Kaminokawa

Researchers are evaluating zorevunersen, an investigational antisense oligonucleotide drug, in children with Dravet syndrome, a rare and severe form of epilepsy. This Phase 3, global, multicenter, randomized, double-blind, sham-controlled study aims to assess the efficacy, safety, and tolerability of zorevunersen by measuring changes in major motor seizure frequency and other important aspects such as behavior, cognition, clinical status, and quality of life. Participants will be randomly assigned to receive either zorevunersen or a sham procedure during Treatment Period 1, which lasts about 52 weeks. Zorevunersen is given by intrathecal injection at specific doses and intervals throughout this period. After Treatment Period 1, all eligible patients enter Treatment Period 2, where everyone receives zorevunersen for additional dosing over several months. Patients who complete the study may have the chance to join an open-label extension to continue receiving the drug. During the study, participants will undergo regular assessments including seizure monitoring, behavioral and cognitive evaluations, and health-related quality of life measurements. The primary outcome is the change in major motor seizure frequency at Week 28, with secondary outcomes assessed at Week 52. Safety and tolerability are also closely monitored. Overall participation lasts through both treatment periods and possible extension, with detailed follow-up to evaluate the drugs potential for disease modification.

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 etrasimod for treating adolescents aged 12 to under 18 years with moderately to severely active ulcerative colitis. This Phase 2 study aims to determine the safety, effectiveness, and how the drug is processed in the body over a 52-week treatment period. Participants who complete this treatment may continue in a long-term extension for up to 4 additional years, totaling 5 years after enrollment. Participants will take etrasimod tablets or granules by mouth once daily for up to 52 weeks. After this period, those who complete the treatment can join an optional long-term extension phase lasting up to 4 years. The study does not include a placebo group and involves a single treatment arm. During the study, participants will be monitored through regular assessments including clinical remission measured by the Modified Mayo Score at week 52. Blood samples will be taken at various times to measure drug levels. Other outcomes include endoscopic improvement, symptomatic remission, and safety evaluations through adverse event tracking. The total participation could last up to 5 years for those continuing in the extension phase.

Researchers are evaluating the efficacy and safety of KarXT in adult Japanese participants who are acutely psychotic with schizophrenia. This Phase 3 study focuses on adults aged 18 to 65 years who meet specific criteria based on psychiatric evaluations and symptom severity scores. The trial aims to understand how KarXT affects schizophrenia symptoms compared to a placebo over a 5-week period, followed by a longer open-label extension. Participants are randomly assigned to receive either KarXT or a placebo during a 5-week double-blind treatment phase. Following this, they may enter a 52-week open-label extension where all participants receive KarXT. The study includes regular dosing on specified days, with careful monitoring throughout both phases. During their participation, individuals undergo assessments of symptom changes using scales like the Positive and Negative Syndrome Scale PANSS and Clinical Global Impressions-Severity CGI-S. Safety is closely monitored through reports of adverse events, physical exams, laboratory tests, heart rate and blood pressure measurements, and other evaluations. The total study duration can last over a year, allowing for detailed observation of treatment effects and safety over time.

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.

Researchers are evaluating ivonescimab as a first-line treatment for patients with metastatic non-small cell lung cancer NSCLC whose tumors show high PD-L1 expression. This phase 3 study compares ivonescimab to pembrolizumab, focusing on overall survival and progression-free survival to understand which treatment may better support patients with this condition. Participants will be randomly assigned to receive either ivonescimab or pembrolizumab through intravenous injection. The study is double-blinded and multiregional, involving continuous treatment and monitoring for up to approximately 36 months. Both groups receive their assigned treatment regularly during this period, with careful observation of treatment responses and side effects. During the trial, participants will undergo evaluations including scans to measure tumor size, laboratory tests, and assessments of adverse events. Researchers will track overall survival, progression-free survival, response rates, disease control, and duration of response. Safety monitoring will continue for up to 24 months after the last dose. The total study period extends until June 2029, allowing long-term data collection on treatment effects and safety.

Researchers are evaluating the effects of mexiletine hydrochloride in men with spinal and bulbar muscular atrophy SBMA, a condition that causes muscle weakness. This clinical trial aims to determine if mexiletine hydrochloride can improve motor function as measured by the ALS Functional Rating Scale-Revised ALSFRS-R score. The study is designed as a randomized, placebo-controlled, double-blind trial to assess both the efficacy and safety of the drug. Participants will be randomly assigned to receive either mexiletine hydrochloride or a placebo. The active treatment involves taking 300 mg of mexiletine hydrochloride orally, divided into three doses after meals each day for 12 weeks. The placebo group will follow the same schedule with an inactive substance. Hospital visits for evaluation will occur every four weeks during the treatment period. During the study, participants will undergo regular assessments including ALSFRS-R scoring at 4 weeks and up to 12 weeks, as well as other tests such as grip strength, tongue pressure, timed walking tests, and respiratory function tests measuring forced vital capacity and peak expiratory flow. These evaluations will help measure motor function and respiratory health. Safety and adherence will be closely monitored throughout the 3-month treatment period.

This research focuses on improving the safety and quality of tracheal intubation practices in pediatric intensive care units PICUs, cardiac ICUs CICUs, neonatal ICUs NICUs, emergency departments EDs, and delivery rooms DRs. It addresses the risks associated with advanced airway interventions in critically ill children, aiming to reduce life- and health-threatening complications during these high-risk procedures. The study is part of a multi-center quality improvement collaborative called NEAR4KIDS. The study collects detailed baseline data on airway management practices across multiple centers to benchmark and compare care. This observational approach helps identify current practices and monitor changes over time. The goal is to use this data to support quality improvement interventions that enhance safety and reduce complications related to tracheal intubation and related airway management techniques. Participants include patients of all ages undergoing advanced airway events in PICU, CICU, NICU, DR, and ED settings. Researchers will track the occurrence of tracheal intubation-associated events TIAEs throughout each patients hospital stay, typically averaging about four weeks. Data collection involves monitoring airway procedures and outcomes to assess changes in complication rates and the overall effectiveness of quality improvement efforts.

Researchers are evaluating the safety and efficacy of JNJ-90301900 NBTXR3 activated by radiotherapy, with or without cetuximab, compared to radiotherapy with or without cetuximab alone. This Phase 3 study focuses on elderly participants aged 60 years and older who have locally advanced head and neck squamous cell carcinoma LA-HNSCC and are ineligible for platinum chemotherapy. The study is randomized and open-label, aiming to provide insights for this specific patient group. Participants will be assigned to one of two groups. One group receives an intratumoral or intranodal injection of JNJ-90301900 NBTXR3 at a dose of 33% of the gross tumor volume, followed by radiotherapy alone or combined with cetuximab, based on the investigators choice. The other group receives radiotherapy alone or combined with cetuximab. Radiotherapy is intensity-modulated, delivering 70 Gray over 35 fractions across 7 weeks. During the study, participants will be monitored for up to 48 months following the first randomized participant. Researchers will assess progression-free survival as the primary outcome, measured at 30 months, along with secondary outcomes such as overall survival, local-regional control, distant control, quality of life, safety, response rates, and head and neck cancer-specific survival. Regular assessments include clinical evaluations and quality of life questionnaires to track treatment effects and safety over time.

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.