Baggiovara

Researchers are evaluating the safety and effectiveness of KarXT in preventing relapse of psychosis symptoms in people aged 55 to 90 years who have psychosis associated with Alzheimer's Disease. This Phase 3 study is randomized, double-blind, placebo-controlled, and conducted at multiple outpatient centers. The main goal is to compare relapse prevention between KarXT treatment and placebo over 38 weeks, while also assessing time to discontinuation, safety, and tolerability. Participants receive either KarXT in varying doses (ranging from 20 mg/2 mg to 66.7 mg/6.67 mg taken three times daily) or placebo capsules. The study lasts 38 weeks, during which participants remain on assigned treatment in an outpatient setting. The randomized, double-blind design ensures neither participants nor researchers know who receives KarXT or placebo during the study. Throughout the study, participants will visit the clinic regularly for assessments of their psychosis symptoms, safety checks, and overall health. Researchers will track the time to relapse of psychosis symptoms as the primary outcome. They will also monitor safety and tolerability through clinical examinations and other evaluations. The total duration of participation is 38 weeks from randomization to the end of the study period.

Researchers are evaluating the effect of muvalaplin on reducing cardiovascular risk in adults with elevated lipoprotein(a) levels who either have atherosclerotic cardiovascular disease or are at risk for a heart attack or stroke. This Phase 3, randomized, double-blind, placebo-controlled study focuses on adults with high Lp(a) levels and prior or potential cardiovascular events. The study aims to assess the time to the first major adverse cardiovascular event over about 5.25 years. Participants will be randomly assigned to receive either muvalaplin or a placebo, both administered orally. The study includes individuals with Lp(a) levels of at least 175 nanomoles per liter who have had a prior cardiovascular event within 10 years or are at risk for a first event due to conditions such as coronary artery disease, carotid stenosis, peripheral artery disease, high coronary artery calcium score, reduced kidney function with diabetes, or other high-risk factors. The treatment period lasts through the study duration, with close monitoring. During the study, participants will be regularly evaluated to track the occurrence of major adverse cardiovascular events, including heart attacks and strokes. Safety assessments will monitor blood pressure, kidney function, and heart failure status among other health indicators. The primary outcome measures the time to the first major cardiovascular event from baseline up to the end of the study, which spans approximately 5.25 years.

Researchers are evaluating the safety and effectiveness of masitinib combined with riluzole compared to a placebo combined with riluzole for treating patients with Amyotrophic Lateral Sclerosis (ALS). Masitinib is an oral drug that targets specific cells involved in neuroinflammation, such as mast cells and microglia, which are believed to play a key role in ALS progression. This Phase 3 study is designed to better understand how masitinib may affect the disease by slowing progression and reducing inflammation in the nervous system. The study is a multicenter, double-blind, randomized, placebo-controlled trial where participants receive either oral masitinib at doses titrated to 4.5 or 6.0 mg/kg/day plus riluzole, or a matching placebo plus riluzole. Riluzole is given as a 50 mg tablet taken by mouth. The treatment period includes two ascending dose titrations for masitinib or placebo groups to compare outcomes and safety. Participants continue their stable riluzole treatment throughout the study. During the trial, participants are monitored for changes in their ALS Functional Rating Scale-Revised (ALSFRS-R) scores over 48 weeks, which measures their physical function and disease progression. Assessments include clinical evaluations and safety monitoring to track the impact of the treatments. The study enrolls adults aged 18 to 81 years who have probable or definite ALS and meet specified disease duration and function criteria. The goal is to gather detailed information on how masitinib in combination with riluzole might alter the course of ALS compared to riluzole alone.

Researchers are tracking patients with Fabry disease through an ongoing international, multi-center observational program called the Fabry Registry. This program collects routine clinical data from patients regardless of their treatment status to better understand the disease's variability, progression, and natural history. It also focuses on enhancing patient care by supporting the development of monitoring recommendations and evaluating the long-term safety and effectiveness of Fabrazyme, a treatment for Fabry disease. The study includes a Fabry Pregnancy Sub-registry, which is a voluntary, international, longitudinal observation program that monitors pregnancy outcomes for women enrolled in the Fabry Registry who are pregnant or have been pregnant. This sub-registry collects medical and obstetric history, pregnancy, and birth data, along with infant growth information up to 36 months postpartum, regardless of the specific treatment received. No experimental treatments are administered in either registry; patients continue receiving routine care as determined by their physicians. Participants contribute data through clinical assessments and standard care evaluations performed by their doctors. The study measures long-term outcomes including safety and effectiveness of Fabrazyme over up to 33 years, as well as pregnancy outcomes and infant growth data. The program helps fulfill regulatory requirements and supports research while tracking patient health over extended periods without altering their usual care.

The International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry is an ongoing global observational program tracking routine clinical outcomes in patients diagnosed with Gaucher disease. It includes patients regardless of their treatment status and aims to improve understanding of the variability, progression, and natural history of Gaucher disease. The Registry also seeks to support the medical community by developing monitoring recommendations, characterizing the patient population, and evaluating long-term treatment effectiveness of imiglucerase and eliglustat. The Registry involves no experimental treatments; patients receive clinical assessments and care as directed by their treating physicians. Additionally, there is a Gaucher Pregnancy Sub-registry that monitors pregnancy outcomes, complications, and infant growth up to 36 months postpartum for women with Gaucher disease. This Sub-registry collects medical and obstetric history and pregnancy data for participants who consent, without altering their standard care. Participants provide data through routine clinical visits, and researchers collect medical information to better understand patient outcomes and optimize care. The Registry tracks outcomes over long periods, including up to 42 years, to support ongoing care improvements. Women in the Pregnancy Sub-registry have additional data collected on pregnancy and infant growth, contributing to comprehensive monitoring of Gaucher disease impacts during and after pregnancy.

The trial investigates the long-term safety and tolerability of KarXT in people with psychosis associated with Alzheimer's Disease. This Phase 3 global, multicenter, open-label extension study lasts 52 weeks and enrolls participants who have completed earlier related studies (CN012-0026, CN012-0027, or CN012-0056). The purpose is to monitor how well patients tolerate KarXT over an extended period and to collect safety data. Participants receive KarXT in varying doses taken three times daily, ranging from 20/2 mg up to 66.7/6.67 mg per dose, corresponding to total daily doses between 60/6 mg and 200/20 mg. This treatment is provided throughout the 52-week open-label extension. The study includes only those who completed the previous related studies and continues to assess their response to KarXT over this longer timeframe. During the study, participants are closely monitored for treatment-emergent adverse events from the first dose through 14 days after the final dose, which may be up to 54 weeks. Regular assessments ensure safety and tolerability, and caregivers are involved to support participants. The study also evaluates participants' ability to continue living in their current setting and requires consent from the participant or their legal representative. Overall, the study tracks long-term safety outcomes in this specific patient group.

Researchers are conducting a multicenter, prospective observational study to gather clinical data on geriatric patients with hip fractures treated in orthogeriatrics and orthopedics units. The study aims to compare management and clinical methods across centers to promote good clinical practices in orthogeriatrics. The study plans to enroll approximately 7,000 patients aged 65 years and older over a 5-year period, with a 4-month follow-up after enrollment ends. Participating centers can choose between two data collection methods: a high-intensity approach involving continuous data collection for all cases throughout the study, or a low-intensity approach collecting data once a year for one month with fewer variables. This flexibility allows both university-affiliated and non-university assistance departments to participate according to their capacity. Centers decide voluntarily on their preferred data collection intensity after consulting with the study's steering committee and biostatisticians. Participants will be followed for 120 days after enrollment to monitor clinical outcomes and management approaches. The study will continuously collect and compare clinical activity data over approximately 5 years, with the goal of improving care standards. There are no exclusion criteria, and participants or their caregivers must give consent and be able to speak Italian. The study monitors clinical methods and management practices without introducing investigational treatments.

This research focuses on patients with Severe Acquired Brain Injuries and aims to identify medium-term factors that can predict their rehabilitation outcomes. The study also seeks to develop a continuous system to assess the quality of care provided by rehabilitation units treating these patients. Identifying these prognostic factors helps understand which patients are likely to experience key rehabilitation milestones. Data will be collected from rehabilitation units and will include information about the units themselves and detailed patient data at admission and again four months after the acute brain injury event. The study will use statistical models to analyze the likelihood of achieving important rehabilitation goals such as removal of tracheostomy tubes, trunk control, and feeding entirely by mouth, all evaluated four months after the injury. Participants will be assessed at the start of rehabilitation and then re-evaluated four months later. The study measures include monitoring tracheostomy tube status, trunk control ability, and feeding methods. This allows researchers to track patient progress and evaluate care quality over time. The total participation duration includes the initial admission and follow-up assessments up to four months post-injury.

Researchers are evaluating the effectiveness of BHV-7000 in treating adults with refractory focal onset epilepsy, a condition where seizures originate in specific areas of the brain and have not responded to previous treatments. This Phase 2/3 trial aims to assess the safety, tolerability, and ability of BHV-7000 to reduce seizure frequency in participants who continue to have seizures despite using anti-seizure medications. The study follows classification criteria set by the International League Against Epilepsy and includes participants aged 18 to 75 years. Participants will be randomly assigned to receive either BHV-7000 at doses of 50 mg or 75 mg once daily, or a matching placebo, in a double-blind setup where neither participants nor researchers know which treatment is given. The treatment period focuses on monitoring changes in seizure frequency over 28-day averages from baseline through weeks 8 to 16. The study design includes careful control and comparison to evaluate the investigational drug's impact. During the study, participants will keep accurate seizure diaries to track their seizures. Researchers will measure changes in the average number of seizures over 28-day periods as the primary outcome. Safety and tolerability will also be monitored closely. The study requires participants to be currently treated with one to three anti-seizure medications and to meet specific epilepsy criteria. Overall participation includes screening, treatment, and follow-up to assess the drug's effects and participant safety.