The primary objective of the SafeBoosC trial is to examine if it is possible to stabilise the cerebral oxygenation of extremely preterm infants during the first 72 hours of life through the application of cerebral NIRS oximetry and implementation of an rStO2-specific clinical treatment guideline. We hypothesise that by using the specified treatment guideline to respond to cerebral monitoring readings outside the target range, we would reduce the burden of hypo- and hyperoxia and consequently reduce brain injury.

This is an investigator-initiated randomised, blinded, multinational, phase II feasibility clinical trial involving preterm infants from 12 European countries.

Intervention type: diagnosis

In a multicenter, open-label, randomized controlled trial we aim to demonstrate that an early switch from intravenous to oral antimicrobial therapy is non-inferior to a conventional 14-days course of intravenous therapy regarding efficacy and safety. An early switch from intravenous to oral therapy would provide several benefits such as earlier discharge, fewer adverse reactions associated with intravenous therapy, increased quality of life, and cost savings.

Population: adult | Intervention type: therapeutic medicinal product

The aim of this study is to establish which treatment is best for children with Electrical Status Epilepticus in Sleep (ESES) syndrome, by treating 130 children with ESES syndrome with steroids (inflammation inhibitors) or clobazam and evaluating change in cognitive functioning after 6 and 18 months.

Intervention type: therapeutic medicinal product

This is a multicentre European double-blind, randomized and controlled trial with 2 parallel groups (1 study medication, 1 placebo) in order to analyse the impact of ACE inhibitors (ACEi) in subjects who carry a mutation but have not yet developed DCM (dilated cardiomyopathy). Objective of the trial: Study the impact of ACE inhibitors (ACEi) in subjects who carry a mutation (leading to a genetic form of heart failure) but have not yet developed DCM.

Population: adult | Intervention type: therapeutic medicinal product

Premature babies are at high-risk of respiratory failure due to respiratory distress syndrome (RDS). Surfactant given into the trachea is an effective treatment for infants who are intubated for RDS. Endotracheal intubation is difficult and is associated with adverse effects. A strategy of supporting premature newborns with continuous positive airway pressure (CPAP) and reserving intubation and surfactant for those who develop respiratory failure despite CPAP yields better results than intubating all infants for surfactant. However, nearly half of infants managed with CPAP are ultimately intubated for surfactant.
We will establish whether giving preterm infants surfactant into their oropharynx at birth, with the intention that they aspirate it, reduces the rate of intubation in the first 5 days of life.Infants born before 29 weeks gestation will be eligible for inclusion in this multi-centre randomised trial. Written informed consent will be obtained before delivery. Infants will be randomly assigned to receive oropharyngeal surfactant immediately at birth in addition to CPAP or standard care with CPAP alone. Caregivers will not be masked to group assignment.
Randomisation will be stratified by centre and gestation at birth (<26 weeks; 26-28 weeks). Primary outcome of the study is intubation for respiratory failure in the first 120 hours of life. Enrolled infants will be intubated for persistent apnoea and/or bradycardia in the delivery room (DR), or for respiratory failure in the neonatal intensive care unit (NICU) defined as ≥ 2 of worsening clinical signs; acidosis; oxygenation – FiO2 > 0.4; hypercarbia; or apnoea treated with mask ventilation. To demonstrate a reduction in the rate of the primary outcome from 48% in the control group to 26% with oropharyngeal surfactant with 80% power and a = 0.05, we need to recruit 250 infants. Adverse events will be recorded and measure secondary outcomes of clinical interest (rate of DR intubation; duration of ventilation and respiratory support; rate of bronchopulmonary dysplasia at day 28 and chronic lung disease at 36 weeks’ corrected age; mortality before hospital discharge). All outcomes will be determined at or before hospital discharge; this duration will vary according to clinical course [e.g. for 1 day (for participants who die shortly after enrolment) to several months]. Poractant alpha (Curosurf, Chiesi Farmeceutici, Parma, Italy) is a natural surfactant prepared from porcine lungs. It is approved for endotracheal use for prevention and/or treatment of RDS in premature infants. It is currently not licensed for oropharyngeal administration, therefore we will examine the off-label use of a licensed product. It is a randomised, parallel group, controlled Phase III trial.

Population: paediatric | Intervention type: therapeutic medicinal product

Achalasia is a rare esophageal disorder which is characterized by a loss of esophageal motoneurons and impaired gastrointestinal peristalsis and food transit. Peroral Endoscopic Myotomy (POEM) is a new endosurgical technique for achalasia in which a surgical myotomy is created using a flexible endoscope through the mouth, completely avoiding any skin incisions. The aim of this study is to compare short and long-term feasibility, safety and efficacy of endoscopic (POEM) with laparoscopic myotomy (Heller myotomy) in the treatment of achalasia.

Intervention type: therapeutic procedural

Prader-Willi syndrome (PWS) is a rare chronically debilitating and life-threatening disease without efficient treatment. It is a genetic neurodevelopmental disorder with neonatal poor feeding, requiring nasogastric tube feeding, and social skills followed by early onset of obesity, severe behavioural disturbances and comorbidities. Pre-clinical data supported the hypothesis that OXT rescues early phenotype and modifies the course of the disease via its potential neuromodulation effect, if administered in a post-natal short window of opportunity. This is a European phase III randomised double-blind placebo trial, already approved by EMA, evaluating the effects of early intranasal OXT in PWS neonates/infants. This trial is coordinated by Pr M Tauber (France) and sponsored by the Centre Hospitalier Universitaire de Toulouse will be conducted in 5 European countries (France, Belgium, Italy, Netherlands, and Germany).

Population: paediatric | Intervention type: therapeutic medicinal product

Cancer cachexia is a multi-factorial syndrome defined by an ongoing loss of skeletal muscle mass (with or without loss of fat mass) that cannot be fully reversed by conventional nutritional support and leads to progressive functional impairment. There is an urgency for improving management, but there is no consensus on the optimal treatment for cancer cachexia. Several single therapies for cancer cachexia have been examined in clinical trials, with disappointing overall results. As multiple factors are responsible for the development of cachexia, it has been argued that optimal cachexia interventions should target all components: multimodal therapy for a multimodal problem. The overall aim of this study is to early prevent the development of cachexia rather than treatment late in the disease trajectory. From a patient perspective a short term effect will be to improve physical and psychological function, to reduce symptom burden and to improve survival. In other words live a longer and better life during and after chemotherapy. Direct effects of the cachexia intervention are expected to be reduction of weight and muscle loss, and improved physical activity and quality of life.

Population: adult | Intervention type: therapeutic medicinal product

Non-alcoholic fatty liver disease (NAFLD) is responsible for an increasing prevalence of liver disease and is becoming the commonest cause of liver disease in the western world. NAFLD is recognised to be the hepatic manifestation of the metabolic syndrome, which is a cluster of metabolic abnormalities characterised by abdominal obesity, insulin resistance, impaired glucose metabolism, hypertension and dyslipidaemia. In its mildest form there is an accumulation of fat in the liver (steatosis) without any liver damage, however in many cases it progresses to non-alcoholic steatohepatitis (NASH), and cirrhosis. Current treatment options for NASH are limited in efficacy, necessitating the development of more effective options. New agents such as Glucagon-like Peptide-1 (GLP-1) agonists that improve diabetic control and facilitate weight loss have been suggested as therapies in NASH. No published studies to date have assessed the impact of the GLP-1 agonist, Liraglutide, on liver histology and metabolism in obese patients with NASH. This study hypothesises that treatment with liraglutide will result in a significant improvement in histological disease activity in overweight patients with NASH, in the presence or absence of Type 2 Diabetes (T2DM)

Population: adult | Intervention type: therapeutic medicinal product

The purpose of this study is to prolong the time to reinfection with Pseudomonas aeruginosa after successfully treated acute or intermittent infection.

Intervention type: therapeutic medicinal product