Omura

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 effectiveness and safety of opevesostat combined with hormone replacement therapy compared to alternative treatments with abiraterone acetate or enzalutamide in people with metastatic castration-resistant prostate cancer (mCRPC) who have already been treated with one next-generation hormonal agent. This Phase 3 study aims to determine whether opevesostat improves radiographic progression-free survival, assessed by independent central review, in participants with or without androgen receptor ligand binding domain mutations. Participants will receive either oral opevesostat along with hormone replacement therapy drugs such as dexamethasone and fludrocortisone acetate, or they will receive alternative oral treatments including abiraterone acetate with prednisone acetate or enzalutamide. Hydrocortisone can be used as a rescue drug if needed. The study is open-label and randomized, comparing these treatment strategies in participants who have progressed after prior hormonal therapy. During the study, participants will undergo assessments including imaging scans to monitor disease progression. Researchers will measure radiographic progression-free survival up to approximately 52 months. Safety and overall survival are also monitored as secondary outcomes. Participants must attend scheduled visits for evaluations, provide tumor tissue samples, and have ongoing monitoring of organ function, hormone levels, and other relevant health parameters throughout the study period.

Researchers are evaluating the investigational drug volixibat to treat itching (pruritus) caused by Primary Biliary Cholangitis (PBC), a liver disease. This study aims to learn more about how volixibat affects itching symptoms and whether it has any impact on the progression of PBC. The trial is a Phase 2 randomized, double-blind, placebo-controlled study designed to assess the drug's safety and effectiveness. Participants will receive either volixibat oral capsules or placebo capsules that look identical but do not contain the active drug. Volixibat is taken twice daily and works as an Ileal Bile Acid Transporter (IBAT) inhibitor. The study compares volixibat to placebo over a treatment period to determine differences in itching severity and safety outcomes. During the study, participants will complete the Adult Itch Reported Outcome (Adult ItchRO) questionnaire to measure daily itch scores from the start of the trial to week 28. Researchers will monitor symptoms and adverse events throughout the study visits. Participants must comply with all scheduled visits and assessments until the study ends to provide data on the drug's effects and safety profile.

Researchers are evaluating the study medicine PF-08046054 compared to the standard chemotherapy drug docetaxel in adults with non-small cell lung cancer (NSCLC) that has spread or cannot be removed with surgery or radiation. Participants must have PD-L1 expression on 1% or more of their tumor cells and have experienced cancer progression during or after treatment with PD-L1 or PD-1 inhibitors, platinum-based chemotherapy, and targeted therapies for those with known genetic mutations. The trial is a Phase 3 randomized study to better understand how well PF-08046054 works alone compared to docetaxel alone. Participants will be randomly assigned to receive either PF-08046054 or docetaxel. Those in the PF-08046054 group will get intravenous (IV) infusions twice every 21-day cycle, while those in the docetaxel group will receive one IV infusion every 21 days. The treatment period may last up to 5 years if their NSCLC responds to the therapy. No other treatments are combined during the study period. Throughout the study, participants will have regular clinic visits for evaluations and monitoring to see how they respond to the treatment. Researchers will collect information on overall survival over approximately 5 years. They will also monitor safety and disease progression during these visits to understand the long-term effects and benefits of the treatments.

Researchers are evaluating the effectiveness and safety of a single inhaled dose of Staccato alprazolam compared to a placebo in quickly stopping prolonged seizure episodes in people aged 12 years and older with stereotypical prolonged seizures. This Phase 3 study aims to determine if the treatment can stop seizures within 90 seconds and prevent seizure recurrence for up to 2 hours after administration. Participants will receive one inhaled dose of either Staccato alprazolam or a placebo during the treatment period. The study is randomized, double-blind, and placebo-controlled, conducted at multiple outpatient centers. The intervention consists of a single administration designed to rapidly terminate seizure episodes. During the study, participants are closely monitored for seizure treatment success within 90 seconds and no seizure recurrence up to 2 hours post-treatment. Researchers will assess seizure control and safety outcomes throughout the treatment period. The study involves baseline assessments, caregiver involvement to observe seizures, and a focus on quick treatment response and safety over several hours following drug administration.

This research aims to evaluate the long-term safety and tolerability of brivaracetam in children and adolescents with epilepsy. It includes pediatric participants who previously took part in neonatal or long-term follow-up studies, as well as new participants from Japan with partial-onset seizures. The study also includes evaluation of pharmacokinetics in Japanese participants. It is a Phase 3, open-label, single-arm, multicenter study focusing on pediatric epilepsy patients treated with brivaracetam as an additional therapy. Participants will receive brivaracetam orally twice daily, either as tablets available in 10 mg, 25 mg, or 50 mg strengths, or as an oral solution with a concentration of 10 mg/ml. The treatment is administered in two equal doses each day. The study includes those previously enrolled in related studies and new enrollees from Japan, with treatment and monitoring continuing over an extended period to assess long-term safety. During the study, researchers will monitor participants from the initial evaluation visit through safety visits that may last up to five years. They will track any adverse events related to treatment, including serious events and those causing discontinuation of the drug. Assessments include clinical evaluations, laboratory tests, and electroencephalogram readings, with ongoing safety monitoring to understand how well participants tolerate the medication over time.

Researchers are conducting a multicenter, single-arm, non-interventional survey to study the safety of Entresto Tablets or Entresto Granules in pediatric patients with chronic heart failure in Japan. The study focuses on observing these patients in real-world clinical settings and aims to collect information about safety concerns, such as hypotension, hyperkalemia, renal impairment, and dehydration. The observation period lasts for up to 52 weeks after starting treatment. The study involves pediatric patients aged 1 to under 18 years who are receiving Entresto for chronic heart failure for the first time. It monitors the administration of Entresto, including details about accidental use of the granules in capsule-shaped containers. This long-term observation helps researchers understand risk factors related to safety events and the overall status of Entresto use in this young population. Participants will be followed for one year, during which researchers will observe and record any events related to the safety of the medication. The main focus is on tracking occurrences of hypotension, high potassium levels, kidney problems, and dehydration. Safety monitoring includes collecting data on these events and understanding their relationship to Entresto use in children with chronic heart failure.

This research observes patients with Paroxysmal Nocturnal Hemoglobinuria who are treated with Fabhalta capsules. It is a multicenter, single-arm, non-interventional study designed to monitor drug use and safety over time. The study uses a central registration and all-case surveillance system to collect data. Participants will be observed for 48 weeks after starting Fabhalta treatment. If treatment stops within this period, any adverse events and use of other medications will be tracked up to 30 days after the last treatment day. There are no additional interventions or comparison groups in this study. During the study, researchers will monitor the occurrence of infections and other adverse events through case report forms. Participants' health and drug usage will be recorded throughout the observation period. The total participation lasts for 48 weeks, focusing on safety and drug use in real-world settings.

Researchers are investigating the effects of opevesostat compared to alternative treatments abiraterone acetate or enzalutamide in people with metastatic castration-resistant prostate cancer (mCRPC). This phase 3, randomized, open-label study focuses on participants who have already been treated with next-generation hormonal agents and taxane-based chemotherapy. The study aims to assess overall survival (OS) in participants with and without androgen receptor ligand binding domain (AR LBD) mutations, hypothesizing that opevesostat may improve survival. Participants will receive either oral opevesostat or an alternative hormonal agent such as abiraterone acetate or enzalutamide. Additional supportive medications like hydrocortisone, fludrocortisone acetate, prednisone, or dexamethasone may be given orally or as needed. The study compares these treatments directly during the trial period. Throughout the study, participants will provide tumor tissue samples and undergo regular assessments including imaging scans, laboratory tests, and performance status evaluations. Researchers will monitor overall survival for up to approximately 54 months, differentiating outcomes based on AR LBD mutation status. Safety and treatment effects will be carefully tracked during this time to evaluate the benefits and risks of the treatments.

Researchers are evaluating whether a new medicine called PF-08634404 combined with chemotherapy is more effective than the current standard treatment, pembrolizumab with chemotherapy, for adults with locally advanced or metastatic non-small cell lung cancer (NSCLC). This Phase 3 study focuses on adults 18 years and older with squamous or non-squamous NSCLC who are not candidates for surgery or curative chemoradiotherapy and have not received prior treatment for advanced disease. The study excludes participants with known actionable genomic alterations and aims to compare overall survival and progression-free survival over approximately 39 and 32 months, respectively. Participants are assigned to two parts based on their tumor type: squamous NSCLC patients in Part 1 and non-squamous NSCLC patients in Part 2. Within each part, participants are randomly assigned to receive either the experimental treatment PF-08634404 or the control treatment pembrolizumab, each combined with a chemotherapy regimen tailored to tumor type. Treatments are given via intravenous infusions in cycles, followed by maintenance therapy with either monotherapy or combination therapy depending on the study part. Treatment continues as long as it is beneficial and side effects remain manageable. During the study, participants will have regular visits for treatment administration and health evaluations. Cancer response will be monitored with tests every 6 weeks for the first 48 weeks and then every 12 weeks afterward. Researchers will assess overall survival and progression-free survival, ensuring thorough monitoring of participants' health and treatment effects throughout the study period.