Nankoku

This research aims to evaluate the tolerability and safety of ONO-7428 in adults with unresectable advanced or recurrent solid tumors, including non-small cell lung cancer (NSCLC). The study focuses on participants who have tumors resistant or intolerant to standard treatments and who have previously received anti-PD-(L)1 antibody therapies. It is a Phase 1, open-label, dose-escalation study designed to understand how well participants tolerate ONO-7428 and to monitor its safety profile. Participants will receive specified doses of ONO-7428 on designated days as part of the treatment protocol. The study includes a backfill cohort specifically for those with advanced or recurrent NSCLC as classified by the UICC-TNM system. The dosing schedule and escalation allow researchers to identify dose-limiting toxicities and monitor adverse events over an extended period. During the study, participants will be closely monitored for dose-limiting toxicities within the first 21 days and adverse events for up to two years. Researchers will collect tumor tissue samples for biomarker testing and assess participants’ performance status. The study involves ongoing safety assessments and long-term follow-up to better understand the effects and risks associated with ONO-7428 treatment.

Researchers are evaluating how well vortioxetine tablets at doses of 10 mg/day or 20 mg/day work compared to placebo tablets for treating depression symptoms in Japanese teenagers aged 12 to 17 years who have been diagnosed with Major Depressive Disorder (MDD). This phase 3 clinical trial aims to assess the drug's effectiveness, safety, and how the body processes the medication in this pediatric population. Participants will be randomly assigned to receive either vortioxetine or a placebo once daily for 14 weeks. The study includes an initial screening period of up to 15 days to determine eligibility, followed by the 14-week treatment phase. After completing treatment, there is a 4-week period dedicated to monitoring any side effects. Throughout the study, participants will visit the clinic 13 times for assessments and medication administration. During the study, participants will undergo evaluations including the Children Depression Rating Scale Revised version (CDRS-R) to measure changes in depression symptoms from baseline to week 14. Other assessments include safety monitoring and pharmacokinetics. Researchers will also collect information on side effects during and after treatment. The total time commitment for participants is about 20 weeks, including screening, treatment, and follow-up periods.

Researchers are evaluating the combination of BMS-986504, pembrolizumab, and chemotherapy compared to placebo plus pembrolizumab and chemotherapy in people with first-line metastatic non-small cell lung cancer who have a homozygous MTAP deletion. The study is a randomized phase 2/3 trial focused on assessing clinical benefits in this specific patient group. Participants will receive either BMS-986504 with pembrolizumab and chemotherapy or placebo with pembrolizumab and chemotherapy. The chemotherapy may include drugs such as cisplatin, carboplatin, pemetrexed, paclitaxel, or nab-paclitaxel, given at specified doses on specified days. This treatment is administered as part of the first-line therapy for metastatic disease. During the study, researchers will monitor progression-free survival up to 2 and 5 years using RECIST v1.1 criteria and overall survival up to 5 years. Participants will be assessed regularly for disease progression and survival outcomes. The study includes detailed monitoring to evaluate the effects and safety of the treatment combination over time.

Researchers are comparing the effectiveness of two treatments for participants with stage IV or recurrent non-squamous non-small cell lung cancer (NSCLC) who have PD-L1 expression of 1% or higher. This phase 3, randomized, open-label study focuses on first-line treatment options and aims to evaluate overall survival over up to five years for participants with PD-L1 levels between 1% and 49%. The trial involves participants with measurable disease and good performance status who have not received prior systemic therapy for advanced disease. The study compares a combination of Nivolumab and Relatlimab plus chemotherapy against Pembrolizumab plus chemotherapy. Chemotherapy drugs include Carboplatin, Pemetrexed, and Cisplatin, administered at specified doses on scheduled days. Participants are randomly assigned to receive either the Nivolumab and Relatlimab combination with chemotherapy or Pembrolizumab with chemotherapy as their initial treatment. Treatment schedules and doses are defined but not detailed here. Participants will be closely monitored throughout the study, which may last up to five years. Researchers will assess overall survival as the primary outcome, along with regular imaging tests like CT or MRI to measure disease status. Eligibility screening includes assessing PD-L1 levels, performance status, and other health factors. Safety monitoring and follow-up will continue to evaluate treatment effects and participant well-being during and after treatment.

This research aims to evaluate the safety and effectiveness of BGB-16673 compared to pirtobrutinib in adults with relapsed or refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) who have previously been treated with a covalent Bruton tyrosine kinase inhibitor (cBTKi). The study is a Phase 3, open-label, randomized trial sponsored by BeOne Medicines. The goal is to better understand treatment options for patients whose disease has returned or did not respond to earlier therapies involving cBTKi. Participants will receive either BGB-16673 or pirtobrutinib, both given orally. The study compares these two treatments to assess their safety and efficacy. The treatment period and dosing details are managed as per the trial protocol to evaluate the outcomes of each drug. The study includes ongoing monitoring and assessments to observe treatment effects over time. During the study, participants will be closely followed for up to approximately 3 years to measure progression-free survival, as assessed by an independent review committee. Researchers will conduct regular evaluations including imaging and laboratory tests to track disease status and safety. Participants' health will be monitored throughout the study to identify any side effects or changes in condition.

Researchers are evaluating the effectiveness and safety of belzutifan monotherapy in participants with advanced pheochromocytoma/paraganglioma (PPGL), pancreatic neuroendocrine tumor (pNET), von Hippel-Lindau (VHL) disease-associated tumors, advanced wild-type gastrointestinal stromal tumor (wt GIST), or advanced solid tumors with HIF-2α related genetic changes. This phase 2 study aims primarily to measure the objective response rate (ORR) of belzutifan using standardized tumor response criteria reviewed independently and blindly. Participants receive belzutifan orally at a dose of 120 mg once daily until their disease progresses or they stop treatment for other reasons. The study includes a specific cohort for VHL disease-associated tumors, which requires participants to be at least 18 years old. The treatment period continues as long as the participant benefits or tolerates the medication. Throughout the study, participants will be regularly assessed by independent central review to measure tumor response. The study monitors safety and efficacy outcomes for up to approximately 5.5 years. Participants are expected to have a life expectancy of at least 3 months and will be observed carefully for any treatment effects or adverse events during this time.

Researchers are evaluating the early use of a once-daily oral drug called empagliflozin 10 mg in patients hospitalized with acute heart failure (AHF) who are at high risk for serious complications. This multicenter, randomized, double-blind, placebo-controlled Phase 3 trial aims to assess the efficacy and safety of empagliflozin compared to a matching placebo in this patient group. The trial focuses on patients requiring intravenous diuretic therapy and exhibiting specific clinical signs and biomarker levels related to heart failure severity. Participants are randomly assigned to receive either empagliflozin 10 mg once daily or a placebo shortly after hospital admission. Treatment begins within 12 hours of hospital presentation and continues during the hospitalization period. The study excludes patients with very low kidney function, recent use of similar drugs, certain heart conditions, and other specific medical issues to ensure safety and clear evaluation of the drug's effects. During the study, patients will be closely monitored for outcomes including death, rehospitalization for heart failure, worsening heart failure during the hospital stay, and urine output within 48 hours of treatment start. Researchers will use a combined measure called the win ratio to assess these outcomes over 90 days. Participants will undergo clinical evaluations, laboratory tests, and safety assessments throughout the study period to track the drug's impact and monitor for any adverse events.

Researchers are tracking patients with Fabry disease through an ongoing international, multi-center observational program called the Fabry Registry. This program collects routine clinical data from patients regardless of their treatment status to better understand the disease's variability, progression, and natural history. It also focuses on enhancing patient care by supporting the development of monitoring recommendations and evaluating the long-term safety and effectiveness of Fabrazyme, a treatment for Fabry disease. The study includes a Fabry Pregnancy Sub-registry, which is a voluntary, international, longitudinal observation program that monitors pregnancy outcomes for women enrolled in the Fabry Registry who are pregnant or have been pregnant. This sub-registry collects medical and obstetric history, pregnancy, and birth data, along with infant growth information up to 36 months postpartum, regardless of the specific treatment received. No experimental treatments are administered in either registry; patients continue receiving routine care as determined by their physicians. Participants contribute data through clinical assessments and standard care evaluations performed by their doctors. The study measures long-term outcomes including safety and effectiveness of Fabrazyme over up to 33 years, as well as pregnancy outcomes and infant growth data. The program helps fulfill regulatory requirements and supports research while tracking patient health over extended periods without altering their usual care.

A phase 3b, Randomized, Controlled Trial of Nadofaragene Firadenovec vs. Observation in Participants with Intermediate Risk Non-Muscle Invasive Bladder Cancer (IR NMIBC)

Researchers are evaluating the effectiveness of nucresiran compared to a placebo in reducing death from any cause and cardiovascular events in patients with transthyretin amyloidosis that affects the heart. The study also aims to assess how nucresiran impacts additional cardiovascular events, mortality, and patient-reported health status and quality of life. This is a Phase 3, global, randomized, double-blind, placebo-controlled trial focusing on this serious heart condition. Participants will receive either nucresiran 300 mg or a placebo (sterile normal saline) via subcutaneous injection every six months. The study treatment period is expected to last about 32 months, with a maximum duration of up to 5 years. Both groups will be monitored closely throughout this time, and patients may continue other approved therapies for transthyretin amyloidosis and heart failure as recommended by their doctors. During the study, participants will undergo regular assessments to track all-cause mortality and recurrent cardiovascular events, including hospitalizations and urgent visits for heart failure. Other evaluations will include patient-reported health outcomes and quality of life measurements. Researchers will monitor safety and treatment effects over the full study period, with a focus on how well nucresiran may improve health and reduce cardiovascular complications in this patient population.