Kodaira

Researchers are evaluating the safety and potential benefits of VHB937 in people aged 50 to 85 years with early Alzheimer's disease, including those diagnosed with Mild Cognitive Impairment due to Alzheimer's or mild Alzheimer's disease. This Phase II, multicenter, randomized, double-blind, placebo-controlled study aims to assess how VHB937 affects memory, thinking abilities, daily activities, and brain changes, while also studying how the body processes and responds to the treatment. The study includes an initial 72-week double-blind phase followed by an extension period. Participants will receive either VHB937 solution for infusion or a placebo solution through infusion during the 72-week double-blind phase. The study compares these two groups to evaluate the effects and safety of VHB937 in early Alzheimer's disease. After the double-blind phase, participants may continue in an extension period for further observation. Treatment involves regular infusions under controlled conditions throughout the study. During the study, participants and their study partners will attend visits for assessments including memory and cognitive tests, evaluations of daily functioning, brain imaging, and biomarker analysis from cerebrospinal fluid or PET scans. Researchers will monitor safety, record any side effects, and track changes using the Clinical Dementia Rating scale (CDR) over 72 weeks. The study requires a reliable partner to accompany participants to visits, and overall participation includes monitoring during treatment and the extension phase to thoroughly assess VHB937's effects and safety.

Researchers are studying participants with Congenital Myasthenic Syndromes (CMS) caused by mutations in the DOK7, MUSK, AGRN, or LRP4 genes. The goal is to understand symptoms, quality of life, and disease activity in these patients over time. This natural history study gathers important information about how CMS affects participants and tracks changes in their health. Participants will attend up to four study visits during which clinical assessments are performed. These assessments focus on evaluating symptoms and overall quality of life related to CMS. No specific treatments or interventions are administered, as the study is observational and aims to collect detailed data about the condition's progression. Throughout the study, researchers collect data retrospectively and prospectively over a period of up to 12 months. This includes information on diagnosis, healthcare use, medications, and changes in health status related to CMS. The study carefully monitors participants to better characterize the natural course of the disease and its impact on daily living.

Researchers are evaluating the safety and effectiveness of brenipatide combined with standard care compared to a placebo with standard care in adults with schizophrenia. This phase 2 study aims to understand how well brenipatide works as an additional treatment for schizophrenia and monitor any side effects. Participants eligible for the study must have schizophrenia and be on stable standard care medication. The trial consists of three main periods: a screening period lasting about one month, a treatment period that can last up to 12 months, and a follow-up period of approximately two months. During the treatment phase, participants receive either brenipatide or placebo administered by subcutaneous injection alongside their standard care. The study includes careful monitoring and adherence to study procedures such as self-injection, keeping diaries, and completing questionnaires. Participants will be involved in regular visits and assessments throughout the entire study duration, which may last up to 15 months. Researchers will measure changes in body weight from baseline to week 52 as a primary outcome. Participants will also be monitored for safety and efficacy through ongoing evaluations, including the use of electronic or paper diaries and required questionnaires to track their progress and response to treatment.

Researchers are evaluating how well vortioxetine tablets at doses of 10 mg/day or 20 mg/day work compared to placebo tablets for treating depression symptoms in Japanese teenagers aged 12 to 17 years who have been diagnosed with Major Depressive Disorder (MDD). This phase 3 clinical trial aims to assess the drug's effectiveness, safety, and how the body processes the medication in this pediatric population. Participants will be randomly assigned to receive either vortioxetine or a placebo once daily for 14 weeks. The study includes an initial screening period of up to 15 days to determine eligibility, followed by the 14-week treatment phase. After completing treatment, there is a 4-week period dedicated to monitoring any side effects. Throughout the study, participants will visit the clinic 13 times for assessments and medication administration. During the study, participants will undergo evaluations including the Children Depression Rating Scale Revised version (CDRS-R) to measure changes in depression symptoms from baseline to week 14. Other assessments include safety monitoring and pharmacokinetics. Researchers will also collect information on side effects during and after treatment. The total time commitment for participants is about 20 weeks, including screening, treatment, and follow-up periods.

Researchers are evaluating the effectiveness and safety of empasiprubart compared to intravenous immunoglobulin (IVIg) in adults diagnosed with Multifocal Motor Neuropathy (MMN). This Phase 3 study includes a double-blinded phase where participants receive either empasiprubart or IVIg, followed by an open-label phase where all receive empasiprubart. The study aims to assess treatment impact over a maximum duration of up to 49 months. Participants receive intravenous infusions of either empasiprubart or IVIg during the double-blinded phase, with matching placebo infusions given to maintain blinding. In the open-label phase, all participants are given empasiprubart infusions. The study carefully monitors treatments and their effects throughout these periods. Participants undergo assessments including measuring changes in grip strength of their most affected hand, using a 3-day moving average at week 24 as the primary outcome. Safety and efficacy are tracked throughout the study duration, with detailed follow-up visits and evaluations to monitor participant response and well-being during the study period.

Researchers are evaluating the long-term safety and tolerability of KarXT in treating mania or mania with mixed features in adults with Bipolar-I disorder. This phase 3, open-label extension study aims to better understand how KarXT performs over an extended period in this population. The study includes participants who either completed previous double-blind placebo-controlled studies or are newly diagnosed with Bipolar-I disorder experiencing manic symptoms. Participants receive KarXT at specified doses on certain days, with some also taking therapeutic doses of Lithium, Valproate, or Lamotrigine as part of their treatment. The study does not mention a placebo group during this extension, focusing instead on monitoring the long-term effects of KarXT alone or in combination with these established therapies. During the study, participants are monitored for adverse events up to week 54 to assess safety. Evaluations include psychiatric assessments using scales such as the Young Mania Rating Scale and CGI-BP score at screening and baseline. Researchers will track treatment-emergent adverse events and overall tolerability throughout the study duration, which lasts up to 54 weeks for each participant.

Researchers are evaluating the safety and effectiveness of adding KarXT (Xanomeline/Trospium Chloride) to standard treatment for mania in adults with Bipolar-I Disorder. This Phase 3, randomized, double-blind study focuses on individuals experiencing acute manic episodes, with or without mixed features, who are already taking lithium, valproate, or lamotrigine. The study aims to measure changes in mania symptoms using the Young Mania Rating Scale at Week 5. Participants will be randomly assigned to receive either KarXT or a placebo alongside their stable dose of lithium, valproate, or lamotrigine. The doses of these medications are specified and given on set days during the study. Only those with stable mood stabilizer doses for at least two weeks prior to screening, and valproate treatment for at least seven months, are eligible. The treatment period lasts for 5 weeks. During the study, participants will be closely monitored through psychiatric evaluations and clinical assessments. Researchers will assess mania severity, safety, and any side effects. The main outcome is the change from baseline in the Young Mania Rating Scale score at Week 5. Participants’ physical health, including liver function and risk of urinary or gastrointestinal issues, will also be monitored to ensure safety throughout the trial.

Researchers are investigating the safety and effectiveness of KarXT in treating manic episodes in adults with Bipolar-I Disorder. This Phase 3 study focuses on individuals experiencing acute mania or mania with mixed features who require hospitalization. The study aims to compare KarXT with a placebo to see how well it reduces manic symptoms during a three-week inpatient period. Participants will be randomly assigned to receive either KarXT or a placebo at specified doses during the inpatient treatment phase. The study is double-blind, so neither the participants nor the researchers know who receives which treatment. The total study duration, including screening, treatment, and safety follow-up, will not exceed seven weeks. During the study, participants will be closely monitored through psychiatric evaluations and rating scales, including the Young Mania Rating Scale (YMRS) to measure changes in mania symptoms by week 3. Other assessments include the Clinical Global Impressions-Bipolar scale and safety evaluations. Researchers will track adherence, symptoms, and any side effects throughout the study period.

Researchers are evaluating the effectiveness and safety of KarXT in Japanese adults aged 18 to 65 who are experiencing acute psychotic episodes due to schizophrenia. The study focuses on adults diagnosed with schizophrenia according to the Diagnostic and Statistical Manual of Mental Disorders-Fifth Edition (DSM-5) and confirmed by a psychiatric interview. Participants must have a specific range of symptom severity measured by the Positive and Negative Syndrome Scale (PANSS) and Clinical Global Impression-Severity (CGI-S) scale. Participants are randomly assigned to receive either KarXT or a placebo during a 5-week double-blind phase where neither the participants nor the researchers know which treatment is given. After this, there is a 52-week open-label extension where all participants receive KarXT. The doses are specified and administered on set days throughout the study. Throughout the study, researchers monitor changes in schizophrenia symptoms using the PANSS score at week 5 and track any treatment-emergent adverse events up to week 52 during the open-label extension. The study involves regular assessments to ensure safety and effectiveness over both the short and long term, with total participation lasting up to 57 weeks.

Researchers are evaluating the efficacy and safety of SAR441566, an oral tablet medication, in adults with moderate-to-severe ulcerative colitis (UC). This Phase 2, multinational, multicenter, randomized, double-blind, placebo-controlled, dose-ranging study aims to determine how well different doses of SAR441566 help patients achieve clinical remission, measured by the modified Mayo Score, after 12 weeks of treatment. The study includes a screening period lasting up to 28 days plus an additional 7 days if needed. The main treatment period lasts 52 weeks and consists of a 12-week double-blind induction phase followed by a 40-week maintenance phase, ending with a 2-week follow-up after treatment. Participants receive either SAR441566 or a matching placebo orally as tablets. After the main study, eligible participants not joining a long-term study may enter an open-label treatment period for up to 40 weeks. The main study involves 12 visits, and the open-label phase includes 8 visits. During the study, participants will be monitored through clinical assessments and scoring of disease activity. Researchers will track remission rates at week 12 and evaluate long-term safety and efficacy during the maintenance and open-label periods. The total time involved for participants can be up to 59 weeks, including screening, treatment, follow-up, and optional open-label extension.