Kirkcaldy

Researchers are evaluating whether reducing the frequency of pembrolizumab treatment after six months of standard therapy is safe and effective for patients with advanced non-small cell lung cancer (NSCLC). Pembrolizumab, an immunotherapy targeting the PD-1 receptor on T cells, has improved outcomes for this condition. Because pembrolizumab remains bound to its target for a long time and dosing frequency may not affect outcomes, this study aims to find out if less frequent dosing can maintain effectiveness while reducing overtreatment and side effects. This phase III study also considers potential benefits like cost savings and improved quality of life due to fewer hospital visits. Participants who have completed six months of pembrolizumab treatment without disease progression and are continuing therapy will be randomly assigned to receive pembrolizumab at the standard six-week interval or at a reduced frequency of 12 weeks. If early results show that the 12-week schedule is not less effective, later participants may be randomized to even longer intervals of 9, 15, or 18 weeks. Pembrolizumab is given intravenously at 400 mg per dose. Patients whose disease progresses on a reduced frequency schedule may return to the standard six-week treatment. During the study, researchers will monitor overall survival at two years after randomization. Participants will undergo regular assessments to track disease status, treatment tolerability, and overall health. The study aims to confirm that less frequent dosing does not reduce survival while potentially improving patient experience. The trial is open to adults aged 18 and older with advanced NSCLC who have already completed six months of pembrolizumab therapy and intend to continue treatment.

Researchers are conducting a non-interventional, observational, retrospective study to describe the characteristics, clinical outcomes, and event rates of participants diagnosed with propionic acidemia (PA). This global, multicenter study focuses on gathering medical record data from participants who meet specific inclusion criteria related to PA diagnosis and clinical history. Medical data will be collected through review of records from medical clinics, hospitals, and academic medical centers. The study includes participants with confirmed PA diagnosis based on molecular genetic testing of specific enzyme subunits. Data must support clinical event adjudication and cover participants' history from birth or January 1, 2015, onward. The study records metabolic decompensation events (MDEs) experienced by participants over up to 10 years. Participant involvement consists of medical record abstraction without intervention or direct treatment. Researchers will evaluate the number of MDEs adjudicated by a clinical event committee. Eligibility requires informed consent and sufficient data availability. Participants with certain prior treatments or conditions, such as gene therapy, organ transplantation, or participation in specific clinical trials, will be excluded. The observation period includes retrospective data spanning multiple years to assess long-term clinical events and outcomes.

The MobileLink hip prosthesis system study focuses on patients with osteoarthritis of the hip who undergo hip replacement surgery. This research aims to collect real-world clinical data on the safety, performance, and patient satisfaction with the MobileLink system, a CE-marked medical device. The study observes the prosthesis's survival and failure over time in routine use. Participants will receive the MobileLink hip prosthesis system, which includes the acetabular cup and a femoral stem by Waldemar Link. After implantation, patients will be followed for up to 10 years to monitor the outcomes of the device under normal conditions. This long-term follow-up period helps assess the device's durability and any need for revision surgery. During the study, patients will have regular assessments to evaluate the function and clinical outcomes of the hip prosthesis. Researchers will measure the device's survival rate, with revision for any reason as the main endpoint after 10 years. Patient satisfaction and other clinical data will also be collected throughout the follow-up period to provide a comprehensive understanding of the treatment's effectiveness and safety.

Researchers are investigating whether the medicine vicadrostat, when taken together with empagliflozin, can lower the risk of heart-related problems in adults who have type 2 diabetes, high blood pressure, and cardiovascular disease but no history of heart failure. This study is a Phase III trial that compares the effects of vicadrostat plus empagliflozin to a placebo plus empagliflozin in people with these conditions. Participants are randomly assigned to one of two groups: one group takes vicadrostat and empagliflozin tablets, and the other group takes placebo tablets that look like vicadrostat along with empagliflozin. All participants take one tablet daily for a period ranging from two and a half years up to four years and three months. Throughout the study, participants continue their usual medications for diabetes, high blood pressure, and cardiovascular disease. During up to 51 months of participation, participants visit the study site regularly where doctors collect health information and blood samples. Researchers track when participants experience cardiovascular events such as heart-related deaths or heart failure events. The study also monitors participants’ overall health and any side effects they may experience to assess the safety and effects of the treatments.

Researchers are conducting Enroll-HD, a large, long-term observational study involving individuals affected by Huntington's Disease (HD), those at risk, and control participants. This study combines data from previous registries in Europe, North America, Australasia, and now includes Latin America. It aims to build a comprehensive database of clinical information and biological samples to support research on disease progression, prognosis, and clinical characteristics, as well as to establish clear endpoints for future interventional studies. Enroll-HD collects detailed clinical and genetic information along with blood samples from participants categorized as carriers of the HD gene mutation, controls without the mutation, and family or community controls. The study enrolls participants from over 150 sites in 23 countries and conducts annual assessments without a set end date. Participant groups include those with manifest HD symptoms, pre-manifest carriers, relatives with unknown genotype, and genotype-negative relatives. Participants undergo yearly evaluations including motor, functional, behavioral, and cognitive assessments using standardized scales such as the Unified Huntington's Disease Rating Scale and the Problem Behaviors Assessment-Short. Researchers track changes over an average of one year or longer through this ongoing registry. Data collected supports multiple research efforts and is accessible to qualified researchers worldwide.

Researchers are collecting cerebrospinal fluid (CSF) and blood plasma samples from people at various stages of Huntington's disease (HD) to help develop new treatments. This study aims to gather high-quality CSF samples for biomarker analysis and to better understand disease pathways. It also seeks to collect matching plasma samples to support HD research and therapeutic development. Participants will take part in an ongoing observational study with annual visits. Each year, they have a Screening Visit to provide medical history and clinical data, followed by a Sampling Visit where blood and CSF samples are collected after fasting for at least six hours. Some participants may be invited to an optional repeat Sampling Visit within 4 to 8 weeks of their first sampling during their first year in the study. Visits continue annually for multiple years with flexibility to skip visits without leaving the study. During each visit, researchers will gather detailed health information and biological samples through blood draws and lumbar punctures. Participants' participation is monitored over time, with the option to re-enroll if they stop attending visits. The main goal is to measure biomarkers relevant to HD progression and treatment development, with careful attention to safety and participant willingness throughout the study duration.

Researchers are evaluating the long-term safety of two drugs, Deucravacitinib and Ustekinumab, in adults with moderate-to-severe plaque psoriasis. This Phase 3b/4 study focuses on participants who are candidates for phototherapy or systemic treatment and have specific cardiovascular risk factors such as smoking, hypertension, diabetes, obesity, or a family history of heart disease. Participants will receive either Deucravacitinib or Ustekinumab at specified doses on set days. This open-label, randomized study compares these treatments over an extended period to monitor their safety profiles, including cardiovascular health. Throughout the study, researchers will track major adverse cardiovascular events, including heart attacks, strokes, and related procedures, for up to five years. Participants will undergo regular assessments to monitor their psoriasis and cardiovascular status, ensuring comprehensive safety evaluation during the long-term treatment.

Researchers are evaluating the real-world effectiveness of nemolizumab for treating moderate-to-severe atopic dermatitis (AD) in adolescents and adults. This study is a prospective, multicenter, non-interventional trial that aims to measure treatment outcomes through physician assessments and patient-reported outcomes over approximately 12 months. The goal is to understand how nemolizumab works in routine clinical practice, focusing on physician evaluations and patient experiences at Month 6. Treatment with nemolizumab is determined solely by the participant's physician before joining the study, with no extra visits, procedures, or lab tests beyond standard care. The study does not define a specific visit schedule; instead, visits follow routine medical practice to collect data systematically. A sub-study in Germany and the UK will have participants complete daily questionnaires on itch severity, sleep disturbance, and pain from Day -1 to Day 14 remotely, without requiring clinic visits. Participants will be involved in routine clinical visits where physician assessments and patient-reported outcome measures will be gathered. Key outcomes measured include the Investigator Global Assessment (IGA) and the Peak Pruritus Numerical Rating Scale (PP NRS) at Month 6. The study observes participant responses and safety under normal care conditions, with data collection lasting about a year to evaluate nemolizumab's effectiveness in everyday treatment settings.