Sunderland

Researchers are conducting an observational study to understand the short-term progression of geographic atrophy GA caused by age-related macular degeneration AMD in adults aged 55 years and older. The study aims to identify participants with progressive GA to measure structural and functional changes and explore whether these changes relate to genetic or lifestyle factors. This multi-center research provides insights into disease progression without introducing any treatment interventions. Participants with bilateral GA secondary to AMD will be observed over time. The study involves assessments at baseline, month 3, and month 6 to measure the progression of GA using imaging techniques such as fundus autofluorescence FAF and optical coherence tomography OCT. Researchers will quantify GA lesion size and evaluate visual function parameters, including best-corrected visual acuity BCVA and retinal sensitivity using microperimetry. During the study, participants will attend visits for eye examinations and imaging at specified time points to monitor GA progression. The research team will use these assessments to analyze short-term changes in GA and explore correlations with genetic and lifestyle factors. The total participation duration is approximately six months, during which safety and vision measures will be carefully monitored.

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 the efficacy and safety of two different dose regimens of pegozafermin compared to a placebo in adults with metabolic dysfunction-associated steatohepatitis MASH who have liver fibrosis stage F2 or F3. This Phase 3 study aims to better understand how pegozafermin may impact liver fibrosis and steatohepatitis in this population. Participants will receive subcutaneous injections of either one of two pegozafermin regimens or a matched placebo. These treatments are given in parallel groups, and participants are randomly assigned to one of the study groups. The study compares the effects of pegozafermin on liver fibrosis and steatohepatitis over a treatment period that includes evaluations up to 52 weeks and monitoring for disease progression up to 5 years. During the study, participants will be monitored through biopsies and blood tests to assess liver fibrosis improvement, resolution of steatohepatitis, changes in liver enzyme levels, and enhanced liver fibrosis scores. Safety and disease progression are also tracked throughout the study period. The total participation duration includes treatment and long-term observation to evaluate outcomes and any potential changes in liver health.

Researchers are evaluating elritercept TAK-226, KER-050, an investigational drug, for treating anemia in adults with very low, low, or intermediate risk myelodysplastic syndromes MDS who need regular red blood cell RBC transfusions. The study is a Phase 3, randomized, double-blind, placebo-controlled trial designed to assess how well elritercept reduces the need for RBC transfusions and to evaluate its safety and tolerability over time. Participants will be randomly assigned in a 21 ratio to receive either elritercept or a matching placebo, both given as subcutaneous injections every 4 weeks. The study includes a Primary Phase lasting 24 weeks and a Secondary Phase lasting an additional 24 weeks, during which participants continue their assigned treatments. Eligible participants may also enter an Extension Phase to continue treatment until individual discontinuation or study unblinding. After treatment ends, a Safety Follow-Up Period of 8 weeks and a long-term follow-up lasting up to 5 years will monitor participants. During the study, participants will have visits approximately every 2 weeks initially, then every 4 weeks, to assess treatment effects and safety. Researchers will evaluate the percentage of participants achieving transfusion independence and monitor adverse events, laboratory values, vital signs, and heart tests. Long-term follow-up will continue through regular check-ins for up to five years or until the participant withdraws or the study ends.

Researchers are evaluating how well elritercept works to improve anemia in adults with myelofibrosis MF who are already taking ruxolitinib. The study compares elritercept to a placebo and aims to see if elritercept can reduce tiredness, improve MF-related symptoms, and help participants perform physical activities more easily. It also looks at elritercepts effects on bone marrow, spleen size, antibody development, and long-term safety. Participants receive either elritercept or a placebo by subcutaneous injection once every 4 weeks during a 36-week double-blinded treatment period. The starting dose of elritercept is 3.75 mgkg, with a possible increase to 5.0 mgkg after the second cycle based on response and safety. After 36 weeks, participants who took placebo may switch to receive elritercept in an extended open-label phase. During the study, participants undergo assessments including blood transfusion independence, symptom and fatigue questionnaires, spleen imaging, and bone marrow evaluation. Researchers monitor safety, antibody formation, and survival for up to 7 years. The main outcome is the proportion of participants who become independent from red blood cell transfusions for at least 12 consecutive weeks during the 36-week treatment. Participants are involved in regular visits and evaluations throughout the treatment and follow-up periods.

Alport syndrome AS is a rare genetic disorder caused by changes in specific genes that produce collagen, leading to kidney disease, hearing loss, and eye problems. People with AS face a high risk of chronic kidney disease CKD, which gradually reduces kidney function and can lead to end-stage kidney disease. A common sign of worsening kidney function is proteinuria, the presence of excess protein in the urine. This study evaluates the effects of BAY 3401016, a monoclonal antibody designed to block the protein Semaphorin 3A Sema3A, which may contribute to kidney damage in AS. Participants are randomly assigned to receive either BAY 3401016 or a placebo once weekly for 24 weeks, alongside their background therapy. The study includes an extension phase to further assess the treatments safety and efficacy. Participants will be involved for at least 24 weeks of treatment, followed by a 90-day follow-up period. Researchers will monitor kidney function by measuring the urinary albumin creatinine ratio UACR at several points during treatment and after its completion. Safety and tolerability of BAY 3401016 will also be carefully assessed throughout the study period.

Researchers are evaluating the effects of a medicine called BI 1815368 in adults aged 18 and older who have a specific eye condition known as centre-involved diabetic macular edema CI-DME. This condition affects vision, and the study aims to find out if BI 1815368 can improve sight and to determine the best dose. The trial is a phase 2, randomized, double-masked, placebo-controlled study designed to assess efficacy, safety, and tolerability. The study has two parts. In the first part, participants are randomly assigned to one of two groups one receiving BI 1815368 tablets and the other receiving placebo tablets that look identical but contain no medicine. In the second part, participants are randomly assigned to one of four groups three groups receive different daily doses of BI 1815368, and one group receives placebo. All participants take the tablets twice daily for about 11 months. Participants stay in the study for about one year, during which they visit the study site 16 times. At these visits, doctors check vision, document any health problems, and take detailed eye pictures. Researchers compare changes over time between the groups to evaluate if the treatment affects vision. The main outcome measured is whether participants gain at least 10 Early Treatment Diabetic Retinopathy Study ETDRS letters in vision at Week 48 compared to baseline.

This study evaluates the long-term safety and tolerability of pelacarsen TQJ230 in people with established cardiovascular disease and elevated Lipoproteina who completed a previous related study. It is an open-label extension trial, meaning all participants receive the study drug without placebo comparison. The trial is sponsored by Novartis Pharmaceuticals and focuses on continued treatment after the completion of the parent study. Participants receive monthly injections of pelacarsen 80 mg subcutaneously for up to 36 months during this extension phase. This phase is designed to provide access to the study drug after the initial trial and to monitor participants closely. The study does not involve randomization or blinding, and all enrolled participants receive the active drug. During the study, participants will undergo regular assessments including monitoring for adverse events and cardiovascular events, as well as measuring Lipoproteina levels at baseline and several time points over 36 months. Safety and tolerability will be closely tracked throughout the treatment period. The total duration of participation corresponds to the length of the extension phase, up to three years.

Severe diabetic macular oedema DMO affects the central part of the retina called the macula, leading to sight loss due to fluid buildup from leaking blood vessels. This condition mainly occurs in adults with type 1 or type 2 diabetes and is measured by how thick the macula becomes in microns. This trial evaluates and compares the current standard treatment of anti-vascular endothelial growth factor anti-VEGF injections alone with a new approach where patients start with anti-VEGF injections and then switch to subthreshold micropulse laser SML treatment once the macula thickness decreases below 400 microns. Participants are randomly assigned to receive either continued anti-VEGF monotherapy or a combination where anti-VEGF treatment is followed by SML applied every 2-3 months after the macula thickness drops below 400 microns. Anti-VEGFs used include ranibizumab, aflibercept, faricimab, and brolucizumab, administered monthly initially and then spaced out as needed. SML is applied according to the study guidelines once the macula thins, aiming to reduce the number of injections required. During their participation, individuals will undergo regular optical coherence tomography OCT scans to measure macular thickness and visual acuity tests up to 104 weeks after randomization. Researchers will assess vision changes, macular thickness, quality of life, safety, treatment use, and participant experience. The study also includes follow-up questionnaires and qualitative interviews to understand patient preferences and the potential for wider implementation of the laser treatment strategy.

This observational study focuses on children and teenagers aged 2 to 16 years, investigating the normal appearances of lymph nodes in the lower abdomen using point of care ultrasound POCUS. The goal is to better understand what normal lymph node numbers and sizes look like in young people who visit the pediatric emergency department for non-abdominal injuries. This information aims to help in identifying abnormalities such as mesenteric adenitis, a condition often suspected but not well-defined by imaging. Participants include children presenting with injuries like head, face, neck, or limb trauma but without abdominal or infectious symptoms. The study uses POCUS to scan the right lower abdomen to record the number and size of lymph nodes. This baseline evaluation will help define normal ranges to support future diagnosis of mesenteric adenitis. During the study, participants undergo abdominal ultrasound scans, with data collected over a six-month period to measure lymph nodes. Researchers will monitor the ultrasound results to describe normal lymph node characteristics. There are no treatments involved, and participation mainly includes one or more ultrasound assessments to gather these measurements safely and non-invasively.