Cerqueira Cesar

Transthyretin amyloidosis ATTR is a condition where the transthyretin protein breaks down and forms amyloid plaques that build up in organs, causing damage. There are two types wild-type ATTR, which occurs with aging, and variant ATTR caused by inherited defective TTR genes. This study focuses on adults who have inherited a pathogenic TTR gene variant but do not yet show symptoms, aiming to see if early treatment can prevent or delay disease development. The trial is a Phase 3, randomized, double-blind, placebo-controlled study evaluating prevention of ATTR cardiomyopathy and polyneuropathy. Participants will be randomly assigned to receive either acoramidis, a drug designed to stabilize the TTR protein and prevent amyloid formation, or a matching placebo. The study drug is taken orally twice daily at a dose equivalent to 800 mg acoramidis HCl BID. Participants will be adults aged 18 to 75 years whose age falls within 10 years younger or older than their predicted age of disease onset based on family history or actuarial tables. The trial will last approximately 7 years from randomization or until study end. During the study, participants will be monitored regularly with evaluations to detect the development of ATTR cardiomyopathy or polyneuropathy. Researchers will assess time to disease onset using centrally adjudicated criteria. Safety and adherence will be tracked throughout the trial. This long-term study will help determine if early treatment with acoramidis can delay or prevent symptoms in asymptomatic carriers of pathogenic TTR variants.

This research aims to compare the effects of oral anticoagulation versus no anticoagulation in patients aged 55 and older who have had recent episodes of transient atrial fibrillation triggered by stress and have additional factors that increase their risk of stroke. The study is a multinational, investigator-initiated, prospective, randomized, open-label trial evaluating the impact of non-vitamin K oral anticoagulants NOACs on preventing stroke and other serious cardiovascular events. Participants will be randomly assigned to either receive one of several NOACs, such as edoxaban, apixaban, dabigatran, or rivaroxaban, prescribed by their physician for the duration of the follow-up, or to a control group that will not receive anticoagulants unless medically indicated during the study. The choice of specific NOAC and dose adjustments are determined by the prescribing doctor. The study excludes patients with certain conditions like chronic atrial fibrillation, severe kidney or liver disease, recent cardiac surgery, or bleeding risks. During the study, participants will be monitored for up to 24 months to assess the occurrence of non-hemorrhagic stroke, systemic embolism, vascular mortality, myocardial infarction, peripheral arterial thrombosis, amputation, and venous thromboembolism. Regular follow-up will include evaluation of these outcomes to measure the effectiveness and safety of anticoagulation compared to no anticoagulation. Participants will provide informed consent and remain under medical supervision throughout the trial.

Researchers are evaluating whether survodutide can help adults with liver diseases called non-alcoholic steatohepatitis NASH or metabolic-associated steatohepatitis MASH who have cirrhosis and a body mass index BMI of 27 kgm2 or higher 25 kgm2 for Asian participants. The study compares survodutide to a placebo to see if it improves liver function and related health outcomes over time. This is a Phase III trial with participants randomly assigned to groups, and it is double-blind, meaning neither participants nor doctors know who gets the medicine or placebo. Participants receive weekly injections of survodutide or placebo under the skin and get regular counseling on diet and exercise. The study lasts up to four and a half years, with visits either in person or via video call every 2, 4, or 6 weeks for about 17 months, then every 3 months thereafter until the study ends. The study collects health data including body weight, liver imaging results, and symptom questionnaires to assess the treatment effects. During the study, doctors monitor participants health and record any side effects. Liver health is checked using imaging methods at certain visits, and participants fill out questionnaires about their symptoms. The primary outcome measures include time to serious liver-related events and overall survival. Secondary outcomes look at changes in liver fibrosis, body weight, blood sugar control, liver stiffness, and other blood markers. The study aims to provide detailed long-term information on survodutides impact on liver disease and safety.

Researchers are evaluating survodutide, a medicine given by weekly injection, in adults aged 18 and older who have obesity and a liver disease called non-alcoholic steatohepatitis NASH or metabolic associated steatohepatitis MASH with moderate or advanced liver fibrosis. The study aims to see if survodutide can improve liver function and slow disease progression. This Phase III trial compares survodutide to a placebo, with participants randomly assigned to one of the two groups, and neither participants nor doctors know who receives which treatment. Participants inject survodutide or placebo under their skin once a week, with doses gradually increasing to a target level. All participants also receive counseling to encourage diet changes and regular exercise. The study has two parts the first focuses on the effect of survodutide on liver fibrosis and MASH over 52 weeks, and the second assesses long-term safety and effectiveness up to 7 years. Participants are involved for up to 7 years, with visits to the study site or remote video calls starting every 2 weeks, then every 4 and 6 weeks, and eventually alternating every 3 months. During visits, doctors monitor health, weight, and digestive effects, perform liver imaging, and collect liver tissue samples at select times. Participants complete questionnaires about symptoms and quality of life. Researchers measure changes in liver disease markers, body weight, blood tests, and monitor safety and serious outcomes like progression to cirrhosis or liver-related events.

This research aims to evaluate the use of a lower international normalized ratio INR target range of 1.5 to 2.5 in patients with a mechanical bileaflet heart valve placed in the aortic position. The study seeks to determine whether lowering the INR target can reduce bleeding risks without increasing the chance of blood clots or stroke. This is important because patients with mechanical heart valves require lifelong warfarin Coumadin treatment to prevent clot formation, but managing the right balance between clot prevention and bleeding risk remains challenging. Participants will be randomly assigned to one of two groups. One group will receive warfarin therapy adjusted to a lower INR target range of 1.5 to 2.5, while the other group will have warfarin adjusted to a standard INR target range as currently recommended. Both groups will receive warfarin after their mechanical valve implant, but the INR targets for blood thinning will differ. The study includes follow-up over an expected mean of 2 to 3 years to monitor outcomes. During the study, participants will be regularly monitored for blood clot events such as thrombosis or stroke, as well as bleeding episodes, including major and minor bleeding. Researchers will track various outcomes like mortality, different types of stroke, heart attacks, pulmonary embolism, and kidney function over the study period. Time in the therapeutic INR range and occurrences of extreme INR values will also be recorded. Participants will be followed through the study duration, which will help determine the safety and risk balance of using a lower INR target in this patient group.

Researchers are evaluating futibatinib in patients with advanced cholangiocarcinoma CCA that have FGFR2 gene fusions or rearrangements. This open-label, multinational Phase 2 study aims to confirm the clinical benefit of a 20 mg dose and assess the safety and effectiveness of a 16 mg dose in patients who have already undergone prior treatment. The study compares these two doses to better understand their impact on this specific type of cancer. Participants will be randomly assigned to one of two groups receiving futibatinib orally either 16 mg or 20 mg daily in repeated 21-day cycles. Treatment continues until disease progression or other withdrawal criteria occur. Both doses involve taking tablets daily with continuous monitoring throughout the treatment period. During the study, participants will undergo regular assessments including imaging scans to measure tumor response, evaluation of disease progression, overall survival, and quality of life questionnaires. Safety is monitored through adverse event reporting. The main outcome is the objective response rate reviewed independently 12 months after study completion, with additional measures such as duration of response and progression-free survival also evaluated. The study continues until December 2026.

This research aims to better understand congenital myotonic dystrophy CDM and childhood myotonic dystrophy ChDM, which are inherited disorders affecting multiple body systems in children. CDM appears at birth with serious symptoms like breathing problems and feeding difficulties, while ChDM begins after birth but before age 10 with similar but generally less severe effects. The study seeks to develop better clinical assessments and biomarkers for these conditions to aid future drug trials, especially since current adult trials cannot be directly applied to children due to differences in symptoms and disease progression. The study will enroll up to 200 children with either CDM or ChDM. No treatments are given as part of this study participants will continue to receive their usual medical care. Study visits will take place at the start of the study Baseline, and then again at 12 months and 24 months. During these visits, researchers will perform various physical and cognitive tests to track changes in mobility, muscle strength, language, sleepiness, behavior, and quality of life. Participants will be involved in detailed assessments including walking tests, motor function evaluations, hand strength measurements, and questionnaires on sleepiness and quality of life. Researchers will also monitor language abilities, repetitive behaviors, and adaptive skills. These evaluations will help measure changes over 24 months and improve the tools used in clinical trials for these diseases. The study is sponsored by Virginia Commonwealth University and is expected to continue through June 2030.