Baggiovara

This trial studies adults aged 55 to 90 who have psychosis linked to Alzheimers Disease. It is a Phase 3, 38-week, randomized, double-blind, placebo-controlled outpatient study. Its main goal is to assess how well KarXT capsules prevent relapse of psychosis compared to placebo. Additional goals include evaluating time to treatment discontinuation or relapse, and monitoring safety and tolerability. Participants receive either KarXT capsules at various doses or placebo capsules. The study involves a randomized assignment and is conducted under quadruple masking. The treatment period lasts 38 weeks during which KarXT or placebo is taken three times daily. Assessments continue up to approximately 42 weeks to monitor adverse events and other safety measures. Throughout the study, participants attend outpatient visits for evaluations including cognitive tests, assessments of psychosis severity, caregiver reports, lab tests, vital signs, and safety monitoring. Researchers measure relapse timing, treatment discontinuation, neuropsychiatric symptoms, movement scales, weight, and signs related to heart and urinary health. Safety is closely tracked with various assessments until about week 42.

Researchers are evaluating the effect of muvalaplin in lowering cardiovascular risks among adults with elevated lipoproteina who either have atherosclerotic cardiovascular disease or are at risk of a first heart attack or stroke. This phase 3, randomized, double-blind study aims to investigate whether muvalaplin can reduce major adverse cardiovascular events compared to placebo in this high-risk population. Participants are randomly assigned to receive either muvalaplin or a placebo, both given orally. The study is designed with parallel groups and will last about 5.25 years, during which the occurrence of cardiovascular events and changes in lipoproteina levels will be closely monitored. Throughout the study, participants will undergo regular assessments including measurement of lipoproteina levels, monitoring of cardiovascular events such as heart attacks or strokes, and evaluation of healthcare resource use. The primary outcome is the time to first major adverse cardiac event, tracked from baseline until the study ends. Safety and pharmacokinetics of muvalaplin will also be evaluated during the trial period.

Researchers are evaluating the effectiveness and safety of masitinib combined with riluzole compared to a placebo combined with riluzole for treating Amyotrophic Lateral Sclerosis ALS. This phase 3 study focuses on patients diagnosed with probable or definite ALS, aiming to understand how masitinib, a drug that targets cells involved in neuroinflammation, might slow disease progression and affect the nervous systems environment. Participants receive oral masitinib at 3.0 mgkgday twice daily, with dose increases to 4.5 mgkgday after 4 weeks, and for some, an additional increase to 6.0 mgkgday after another 4 weeks. Each dose increase includes safety monitoring. Masitinib is given alongside riluzole, a standard ALS treatment at 50 mg twice daily. The control group receives a matching placebo with riluzole. The study is randomized, double-blind, and includes two different masitinib dose escalation schedules. During the 48-week study period, participants undergo regular assessments including functional rating scales ALSFRS-R, quality of life questionnaires ALSAQ-40, survival and progression monitoring, lung function tests FVC, and muscle strength measurements HHD. Researchers measure changes in combined function and survival scores. Safety is closely monitored throughout, with follow-up extending up to 36 months for disease progression or death. Total participation includes baseline screening, treatment, and long-term observation.

Researchers are tracking patients with Fabry disease through an ongoing international observational program called the Fabry Registry. This registry collects routine clinical outcomes for patients regardless of whether they are receiving treatment. The study aims to better understand the diseases variability, progression, and natural history, including in women who carry one copy of the gene, and to help improve patient care by developing monitoring recommendations and reporting outcomes. Additionally, the registry evaluates the long-term safety and effectiveness of Fabrazyme4, a treatment used in Fabry disease. The registry includes a special pregnancy sub-registry for women with Fabry disease who are pregnant or have been pregnant. This sub-registry observes pregnancy outcomes and infant growth up to 36 months after birth, collecting medical and obstetric history and treatment details. No experimental treatments are given participants continue to receive their usual care as determined by their physicians. Data from both registries support regulatory requirements and ongoing research. Participants undergo regular clinical assessments and receive standard care from their doctors throughout the study. The research team collects data on disease progression, treatment effectiveness, pregnancy outcomes, and infant development. The study is observational, meaning no study drugs or procedures are administered. The total participation can last up to 33 years, allowing for long-term monitoring of safety and outcomes related to Fabry disease and pregnancy.

Researchers are tracking the natural history and clinical outcomes of patients with Gaucher disease through the ICGG Gaucher Registry, an international, multi-center observational program. This registry does not involve any experimental treatments, but collects information to better understand the variability, progression, and identification of Gaucher disease, aiming to improve patient care and therapeutic guidance. It also evaluates the long-term use of treatments like imiglucerase and eliglustat. The study includes two groups patients with Gaucher disease who receive routine clinical assessments and standard care as determined by their physicians, and a Pregnancy Sub-registry for women with Gaucher disease who are pregnant or have been pregnant. The Pregnancy Sub-registry collects information on pregnancy outcomes, complications, and infant growth up to 36 months postpartum, regardless of whether the women receive disease-specific therapy. Participants undergo clinical assessments and receive care according to their treating physicians decisions. Data collected includes medical history, pregnancy and birth details for the sub-registry, and patient outcomes over time. The primary goals are to provide recommendations for monitoring Gaucher disease patients, report outcomes to optimize care, and track pregnancy and infant growth outcomes. This ongoing registry allows long-term follow-up without experimental interventions.

This research focuses on people with psychosis linked to Alzheimers Disease who have completed earlier studies CN012-0026, CN012-0027, or CN012-0056. It is a Phase 3 global, multicenter, open-label extension study lasting 52 weeks that aims to evaluate the long-term safety and tolerability of KarXT in this population. Participants will receive KarXT capsules containing Xanomeline and Trospium Chloride. The study includes various dosing levels ranging from 202 mg to 66.76.67 mg taken three times daily. This open-label extension follows completion of previous studies and continues for up to 54 weeks from the initial dose, including a 14-day safety follow-up after the final dose. During the study, participants will be monitored for treatment-emergent adverse events and serious adverse events. Safety assessments include clinical evaluations throughout the treatment and 14 days after the last dose. The total participation time is approximately one year, allowing researchers to understand long-term effects of KarXT in this group.

This research aims to collect and analyze clinical activity and management data from Orthogeriatrics and Orthopedics Units treating geriatric patients with hip fractures. By continuously gathering data over a long period, the study seeks to compare how different centers manage care and to guide improvements in clinical practice within orthogeriatrics. The study is a multicenter, prospective observational cohort study enrolling an estimated 7,000 patients aged 65 and older over five years. Data collection can be done either continuously with high intensity on all new cases or yearly with low intensity involving a smaller set of variables over one month. Centers voluntarily choose their preferred data collection method. Participants will be followed for 120 days after enrollment, with an additional four months allowed for the last patients to complete follow-up. Researchers will collect clinical data to assess management approaches, track fracture patterns and patient profiles by age, and evaluate improvements in clinical practice and interdisciplinary collaboration. The study will run for approximately five years and monitor outcomes related to orthogeriatric care models and sensitivity to elderly patient needs.

This research aims to identify medium-term factors that influence recovery for patients with Severe Acquired Brain Injuries. It also seeks to develop a continuous system to assess the quality of care provided by rehabilitation units treating these patients. The study focuses on understanding key outcomes important to rehabilitation success. Data will be collected from each participating rehabilitation unit, including information about their structure and organization. Detailed patient data will be gathered upon admission to these units and then re-evaluated four months after their acute brain injury event. Statistical models will be used to predict the likelihood of achieving three key rehabilitation goals removal of tracheostomy tubes, trunk control, and full feeding by mouth. Participants will be adults admitted to these specialized rehabilitation units between July 2021 and December 2023. Patients admitted more than 14 weeks after their acute injury will not be included. Researchers will monitor patients for up to four months post-injury to evaluate the main outcomes. The study will help understand patient progress and care quality in these rehabilitation settings.