Glencoe, Minnesota

Effectiveness of Decision Support for Cardiovascular Risk Management in People With Cardiovascular Disease

A Multicenter Study on Atrioventricular Regurgitation in Transthyretin Amyloid Cardiomyopathy: Definition, Prevalence, and Prognostic Impact.

Implementation of Home Monitoring in Patients with Pulmonary Fibrosis

Patients will be randomly assigned to receive either hospital-based care or a home monitoring program integrated into hospital-based care. Hospital-based care involves outpatient clinic visits every three months including lung function testing. Home monitoring involves weekly measurements of both physiological- and patient reported outcomes (PROs) in a mobile health care application with half of the outpatient clinic visits alternately being replaced by remote video consultations. The home monitoring program includes home spirometry, pulse-oximetry, PROs, video consultations, a medication coach, and an infotheque showing disease-specific information. Questionnaires will be filled out by both study groups at set time points. The total study duration for individual patients will be 12 months.

The Dragon PLC Trial (DRAGON-PLC)

Primary liver cancer (PLC) is the third most common cause of cancer death worldwide. Surgical resection is the mainstay for a curative approach as contemporary chemotherapy and immune-based therapies only lead to a median survival of 10-14 months. A complete surgical resection increases the median survival to 42 months (range 32-52 months). However, PLC is mainly diagnosed at an advanced stage and >70% of PLC patients are ineligible for an immediate surgical approach. There are different reasons that make a patient ineligible for surgery, one important reason is the risk of liver failure after the surgery due to a small remnant liver. This study aims to improve the oncological, radiological and surgical strategy to allow more patients to undergo liver resection safely, to improve quality of life and to extend overall survival at acceptable costs. Adequate function of the future liver remnant (FLR) is a prerequisite for surgical resectability. This is necessary in order to avoid liver failure after surgery, a major cause of morbidity (38%) and mortality (27%). To mitigate this risk, regenerative strategies based on preoperative calculation of the FLR volume and function are essential. Patients with technically resectable disease but predicted insufficient FLR volume or function are referred to as primarily unresectable or potentially resectable (PU/PR). These patients can undergo strategies that capitalize on the regenerative capacity of the liver which aim to preoperatively increase the FLR volume and function in order to allow surgery. Many of the patients that are primarily unresectable due to an insufficient FLR can become ultimately and safely resectable after the induction of adequate FLR-hypertrophy by the current standard, portal vein embolisation (PVE). However, 25% of patients do not show sufficient FLR growth after PVE and are unable to safely undergo resection. A new approach has been developed to improve this. Combined portal and hepatic vein embolisation (PVE/HVE) has great promise in terms of increasing FLR growth, resection rate (RR), safety and potentially, overall survival. Establishing PVE/HVE as the new standard could result in increased survival and a better quality of life (QoL) for patients.

A Study to Learn How a Tablet Compared With an IV Infusion of the Study Medicine Called Vepdegestrant is Taken up Into the Blood in Healthy Adults

FTD-TPI, Bevacizumab, and Radioembolization With 166Ho-microspheres in Refractory Metastatic Colorectal Cancer

UCAN CAN-DU: Canada-Netherlands Personalized Medicine Network in Childhood Arthritis and Rheumatic Disease

UCAN CAN-DU is a multicenter observational cohort study that will collect prospective data from children with arthritis. Biologic samples, clinical data and patient reported outcomes will be collected. In addition, the study will also include a health economics component which will include a number of complementary approaches for quantifying and comparing benefits and risks that promote evidence-based, patient centered health care. This will address both the personal and societal economic burden of disease and include qualitative methods to inform the measurement of preferences, economic and simulation modelling to assess the value of biomarker testing. The socioeconomic impact of biomarker based treatment will be evaluated. All clinical, biological and patient-derived data will be collected at an aggregation point housed and managed by High Performance Computing 4 Health (HPC4Health), a private hospital-only secure cloud-computing service within Compute Canada and physically located at SickKids/UHN. These databases and apps include biospecimen data and data collected through the eHealth platform. This will enable the study team to share and integrate data in near real-time into analytic models throughout the study course; hence providing a near real-time feedback from bench to bedside and vice versa. The analysis of the cohorts will help define and confirm the biologic pathways predictive of disease course, treatment response and disease remission. This knowledge will then be used to develop a comprehensive clinical predictive tool to guide effective and safe treatment of childhood arthritis.

A Phase 3, Placebo-controlled, Double-blind Study Assessing Rocatinlimab in Prurigo Nodularis

Adult Congenital Heart Disease International EValuation of the Effectiveness of SGLT2i Registry

In the adult congenital heart disease (ACHD) population, heart failure currently represents the main cause of morbidity and mortality. The etiology of ACHD-related heart failure is heterogenous, and there is limited evidence for pharmacological treatment options for this population. Sodium-glucose cotransporter 2 inhibitors (SGLT2i) are a novel pillar in the treatment of conventional LV heart failure. SGLT2i have been shown to reduce the risk of worsening heart failure and cardiovascular-related death in patients with LV heart failure. While the exact mechanisms of action are still to be elucidated, SGLT2i seem to address heart failure by targeting several pathways. These include but are not limited to; a decrease in renin-angiotensin and sympathetic nervous system activation, a decrease in pressure overload-induced myocardial fibrosis, reverse cardiac remodeling, and improvement in myocardial energetics. Given the compelling evidence on the effectiveness of SGLT2i over a broad range of cardiac dysfunction and initial promising reports of its utilization in the field of ACHD, SGLT2i deserve further exploration in the group of ACHD patients. This real-world, international registry aims to evaluate the current experience with SGLT2i in ACHD patients by investigating the prescription patterns, safety, tolerability, and potential beneficial effects on heart failure-related outcomes. Project design: Data of all ACHD patients who were started on an SGLT2i will be collected in a real-world, international registry. Only data resulting from routine clinical care will be collected from the electronic health records at the participating centers, and participants will not undergo any interventions for this project. Data will be collected from 1 year before starting with the SGLT2i to most recent follow-up after starting with the SGLT2i, to evaluate if SGLT2i therapy halts the progression of clinical deterioration in ACHD patients with heart failure and can improve heart failure-related outcomes.

Dutch Registry of Pediatric Cardiac Arrest

The PROGNOSE study (Pediatric Resuscitation Neuroprognostication and Outcomes Registry) is a prospective, multicenter, observational patient registry established to improve understanding of short- and long-term outcomes following pediatric cardiac arrest (CA) in the Netherlands. The study focuses on both out-of-hospital (OHCA) and in-hospital cardiac arrest (IHCA) in children under 18 years of age. Given the rarity yet severity of pediatric CA, the registry aims to collect a large dataset to identify patterns in care, outcomes, and recovery trajectories to ultimately improve treatment strategies and prognosis. The study is considered non-WMO (not subject to the Dutch Medical Research Involving Human Subjects Act), as it involves no additional interventions beyond standard clinical care. Registry Objectives and Scope The primary objective is to evaluate diagnostic practices and long-term functional and neuropsychological outcomes in pediatric patients post-cardiac arrest. Key secondary objectives include determining the incidence and etiology of pediatric CA in the Netherlands, analyzing survival to discharge, and characterizing post-Return of Circulation (ROC) care. Data will be collected longitudinally during standard outpatient follow-up visits at specified intervals (3, 12, and 24 months post-arrest; and at the ages of 5, 8, 12, and 17 years, depending on the age at CA event). No study-specific interventions will be performed. Data Collection and Source Verification All data collected in this registry will be sourced from routine clinical care and medical records from seven Dutch academic (pediatric) hospitals. Data will be abstracted from: Emergency services records Inpatient hospital and ICU documentation Follow-up outpatient clinic assessments Neuropsychological and functional evaluations using validated tools (e.g., PCPC, POPC, FSS) The data will be pseudo-anonymized using unique study IDs. Only site-specific investigators will have access to the link between study ID and patient identity. Quality Assurance and Monitoring A robust quality assurance framework has been put in place to ensure data integrity, including: Data validation procedures: Data entries will be checked against predefined rules for logic, consistency, and range validation in the electronic data capture (EDC) system (Castor EDC). Source data verification (SDV): Periodic audits by local site PIs will compare registry entries against source data (e.g., EHRs) for completeness and accuracy. Site monitoring: The coordinating center (Erasmus MC) will provide oversight and perform routine cross-site reviews to ensure harmonized data collection. Data dictionary: A detailed data dictionary defines all collected variables, including their source, coding schemes (e.g., ICD, MedDRA where applicable), and interpretation ranges. Standard Operating Procedures (SOPs) Standard operating procedures have been established to guide the entire registry lifecycle, including: Site initiation and training Patient screening and enrollment processes Data entry and validation procedures Procedures for obtaining (delayed) informed consent at follow-up Change management and version control of registry forms Procedures for data sharing and publication Statistical Analysis Plan Given its quality improvement nature, no formal sample size calculation was performed. Instead, the registry aims to enroll all eligible pediatric CA cases nationwide, allowing the creation of a comprehensive, hypothesis-generating dataset. Descriptive statistics will summarize demographics, event characteristics, interventions, and outcomes. Longitudinal analysis of neuropsychological function and functional status will be performed using repeated-measures ANOVA or mixed-effects modeling. Comparative statistics (e.g., logistic regression) will be used to identify predictors of survival and favorable neurological outcome. Covariates will be selected based on literature and tested for collinearity. Multivariate models will report odds ratios with 95% confidence intervals. Missing data will be addressed using appropriate imputation techniques, depending on the mechanism of missingness (e.g., multiple imputation for random missing data). Plan for Missing Data To manage incomplete records and potential attrition in follow-up: All missing values will be coded according to reason (e.g., not applicable, patient deceased, declined follow-up). Patterns of missingness will be explored. If data are missing at random, multiple imputation may be used in analysis. Data collection tools prompt required fields and flag missing values to reduce omissions during entry. Informed Consent and Ethics Due to the emergent nature of CA and the high mortality rate, an exception from consent procedure is employed for initial data collection. For survivors, informed consent is sought at follow-up outpatient visits, as part of standard care. Data are de-identified and used solely for research purposes. No images or human materials are collected. The study complies with the General Data Protection Regulation (GDPR), the Dutch "Code Goed Gedrag," and the Declaration of Helsinki. Data Storage and Access Pseudo-anonymized data are stored securely in Castor EDC, managed by Erasmus MC. Each center has access only to its own data. A Data Transfer Agreement (DTA) governs anonymized data sharing for multicenter publications. Data will not be shared outside the EU. No end date is set for the registry; it will remain open for ongoing data entry and hypothesis generation. Dissemination Plan Findings from the registry will be disseminated through peer-reviewed publications, with a goal of generating multicenter analyses and contributing to national and international guideline development. A first multicenter manuscript is expected within approximately five years from the start of the registry.