study ends. An independent data monitoring committee will review safety and tolerability of Saruparib plus NHA. The main outcome measured is radiographic progression-free survival, tracked for up to approximately 50 months, to evaluate how well the treatments control cancer progression.

Researchers are investigating the antibody-drug conjugate M9140 in adults with advanced solid tumors that express CEACAM5, including gastric cancer, non-small cell lung cancer (NSCLC), and pancreatic cancer. This Phase 1b/2 study uses a master protocol with three parallel substudies focusing on each tumor type. The study aims to evaluate the antitumor activity, safety, tolerability, and how the body processes M9140 when given alone or with other treatments. Participants will receive M9140 intravenously at a dose of 2.8 mg/kg every three weeks on Day 1 of each 21-day cycle. Each substudy has a different average duration: about 10 months for gastric cancer, 12 months for NSCLC, and 8 months for pancreatic cancer. Every participant undergoes a 28-day screening period before treatment and a safety follow-up visit approximately 30 days after the last dose. During the study, participants will be monitored regularly through clinical assessments, laboratory tests, and evaluations of tumor response using RECIST criteria. Researchers will track safety and side effects throughout the treatment and follow-up periods. The main outcome measured is the objective response rate, assessed up to about 48 months from the first treatment. This long-term monitoring helps understand the treatment's effects and safety over time.

Researchers are evaluating the safety and effectiveness of a new oral drug called daraxonrasib compared to the chemotherapy drug docetaxel in patients with non-small cell lung cancer (NSCLC) that has a specific RAS mutation. This Phase 3 global study focuses on patients whose cancer has locally advanced or spread and who have already received prior treatments. The goal is to see if daraxonrasib can improve the time patients live without their cancer worsening and overall survival. Participants will be randomly assigned to receive either daraxonrasib tablets taken by mouth or docetaxel given by intravenous infusion. The study is open-label, meaning both doctors and patients know which treatment is given. Treatment continues as long as it is appropriate, and patients are monitored throughout the study period. During the trial, patients will undergo regular assessments to measure disease progression and survival up to about four years. Researchers will evaluate progression-free survival and overall survival as the main outcomes. Patients must have measurable disease and meet health criteria, and their RAS mutation status will be confirmed. Safety and effectiveness will be closely monitored throughout the study.