Lohmar, Germany

EUthyroid2: the Next Step Towards the Elimination of Iodine Deficiency and Preventable Iodine-related Disorders in Europe

BACKGROUND: Iodine deficiency (ID) is a leading risk factor for the development of thyroid disorders, of which in particular women are affected. During pregnancy, ID can increase the risk of developmental disorders in the offspring, however, it is considered as one of the most preventable causes of mental impairment in children. Therefore, the EUthyroid2 project aims to contribute to the prevention of ID and associated symptom and disease burden in adolescence and young women in Europe and beyond. AIM: An educational intervention is to be developed, implemented in ambulatory care settings and evaluated to effectively raise awareness for the risks of ID among young women (18-24 years) in three European countries (Norway, Poland, UK) as well as Bangladesh and Pakistan. METHODS: A cluster-randomised controlled trial is to be conducted in each of the five countries. 10 clusters per country (5 intervention group clusters and 5 control group clusters) are planned to achieve a final sample size of 200 study participants per implementing country with one baseline (before the intervention) and two follow-up measurements (2-4 weeks and 6-8 months after the intervention). In all, 1000 participants are to be recruited and data for all measurement points collected. Due to differences in the healthcare systems, the ambulatory care units, where the intervention will be implemented, may vary across the countries. Before recruiting the women, the healthcare professionals who carry out the intervention will receive a specially designed training program. To assure the intervention's functionality and effectiveness, recommendations for the development of complex interventions, appropriate theories and frameworks will be considered and a context analysis will be conducted. The primary outcome of the study is iodine awareness and knowledge measured by a newly developed questionnaire. Other outcomes includes measurement of iodine status (urinary iodine concentration), intake of dietary iodine sources (measured by a food frequency questionnaire), and iodine related behaviours. Sociodemographic characteristics and general dietary habits will also be measured. Descriptive analyses for all variables will be performed. Intervention groups will be compared to control groups over time to test effectiveness. Subgroup and country-specific analyses will also be computed. A process evaluation will be conducted to evaluate the implementation process with a convergent parallel mixed methods design. For this, healthcare professionals in the ambulatory care settings and the women who received the intervention will be invited to participate in an online survey. Further, ca. 20-30 semi-structured interviews will be conducted with women and healthcare professionals. CONCLUSION / OUTLOOK: The project results may support health authorities across countries to implement effective measures to reduce ID and associated risks. This may sustainably reduce the disease burden induced by ID for young women, pregnant women and their offspring.

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 Compare the Relative Potency of Salbutamol Administered Via Metered Dose Inhalers (MDI) Containing Propellants HFA-152a to HFA-134a in Mild Asthmatics Aged 18 to 65 Inclusive

Study to Assess the Efficacy, Safety, and Tolerability of NOC-110 in Adults with Refractory or Unexplained Chronic Cough

Approximately 325 participants will take part in the study. It is anticipated that up to 600 participants will be screened. Participation will be approximately 13 weeks.

Hydra Registry - UK

This is a prospective, observational, multi-centre study. The aim of this study is to collect clinical, procedural, and follow-up data in order to evaluate the efficacy and safety of the Hydra THV series (Vascular Innovations Co. Ltd., Nonthaburi, Thailand). Additionally, the study will conduct the following evaluation: 1. The remote ambulatory cardiac monitoring (rACM) using the Checkpoint Cardio System, consisting of 72 hours of continuous electrocardiogram (ECG) monitoring, will be evaluated post hospital discharge where it is clinically appropriate. 2. Coronary access will be evaluated immediately post-TAVI by attempting to selectively engage both the right and left coronary arteries through standardised angiographic views. This will be done in a sub-set of patients who are either known to have coronary artery disease, did not have coronary angiogram prior to TAVI or where it is felt to be clinically appropriate for other reasons post implant. Following discharge, clinical follow-ups are scheduled at 45 days (±14 days), and 1-year (±30 days).

Revumenib in Combination With Azacitidine + Venetoclax in Patients NPM1-mutated or KMT2A-rearranged AML

Cervical Spine Focused Treatment for Patients with Persistent Concussion Symptoms and Neck Pain

This is a a single site pilot randomized controlled trial consisting of a total of 40 participants with concussion and neck pain, randomized to one of two groups (n=20 per group): 1. early cervical spine focused treatment combined with standard concussion care. 2. standard concussion care alone for 4 weeks, followed by delayed introduction (after 4 weeks) of cervical spine focused treatment (which is the usual care workflow at our clinical partner site.) Both groups will receive the combination of cervical spine focused treatment and standard concussion care; the only variation being the timing of the introduction of the cervical spine care. The primary endpoint will be 4 weeks from start of cervical rehabilitation intervention. The secondary would be the point of discharge. The duration of active participation in the study will be 8 weeks for the participants.

A Study on the Immune Response, Safety and the Occurrence of Respiratory Syncytial Virus (RSV)-Associated Respiratory Tract Illness After Administration of RSV OA Vaccine in Adults 60 Years and Older

Clinical Epidemiology in Contemporary Patients With Myelofibrosis.

Myeloproliferative neoplasms (MPNs) are rare bone marrow disorders characterized by clonal proliferation of hematopoietic cell lineages, and include polycythemia vera (PV), essential thrombocythemia (ET), and myelofibrosis (MF). MF has worse prognosis, with main causes of death including acute leukemia transformation, comorbid conditions, and consequences of cytopenia. MF is characterized by progressive anemia, bone marrow fibrosis, and extramedullary hematopoiesis with splenomegaly. Moreover, the disease is associated with a heavy symptom burden including night sweats, fever, bone pain, and weight loss and worsening the quality of life. On the beginning of 2013 the European Registry for Myeloproliferative Neoplasms (ERNEST) observational study was launched and approved by several IRBs of European hematological centers. The study focused on overt Primary (PMF) and Secondary myelofibrosis (SMF; i.e., post-Essential Thrombocythemia myelofibrosis (post-ET MF) and post-polycythemia (post-PV MF)) and aimed at describing the clinical epidemiology of large series of patients observed in clinical practice. This research was justified as the landscape of both pathophysiological and clinical knowledge in MPNs was rapidly evolving, prompting to revise diagnostic criteria, prognostication and therapy recommendations. ERNEST retrospectively enrolled 1292 patients in whom the proposed prognostic models were confirmed to differentiate treatments in clinical practice, while ERNEST-2 reported results on critical events observed in 1010 of these cases during a median follow-up period of 5.4 years.4,5 The two studies closed in December 2022. In the last decade, new diagnostic and prognostic findings have been accumulated and the availability of new approved drugs, based on results of several new clinical trials, influenced the therapy decision making in the real-world clinical practice. Therefore, the continuation of observational studies in present ERNEST-3 on large multicenter case series of patients with MF is timely and might refine the results of clinical trials. The purpose of this study observational retrospective/prospective study is to gain information on MF associated cytopenias that represent a significant challenge in the contemporary patients with MF. Currently, there are few agents aimed at treating cytopenic MF, including immunomodulatory drugs, hypomethylating agents, and JAK inhibitors such as momelotinib and pacritinib, and development of new agents specifically tailored to this patient population remains an unmet need. Therefore, this study can provide data on these patients, focusing on clinical status, quality of life, comorbidities, and treatment results over time.

A Study on the Safety of TAK-279 and Whether it Can Reduce Inflammation in the Bowel of Participants With Moderately to Severely Active Crohn's Disease

The drug being tested in this study is TAK-279. TAK-279 is being tested to treat participants with moderately to severely active Crohn's disease. The study will look at the efficacy and safety of TAK-279. The study will enroll approximately 268 participants. During the Induction Period participants will be randomly assigned to one of the following treatment groups in a ratio of 1:1:1:1 to receive TAK-279 or placebo which will remain undisclosed to the participant and study doctor during the study (unless there is an urgent medical need): 1. TAK-279 Dose 1 2. TAK-279 Dose 2 3. TAK-279 Dose 3 4. Placebo This multi-center trial will be conducted globally. The overall study duration is approximately 60 weeks including a 4-week safety follow-up period.