Paediatric Studies

Emergency treatment with levetiracetam or phenytoin in status epilepticus (1603216)

Most epileptic seizures and convulsions in children last less than three minutes and will stop on their own accord. However, on occasion, a seizure may continue for longer than three minutes and eventually become what is called convulsive status epilepticus (CSE). This is a medical emergency. To prevent CSE from happening, children are given an antiepileptic medicine called an emergency or rescue medicine (also known as first-line treatment). owever, this treatment will only be successful in around half of all children. In those cases where the rescue medicine is not successful, the children need to be taken to the Accident and Emergency Department (AED) of their local hospital. Once there, if the child is still in the seizure, they are given a different rescue medicine. This again will be successful in stopping the seizure in about half of the children. For those that are still in seizure, a different medicine is then given (this medicine is known as second-line treatment). The usual medicine given at this stage is called phenytoin. 

Clinical Safety & Efficacy of a New Infant Formula With Specific Medical Purpose Containing Human Milk Oligosaccharides (1706444)

Infants with physician diagnosed CMPA, aged between birth and 6 months of age will take either the control or new test infant formula for 4 months and if judged suitable by physician, up to maximum of 12 months of age. Growth, adverse events, medication use and tolerance to formula will be assessed. As part of exploratory objectives, the study will also explore possible mode of action of the Test formula in CMPA infants, by assessing whether consumption of Test formula by CMPA infants affects stool microbiota and metabolic signatures as well as urine metabolic signatures and whether such changes can be associated to the intestinal inflammatory/health status, and the clinical measures.

A study to test the methods and costs of an online programme for adolescents who have survived a brain injury: Teen online problem solving (TOPS-UK) (1705425)

The clinical and cost-effectiveness of Teen Online Problem Solving for adolescents who have survived an acquired brain injury in the UK (TOPS- UK): A feasibility study.

RESULT: Reliable, Emergent Solution Using Liprotamase Treatment (1802572)

Liprotamase powder is a non-porcine, soluble and stable mixture of biotechnology-derived lipase, protease, and amylase digestive enzymes. The purpose of the present study is to to evaluate the non-inferiority of liprotamase compared with porcine-derived, enterically-coated pancreatic enzyme replacement therapy (PERT). The primary efficacy endpoint of the study will be comparative efficacy measured as the change from baseline in the coefficient of fat absorption (CFA) in Cystic Fibrosis patients with exocrine pancreatic insufficiency (EPI).

United Kingdom Paediatric Chronic ITP Registry (806162)

The objective of the registry is to establish a prospective registry of children with immune thrombocytopenic purpura (ITP) in the UK . The prime aim is to relate the long term consequences of a low platelet count on the frequency and severity of bleeding symptoms and requirement for treatment. Adults are not included in this application as there is already a registry established for adults in the UK.

UK TTP Registry (1002349)

The UK TTP registry, involves all sites treating newly presenting Thrombotic Thrombocytopenic Purpura (TTP). From this registry, important epidemiological data will be obtained. Admission and remission samples will be collected. DNA will be collected and analysed from patients wishing to participate to determine if any link exists between mutations/polymorphisms and the risk of TTP.

University College London (UCL) Haemostasis Research Unit (HRU) will collect and collate the data and help administrate for those sites participating in the registry. 

PREDNOS 2 (1310849)

Steroid-sensitive nephrotic syndrome (SSNS) is the most common kidney disease of childhood. Large amounts of protein are leaked into the urine resulting in generalised oedema (swelling). It is treated with high-dose oral prednisolone, a steroid drug which is effective, though associated with a number of serious side effects. Following successful initial treatment, 70-80% of children develop relapses where leakage of protein into the urine recurs. These are associated with a risk of significant complications. Relapse of nephrotic syndrome is treated with a further course of high-dose prednisolone, further increasing the risk of side effects. Children are kept off school, resulting in educational impairment and parental absence from work.

RAACENO (1802575)

Asthma is a long-term condition which affects the airways. When a person is suffering from asthma, the airways are extremely sensitive (hyperresponsive) to both natural chemicals the body produces and irritants outside the body, such as dust or pollen. Coming into contact with these substances can cause an asthma attack (also known as an exacerbation), which involves feelings of tightness in the chest as the airways become inflamed (swollen), causing coughing, wheezing, chest tightness and difficulty breathing.

Every year in the UK, 150,000 children see their family doctor for an asthma exacerbation and 25,000 are hospitalised. One third of the £1 billion NHS budget for asthma is spent on provision for unscheduled care of which about one half is for childhood exacerbations. Exacerbations are relatively infrequent and short-lived but their importance to patients is emphasised in the Global Initiative for Asthma whose major goals include “to prevent asthma exacerbations”.

Everyone breathes out a gas called nitric oxide. Exhaled nitric oxide can be measured using a special breathing device. People with asthma breathe out more nitric oxide than people without asthma because nitric oxide is produced by the allergic cells which are present in the lungs of people with asthma. These allergic cells build up before an asthma attack. The aim of this study is to find out whether measuring fractional exhaled nitric oxide (FeNO) can guide asthma treatment and help prevent asthma attacks.