Nishinomiya

Researchers are evaluating whether the drugs retatrutide and tirzepatide can prevent major adverse liver outcomes (MALO) in adults with metabolic dysfunction-associated steatotic liver disease (MASLD) who are at high risk. This Phase 3 trial enrolls about 4,500 adults with MASLD identified by non-invasive tests indicating an increased likelihood of developing serious liver problems. The study aims to understand how these treatments might affect liver health over time compared to a placebo. Participants will be randomly assigned to receive either retatrutide, tirzepatide, or a placebo, all given by subcutaneous injection. The study will last approximately 224 weeks, during which participants may attend 25 to 30 clinic visits for monitoring and assessment. After the main study, eligible participants can join an optional 2-year extension where all will receive either retatrutide or tirzepatide regardless of their original group. Throughout the trial, participants’ liver function and disease progression will be closely monitored through various health assessments. Researchers will track the time to the first major adverse liver event as the main outcome. Safety and health status will be evaluated regularly during clinic visits, ensuring thorough observation over the long study period.

Researchers are evaluating the effects of iptacopan compared with a placebo in adults aged 18 to 85 years who have generalized Myasthenia Gravis positive for acetylcholine receptor antibodies (AChR+ gMG). This Phase III, randomized, double-blind, placebo-controlled, multicenter study aims to assess the efficacy, safety, and tolerability of iptacopan while participants continue their stable standard of care treatments. The study includes participants with moderate to severe gMG symptoms and positive diagnostic criteria. Participants will be randomly assigned in a 1:1 ratio to receive either iptacopan or a matching placebo in the form of hard gelatin capsules for six months (180 days). During this time, they will continue their stable standard of care treatments. After the double-blind treatment period, a maximum 60-month open-label extension phase is offered. Safety follow-up assessments will occur one week and one month after the last dose of study treatment. During the study, participants will be evaluated for changes in their Myasthenia Gravis Activity of Daily Living (MG-ADL) total score from baseline to month 6. Researchers will monitor safety and tolerability throughout the treatment and extension periods. Vaccination status, infection monitoring, and regular clinical assessments will be part of participant evaluations to ensure safety and track disease symptoms over time.

Researchers are evaluating the effects of E6742, an oral tablet medication, compared to a placebo in adults with systemic lupus erythematosus (SLE). The main goal is to see how well participants respond to treatment based on a specific lupus assessment called the British Isles Lupus Assessment Group (BILAG) based Composite Lupus Assessment (BICLA), focusing on those who maintain a low dose of oral corticosteroids by Week 24. This is a Phase 2, randomized, double-blind, placebo-controlled study aimed at finding the best dose response of E6742 for treating SLE. Participants will receive either E6742 oral tablets or placebo tablets during the study. The study evaluates different doses to understand the medication's effects and safety. Before treatment, participants must have active lupus with specific disease activity scores and stable doses of certain lupus medications. The study includes a planned treatment period lasting at least 24 weeks, during which the response to therapy and safety are closely monitored. Throughout the study, participants will undergo regular assessments to measure lupus disease activity using BILAG and other scoring systems. Researchers will monitor medication adherence, side effects, laboratory tests, and physical exams to evaluate safety and effectiveness. The primary outcome is the percentage of participants achieving a BICLA response while on a low dose of corticosteroids at Week 24. The study also includes ongoing safety monitoring to ensure participant well-being during and after treatment.

Researchers are evaluating the safety and effectiveness of two drugs, eltrekibart and mirikizumab, in adults with moderately to severely active ulcerative colitis (UC). This study is a phase 2 trial lasting about 4 to 5 years, aiming to understand how well these treatments work alone or together for this chronic condition. Participants will receive either eltrekibart alone, mirikizumab alone, a combination of both, or a placebo. The treatments are administered as drugs, and the study includes a screening period of up to 35 days before enrollment. The total participation time for each person is approximately 69 weeks, which includes the screening and treatment periods. During the trial, participants will be closely monitored to assess the percentage who achieve clinical remission by week 12. Researchers will conduct regular evaluations, which may include medical assessments and questionnaires, to track the safety and effects of the treatments. The study emphasizes careful follow-up to ensure participant safety and to gather detailed information about the therapies over the entire study duration.

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.

This research aims to evaluate the safety and effectiveness of TAK-279 in people with moderately to severely active Crohn's disease, a long-term condition that causes inflammation anywhere in the gut. The study seeks to determine if three different doses of TAK-279 can reduce bowel inflammation and ulcers compared to a placebo after 12 weeks of treatment. Participants will be assessed using endoscopy to check the level of bowel inflammation. Participants will be randomly assigned to one of four groups: three different doses of TAK-279 or a placebo. They will receive the assigned treatment capsules for a total of 52 weeks (1 year). The study is double-blind, meaning neither the participants nor the doctors will know which treatment is given unless needed for urgent medical reasons. The trial will be conducted at multiple centers worldwide and involves 15 clinic visits. Throughout the study, participants will undergo assessments including endoscopy to measure treatment response based on the Simple Endoscopic Score for Crohn's Disease at week 12. Safety will also be monitored over approximately 60 weeks, including a 4-week safety follow-up period after treatment ends. Researchers will compare the medical problems experienced and how well participants tolerate the treatments.

Researchers are evaluating the safety and effectiveness of low-dose and high-dose atogepant in children and adolescents aged 6 to 17 who experience episodic migraine. Migraines are moderate to severe headaches often accompanied by symptoms such as throbbing pain, nausea, and sensitivity to light and sound. While several treatments exist for adults, options for younger patients are limited, making this Phase 3 study important to understand how atogepant works in this younger population. Participants aged 6 to 17 will be randomly assigned to one of six groups to receive either placebo, low-dose atogepant, or high-dose atogepant tablets taken once daily by mouth for 12 weeks. The exact doses for children aged 6 to 11 will be decided after a pharmacokinetic substudy. After 12 weeks, participants may either have a follow-up visit 4 weeks after stopping the treatment or join an extension study to continue taking atogepant for an additional 52 weeks. During the study, participants will attend regular visits at hospitals or clinics for medical assessments, blood tests, and to monitor for any side effects. They will also complete questionnaires to evaluate how treatment affects their migraines. The main outcomes measured are changes in the number of monthly migraine days over 12 weeks and the number of participants experiencing adverse events during the first 16 weeks. About 450 participants will be enrolled across roughly 100 sites worldwide.

This research investigates the effectiveness and safety of arlo-cel (BMS-986393), a GPRC5D-directed CAR-T cell therapy, compared to standard treatment regimens in adults with relapsed or refractory multiple myeloma who have been previously treated with lenalidomide. The study is a Phase 3, randomized, open-label trial conducted at multiple centers. It aims to provide new options for patients whose disease has returned or did not respond after prior therapies. Participants will receive either arlo-cel or one of several standard regimens that include drugs such as cyclophosphamide, fludarabine, daratumumab, pomalidomide, dexamethasone, or carfilzomib. Each medication is given in specified doses on designated days as part of the treatment plan. The study compares these approaches to assess which is more effective in managing the disease. Throughout the study, researchers will monitor participants for progression-free survival up to five years after the last participant is enrolled and the presence of minimal residual disease negativity within one year. Participants will undergo assessments to measure disease status, organ function, and performance status. Safety and treatment effects will be tracked during the study period to understand the impact of the therapies over time.

Researchers are evaluating the safety, effectiveness, and how the body processes etrasimod in treating adolescents aged 12 to under 18 years with moderately to severely active ulcerative colitis. This phase 2 study aims to understand the impact of this treatment on this younger population. Participants who complete the initial 52-week treatment may continue in a long-term extension period lasting up to 4 additional years, totaling 5 years after enrollment. Participants will take etrasimod orally once daily as either a tablet or granules for up to 52 weeks. After this treatment period, those who finish may opt to join a long-term extension to continue treatment and monitoring. The study is open-label and single-arm, meaning all participants receive the study drug and both effects and safety are observed over time. During the study, researchers will regularly assess participants' health, focusing on the proportion achieving clinical remission based on the Modified Mayo Score at week 52. Monitoring includes tracking safety, drug levels in the body, and overall treatment effects. The extended monitoring in the long-term extension allows for continued evaluation of safety and effectiveness over several years.

Researchers are evaluating the effectiveness, safety, and behavior of a new treatment called sefaxersen (RO7434656), an Antisense Oligonucleotide (ASO) therapy, for people with primary IgA nephropathy (IgAN). The study focuses on participants who have a high risk of their kidney disease worsening despite receiving the best available supportive care. This is a Phase III, randomized, double-blind, placebo-controlled trial conducted at multiple centers. Participants will receive either sefaxersen or a matching placebo through subcutaneous injections according to a specified schedule. The study compares these two groups to see how the treatment affects kidney function over time. The intervention is designed to inhibit Complement Factor B, which is involved in the disease process. The study includes vaccination requirements and contraceptive use for women of childbearing potential to ensure safety. During the study, participants will be monitored for changes in their urine protein-to-creatinine ratio (UPCR) at baseline and at week 37, which is the primary measure of kidney function improvement. Other assessments include kidney biopsy results, kidney function tests estimating glomerular filtration rate (eGFR), and ongoing safety evaluations. The trial tracks participants' health closely to assess the treatment's effect and any side effects throughout the study period.