Clinical Research Directory

Efficacy and Safety of Subcutaneous Dupilumab in Participants With Asthma/Asthmatic Wheeze Aged 2 to <6 Years (LIBERTY ASTHMA TREKIDS)

This is a parallel, Phase 3, 2-arm study to evaluate the efficacy and long-term safety of dupilumab treatment in children 2 to \<6 years of age with uncontrolled asthma and/or recurrent severe asthmatic wheeze. The study will be conducted in 2 parts. Part A will be a 52-week, randomized, double-blind, placebo-controlled study to assess the safety and efficacy of dupilumab in children aged 2 to \<6 years old with uncontrolled asthma and/or recurrent severe asthmatic wheeze. At the end of Part A, all eligible participants will be offered participation in Part B, an optional open-label extension phase. Study details include: Part A: The study duration of part A will be up to 68 weeks consisting of a 4-week Screening, a 52week treatment period, and a 12-week post-treatment follow-up period. For participants who will chose to participate in Part B, the study duration will be up to 120 weeks (additional 52-week treatment period). Part B: For participants who will choose to participate in Part B, the study duration will be up to 120 weeks (Part A \[4-week Screening and a 52-week treatment period\] plus additional 52-week treatment period and a 12-week post-treatment follow-up period).

Bacterial Lysate In Preventing Asthma

Bronchiolitis is a common viral infection of the small airways of infants and some affected infants will require hospital admission. Severe bronchiolitis is a marker for greatly increased risk of developing both preschool wheeze and subsequent school age asthma. Since epidemiological studies suggest that exposure to microbial products protects against preschool wheeze, lysates of bacteria may prevent the development of wheeze after bronchiolitis, with long-term beneficial consequences. BLIPA is a phase 2b, randomised, double blind, placebo-controlled study, investigating the efficacy superiority of bacterial lysate (Broncho Vaxom) capsules over placebo, in reducing wheeze in infants after severe bronchiolitis. The primary end point of the study to establish whether there is superiority of oral Broncho-Vaxom over placebo in reducing the number of parent-reported wheeze episodes by 12 months post IMP/placebo initiation. The study aims to test bacterial lysate capsules (3.5mg over 12-24 months) for safety, efficacy, and to advance mechanistic understanding of its action.

Breast Milk: Influence of the Micro-transcriptome Profile on Atopy in Children Over Time

This is an observational cohort study of 221 breast-feeding mother-infant dyads delivered at term. The goal of the study is to investigate whether levels of immune-related microRNAs (miRNAs) in maternal breast milk (MBM) influence child atopy risk in the first 12 months, defined as atopic dermatitis, wheezing, or food allergy. Infant exposure to individual miRNA components will be quantified at 0, 4, and 16-weeks after delivery using high throughput RNA sequencing of MBM samples and detailed dietary logs employing the Infant Feeding Practices (IFP) survey. The relationship of individual miRNA exposures (parts per million) and presence/absence of atopy in the 48 weeks after delivery will be assessed, while controlling for environmental exposures (National Survey of Lead hazards and Allergens in Housing), maternal diet, and genetic predisposition. Potential transfer of MBM miRNAs to the infant oropharynx and subsequent impact on immune reactivity will also be explored through RNA sequencing of infant saliva and quantification of cytokine profiles.

Soy Isoflavones For Inner City Infants At Risk For Asthma (SIRA) Study

The goal of this clinical trial is to compare soy isoflavones to placebo in children who at risk of asthma and have a genetic variation which results in them making more of a pro-inflammatory protein, plasminogen activator inhibitor-1. The main questions this trail seeks to answer is: will soy isoflavones decrease the proportion of infants with aT2 high endotype at the end of treatment, and secondarily decrease the number of wheezing episodes in these children when given in the first year of life. Participants will be asked to ingest soy isoflavone or placebo twice daily mixed into a liquid or puree vehicle for 7 months from randomization. There will be 3 mandatory in-person visits, and 6 virtual visits in the first year. There will also be 11 monthly questionnaires and 1 in person visit in the observation year. Participants will have 4 nasal swabs, 3 blood draws, and also provide 4 stool samples over the course of the study.

Critical Windows in the Development of Asthma Endotypes and Phenotypes in High-Risk Toddlers

The purpose of this study is to learn more about how asthma develops in early childhood. This will help doctors understand how to prevent and treat asthma better.

Fish Oil in pREgnancY for Personalized Prevention of Early Childhood Asthma

The overall aim of the study is to develop a nutritional preventive fish oil supplementation strategy in pregnancy for early childhood asthma/persistent wheeze during the first three years of life as we hypothesize that both supplementations in higher doses than recommended could reduce the risk of disease development.

VItamin D in pregnanCy for prevenTion Of eaRlY Childhood Asthma

The overall aim of the study is to develop a nutritional preventive vitamin D supplementation strategy in pregnancy for early childhood asthma/persistent wheeze during the first three years of life as we hypothesize that supplementation in higher doses than recommended could reduce the risk of disease development.

Validate the Efficacy and the Safety of the Keikku Electronic Stethoscope for Its Indication and Intended Purpose, Under the Intended Conditions of Use.

This study is testing a digital stethoscope called the Keikku electronic stethoscope. Doctors use stethoscopes to listen to the heart, and this new device records heart sounds using a smartphone app. The purpose of the study is to check how well this new device works and whether it is safe to use. The study will include 149 participants of all ages (from birth to 99 years), who are undergoing routine (non-urgent) heart checks. After giving consent, each participant will have their heart sounds listened to twice: once with the Keikku electronic stethoscope and once with a regular stethoscope. Two different doctors will do the listening, and they won't share information with each other. The doctors will record whether they hear a heart murmur and how clear the sounds were. The study will compare the findings from the new device to those from the regular stethoscope. Researchers will also check for any device problems or side effects. The study takes place at a single site in Peru and will take about one day for each participant. The goal is to find out if the Keikku electronic stethoscope is accurate and safe for doctors to use in real medical settings.

Swiss Paediatric Airway Cohort

The Swiss Paediatric Airway Cohort (SPAC) is a national, prospective clinical cohort of children and adolescents who visit physicians in Switzerland for recurrent wheeze, cough, and exercise- or sleep-related respiratory problems. SPAC aims to answer important questions on clinical phenotypes, prognosis, diagnosis and treatment. SPAC is part of routine care, and only clinically indicated investigations are done. The comprehensive baseline assessment includes a detailed questionnaire to families, plus test results, diagnoses and treatments from hospital records. Follow-up is via monthly questionnaires the first 12 months and thereafter annual questionnaires to families, and data from follow-up visits. Currently, 4344 patients from 10 clinics and hospitals in Switzerland (Aarau, Basel, Bern, Chur, Horgen, Lausanne, Luzern, St. Gallen, Worb, Zurich) have been enrolled. SPAC provides real-life data on children visiting the Swiss health care system for common respiratory problems. It will provide a research platform for health services research, and for nested clinical and transitional studies. Publications and plain language summaries are listed on the study website: https://www.spac-study.ch/publikationen/

PREcision Medicine Directed Corticosteroids In Children With preSchool Wheeze

Young children frequently attend the emergency department (ED) with wheeze which is usually triggered by a virus infection, such as the common cold. Wheeze can be treated with inhaled medications and sometimes oral steroid medicines are also given to reduce swelling within the lungs. Unfortunately, oral steroids can have side effects. Despite lots of research there is no clear evidence that oral steroids work in young children with a wheeze attack. It is likely that some children get better quicker with oral steroids but deciding who to treat is difficult. This results in differences in care with some children receiving unnecessary oral steroids and others not receiving them when they could benefit. The investigators hypothesise that in a subgroup of children with mild-to-moderate acute wheeze attacks, the viral trigger can predict their response to steroid medications. Previously, respiratory virus testing was too slow to be used to inform treatments in the ED. Now, point-of-care (POC) viral tests can provide results within 30 minutes. There is a research gap regarding the role of these tests in determining steroid responsiveness and outcomes in children presenting with acute wheeze in the pre-school population. In order to address the hypothesis in a future definitive trial, the feasibility of using (POC) viral tests to randomise steroid treatments for children in a clinical study in the ED setting must be ascertained. The PRECISE Study will therefore be a single centre randomised, feasibility study enrolling approximately 60 pre-school aged children to inform a future definitive multi-centre Randomised Controlled Trial.

Vitamin C to Decrease Effects of Smoking in Pregnancy on Infant Lung Function (VCSIP) Longer Term Follow Up

The overall aims of this protocol are to determine whether prenatal supplementation with vitamin C to pregnant smokers can improve pulmonary function at 10 years of age in their offspring. This is an additional continuation of the Vitamin C to Decrease Effects of Smoking in Pregnancy on Infant Lung Function (VCSIP) trial, to follow the offspring through 10 years of age. The hypothesis for this protocol is an extension of the VCSIP trial that supplemental vitamin C in pregnant smokers can significantly improve their children's airway function tests. The investigators aim to demonstrate sustained improvement in airway/pulmonary function and trajectory through 10 years of age.

Video Assisted Study of Salbutamol Response in Viral Wheezing

The study is video assisted cross-over study evaluating the effect of inhaled salbutamol in wheeze in children 6-24 months old.

Phenotypes of Preschool Wheezing Among Children Attending Sohag University Hospital

Wheezing in preschool children is very common, with a wide differential diagnosis. It is essential to be sure of the exact sound that parents are describing; the term 'wheeze' is often applied to non-specific sound.Approximately one-third of children are diagnosed with wheeze in the first 3 years of life, making wheeze one of the commonest respiratory symptoms.The differential diagnosis of wheeze is wide, and different management strategies are needed depending on the underlying phenotype. The word 'wheeze' is used to describe many different sounds. That can be heard by both clincians and parent. Even if true wheeze is heard, this should not be automatically assumed to be due to bronchospasm. Airway narrowing by mucus will produce true wheeze but does not respond to bronchodilators. Similarly, airway malacia, either related to intrinsic airway wall defects or loss of alveolar tethering points, are also causes of bronchodilatorunresponsive wheeze; indeed, bronchodilators, by reducing airway smooth muscle tone, may actually worsen airway narrowing.

Protecting Preterm Infants From Respiratory Tract Infections and Wheeze by Using Bacterial Lysates.

The primary objective of this study is to reduce respiratory tract infections and wheezing in moderate-late preterms in the first years of life by bacterial lysate administration. Next to determine the correlation of biological markers with respiratory symptoms, immune protection and treatment effect.

Prospective Multicentre Study on Symptoms in First-onset Bronchial Asthma in Children and Adolescents

Bronchial asthma may present with symptoms other than the commonly reported complaints (cough, chest tightness, shortness of breath and wheezing). Less common symptoms include chronic or recurrent productive cough, inspiratory dyspnoea or recurrent pneumonia. Children presenting with these symptoms are often diagnosed with asthma bronchiale and benefit from antiasthmatic management.

Luchtbrug Junior: the Next Step

Rationale: The introduction of e-health in the monitoring and management of patients with chronic conditions can be beneficial and efficient. The introduction of an online monitoring and management tool, "Luchtbrug", for children 6-16 yrs of age with asthma while reducing 50% of visits to the outpatient clinic, results in similar or improved asthma control while reducing costs. It is highly likely that this concept of e-health is suitable for other chronic conditions. Therefore the aim of the study is to investigate the added value of online monitoring and management of children with preschool wheeze (2-6 yrs of age). Objective: To assess whether the number of symptom free days can be improved using online monitoring and disease management via Luchtbrug Junior, while reducing the number of outpatient clinic visits by 50% Study design: Prospective multicentre (n=6) randomised trial in which disease management via Luchtbrug Junior will be compared with usual care. Follow-up: 12 months. Study population: children aged 2-6 yrs with preschool wheezing (multiple episode of cough, wheeze and dyspnoea). Intervention (if applicable): Children will be randomised into usual care (n=135) or partly online care via Luchtbrug Junior (n = 135) Main study parameters/endpoints: Primary objective: Number of symptom free days (SFDs) based on the TRACK questionnaire, during the last four weeks of the study (measured at 12 months). Secondary objectives: Healthcare consumption: unscheduled visits Emergency Department (ED) or outpatient clinic, unscheduled phone calls, hospital admissions) Questionnaires into: Quality of Life (QoL), cost-effectiveness (direct and indirect costs), self-management of patients, adherence to treatment, satisfaction of parents/caregivers. Usual care consists of regular visits to the outpatient clinic every 3 months after starting the study. In between visits contact with the healthcare team is by telephone as needed. At 3, 6, 9 and 12 months, patients will be asked to complete the TRACK questionnaire. Online care using Luchtbrug Junior will include visits to the outpatient clinic every 6 months after starting the study. In between visits, parents/caregivers of the patient will be asked to answer the digital version of TRACK questionnaire monthly, therefore the parents/caregivers will received email-reminders. Appropriate feedback on their TRACK-scores will be provided by their healthcare team within two working days. Any interventions such as adjustment of treatment are at the judgment of the treating pediatrician. Therapy adherence will be monitored during the study by using smart inhalers.

Early Life Intervention in Pediatrics Supported by E-health - SMOKE

Unrestricted parental smoking habits are lifestyle risk behaviors associated with chronic, systemic inflammation, which promotes the pathogenesis of non-communicable diseases (NCDs). Exposure to secondhand tobacco smoke in early life contributes to the development of specific NCDs in children, i.e. asthma. Early preventive measures to improve lifestyle behavior in parents are therefore of utmost importance. The aim of ELIPSE-II is to assess whether an eHealth application intervention for parents is feasible and efficacious in lowering levels of urinary cotinine as a measure of second-hand smoke exposure in their children .

Longitudinal Preschool Wheeze Biomarker Study

Preschool children (aged 1-5 years) account for 75% of all UK childhood wheezing hospitalisations. This has not changed over 20 years, meaning current treatments are not working and a new approach is needed. Currently, all preschool wheezers are treated with inhaled steroids. However, only about 25% of patients, with allergies, respond well to inhaled steroids; for the other 75%, they are ineffective. This research group has found that some preschool wheezers may have other causes but there are no specific, non-invasive tests to match the right treatment to each child. The goal of this observational study is to test various bedside tests for this purpose in preschool children with wheeze, to see if they are feasible, accurate and acceptable in this age group. The research team would like to investigate the following aims: Aim 1 - To test the proposed panel of simple bedside tests below, to see how accurately they corelate with lower airway infection or inflammation. Aim 2 - To test the acceptability of these bedside tests are to parents and children, and if they reflect the child's symptoms, symptoms control and medication use. Aim 3- A small proof-of -concept study, to test if these simple bedside tests, can be used to determine treatment for each individual child. The panel of simple non-invasive tests that the research team are proposing are: 1. Skin prick tests to common allergies (house dust mite, cat, dog, grass, tree pollen, mixed moulds) 2. Finger prick blood test 3. Phlegm test for bacteria 4. Nose and throat swab for bacteria 5. Lung function test called forced oscillation technique (FOT)

The Gut-Lung Axis and Respiratory Illness in Children

The goal of this single-centre observational study conducted at the Royal Hospital for Children in Glasgow, Scotland, is to employ a multi-omics approach to investigate the "gut-lung axis" in health and disease. Part A is a cross-sectional study design investigating the postulated bidirectional link between the gut and lung microbiomes in children suffering from respiratory or gastrointestinal conditions. Children with no GI or respiratory issues attending for orthopaedic care will be used as a benchmark for a healthy gut-lung axis. The main questions we aim to answer are: * What does a healthy gut-lung axis look like? * Do children with respiratory issues show an altered gut microbiome? * Do children with GI issues show an altered lung microbiome? Part B is a longitudinal study design, that aims to assess the effects of biologics on the gut-lung axis by comparing the gut and lung microbiomes in children with asthma at two time-points who are indicated to start biologics therapy (Asthma treatment) or will not receive biologics therapy (asthma control). Participants will provide: * airway samples (to investigate the lung microbiome) * blood samples (to assess inflammatory and metabolic factors which may mediate communication between the two sites) whilst under general anaesthetic for a treatment related to their standard of care * stool samples (to assess gut microbiome) * dietary information (food diary and/or food frequency questionnaire) to assess relationships between diet and the gut-lung axis.