Cefalu

Researchers are studying the safety and effects of VHB937 in people with early Alzheimers disease, including those with Mild Cognitive Impairment due to Alzheimers or mild Alzheimers itself. This randomized, double-blind, placebo-controlled Phase II trial aims to evaluate whether VHB937 can benefit memory, thinking abilities, daily functioning, and brain changes. The study also looks at how the body processes VHB937 and responds to it. Participants receive intravenous infusions of either a low dose or high dose of VHB937, or a placebo, over a 72-week double-blind period. After this, an extension phase follows for further observation. The treatments are given through infusions, and participants are randomly assigned to one of the three groups in parallel. Throughout the study, participants and their study partners attend regular visits for assessments including clinical dementia rating scales, cognitive tests, daily living activities evaluation, and brain imaging biomarkers. Safety is monitored by tracking adverse events and serious adverse events. Blood samples are collected to measure VHB937 levels and immune responses. The total study duration includes the 72-week treatment period plus additional time in the extension phase.

Researchers are evaluating nipocalimab, a drug given intravenously or subcutaneously, for adults with generalized myasthenia gravis gMG, a condition causing muscle weakness. This Phase 3 study aims to compare the effectiveness and safety of nipocalimab against a placebo and to understand how the drug works in the body when given by injection under the skin versus through a vein. The study includes a subcutaneous substudy to explore these differences in delivery methods. Participants receive nipocalimab intravenous infusions every two weeks for up to 24 weeks during a double-blind placebo-controlled phase. Those completing this phase can enter an open-label extension, choosing to continue receiving intravenous nipocalimab or switch to the subcutaneous form given weekly for eight weeks, with the option to continue longer in a long-term extension. Placebo is also given intravenously every two weeks in the control group during the double-blind phase. Throughout the study, participants undergo regular assessments including muscle function scores, quality of life evaluations, and blood tests measuring antibody levels and drug concentration. Researchers monitor safety by tracking adverse events and changes in clinical and laboratory parameters. The study measures changes in activities of daily living related to myasthenia gravis over time. Participation may last several years, as long-term monitoring continues after the initial treatment phases to evaluate ongoing effects and safety.

Generalized myasthenia gravis gMG is an autoimmune disorder that causes muscle weakness due to autoantibodies affecting nerve-to-muscle communication. This research evaluates the safety and effectiveness of telitacicept, a drug designed to target immune system proteins involved in the disease. The study is a Phase 3, randomized, double-blind, placebo-controlled trial with an open-label extension to further assess telitacicepts impact on gMG symptoms. Participants receive either telitacicept or a placebo through subcutaneous injections during the 24-week double-blind treatment period. Afterward, eligible participants may continue in a 48-week open-label extension where all receive telitacicept, followed by a variable extended open-label period until telitacicept is approved or further development ends. The study includes a 4-week screening phase before treatment and an 8-week follow-up after treatment completion. Throughout the trial, participants undergo assessments including muscle strength and daily living activity scores to measure treatment effects. Researchers monitor safety, quality of life, and muscle function using tools like the Myasthenia Gravis-Activities of Daily Living MG-ADL and Quantitative Myasthenia Gravis QMG scores. Study visits and evaluations track progress over the treatment and extension phases, with a total study duration depending on the participants time in the extended open-label period.

Researchers are evaluating ACP-204, a drug targeting serotonin receptor subtype 2A, in adults aged 55 to 95 years with psychosis related to Alzheimers disease. This master protocol includes three independent, randomized, double-blind, placebo-controlled studies aimed at assessing the drugs efficacy and dose response. Substudy 1 is a Phase 2 trial comparing two doses of ACP-204 30 mg and 60 mg to placebo, followed by two Phase 3 confirmatory studies Substudies 2A and 2B that will independently evaluate the doses or a selected dose against placebo. Participants will undergo a screening period lasting up to 49 days before starting a six-week double-blind treatment phase where they will receive daily doses of ACP-204 or placebo, taken once a day at about the same time with or without food. After treatment, there is a 30-day safety follow-up for those not entering an open-label extension, and vital status follow-up for those who end the study early. Each substudy is analyzed separately to assess the drugs impact. During the trial, participants are closely monitored with assessments including the Scale for the Assessment of Positive Symptoms-Hallucinations and Delusions SAPS-HD from baseline to week 6. Other evaluations include clinical global impression scales and safety monitoring. Participants must have a study partner or caregiver to assist with visits and adherence. The study is designed to track changes in psychosis symptoms and overall safety through the treatment and follow-up periods.

Researchers are studying the effects of a nutritional intervention combining ketogenic medium-chain triglycerides kMCT and B-vitamins on cognitive function in older adults diagnosed with mild cognitive impairment MCI. This randomized, double-blind, placebo-controlled trial aims to evaluate how this nutritional approach impacts cognitive performance over a 12-month period. The study is sponsored by Socit des Produits Nestl and involves multiple centers and countries. Participants will be randomly assigned to receive either BrainXpert, a dietary supplement containing 15 g of kMCT and B-vitamins in powder form, or a placebo consisting of a calorie-equivalent, non-ketogenic high-oleic acid sunflower oil powder. Both are provided in sachetstickpack format without preservatives, flavors, sweeteners, or colorants. The intervention is administered daily during the study period. During the trial, participants and their informants will attend clinic visits at baseline, 12 months, and 18 months. Cognitive function will be assessed using tools such as the Preclinical Alzheimers Cognitive Composite PACC, Montreal Cognitive Assessment MoCA, Wechsler Memory Scale, and Mini-Mental State Examination MMSE. Safety and tolerability will also be monitored throughout the 12 months. The study includes adherence assessments and requires participants to comply with study procedures and medication restrictions.

Cerebrovascular diseases CVDs are a major cause of illness and death worldwide, with over 30% of strokes having unknown causes. Rare cerebrovascular diseases rCVDs, including inherited conditions like CADASIL, COL4A1 syndrome, and Fabry disease, as well as acquired ones like Sneddon syndrome and Moyamoya arteriopathy, contribute to some of these strokes. These rare diseases are often misdiagnosed due to difficulty in recognition, yet identifying them is crucial for proper management, genetic counseling, and potential therapy. Currently, diagnosis and care are limited to a few specialized centers, mainly in northern Italy, causing patients to travel long distances for care. This observational study aims to create a clinical and research network across Italy to improve diagnosis and understanding of rCVDs, especially in southern regions. The study will describe the clinical features and natural course of these diseases during the first 12 months. Later, between 12 and 30 months, it will explore molecular mechanisms, identify biomarkers, and work towards better clinical and therapeutic management. Participants diagnosed with rCVDs such as CADASIL, Fabry disease, COL4A1, Sneddon syndrome, or Moyamoya arteriopathy who have had at least one brain MRI will be included. Researchers will collect clinical, genetic, and neuroradiological data to characterize these patients and monitor their disease progression. The study does not involve treatments but focuses on gathering information to improve diagnosis and care. Participation may last up to 30 months with ongoing assessments and data analysis.

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 adequate medical treatment. This observational cohort study, both retrospective and prospective, focuses on subjects already receiving CCM as part of their regular care. The study aims to assess clinical response, hospitalizations, quality of life, walking distance, and changes in heart biomarkers over 12 months. Participants carry the OPTIMIZER Smart Mini CCM device, a programmable implantable pulse generator connected to leads in the heart. The study groups are defined by their response to low-dose dobutamine stress echocardiography before implantation, with one group showing a reduction of 15% or more in left ventricular end-systolic volume LVESV and the other less than 15%. The low-dose dobutamine test involves incremental infusions of the drug to assess heart contractile reserve. During the 12-month follow-up, researchers will monitor changes in New York Heart Association NYHA class, hospitalizations, quality of life using the Minnesota Living with Heart Failure Questionnaire, walking test distance, and NT-proBNP biomarker levels. Additional assessments include echocardiographic measures and recording adverse events, cardiac device implants, or surgery. This comprehensive monitoring helps evaluate the clinical and functional effects of CCM therapy over time.