Maceio

Researchers are evaluating the safety and effects of a new medicine called NNC0487-0111 in people who have Heart Failure with preserved Ejection Fraction HFpEF or Heart Failure with mildly reduced Ejection Fraction HFmrEF and excess body weight. This phase 3 clinical trial aims to find out if NNC0487-0111 is safe and effective for treating these conditions compared to a placebo. Participants have HFpEF or HFmrEF and a body mass index of 30 or above. The study is sponsored by Novo Nordisk AS and uses a randomized, quadruple-masked design. Participants will receive either NNC0487-0111 or a matching placebo by injection under the skin once a week. The NNC0487-0111 is given in increasing doses over time. The study is parallel in design, meaning participants are randomly assigned to one of the two groups and receive that treatment throughout the trial. This treatment period extends for up to about 165 weeks. The study evaluates the time to certain heart failure events, hospitalizations, cardiovascular deaths, and other major cardiovascular events. During the study, participants will be monitored regularly to assess heart failure outcomes and kidney function, as well as quality of life using questionnaires like the Kansas City Cardiomyopathy Questionnaire. Safety and effectiveness are assessed through hospital visits, heart failure event tracking, and blood tests including kidney function and blood sugar levels. The total participation spans over three years, with ongoing evaluations to measure the time to heart failure events and cardiovascular outcomes. Participants receive close medical monitoring throughout the study period.

Researchers are evaluating finerenone compared to a placebo to assess the effectiveness and safety of treatment in patients with heart failure and reduced ejection fraction HFrEF who cannot tolerate or are not eligible for steroidal mineralocorticoid receptor antagonists sMRA. This international study is a randomized, double-blind, placebo-controlled trial focused on this specific group of heart failure patients. Participants will be randomly assigned to receive either oral finerenone or a matching placebo. The study uses a parallel design and treatment will be monitored for up to about 30 months. During this time, researchers will track cardiovascular events, heart failure events, and any serious or adverse events leading to discontinuation of the study drug. Throughout the study, participants will undergo regular assessments including symptom questionnaires and monitoring for cardiovascular outcomes and safety. The main outcomes include the time to the first cardiovascular death or heart failure event and the number of serious adverse events. The study also tracks changes in symptom scores over six months and overall survival. Participants will be followed closely during treatment and after to understand both efficacy and safety.

Researchers are evaluating zongertinib, an oral drug targeting HER2 mutations, to see if it can improve outcomes compared with standard adjuvant treatments in adults who have had complete surgical removal of Stage II-IIIB non-small cell lung cancer NSCLC with activating HER2 tyrosine kinase domain mutations. This Phase 3, global, randomized, open-label study involves participants who have already received appropriate perioperative systemic therapy, including neoadjuvant or adjuvant platinum-based chemotherapy, with or without immunotherapy. Participants are randomly assigned in equal numbers to receive either zongertinib once daily by mouth for up to 3 years or standard treatment, which may include approved adjuvant immunotherapy or observation depending on local guidelines and patient status. The study compares disease-free survival between these groups and also monitors safety and patient-reported outcomes. Tumor samples are collected centrally to confirm HER2 status. During the study, participants undergo regular assessments to monitor disease status, safety, and quality of life. The primary outcome is disease-free survival, tracked for up to 8 years and 5 months. Secondary outcomes include overall survival and occurrence of significant adverse events over up to 3 years. Participant involvement may last several years, with follow-up continuing after treatment to assess long-term outcomes and safety.

Researchers are evaluating the effects of a medicine called BI 690517 combined with empagliflozin in adults with chronic kidney disease CKD who are at risk of their kidney condition getting worse. The study includes people with or without type 2 diabetes and those who may already be taking medicines like angiotensin converting enzyme inhibitors ACEi, angiotensin receptor blockers ARB, or sodium-glucose cotransporter-2 inhibitors SGLT2i. The goal is to understand if adding BI 690517 can help delay worsening kidney function, hospitalizations due to heart failure, or cardiovascular death. After a run-in period where all participants take empagliflozin and other standard medications, participants are randomly assigned to receive either BI 690517 tablets or placebo tablets once daily alongside empagliflozin. The run-in period confirms that participants are stabilized on empagliflozin before randomization. The treatment phase continues for about three to four years until enough kidney or heart-related events have occurred to compare outcomes between the two groups. During the study, participants visit the study site about five times in the first six months and then every six months thereafter. At these visits, health is regularly checked through blood and urine tests, blood pressure and weight measurements, kidney function monitoring, and collection of any side effect information. The main outcome measured is the time until the first occurrence of kidney disease progression, hospitalization for heart failure, or cardiovascular death.

Researchers are evaluating the effects of vicadrostat combined with empagliflozin in adults who have type 2 diabetes, high blood pressure, and cardiovascular disease but no history of heart failure. The study aims to assess whether this combination can help reduce cardiovascular risks compared to a placebo with empagliflozin. This Phase III trial involves adults with these conditions who are already receiving treatment for them. Participants are randomly assigned to one of two groups. One group takes vicadrostat and empagliflozin tablets daily, while the other group takes placebo tablets that look like vicadrostat but have no active medicine, alongside empagliflozin. Treatment lasts from two and a half years up to four years and three months. All participants continue their usual medications for diabetes, blood pressure, and heart disease during the study. Throughout the study, lasting up to four years and three months, participants visit the study site regularly for health checks and blood samples. Doctors monitor cardiovascular events and any side effects experienced. The main outcome measured is the time until the first cardiovascular death or heart failure event. Other health indicators like blood pressure and kidney function are also tracked to understand the effects of the treatment combination.

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.

Researchers are evaluating the safety and effectiveness of LY4268989 combined with mirikizumab compared to mirikizumab alone in adults with moderately to severely active ulcerative colitis. This phase 2 study aims to understand if the combination can better manage the condition. The study is sponsored by Eli Lilly and Company and involves a randomized, double-blind design with parallel groups. Participants receive either LY4268989 orally along with mirikizumab given intravenously and then subcutaneously, or mirikizumab with a placebo pill matching LY4268989. Responders to treatment will be re-randomized for a second study period. Treatment lasts approximately 104 weeks with up to 21 visits planned throughout the study. During the study, participants undergo assessments including clinical remission evaluation using the modified Mayo Score at weeks 12, 24, and 48, endoscopic improvement, and symptomatic remission measurements. Safety and response are closely monitored through scheduled visits. Overall participation lasts about 118 weeks, allowing researchers to gather long-term data on the treatments being studied.

Researchers are developing a registry of Brazilian patients with hereditary cardiovascular diseases by combining clinical information and genetic data. The study aims to identify which genes are most commonly affected and the frequency of these genetic changes within this population. The registry will help advance understanding and healthcare for hereditary heart conditions in Brazils public health system. Participants will be interviewed during their routine medical visits, and their DNA will be collected using buccal swabs for whole genome sequencing. This observational study does not involve any treatment but focuses on gathering detailed genetic and clinical data to better characterize hereditary cardiovascular diseases. The study is planned to run for 30 months starting from April 2025. During the study, participants will provide information through interviews and give DNA samples for sequencing. Researchers will measure diagnostic yield, genetic diversity, and variant frequency over the 30 months. The study does not involve additional interventions beyond data and sample collection, and participants will continue their usual medical care throughout the study period.

Researchers are investigating the effects of a polypill combined with colchicine in people with established atherosclerotic cardiovascular disease ASCVD, a major cause of illness and death worldwide, including Brazil. The trial aims to assess whether this combination can better prevent heart and blood vessel events compared to usual care. The study addresses the need for improved control of cardiovascular risk factors and adherence to treatments in a diverse population. Participants will be randomly assigned to one of four groups cardiovascular polypill plus colchicine, cardiovascular polypill plus placebo, usual care plus colchicine, or usual care plus placebo. The polypill contains fixed doses of valsartan, atorvastatin, and aspirin, taken daily. Colchicine or its placebo is given once daily. The study will last about three years on average, with the goal of evaluating the combined effects of these treatments on cardiovascular health. During the study, participants will be monitored for major adverse cardiovascular and limb events, including heart attacks, strokes, and cardiovascular deaths. Researchers will collect data throughout the trial to assess treatment effects and safety. The study design includes a randomized, quadruple-masked approach to ensure unbiased results. Participants can expect regular evaluations over the study period, which ends around May 2031.

Researchers are conducting a phase III, randomized, multi-center clinical trial to evaluate whether intravenous Tenecteplase TNK treatment is better than a placebo for patients who have suffered a non-large vessel occlusion ischemic stroke between 4.5 and 12 hours after they were last seen well. The study focuses on patients with evidence of salvageable brain tissue or a clinical-radiological mismatch on perfusion imaging. The goal is to determine if TNK can improve functional outcomes compared to placebo. Participants will be randomly assigned in a 11 ratio to receive either a single intravenous bolus injection of Tenecteplase at a dose of 0.25 mgkg maximum 25 mg or a placebo bolus injection over 5 seconds. The study uses a double-blind design, and randomization considers factors such as age, stroke severity, and timing since last seen well. The planned enrollment is 466 participants, split evenly between the TNK and placebo groups, with interim analyses to assess study progress. During the 90-day follow-up, participants will undergo assessments including modified Rankin Scale scoring to evaluate functional outcomes, brain imaging to measure infarct volume and reperfusion, and quality of life questionnaires at 3 months, 6 months, and one year. Safety outcomes like intracranial hemorrhage and mortality rates will also be monitored. These evaluations aim to measure the effectiveness and safety of TNK treatment compared to placebo after ischemic stroke.