Tochigi

Researchers are evaluating ziftomenib in Japanese patients with relapsed or refractory acute myeloid leukemia AML that has an NPM1 mutation. This Phase 2 study aims to assess the drugs effectiveness, safety, and how it behaves in the body. It is the first trial to administer ziftomenib to this patient group in Japan, with the goal of understanding its potential benefits for patients who have limited treatment options. Participants will take ziftomenib orally once daily. The study will monitor responses every 28 days, continuing treatment until the disease progresses or the patient withdraws. Several secondary measures will assess deeper remission rates, transfusion needs, and duration of response. The study also includes detailed monitoring of adverse events, drug levels in the blood, and patient health indicators during treatment and after discontinuation. During the trial, participants will have regular assessments including disease response evaluations every 28 days and safety checks. Follow-up will continue for up to one year after treatment ends to track overall survival and late effects. Participants will be monitored for side effects, changes in laboratory values, heart function, and overall physical status. The study duration varies but includes ongoing treatment and post-treatment observation to gather comprehensive data on ziftomenibs impact.

Researchers are evaluating the safety and effectiveness of KarXT combined with KarX-EC in adults aged 55 to 90 who have agitation related to Alzheimers Disease. This Phase 3, randomized, double-blind, placebo-controlled study aims to address agitation symptoms in this population by comparing the investigational drugs with a placebo. The study is sponsored by Bristol-Myers Squibb and uses established diagnostic criteria for Alzheimers Disease. Participants will receive either the combination of XanomelineTrospium Chloride capsules KarXT KarX-EC or a placebo with specified doses on designated days. The study includes a parallel group design and treatment lasts for 14 weeks. The main focus is to assess changes in agitation using the Cohen-Mansfield Agitation Inventory-International Psychogeriatric Association CMAI-IPA total score. During the study, participants will undergo regular assessments including cognitive and behavioral evaluations, safety monitoring through vital signs, laboratory tests, electrocardiograms, and rating scales for movement disorders and suicidal ideation. Caregivers will be involved to help monitor participant status and medication compliance. The primary outcome is measured at Week 14, with safety follow-up extending to Week 18. Participants are expected to be engaged throughout the treatment period and follow-up assessments.

Researchers are evaluating Mim8, a new medicine designed to help people with haemophilia A, including those with or without inhibitors. Mim8 aims to prevent bleeding episodes by replacing the function of the missing clotting factor VIII. This long-term study will last up to 5.5 years, ending either when Mim8 is approved in the participants country or by June 2028, whichever comes first. The study includes participants who have been involved in earlier related studies or are new infants with severe haemophilia A. Participants will receive Mim8 as a preventive treatment through subcutaneous injections. Depending on their entry point, participants may use an enhanced cartridge or a DV3407 pen-injector device for administering Mim8. The treatment is given regularly over the study period, with participants potentially receiving up to 262 injections. In the event of bleeding, additional haemostatic medications may be used as agreed with the study doctor. Female participants who are pregnant, breastfeeding, or planning pregnancy during the study are not eligible. During the study, participants will be monitored for any side effects, including injection site reactions and the development of antibodies against Mim8. Researchers will also track bleeding episodes, Mim8 blood levels, and device handling for some participants. Participants and their representatives will complete diaries and questionnaires about their treatment and health. Safety will be carefully followed throughout the study, which may last several years depending on individual enrollment and study progress.

Researchers are evaluating the effects of TAK-226 on symptoms of transfusion-dependent anemia in Japanese adults with lower-risk myelodysplastic syndromes MDS. This Phase 2, open-label study focuses on patients classified as very low, low, or intermediate risk according to a specific scoring system. The trial aims to understand how TAK-226 may improve anemia-related symptoms and reduce the need for blood transfusions in this population. Participants receive TAK-226 through subcutaneous injections every four weeks for about one year during the Treatment Period. The study begins with a Screening Period lasting up to six weeks to confirm eligibility. After treatment, participants enter an 8-week Safety Follow-Up Period to monitor side effects, followed by a Long-Term Follow-Up Period lasting up to five years from the first dose or three years after the last dose, whichever is longer. During the Treatment Period, participants visit the clinic approximately every two to four weeks. Throughout the study, participants undergo regular visits for assessments including blood tests and evaluations of hemoglobin levels and transfusion needs. Researchers will monitor various blood parameters and adverse events up to about six years. The primary outcomes focus on achieving transfusion independence or meaningful increases in hemoglobin over periods of up to 24 weeks. Overall, participants may be involved in the study for up to six years, allowing long-term observation of treatment effects and safety.

Researchers are evaluating the efficacy, safety, and pharmacokinetics of sefaxersen RO7434656, a new Antisense Oligonucleotide ASO therapy, in adults with primary IgA nephropathy IgAN who are at high risk of worsening kidney disease despite receiving optimized supportive care. This phase III study focuses on participants who continue to face disease progression despite standard treatments. Participants will receive subcutaneous injections of either sefaxersen or a matching placebo. The dosing schedule includes injections on Days 1, 15, and 29, followed by doses once every four weeks until Week 105. After Week 105 or the primary data cut-off, eligible participants may switch to open-label sefaxersen treatment at the investigators discretion. Throughout the study, participants will undergo assessments to measure changes in urine protein-to-creatinine ratio at Week 37, kidney function eGFR slope at Week 105, and monitor for hematuria resolution, kidney failure events, fatigue, and treatment-emergent adverse events. Blood samples will be collected to measure plasma sefaxersen levels. The total study duration extends up to approximately 36 months, with ongoing safety and efficacy monitoring.

This research aims to evaluate the long-term efficacy and safety of a combined formulation of xanomeline tartratetrospium chloride in an immediate release capsule KarXT and xanomeline enteric capsules KarX-EC for treating agitation in participants with Alzheimers Disease. The study focuses on individuals who have completed prior parent studies CN012-0023 or CN012-0024 and seeks to understand treatment effects over an extended period. Participants will receive specified doses of KarXT and KarX-EC on designated days as part of this single-group, non-randomized study. The treatment phase lasts up to approximately 30 weeks, during which the combined drugs are administered and monitored for safety and effectiveness related to agitation management in Alzheimers Disease. Throughout the study, participants will be monitored for treatment-emergent adverse events and other safety measures. Assessments include tracking adverse events, serious adverse events, changes in vital signs, laboratory evaluations, electrocardiograms, cognitive function tests like the Mini-mental State Examination and ADAS-Cog-13, as well as symptom severity scales. Caregiver involvement is required to provide support and facilitate study participation. The total study duration extends up to about 30 weeks with ongoing safety and efficacy evaluations.

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 the safety and clinical effects of Awiqli Insulin Icodec in people with diabetes mellitus in Japan through a non-interventional observational study. The study focuses on real-world use of Awiqli prescribed by doctors as part of routine clinical care, with no restrictions on diabetes type or prior treatments. This research aims to gather information about adverse reactions and clinical outcomes over a one-year period. Participants will receive commercially available Awiqli once weekly, with treatment decisions made by their doctors following normal clinical practice. The study does not assign treatment but observes patients using Awiqli as prescribed. Data will be collected throughout the 52-week period to monitor safety and clinical parameters under real-world conditions. During the study, participants will be monitored for adverse reactions, serious adverse events, and changes in blood sugar control, including glycosylated hemoglobin HbA1c and fasting plasma glucose. Quality of life related to diabetes therapy will also be assessed. The study involves regular data collection over approximately one year, and participants continue with their usual care while contributing information to the study.

This clinical trial is studying adults who have experienced a small ischemic stroke, also known as lacunar stroke. It aims to determine if a combination of antiplatelet drugs dual antiplatelet therapy works as well as the standard treatment with intravenous tissue plasminogen activator rt-PA. Researchers also want to see if the combination reduces bleeding complications compared to rt-PA. Participants will be randomly assigned to receive either the dual antiplatelet therapy, which includes aspirin 200 mg and clopidogrel 300 mg, or intravenous rt-PA at a low dose of 0.6 mgkg alteplase, which is approved in Japan. The study follows a parallel design and is conducted at multiple centers. Treatment is given during the hyperacute phase within 4.5 hours of stroke onset. During the study, participants will be monitored for neurological status at 3 months after stroke through in-person visits, phone calls, or mail. Researchers will measure outcomes such as excellent recovery at 3 months, infarct growth, early neurological changes, stroke recurrence, and cost effectiveness. The total study period extends through March 2029, with primary outcome assessment at 3 months post-stroke.

Researchers are evaluating whether low treatment intensity 12 mLkghr or medium treatment intensity 25 mLkghr continuous kidney replacement therapy is more effective and safer for critically ill patients with acute kidney injury. This interventional clinical trial is sponsored by Jikei University School of Medicine and uses a randomized, parallel study design to compare these two dosing strategies in an intensive care setting. The study is conducted to understand the best approach to continuous renal replacement therapy for patients requiring this treatment. Participants are randomly assigned to receive either low-intensity or medium-intensity continuous hemodialysis andor hemofiltration, delivered through dialysate fluid and filtration replacement fluid. The study compares these two dosing levels, with interventions administered continuously while patients are in intensive care units. The trial includes phases 2 and 3 and is planned to complete by June 2030. During the study, participants are monitored for outcomes such as death and duration of kidney replacement therapy over 28 days or until hospital discharge, whichever comes first. Additional evaluations include ICU and hospital mortality, dialysis dependence, vasopressor-free days, ventilator-free days, serum creatinine levels, and urine output at kidney replacement therapy end. The total participation timeline extends up to hospital discharge or 90 days, with assessments performed at multiple time points to measure treatment effects and safety.