Cerqueira Cesar

Researchers are evaluating the safety and effectiveness of zodasiran injections in adolescents and adults aged 12 years and older who have genetically or clinically diagnosed Homozygous Familial Hypercholesterolemia (HoFH). This phase 3, randomized, placebo-controlled study aims to understand how the treatment affects levels of LDL cholesterol, a harmful type of cholesterol, in the blood over time. Participants receive either zodasiran or a placebo through subcutaneous injections during the double-blind treatment period. After completing this phase, those who wish can enter an optional open-label extension where all placebo participants have the chance to switch to the active drug. The study involves ongoing monitoring to compare the effects of zodasiran versus placebo. Throughout the study, participants will have their LDL cholesterol levels measured at the start and after 12 months to assess changes. Researchers will also monitor safety through regular clinical assessments and laboratory tests. The total duration includes the double-blind period plus the optional extension, providing detailed information on treatment impact and participant health over time.

Researchers are studying the effects of felzartamab, a laboratory-made antibody, in adults who have received kidney transplants and are experiencing antibody-mediated rejection (AMR). AMR happens when the immune system mistakenly attacks the new kidney, causing damage that can lead to organ failure. This phase 3 trial aims to compare felzartamab to a placebo to see how well it helps treat AMR in kidney transplant patients. The study focuses on how many participants have kidney tissue that looks normal or nearly normal after 24 weeks of treatment. Participants will be randomly assigned to receive up to 9 doses of either felzartamab or a placebo through an intravenous infusion based on their body weight during the first part of the study, which lasts up to 24 weeks and is double-blind. In the second part, lasting up to 28 weeks, all participants will receive felzartamab in an open-label phase where everyone knows the treatment being given. In total, participants will have up to 21 study visits over about one year. During the study, participants will undergo kidney biopsies, blood and urine tests, electrocardiograms, and other assessments to monitor kidney function, immune response, and safety. Researchers will measure how long the transplanted kidney works, changes in antibody levels, signs of rejection, and side effects. The study also monitors how felzartamab is processed by the body and whether participants develop antibodies against the drug. Screening before enrollment can take up to 42 days.

Transthyretin amyloidosis (ATTR) is a condition where the transthyretin (TTR) protein breaks down and forms amyloid plaques that build up in organs, causing damage. This can happen either as people age (wild-type ATTR) or due to inherited defective TTR genes (variant ATTR). When amyloid deposits affect the heart, it leads to transthyretin amyloid cardiomyopathy (ATTR-CM), and when it affects nerves, it causes transthyretin amyloid polyneuropathy (ATTR-PN). Researchers are evaluating acoramidis, a drug designed to stabilize the TTR protein to prevent or delay these conditions in adults who carry a pathogenic TTR gene variant but do not yet show symptoms. The study is a Phase 3, randomized, double-blind, placebo-controlled trial involving asymptomatic adults aged 18 to 75 years who carry a known pathogenic TTR variant. Participants receive either acoramidis or a placebo pill taken orally twice daily. Participants are selected based on their age being within 10 years younger or older than their predicted age of disease onset, which is estimated from family history or published data. The study aims to prevent or delay the development of ATTR-CM or ATTR-PN over approximately 7 years. Participants will be closely monitored throughout the study period for the time to development of ATTR, either cardiomyopathic or polyneuropathic forms, as determined by central adjudication. Assessments include genetic testing confirmation, cardiac magnetic resonance testing, and evaluation for any signs of disease progression. Safety and treatment adherence will also be monitored. The study may last up to 7 years or until it is declared over, with careful follow-up to detect any early signs of disease or treatment effects.

Researchers are evaluating the use of non-vitamin K oral anticoagulants (NOACs) compared to no anticoagulation in people who have experienced transient atrial fibrillation episodes triggered by stress and have additional risk factors for stroke. This multinational, investigator-initiated Phase 4 trial aims to prevent stroke and other serious cardiovascular events in this group by assessing the effects of NOACs on two main outcomes: the occurrence of non-hemorrhagic stroke or systemic embolism, and a combination of vascular death and other major cardiovascular problems, over a follow-up period lasting until the last participant reaches 24 months of observation. Participants in the study are randomly assigned to either receive one of several NOAC medications—edoxaban, apixaban, dabigatran, or rivaroxaban—with dosing adjusted as needed and chosen by their prescribing doctor, or to receive no oral anticoagulation. The treatment continues throughout the follow-up period. The trial is open-label, meaning both researchers and participants know which treatment is given. The study specifically focuses on patients who had transient atrial fibrillation related to stress, such as after certain surgeries or acute medical illness. During the study, participants undergo regular monitoring to track the incidence of stroke, embolism, vascular death, heart attacks, blood clots, and other cardiovascular events. Researchers collect information over up to two years to evaluate these outcomes. Safety and adherence to treatment are also monitored. This thorough follow-up helps determine the impact of NOAC treatment compared to no anticoagulation in this particular patient population.

Researchers are evaluating the effects of survodutide in adults aged 18 years and older who have a confirmed liver condition called non-alcoholic steatohepatitis (NASH) or metabolic-associated steatohepatitis (MASH). Eligible participants must have a body mass index (BMI) of 27 kg/m2 or higher, or at least 25 kg/m2 if they are Asian. The study excludes those with other chronic liver diseases or a history of significant alcohol use. The main goal is to see if survodutide can improve liver function and delay progression of liver damage over time. Participants are randomly assigned to receive either survodutide or a placebo, with twice the chance of receiving survodutide. Both treatments are given as weekly injections under the skin using a pre-filled syringe. Alongside treatment, all participants receive regular counseling to encourage healthy diet and exercise habits. The study lasts up to four and a half years, with frequent visits or remote video calls during the first year and five months, then quarterly visits thereafter. During the study, doctors monitor participants' health, including body weight and liver function using imaging tests at certain visits. Participants complete symptom questionnaires to help assess their condition. Researchers track outcomes such as survival, need for liver transplant, worsening liver disease, and liver-related complications. Safety and any side effects are closely watched throughout the study period to understand the treatment's impact.

Researchers are evaluating the effects of survodutide on adults living with obesity who have a liver disease called non-alcoholic steatohepatitis (NASH) or metabolic associated steatohepatitis (MASH), along with moderate or advanced liver fibrosis. The study focuses on whether survodutide can improve liver function and reduce liver damage in these participants. This Phase III trial aims to assess both the effectiveness and safety of survodutide over a long-term period. Participants are randomly assigned to one of two groups: one receiving weekly injections of survodutide and the other receiving placebo injections that look like the medicine but contain no active drug. The doses of survodutide are gradually increased until the target dose is reached. All participants receive counseling to support healthy diet changes and regular exercise throughout the study. The study lasts up to 7 years, with frequent visits to the study site or remote video calls. In the first year, visits occur every 2 weeks, then every 4 to 6 weeks, and later every 3 months alternating between in-person and remote. Throughout the study, researchers monitor participants' health, liver condition through imaging and biopsies, body weight, digestive system effects, and questionnaires about symptoms and quality of life. The main outcomes include liver fibrosis improvement, resolution of MASH without worsening fibrosis, and long-term safety and efficacy measures.

Researchers are evaluating the use of a lower INR target range (1.5 to 2.5) in patients who have a mechanical bileaflet heart valve in the aortic position. The study aims to determine whether this lower INR target can reduce the risk of bleeding without increasing the risk of blood clot formation or stroke. This is important because patients with mechanical heart valves need lifelong warfarin treatment, and finding the right INR balance could lessen bleeding complications. Participants will continue warfarin therapy after their mechanical valve replacement, but with different INR target ranges depending on their study group. The study compares the standard higher INR targets to the lower 1.5 to 2.5 range to assess safety and effectiveness. This is a Phase 3 clinical trial focusing on patients who had their bileaflet mechanical heart valve implanted at least three months prior. During the study, participants will be monitored for thrombosis or thromboembolism and major bleeding events over a period expected to last 2 to 3 years. Researchers will regularly measure INR levels to guide warfarin dosing and track outcomes related to bleeding and clotting. The results are intended to help doctors better manage warfarin dosing to reduce bleeding risks while preventing clots in this patient group.

Researchers are evaluating the safety and effectiveness of two doses of futibatinib, 16 mg and 20 mg, in patients with advanced cholangiocarcinoma (CCA) that have FGFR2 gene fusions or rearrangements. This Phase 2, open-label, multinational, randomized study focuses on patients who have previously received treatment for their condition. The goal is to confirm the clinical benefit of the 20 mg dose and to assess the safety and efficacy of the 16 mg dose in this patient group. Eligible patients are randomly assigned in a 1:1 ratio to receive either 16 mg or 20 mg of oral futibatinib daily, taken every day in 21-day cycles. Treatment continues until the disease progresses according to RECIST 1.1 criteria or until other withdrawal conditions are met. Futibatinib is an FGFR inhibitor given by mouth, and patients remain on continuous treatment as long as they meet these criteria. During the study, participants undergo regular assessments including radiographic imaging to measure disease progression and response. Researchers monitor safety and efficacy outcomes, including the overall response rate assessed by an independent central review 12 months after study completion. The study also involves evaluations of organ function, performance status, and other health parameters to ensure participant well-being throughout the trial.