Omura

Researchers are evaluating whether retatrutide and tirzepatide can prevent major adverse liver outcomes in adults with metabolic dysfunction-associated steatotic liver disease MASLD who are at high risk based on non-invasive tests. This Phase 3 randomized controlled trial aims to assess these treatments compared to placebo in about 4,500 adults over approximately 224 weeks. The study is sponsored by Eli Lilly and Company and focuses on liver disease progression and related health measures. Participants will be randomly assigned to receive retatrutide, tirzepatide, or placebo, all administered by subcutaneous injection. The trial includes two placebo groups corresponding to each experimental drug. After completing the main study, eligible participants may join a 2-year extension where all will receive either retatrutide or tirzepatide regardless of their initial assignment. During the study, participants may attend around 25 to 30 clinic visits for health monitoring, study procedures, and assessments of liver function and disease status. Researchers will measure the time to major adverse liver outcomes, changes in liver fibrosis scores, liver stiffness, liver fat content, liver enzyme levels, body weight, and cardiovascular events. Monitoring will continue from baseline through study completion, with detailed evaluations at multiple timepoints including week 104.

Researchers are evaluating the efficacy and safety of opevesostat combined with daily corticosteroids compared to alternative treatments abiraterone acetate or enzalutamide in participants with metastatic castration-resistant prostate cancer mCRPC who have previously been treated with one next-generation hormonal agent NHA. The study aims to determine if opevesostat offers better control of disease progression assessed by radiographic progression-free survival, including participants with and without androgen receptor ligand binding domain mutations. Overall survival has also been included as a secondary outcome measure. Participants are randomly assigned to one of two groups. One group receives opevesostat 5 mg orally twice daily, plus dexamethasone 1.5 mg and fludrocortisone acetate 0.1 mg orally once daily, continuing until disease progression. Hydrocortisone is available as a rescue medication if needed. The other group receives either abiraterone 1000 mg once daily with prednisone 5 mg twice daily or enzalutamide 160 mg once daily, also until disease progression. This open-label, phase 3 study compares these two treatment approaches in a parallel design. During the study, participants undergo regular assessments including imaging scans to measure disease progression, safety monitoring, and evaluations of overall survival and quality of life. Researchers track radiographic progression-free survival for up to 52 months and secondary outcomes such as overall survival, time to new treatments, pain progression, and prostate-specific antigen PSA responses for up to approximately 82 months. Participants are closely monitored for adverse events and treatment tolerability throughout the study duration, which spans several years.

Researchers are evaluating the investigational drug volixibat for treating itching pruritus caused by Primary Biliary Cholangitis PBC, a liver disease. This Phase 2 clinical trial aims to learn more about volixibats effects on itching and its potential impact on PBC disease progression. The study is sponsored by Mirum Pharmaceuticals, Inc. Participants are randomly assigned to one of several groups receiving either volixibat capsules at doses of 20mg or 80mg twice daily, or placebo capsules without the active drug, also taken twice daily. The trial includes two parts, with some participants receiving volixibat 20mg twice daily and others receiving matching placebo capsules. The study is double-blind, meaning neither participants nor researchers know which treatment is given. During the study, participants itching levels are monitored using the Adult Itch Reported Outcome questionnaire over 28 weeks. Researchers also assess quality of life, fatigue, sleep disturbance, liver function tests, bile acid levels, and adverse events. Participants will attend regular visits for assessments, and the main outcome measured is the change in daily itch scores from baseline to week 28. The study excludes healthy volunteers and focuses on adults aged 18 years and older with confirmed PBC.

Researchers are evaluating the effectiveness, safety, and tolerability of a vaccine designed to reduce Clostridioides difficile C. difficile infections in adults aged 65 years and older. This phase 3 study compares the vaccine to a placebo in a group of older adults who have recent or planned contact with healthcare systems or recent antibiotic use. The purpose is to understand how well the vaccine works to prevent infections and to monitor any side effects or reactions. Participants will receive either the C. difficile vaccine or a placebo shot injected into the upper arm muscle. The study is randomized, double-blinded, and placebo-controlled. The vaccination period includes two doses and participants will be monitored for up to about three and a half years. Follow-up includes three planned clinical visits and three phone visits initially, then yearly clinic visits until the study ends. Participants are asked to report and save stool samples if they experience three or more loose stools in 24 hours to check for possible infection. During the study, researchers will track local and systemic reactions to the vaccine within seven days after each shot, adverse events up to one month after each vaccination, and serious adverse events for up to 18 months after the last dose. The main measurement is the occurrence of medically attended primary C. difficile infections from two weeks after the second vaccination through the surveillance period. Safety monitoring and annual visits will continue until the study is completed, which could be sooner or later depending on infection rates.

Researchers are evaluating the study medicine PF-08046054 compared to the standard treatment docetaxel in adults with non-small cell lung cancer NSCLC that has PD-L1 expression of 1% or higher. These participants have cancer that has spread or cannot be treated with surgery or definitive radiation and have shown disease progression during or after previous treatments including PD-L1 or PD-1 inhibitors, platinum-based chemotherapy, and targeted therapies for known genomic alterations. The study is a randomized phase 3 trial assessing treatment options for advanced NSCLC. Participants are randomly assigned to one of two groups one receives PF-08046054 as an intravenous IV infusion twice during each 21-day cycle, and the other receives docetaxel as an IV infusion once every 21 days. The study treatment may continue for up to 5 years if the participants cancer responds to therapy. Both treatments are given in cycles, and participants receive the medicine through infusions during clinic visits. During the study, participants will have regular clinic visits to monitor their health and how well the treatment is working. Assessments include measuring overall survival, progression-free survival, tumor response rates, and quality of life through questionnaires. Safety is monitored for adverse events up to 90 days after treatment ends. Blood samples are also taken to study the medicines levels and immune response. The total study duration can be up to 5 years depending on individual responses and outcomes.

Researchers are evaluating the effectiveness of a single dose of Staccato alprazolam compared with a placebo to quickly stop prolonged seizure episodes in people aged 12 years and older with stereotypical prolonged seizures. The study aims to determine if the treatment can stop seizures within 90 seconds and prevent recurrence for up to 2 hours after administration. This is a phase 3, randomized, double-blind clinical trial sponsored by UCB Biopharma SRL. Participants are randomly assigned to receive one dose of either Staccato alprazolam or placebo by inhalation during the treatment period. The study involves a parallel-group design with one treatment administration. Participants are observed for the treatments effect on seizure cessation and recurrence for up to 6 hours. During the study, participants and their caregivers will be monitored for seizure activity and safety outcomes. Various assessments include measuring treatment success within 90 seconds and seizure recurrence at 2, 4, and 6 hours after treatment. Safety is tracked through adverse event monitoring, with follow-up extending to 19 weeks after treatment. The total participation duration is based on these assessments and monitoring periods.

This research aims to evaluate the long-term safety and tolerability of brivaracetam in children and adolescents with epilepsy, including those who participated in earlier studies or are newly enrolled in Japan with partial-onset seizures. The study also seeks to assess pharmacokinetic data in Japanese participants. The study is an open-label, single-arm, multicenter trial focusing on pediatric epilepsy treatment with brivaracetam. Participants will receive brivaracetam tablets or oral solution administered twice daily in two equal doses. Dosage varies based on weight up to 5 mgkgday for those weighing 11 to less than 20 kg, up to 4 mgkgday for those weighing 20 to less than 50 kg, and no more than 200 mgday. Directly enrolled participants in Japan will receive 1 to 4 mgkgday, not exceeding 200 mgday. The study includes long-term follow-up for up to 5 years. During the study, participants will be monitored for treatment-emergent adverse events, serious adverse events, and adverse events leading to discontinuation of the drug. Evaluations occur from Day 1 through safety visits over the 5-year period. Researchers will assess safety, tolerability, and pharmacokinetics. The total participation duration extends up to 5 years, with ongoing safety evaluations throughout this time.

This research aims to evaluate the safety of Entresto Tablets and Granules in children with chronic heart failure. The study is conducted across multiple centers and observes pediatric patients in real-world clinical settings in Japan. The goal is to monitor safety-related events and understand risk factors during up to one year of treatment. Participants receive Entresto, a specified drug used to treat chronic heart failure, and the study carefully tracks how the medication is administered, including any accidental use of the granule form in capsule-shaped containers. The study is non-interventional and centrally enrolled, focusing on children aged 1 to under 18 years old, with a total observation period lasting 52 weeks. During the study, researchers will monitor for events like low blood pressure, high potassium levels, kidney problems, and dehydration. They will also document any adverse reactions and how Entresto is used throughout the year. The study involves ongoing safety assessments to ensure thorough observation of participants health over the full duration.

This research investigates response-guided therapy after neoadjuvant endocrine treatment to improve adjuvant care in premenopausal women with hormone receptor-positive, HER2-negative breast cancer. It focuses on optimizing treatment by identifying patients who may safely avoid chemotherapy based on their response to endocrine therapy. The study is a multicenter, randomized phase III trial aiming to compare this approach to standard surgery followed by adjuvant therapy. Participants are randomly assigned to one of two groups one group undergoes upfront breast surgery followed by adjuvant systemic therapy based on pathological findings, and the other group receives neoadjuvant endocrine therapy with an aromatase inhibitor and ovarian function suppression for three months before surgery. Adjuvant treatments vary depending on the patients Oncotype DX recurrence score and endocrine therapy response, with options including endocrine therapy alone, chemotherapy combined with endocrine therapy, or tamoxifen. During the study, participants are monitored for outcomes such as event-free survival, overall survival, relapse-free survival, and distant recurrence-free survival over up to 12 years. Researchers also assess quality of life, safety, and the rate of endocrine therapy use without chemotherapy. The study involves long-term follow-up to evaluate the effectiveness of response-guided therapy in improving treatment decisions for premenopausal women with this breast cancer subtype.

Researchers are evaluating the drug opevesostat in a phase 3, randomized, open-label study for men with metastatic castration-resistant prostate cancer mCRPC who have previously been treated with next-generation hormonal agents and taxane-based chemotherapy. The study aims to compare overall survival between participants receiving opevesostat and those receiving alternative treatments, including abiraterone acetate or enzalutamide. The research focuses on participants with specific androgen receptor mutations to determine if opevesostat provides better outcomes. Participants are assigned to one of two groups one group receives opevesostat 5 mg orally twice daily along with dexamethasone and fludrocortisone acetate daily, with hydrocortisone available as rescue medication. The other group receives either abiraterone acetate 1000 mg daily plus prednisone 5 mg twice daily or enzalutamide 160 mg daily. Treatments continue until disease progression, with prior prednisone use replaced by hydrocortisone in later protocol versions. During the study, participants undergo regular assessments including scans and laboratory tests to monitor prostate cancer progression and treatment effects. Researchers measure overall survival and several secondary outcomes such as progression-free survival, response duration, pain progression, and adverse events for up to approximately 54 months. The study also tracks safety and treatment discontinuation, with ongoing evaluations throughout the treatment period and follow-up.