Pordenone

Researchers are evaluating the effects of pelacarsen (TQJ230), given as a monthly injection under the skin, in people with mild to moderate calcific aortic valve stenosis. This study aims to see if pelacarsen can safely slow the progression of this heart valve condition compared to a placebo. The trial is a phase 2, randomized, double-blind, placebo-controlled study conducted at multiple centers. Participants will receive either pelacarsen 80 mg or a matching placebo once a month. Before starting the treatment, they must have elevated lipoprotein(a) levels and be optimally treated for existing cardiovascular risk factors. The study focuses on those aged 50 to under 80 years with mild or moderate calcific aortic valve stenosis. During the 36 months of participation, researchers will monitor changes in peak aortic jet velocity and aortic valve calcium score to assess disease progression. Safety, tolerability, and the impact of the treatment will be evaluated. Participants will undergo regular assessments, including laboratory tests and clinical evaluations, to track heart valve condition and overall health throughout the study.

Researchers are investigating the effectiveness, safety, and tolerability of combining baxdrostat with dapagliflozin compared to dapagliflozin alone in people with chronic kidney disease (CKD) and high blood pressure. This Phase III, international, multicenter, double-blind, placebo-controlled study aims to see if this combination reduces risks such as significant kidney function decline, kidney failure, heart failure events, or cardiovascular death. The study includes a 4-week run-in period where participants not previously treated with SGLT2 inhibitors receive dapagliflozin alone. After this, participants are randomly assigned to receive either baxdrostat plus dapagliflozin or placebo plus dapagliflozin in a double-blinded manner. Study visits occur frequently initially (at 2, 4, 8, 16, 34, and 52 weeks after randomization) and then approximately every 4 months. If participants stop the blinded treatment early, they continue dapagliflozin alone unless specific criteria require its discontinuation. Participants will undergo regular assessments including blood pressure monitoring and laboratory tests related to kidney function and cardiovascular health. The primary outcome measures the reduction in risk of major kidney and heart events over up to 37 months. Even if participants stop the study treatment, they will continue follow-up visits and data collection to ensure comprehensive safety and efficacy evaluation throughout the study duration.

Researchers are evaluating AZD0780, an oral PCSK9 inhibitor, in a phase 3, randomized, placebo-controlled study to see if it can reduce the risk of major adverse cardiovascular events (MACE-PLUS) in adults with established atherosclerotic cardiovascular disease (ASCVD) or those at high risk for a first ASCVD event. The study compares AZD0780 to a placebo and monitors participants from randomization until the primary analysis censoring date, followed by a final study closure visit. Participants will be randomly assigned to receive either oral AZD0780 or an oral placebo once daily. The treatment period lasts until the primary analysis censoring date, after which a study closure visit will occur. The study is event-driven and designed to assess the time to the first major cardiovascular event during treatment. During the study, participants will be closely monitored with various assessments to evaluate cardiovascular outcomes and safety over approximately 54 months. Researchers will track the time to first event of any component of MACE-PLUS and collect data to assess the effect of AZD0780 compared to placebo. The study includes regular visits and evaluations to ensure participant safety and adherence to treatment.

Researchers are evaluating repotrectinib in adults and adolescents with advanced or metastatic solid tumors that have specific gene rearrangements (ALK, ROS1, NTRK1, NTRK2, or NTRK3). The study includes a Phase 1 dose escalation to determine safe dosage levels and side effects, and a Phase 2 expansion to assess the drug's overall response rate in different patient groups. The trial also investigates the drug's effect on liver enzymes in a substudy and measures outcomes like duration of response and survival. The treatment involves oral repotrectinib capsules given to participants. Phase 1 focuses on finding the maximum tolerated dose and recommended dose for Phase 2 by monitoring toxicities within 28 days of dosing. Phase 2 enrolls subjects into six groups based on prior treatments and tumor gene status, including those who are new to ROS1 or TRK targeted therapies and those who have received previous therapies. Each group receives repotrectinib to evaluate its effects in their specific conditions. Participants will undergo assessments including tumor measurements by imaging reviewed independently, laboratory tests, and monitoring for side effects. Researchers will track dose-limiting toxicities, response rates over two to three years, and survival outcomes. Safety and tolerability are closely observed during treatment. The study requires ongoing follow-up, including confirmation of tumor gene status and evaluation of response and progression over time.

Researchers are studying patients with early to locally advanced stages (I-III) of non-small cell lung cancer (NSCLC) to better understand how diagnosis and treatments are managed in real-world settings. Because patients with these stages of NSCLC can vary widely, there is no single standard treatment plan, and practices differ by country and medical center. This study looks closely at how surgery, chemotherapy, and radiotherapy are timed and combined for these patients without interfering with their treatment. This is a non-interventional, retrospective observational study analyzing medical records of patients with stage I-III NSCLC who received curative surgery or radiotherapy between January 2018 and June 2019. Researchers will examine diagnostic and treatment paths, focusing on the use and timing of therapies. The study does not involve new treatments or interventions but reviews past patient data to gather insights. Participants' medical records from January 2018 to January 2021 will be reviewed to assess treatment approaches, discussions in multidisciplinary teams, healthcare resource use, and direct medical costs related to NSCLC care. The study involves collecting data from available charts and follow-up information to understand treatment patterns and outcomes better. No active treatment or patient visits are required as this study relies on existing data.

Researchers are evaluating whether ziltivekimab can help people who were hospitalized due to a heart attack by potentially reducing the development of heart disease and preventing new heart attacks or strokes. This Phase 3 study compares ziltivekimab with a placebo, which is a dummy medicine that has no effect on the body. Both treatments are given by chance, with equal likelihood for participants to receive either ziltivekimab or placebo. Participants will inject the study medicine once a month under the skin in the stomach, thigh, or upper arm. Ziltivekimab is given as an initial loading dose followed by monthly maintenance doses. The placebo group receives a matching injection schedule. The study duration is about two years. During the study, researchers will monitor participants for the time until the first serious heart-related event, including cardiovascular death, non-fatal heart attack, or non-fatal stroke. Participants will be closely observed from the start of randomization up to 25 months. The study includes regular follow-ups to assess safety and effectiveness of the treatments throughout this period.

Researchers are evaluating whether ventilation with argon gas combined with oxygen can help reduce brain injury after cardiac arrest in patients who have been successfully resuscitated. This phase II, multicenter, randomized, controlled, and single-blinded trial focuses on patients who suffered an out-of-hospital cardiac arrest of presumed cardiac cause with shockable rhythm and remained unconscious after return of spontaneous circulation. Earlier studies in animals have shown argon may protect brain and heart cells, reduce damage size, and speed recovery, and initial human safety data from early phases were reassuring. Participants will receive ventilation with a gas mixture of 70% argon and 30% oxygen using an experimental mechanical ventilator during the study treatment period lasting four hours. All patients will receive standard care according to European resuscitation guidelines, and randomization ensures balanced comparison for safety and efficacy outcomes. The study assesses effects during the treatment and continues monitoring for up to six months to track related clinical events. During the study, patients will be closely monitored including neurological assessments to measure brain cell preservation 48 hours after treatment. Safety data will be collected and evaluated by a blinded events committee. The follow-up period extends to six months after the intervention to observe longer-term outcomes. The total participation involves initial intensive care admission, the four-hour treatment window, and the extended follow-up period to capture neurological and clinical effects.

This research focuses on invasive bacterial diseases caused by Streptococcus pneumoniae, Neisseria meningitidis, and Haemophilus influenzae, which lead to severe health problems and frequent serious complications. The study aims to understand the epidemiology of these infections, including the distribution of bacterial strains, to better plan prevention and treatment strategies. It highlights the importance of monitoring these infections to guide public health actions, detect vaccine failures, and assess the impact of vaccination programs. The study uses molecular diagnostic testing on blood samples to identify the pathogens, even when traditional culture methods fail due to prior antibiotic use or sample handling issues. Molecular diagnosis allows for more accurate detection of these bacteria regardless of their viability in samples. This testing is conducted on patients from hospitals within and outside the Tuscany region as part of ongoing surveillance. Participants include both pediatric and adult patients diagnosed with invasive bacterial diseases caused by these bacteria. Researchers will track the incidence rates of infections caused by each pathogen over about one year. The study involves collecting biological samples for molecular testing and monitoring the presence and types of bacteria. This will help improve understanding of infection trends and support the development of better prevention and treatment approaches.

Researchers are investigating the effects of pelacarsen (TQJ230) compared to a placebo in adults with atherosclerotic cardiovascular disease (ASCVD) who have high levels of lipoprotein(a) (Lp(a)) and are also receiving inclisiran treatment for elevated low-density lipoprotein cholesterol (LDL-C). The study is designed as a Phase 3 randomized, double-blind, placebo-controlled, multicenter trial with a parallel group structure, followed by an open-label treatment period. The aim is to assess the efficacy, safety, and tolerability of pelacarsen in this population. Participants will receive pelacarsen or placebo as a solution for subcutaneous injection using prefilled syringes. All participants will be given background treatment with inclisiran, starting with two loading doses spaced three months apart during the run-in period. Afterward, inclisiran will be administered every six months at Month 5 and Month 11. Following the double-blind phase, an open-label treatment period will continue, allowing further evaluation of the treatments. Throughout the study, participants will undergo assessments including measurement of lipoprotein(a) levels, with the primary outcome focusing on change in log-transformed Lp(a) concentration from baseline to six months. Laboratory tests will monitor LDL-C and other relevant markers. Safety and tolerability will be tracked continually, and standard care for cardiovascular risk factors such as hypertension and diabetes will be maintained. The study includes adults aged 18 to 80 years with established ASCVD and elevated lipid levels, ensuring ongoing monitoring and evaluation of treatment effects.

Acute intoxications are a significant public health concern, especially in children who are more vulnerable and at higher risk for unintentional and preventable poisonings. This research aims to study the patterns and social and care-related factors of acute intoxications in children to improve diagnosis and treatment approaches nationally. The study is a prospective, non-profit, multicenter observational cohort focusing on acute intoxications in pediatric patients, conducted by AMIETOX at Poison Control Centres and pediatric emergency rooms. The study observes children aged from 1 month up to 16 years who have experienced acute intoxication, defined as exposure to toxic substances or harmful amounts of substances via unintended routes. There are no specific treatments or interventions administered as it is an observational study. Participants are identified through visits or telephone contacts to participating centers. The study spans an average duration of one year to gather incidence and prevalence data. Participants will be monitored throughout the study to collect data on the occurrence and characteristics of acute intoxications. Researchers will assess the incidence and prevalence of intoxication in pediatric patients during the study period. Data collection includes social and care factors related to each case. The study ensures informed consent is acquired before including any child. Safety and follow-up are integral to the observational process to understand and eventually improve management of acute intoxications in children.