Inverness

Researchers are evaluating the effects of iptacopan compared with a placebo in adults aged 18 to 85 years who have generalized Myasthenia Gravis positive for acetylcholine receptor antibodies (AChR+ gMG). This Phase III, randomized, double-blind, placebo-controlled, multicenter study aims to assess the efficacy, safety, and tolerability of iptacopan while participants continue their stable standard of care treatments. The study includes participants with moderate to severe gMG symptoms and positive diagnostic criteria. Participants will be randomly assigned in a 1:1 ratio to receive either iptacopan or a matching placebo in the form of hard gelatin capsules for six months (180 days). During this time, they will continue their stable standard of care treatments. After the double-blind treatment period, a maximum 60-month open-label extension phase is offered. Safety follow-up assessments will occur one week and one month after the last dose of study treatment. During the study, participants will be evaluated for changes in their Myasthenia Gravis Activity of Daily Living (MG-ADL) total score from baseline to month 6. Researchers will monitor safety and tolerability throughout the treatment and extension periods. Vaccination status, infection monitoring, and regular clinical assessments will be part of participant evaluations to ensure safety and track disease symptoms over time.

Researchers are investigating whether encapsulated faecal microbiota transplantation (FMT) can help reduce infections and mortality in adults with alcohol-related or metabolic dysfunction-associated steatotic liver disease (MASLD) cirrhosis. Cirrhosis, a severe scarring of the liver, is a growing health crisis, leading to high rates of early death, with infections being a major complication. Previous trials showed that FMT delivered endoscopically is safe, and this study aims to test a capsule form of FMT to improve patient outcomes without the need for invasive procedures. Participants will be randomly assigned to receive either encapsulated FMT or placebo capsules that look identical. They will take five capsules every three months for up to 21 months or until they develop an infection requiring hospital admission. This double-blind trial will follow participants for up to 24 months to compare infection rates and time to hospitalization between the two groups. The study will also explore if FMT improves liver function, immune response, and reduces harmful bacteria linked to cirrhosis complications. During the study, participants will be closely monitored for infections and other cirrhosis-related complications through hospital visits and assessments. Researchers will measure the time until the first infection or decompensation leading to emergency or hospital admission. Laboratory tests will evaluate immune system changes and the presence of antibiotic-resistant bacteria. The trial will last up to two years, aiming to provide insights into new, antibiotic-free treatments for cirrhosis and influence future care and policy.

Researchers are investigating whether ziltivekimab can treat people living with heart failure and inflammation. The study compares ziltivekimab, a new medicine not yet approved anywhere, to a placebo, an inactive substance that looks like the medicine but contains no active drug. Participants have an equal chance of receiving either treatment. The study is expected to last up to one year and four months and focuses on people with heart failure who also have systemic inflammation. Participants will receive either ziltivekimab or placebo by monthly injections under the skin. The doses are given once a month throughout the study period. The study lasts for 12 months of treatment following randomization, during which the effects of the medicine compared to placebo will be closely monitored. During the study, participants will undergo various assessments including a heart failure questionnaire called the Kansas City Cardiomyopathy Questionnaire (KCCQ) to measure symptoms and physical function over the 12 months. Other evaluations may include walking tests and heart function tests. Safety and health will be monitored regularly to understand how participants respond to the treatments and to track any side effects or changes in heart failure symptoms.

Researchers are conducting a Phase 3 study to compare two front-line treatments for adults with nonsquamous non-small cell lung cancer (NSCLC) that is stage IV or advanced stage IIIB/C. The study focuses on patients whose tumors have a KRAS p.G12C mutation and are negative for PD-L1 expression. The main goal is to evaluate how each treatment affects progression-free survival and overall survival over about 2.5 years. Participants will be randomly assigned to receive either sotorasib combined with platinum doublet chemotherapy or pembrolizumab combined with platinum doublet chemotherapy. Sotorasib is given orally, while pembrolizumab is given intravenously. Both groups will receive the combination therapies as their initial treatment for advanced NSCLC. During the study, participants will be monitored regularly to assess treatment effects and safety. Researchers will track how long patients live without the cancer worsening and overall survival over approximately 2.5 years. The study includes evaluations to determine eligibility and ongoing assessments to monitor health and treatment response throughout the trial period.

Researchers are evaluating the effectiveness of zanubrutinib combined with anti-CD20 antibodies compared to lenalidomide plus rituximab (R2) in adults with relapsed or refractory follicular lymphoma (FL) or marginal zone lymphoma (MZL). The study aims to measure progression-free survival using independent review committees and established lymphoma response criteria based on PET/CT and CT imaging. Participants will receive zanubrutinib orally either as 160 mg twice daily or 320 mg once daily in continuous 28-day cycles. In the zanubrutinib plus rituximab group, rituximab is given intravenously at 375 mg/m2 on Days 1, 8, 15, and 22 of Cycle 1 and Day 1 of Cycles 2 to 5, each cycle lasting 28 days. The comparator group receives lenalidomide orally at 20 mg daily on Days 1 to 21 of each 28-day cycle for 12 cycles, plus obinutuzumab intravenously at 1000 mg on Cycle 1 Days 1, 8, 15 and Cycles 2 to 6 Day 1. During the study, participants will undergo imaging assessments such as PET/CT and CT scans to evaluate disease progression. Researchers will monitor treatment response and safety over approximately 78 months. Progression-free survival is the primary outcome, measured by a blinded independent review committee. Participants are expected to have measurable disease and adequate organ function at enrollment, with ongoing assessments to track treatment effects and adverse events.

Researchers are evaluating the effectiveness of riliprubart compared to the usual treatment of intravenous immunoglobulin (IVIg) in adults with Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) who are already receiving maintenance IVIg treatment. This Phase 3 study aims to assess how well riliprubart works and its safety in this population. Participants must meet specific CIDP diagnostic criteria and have a history of responding to IVIg treatment. The study involves administering riliprubart or placebo through intravenous (IV) or subcutaneous (SC) solutions, alongside the usual IVIg treatment. The treatment phase includes a randomized, double-blind comparison of riliprubart versus IVIg, followed by an open-label extension period for continued evaluation. The entire study lasts up to 109 weeks, encompassing screening, treatment, and follow-up phases. Participants will have regular assessments to monitor their response to treatment, including measurements of disability and disease activity scores. Researchers will track the percentage of participants showing a response from baseline to week 24 and those maintaining response through week 48 during the extension. Safety and long-term effects will also be observed throughout the study duration, ensuring comprehensive monitoring of participant health and treatment outcomes.

Researchers are evaluating the effect of muvalaplin on reducing cardiovascular risk in adults with elevated lipoprotein(a) levels who either have atherosclerotic cardiovascular disease or are at risk for a heart attack or stroke. This Phase 3, randomized, double-blind, placebo-controlled study focuses on adults with high Lp(a) levels and prior or potential cardiovascular events. The study aims to assess the time to the first major adverse cardiovascular event over about 5.25 years. Participants will be randomly assigned to receive either muvalaplin or a placebo, both administered orally. The study includes individuals with Lp(a) levels of at least 175 nanomoles per liter who have had a prior cardiovascular event within 10 years or are at risk for a first event due to conditions such as coronary artery disease, carotid stenosis, peripheral artery disease, high coronary artery calcium score, reduced kidney function with diabetes, or other high-risk factors. The treatment period lasts through the study duration, with close monitoring. During the study, participants will be regularly evaluated to track the occurrence of major adverse cardiovascular events, including heart attacks and strokes. Safety assessments will monitor blood pressure, kidney function, and heart failure status among other health indicators. The primary outcome measures the time to the first major cardiovascular event from baseline up to the end of the study, which spans approximately 5.25 years.

Researchers are evaluating whether avoiding further axillary treatment after neoadjuvant chemotherapy (NACT) is as effective as standard axillary treatment for patients with early stage breast cancer who initially had cancer in the lymph nodes confirmed by needle biopsy but show no residual cancer in the lymph nodes after NACT. The study aims to determine if skipping axillary lymph node dissection (ALND) or axillary radiotherapy (ART) affects disease free survival (DFS) and whether it reduces the risk of lymphoedema five years after treatment. This phase 3, open-label, randomized trial includes patients with T1-3N1M0 breast cancer and confirmed nodal metastases who have undergone sentinel node biopsy removing at least three lymph nodes post-NACT.

Researchers are studying patients with known or suspected angina who do not have obstructive coronary artery disease to see if a special diagnostic test can help guide personalized treatment. This condition includes ischaemic heart disease, microvascular angina, and vasospastic angina. The trial builds on earlier research suggesting that tailoring treatment based on coronary vascular function tests may improve symptoms and quality of life. This large, multicentre, blinded, randomized study aims to confirm these findings and assess effects on health and wellbeing over a longer period. Participants undergo invasive coronary angiography along with an adjunctive interventional diagnostic procedure (IDP) that measures coronary vascular function using a guidewire technique. Patients with no significant artery blockage are randomized into two groups: one where IDP results are disclosed to guide treatment decisions, and a control group with concealed results receiving standard care. Those with abnormal vascular function in the intervention group may have repeated assessments to tailor medications, such as calcium channel blockers. Patients with obstructive artery disease or other exclusions may join a registry for follow-up and assessment. Throughout the study, participants complete questionnaires about angina symptoms, quality of life, activity levels, treatment satisfaction, and pain. Researchers monitor clinical outcomes for at least 12 months, including major cardiovascular events. The study also evaluates the safety and usefulness of the diagnostic procedure in multiple hospitals across Europe. Blood samples are collected to explore disease mechanisms. Participants and their doctors remain blinded to group assignments, but diagnoses are shared to help guide care following current guidelines.

Autosomal Dominant Polycystic Kidney Disease (ADPKD) affects millions worldwide and is a leading cause of kidney failure. This research aims to evaluate whether metformin, a common diabetes medication, can be repurposed to slow kidney function decline in adults with early-stage ADPKD. The trial addresses the limited treatment options currently available and the significant impact of ADPKD on quality of life, anxiety, and depression. Participants will be randomly assigned to receive either extended-release metformin or a placebo with inactive tablets identical in appearance. This global Phase III study plans to enroll 1,174 adults aged 18 to 70 years diagnosed with ADPKD. The study will carefully monitor kidney function over 24 months to assess the effects of metformin on disease progression. During the study, participants will undergo evaluations including kidney function tests measured by estimated glomerular filtration rate (eGFR). Researchers will track changes over two years to determine if metformin slows kidney decline. Safety assessments and adherence monitoring will also be part of the study to ensure participant well-being throughout the trial period.