Cefalu

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 evaluating the effectiveness and safety of nipocalimab compared to placebo in adults with generalized myasthenia gravis (gMG), a condition causing muscle weakness. This phase 3, multicenter, randomized, double-blind study also includes a subcutaneous substudy to assess how nipocalimab works in the body when given as an injection under the skin compared to intravenous infusion. Participants will receive nipocalimab or a matching placebo through intravenous infusion. In the subcutaneous substudy, nipocalimab will be administered under the skin. The study includes groups receiving different forms of the drug, with dosing schedules detailed in the protocol. The subcutaneous substudy requires participants to maintain stable doses of corticosteroids and/or immunosuppressants for the first 8 weeks. During the study, participants will undergo assessments including the Myasthenia Gravis - Activities of Daily Living (MG-ADL) score measured at baseline and weeks 22 to 24. Blood samples will be collected to measure antibody levels and total IgG from before the first dose up to week 8 in the sub-study. Safety and efficacy will be closely monitored throughout the trial period.

This research aims to evaluate the efficacy and safety of telitacicept in treating generalized myasthenia gravis (gMG), an autoimmune disease where autoantibodies disrupt nerve-to-muscle communication, causing muscle weakness that worsens with activity. The study addresses the challenge of limited effective therapies for this condition. Telitacicept is a fully human fusion protein designed to block specific immune system signals that promote B-cell growth and maturation, potentially reducing autoimmune symptoms in gMG. The study is a Phase 3, randomized, double-blind, placebo-controlled trial with an open-label extension. Participants will receive subcutaneous injections of either telitacicept or placebo. The study includes a 4-week screening period, a 24-week double-blind treatment phase, a 48-week open-label extension, followed by a variable-duration extended open-label extension until telitacicept is approved or development ends, and an 8-week end-of-study follow-up. Participants will undergo assessments including the Myasthenia Gravis-Activities of Daily Living (MG-ADL) score to measure changes in daily functioning by Week 24. The study also monitors safety and efficacy over the treatment and extension periods. Throughout the trial, various clinical evaluations will be conducted to track disease status and response to treatment, ensuring comprehensive monitoring of participant health and outcomes.

Researchers are evaluating ACP-204, a drug that blocks a specific serotonin receptor, in adults aged 55 to 95 with Alzheimer's Disease Psychosis (ADP). The study is designed as a master protocol with three independent, multicenter, randomized, double-blind, placebo-controlled trials. The trials include Phase 2 and Phase 3 studies to assess the drug's effectiveness and safety in treating psychotic symptoms associated with ADP. The research involves three substudies. Substudy 1 (Phase 2) tests two doses of ACP-204, 30 mg and 60 mg, against a placebo to evaluate dose response. Substudies 2A and 2B (both Phase 3) will independently confirm the effects of either both doses or a single dose from Part 1 compared to placebo. Each substudy includes a screening period of up to 49 days, a six-week double-blind treatment phase, and a 30-day safety follow-up for those not continuing into an open-label extension. Vital status follow-up is conducted for participants who end the study early. Participants will receive regular assessments, including evaluations of psychotic symptoms using the Scale for the Assessment of Positive Symptoms-Hallucinations and Delusions subscales from baseline to Week 6. Other study involvement includes brain imaging scans and biomarker tests to confirm Alzheimer's disease diagnosis, cognitive testing, and monitoring of safety and vital status throughout the study periods. Stable living arrangements and support from a caregiver are required to complete all study visits.

Researchers are conducting an observational, prospective, multicenter study in Italian cardiology centers to evaluate how well patients with Heart Failure with Reduced Ejection Fraction (HFrEF) follow guideline-recommended treatments. The study also aims to assess the safety of these treatments, monitor treatment patterns in patients with acute heart failure, and observe treatment approaches in all chronic heart failure patients regardless of their ejection fraction levels. The study involves two phases of educational interventions and data collection. Initially, healthcare providers will receive education on guideline recommendations and treatment patterns, followed by 3 months of patient data collection or up to 30 consecutive patients with chronic or acute heart failure. After 6 months, treatment modifications and outcomes will be evaluated. Then, a second educational session will highlight gaps between guidelines and practice, followed by another 3 months of data collection. Patients will be followed for 12 months total, with ongoing monitoring of treatment changes and outcomes. Participants will be assessed at enrollment and during the follow-up periods through clinical evaluations and data collection on treatment adherence and safety. The main outcome measured is adherence to guideline-directed medical therapies over 6 months. The study includes evaluations at 6 and 12 months after enrollment, with close monitoring of treatment patterns and patient health status throughout the study duration.

Researchers are evaluating patients who have experienced athero-thrombotic events such as coronary artery disease, cerebrovascular disease, or peripheral artery disease. The study aims to assess how well patients follow guideline recommendations, particularly focusing on improving cholesterol levels and other modifiable risk factors to reduce the chance of cardiovascular event recurrence. This observational and prospective study takes place across multiple cardiology centers in Italy to represent a broad patient population. The study includes several phases starting with an educational intervention to discuss guideline recommendations for secondary prevention. Following this, data is collected for three months or until 30 patients with documented cardiovascular conditions are enrolled, using a web-based case record form that identifies when guidelines are not followed and records reasons for non-adherence. After six months, primary and secondary outcomes are evaluated. A second educational intervention then shares findings from the first phase to highlight gaps in clinical practice, followed by another three-month data collection period and a further six-month outcome assessment. Finally, all patients are followed for 12 months to monitor longer-term results. Participants provide informed consent and are monitored through data collection forms that track adherence to guidelines and clinical outcomes. The main outcome measured is adherence to cholesterol management guidelines over six months. Additional assessments include adherence to recommendations for other cardiovascular risk factors. Throughout the study, researchers gather data to understand how guideline adherence affects patient health and to identify barriers to following best practices, with continuous follow-up over a year to evaluate sustained effects.

This research evaluates the effects of a nutritional intervention using ketogenic medium-chain triglycerides (kMCT) and B-vitamins on cognitive functioning in older adults who have mild cognitive impairment (MCI). The trial is designed as a prospective, randomized, double-blind, placebo-controlled, multi-center, and multi-country pivotal study. It aims to study cognitive changes in this population, particularly focusing on those with memory complaints lasting more than three months and clinical diagnoses consistent with MCI. Participants receive either BrainXpert, a dietary supplement sachet containing 25 g of ketogenic medium-chain triglycerides without preservatives or additives, or a placebo consisting of a calorie-equivalent non-ketogenic vegetable oil powder in a similar sachet form. The study includes multiple clinic visits over an 18-month period, with key assessments at randomization, 12 months, and 18 months. The placebo is high-oleic acid sunflower oil without added vitamins, ensuring blinding between groups. During the study, participants and their informants attend scheduled visits where cognitive performance is assessed, including the Preclinical Alzheimer's Cognitive Composite (PACC) score measured at 12 months. Researchers monitor participants' daily living autonomy, cognitive complaints, and compliance with the intervention. Safety and eligibility are closely observed, with attention to medication use and other health factors. The total involvement spans at least 18 months with multiple assessments to evaluate the nutritional intervention's impact on cognition.

This research focuses on rare cerebrovascular diseases (rCVDs) such as CADASIL, Fabry disease, COL4A1 syndrome, Sneddon syndrome, and Moyamoya arteriopathy. These rare conditions contribute to a portion of strokes that often remain undiagnosed due to challenges in recognition by clinicians. The study aims to better understand the clinical features and natural progression of these diseases and to improve diagnosis and care through a large Italian network, addressing the limited knowledge and geographical disparities in expertise across Italy. The study does not specify particular interventions but involves creating a clinical and research network to empower diagnostic pathways for rCVDs. This network will help gather detailed clinical and genetic data from patients diagnosed with these rare conditions, who have undergone at least one brain MRI study. The initiative seeks to enhance diagnostic accuracy, share knowledge, and support appropriate management including genetic counseling. Participants will be monitored for up to 12 months to describe their phenotypic characteristics and observe the natural history of their disease. Evaluations include clinical, genetic, and neuroradiological assessments based on existing diagnoses. The study supports improved patient management through better understanding of disease features and progression, aiming to fill gaps in diagnosis and care, especially for patients in Southern Italy.

Researchers are evaluating the long-term clinical and instrumental response to Cardiac Contractility Modulation (CCM) therapy in adults with symptomatic heart failure caused by systolic left ventricular dysfunction, despite receiving optimal medical treatment. This observational cohort study includes both retrospective and prospective aspects. It aims to determine the proportion of patients who experience improvement in their New York Heart Association (NYHA) class by at least one level after 12 months, reductions in hospitalizations and emergency visits, changes in quality of life using the Minnesota Living with Heart Failure Questionnaire, walking distance improvements, and differences in NT-proBNP levels. Participants either already have or will receive the CCM device called "OPTIMIZER Smart Mini," which is implanted with leads in the heart's right ventricle and optionally the right atrium. This programmable implantable pulse generator delivers electrical impulses to improve heart function. The device is intended for patients over 18 years with symptomatic heart failure and reduced left ventricular function who have not responded sufficiently to medical therapy. The study evaluates responses following implantation, guided by preimplantation low-dose Dobutamine stress echocardiography. During the study, participants' clinical status and heart function are monitored through echocardiographic tests, quality-of-life questionnaires, and walking tests over a 12-month follow-up. Researchers track hospitalizations, emergency visits, and biomarker levels as well. The primary outcome is the change in NYHA class from enrollment to study end. Safety and effectiveness data are collected as part of participants' routine medical care, with the goal of better understanding who benefits most from CCM therapy.