Wakefield

Researchers are evaluating AZD0780, an oral PCSK9 inhibitor, in a phase 3, randomized, placebo-controlled study to see if it can reduce the risk of major adverse cardiovascular events (MACE-PLUS) in adults with established atherosclerotic cardiovascular disease (ASCVD) or those at high risk for a first ASCVD event. The study compares AZD0780 to a placebo and monitors participants from randomization until the primary analysis censoring date, followed by a final study closure visit. Participants will be randomly assigned to receive either oral AZD0780 or an oral placebo once daily. The treatment period lasts until the primary analysis censoring date, after which a study closure visit will occur. The study is event-driven and designed to assess the time to the first major cardiovascular event during treatment. During the study, participants will be closely monitored with various assessments to evaluate cardiovascular outcomes and safety over approximately 54 months. Researchers will track the time to first event of any component of MACE-PLUS and collect data to assess the effect of AZD0780 compared to placebo. The study includes regular visits and evaluations to ensure participant safety and adherence to treatment.

Researchers are conducting a global study to understand the impact of moderate to severe alopecia areata (AA), non-segmental vitiligo (NSV), and hidradenitis suppurativa (HS) on adolescents and adults. This study aims to assess the burden these conditions place on patients' quality of life and daily functioning in a large real-world population. The study involves participants diagnosed by a physician with one of the three conditions: AA, NSV, or HS. There are no interventional treatments or medications being tested in this study, as it is observational in nature. Data collection focuses on patient-reported outcomes and measures that evaluate disease severity and its effects. Participants will complete various questionnaires and assessments related to their condition, such as the Alopecia Areata Symptom Impact Scale (AASIS) for AA, the Severity of Alopecia Tool (SALT) for scalp hair loss in AA, the Facial Vitiligo Area Scoring Index (F-VASI) and Vitiligo Quality of Life Score (VitiQoL) for vitiligo, and the Dermatology Life Quality Index (DLQI) and International Hidradenitis Suppurativa Severity Scoring System (IHS4) for HS. These tools help researchers understand how symptoms affect quality of life and disease severity. The study collects information up to the day of the study visit.

Researchers are evaluating the efficacy and safety of belimumab compared to a placebo, alongside standard therapy, for adults with systemic sclerosis associated interstitial lung disease (SSc-ILD). This Phase 2/3 randomized, double-blind study aims to assess how belimumab affects lung function and other disease symptoms such as skin thickening and fatigue, which impact quality of life. Participants will receive either belimumab or placebo administered subcutaneously. The study focuses on those diagnosed with diffuse cutaneous systemic sclerosis and active or progressive disease. Treatment is given under blinded conditions, and participants must be capable of self-administering the medication or have a caregiver to assist. During the study, lung function will be monitored by measuring changes in forced vital capacity (FVC) from baseline to Week 52. Researchers will also assess skin involvement and general symptoms. Safety and tolerability will be closely followed throughout the trial, which includes screening and regular assessments to evaluate treatment effects and participant well-being.

Researchers are investigating the safety, tolerability, and effectiveness of XEN1101 as an additional treatment for people with primary generalized tonic-clonic seizures (PGTCS) who have generalized epilepsy and are already taking 1 to 3 anti-seizure medications. This phase 3, multicenter, randomized, double-blind, placebo-controlled study includes participants aged 12 years and older and aims to better understand how XEN1101 affects seizure frequency compared to placebo. Participants will be randomly assigned to receive either XEN1101 or a placebo capsule once daily with their evening meal during a 12-week double-blind treatment period. Those aged 18 and older will take a 25 mg dose of XEN1101 or placebo, while those aged 12 to under 18 may receive 15 mg, 25 mg, or placebo. Before this period, participants will have up to 9.5 weeks to record their baseline seizure frequency. After completing the double-blind period, participants can join an open-label extension study for continued XEN1101 treatment or enter an 8-week follow-up phase if they do not enroll in the extension. During the study, participants will keep detailed seizure diaries and maintain stable doses of their anti-seizure medications. Researchers will monitor seizure frequency changes, safety, and tolerability throughout the treatment. The main measurement is the median percent change in monthly primary generalized tonic-clonic seizure frequency from baseline through the 12-week treatment. Safety follow-up and monitoring will continue during the post-treatment follow-up or open-label extension periods, with total participation lasting several months depending on extension enrollment.

Researchers are evaluating the safety and effectiveness of tezepelumab in adults aged 40 to 80 years with moderate to very severe chronic obstructive pulmonary disease (COPD). These participants must have a history of COPD for at least one year and have experienced multiple COPD exacerbations despite using inhaled maintenance therapy. This Phase 3, multicenter, randomized, double-blind, placebo-controlled study focuses on those who have had at least two moderate or one severe exacerbation in the prior year while on inhaled triple or dual therapy. Participants will receive monthly subcutaneous injections of either one of two doses of tezepelumab or a placebo. Treatment will last for a minimum of 52 weeks and up to 76 weeks. After the treatment period, there will be a 12-week off-treatment safety follow-up to monitor any lasting effects or safety concerns. During the study, researchers will assess the participants' lung function and monitor the annual rate of moderate or severe COPD exacerbations. Participants will undergo screening to confirm eligibility based on lung function tests, eosinophil counts, and symptom scores. Safety will be closely monitored throughout the treatment and follow-up periods to evaluate adverse effects and overall participant health.

Researchers are evaluating the safety and effects of a study medicine called PF-07275315 for treating adults with moderate-to-severe asthma that is not well controlled. This condition makes breathing difficult and affects quality of life. The study is a Phase 2, randomized, double-blind, placebo-controlled trial aiming to determine if PF-07275315 is safe and effective for this group. Participants will receive either PF-07275315 or a placebo through multiple subcutaneous injections administered in the clinic over 12 weeks. The study compares these two groups to assess treatment responses. The trial includes a total of 9 clinic visits and lasts about 7.5 months for each participant. During the study, participants will undergo various assessments including lung function tests to measure forced expiratory volume in 1 second (FEV1), safety monitoring through adverse event tracking, laboratory tests, vital sign checks, and electrocardiograms. These evaluations occur from baseline through 24 weeks to observe changes and treatment tolerability.

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.

This research aims to understand the genetic factors that contribute to the risk of giant cell arteritis (GCA) and polymyalgia rheumatica (PMR). GCA is a serious inflammatory disease affecting blood vessels, mainly in people over 50, which can cause severe complications like vision loss or stroke if untreated. PMR causes pain and stiffness in the limbs with signs of inflammation. The study involves both patients recently suspected of having GCA and those with confirmed diagnoses from the past. It seeks to provide new insights into disease causes and improve diagnosis and treatment approaches. Participants are observed in a multi-center study collecting clinical and genetic data. The study includes both prospective patients with suspected GCA and retrospective patients with confirmed GCA or PMR diagnoses. Some retrospective participants receiving tocilizumab for recurring or difficult-to-treat GCA are also included in a safety monitoring registry. Data collected include clinical features, imaging, tissue and blood samples, and advanced genetic testing. The study also follows patients over time to assess disease impact, quality of life, and long-term outcomes. During the study, participants provide medical information, biological samples, and complete questionnaires about their symptoms and quality of life. Researchers monitor disease activity and treatment effects, especially among those starting certain immune-modifying drugs. The main measurements focus on genetic susceptibility at the study start, with ongoing evaluation of diagnosis, prognosis, and disease progression. The study is designed to improve understanding and management of GCA and PMR over time.

Researchers are evaluating whether adding duroplasty, a surgical procedure that expands the dura (the tough membrane around the spinal cord), to standard spinal surgery improves muscle strength and outcomes after severe traumatic spinal cord injury in the neck. This condition often causes permanent disabilities like paralysis and loss of bladder and bowel control, and currently, no treatments have proven to improve recovery. The study focuses on adults with severe cervical spinal cord injury who need surgery within 72 hours of injury. Participants will be randomly assigned to receive either standard spinal surgery, which includes laminectomy (removal of part of the vertebra), or spinal surgery plus duroplasty. The duroplasty involves opening the dura and stitching a patch to relieve pressure on the swollen cord. A subgroup of patients will also take part in a smaller mechanistic study using probes at the injury site to monitor pressure, blood flow, metabolism, and inflammation. The study will recruit 222 adults over 4 years from major trauma centers in the UK. During the study, patients will be assessed at baseline, 3, 6, and 12 months after surgery for muscle strength, hand function, walking ability, bladder and bowel control, quality of life, complications, and survival. Some patients will have additional monitoring at the injury site. The main outcome measured is the change in motor function at 6 months compared to baseline. Participants will be followed for one year to evaluate recovery and safety.

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.