Clinical Research Directory

Use of Mesenchymal Stem Cells in Pre-term Patients With Bronchopulmonary Dysplasia.

Bronchopulmonary dysplasia (BPD) is a disease that affects preterm newborn patients, preventing their lungs from developing properly. Allogeneic fetal stem mesenchymal cells from umbilical cord could reduce the prevalence of BPD in this patients.

Efficacy and Safety of Zelpultide Alfa in Preterm Neonates at High Risk of Developing Bronchopulmonary Dysplasia (BPD)

This is a randomized, parallel-group, double-blind, placebo-controlled multicenter phase 2b/3 study with an adaptive seamless design. The goal fo this study is to determine if an investigational drug, Zelpultide Alfa, can reduce the occurrence of Bronchopulmonary Dysplasia (BPD) in extremely premature babies. The study comprises 2 parts: * Part 1: Phase 2b, dose selection and exploratory efficacy and safety. * Part 2: Phase 3, confirmatory efficacy and safety. In Part 1, the study subjects will be randomized with a 1:1:1 allocation ratio to either : 1. Standard of care + zelpultide alfa 4 mg/kg or, 2. Standard of care + zelpultide alfa 6 mg/kg or, 3. Standard of care + placebo (air-sham). In Part 1, all three arms will be evaluated descriptively to support dose selection based on safety, tolerability, and exploratory efficacy signals. Upon completion of Part 1, the DSMC will recommend which Phase 2b dose ("selected dose") to progress into Part 2 to the Study Steering Committee, which will decide the dose for Part 2 (Phase 3). A sample size reassessment will be performed after Part 1 completion. In Part 2, the selected dose of zelpultide alfa will be compared against placebo (air-sham) in a confirmatory analysis on the primary and key secondary endpoints. The study subjects will be randomized with a 1:1 allocation ratio to either: 1. Standard of care + zelpultide alfa (selected dose from Part 1), or 2. Standard of care + placebo (air-sham). The main objective in part 2 is to compare the efficacy of zelpultide alfa added to standard of care versus standard of care plus placebo (air-sham) in terms of incidence of grade 2 and grade 3 bronchopulmonary dysplasia (BPD) and death in neonates at high risk for developing BPD. In both parts, treatment will be administered intratracheally. Participants will receive up to 7 administrations of zelpultide alfa at (4mg/kg or 6 mg/kg) or air-sham in 24 h intervals while the subjects are still intubated per standard of care.

Pulmonary Hypertension and Oxygen Saturation Targeting in Preterm Infants

Around 50% of infants born extremely preterm develop a chronic lung disease known as bronchopulmonary dysplasia of which some infants will also develop pulmonary hypertension of which 50% of children will die before the age of 2. Physicians are currently limited in their ability to select the most appropriate oxygen targets that will improve outcomes in infants with this condition. This clinical trial will determine whether using different amounts of oxygen improve outcomes in infants with this disease.

Preliminary Study on Microplastics Deposition in Ventilator Circuit and the Risk of Bronchopulmonary Dysplasia in Premature Infants

In this study, the premature infants with gestational age less than 30 weeks were taken as the object, and the deposition characteristics of microplastics in ventilator circuit and its correlation with the risk of bronchopulmonary dysplasia were systematically discussed through prospective observation design. The research is divided into two parts: firstly, the release and deposition of microplastics in the ventilator circuit (water storage tank and simulated lung) are detected; Secondly, the changes of microplastics load in airway secretions of children with bronchopulmonary dysplasia on the 0-7th day, 14th day and 28th day were dynamically monitored, and samples of bottle milk and newborn blood were collected simultaneously to evaluate the exposure level of multi-channel microplastics. Microplastics was qualitatively and quantitatively analyzed by confocal micro-Raman spectroscopy, and the dose-response relationship between microplastics load and bronchopulmonary dysplasia was analyzed in combination with clinical data, so as to provide scientific basis for risk prevention and control of iatrogenic microplastics exposure in neonatal intensive care unit. In this study, the blood sample only used the remaining venous blood collected by routine clinical diagnosis and treatment (such as blood routine and blood gas analysis) on the 7th, 14th and 28th day (2 days) after the newborn was admitted to the hospital, and the dosage was 0.5-1 mL, which was placed in a glass anticoagulant tube. Each child is collected at most once. If there is no clinical blood collection at this time point, no additional puncture blood collection will be carried out to ensure that the operating burden and risk of children will not be increased.

High-energy Human Milk Diets in the First Two Weeks After Birth to Reduce BPD in Extremely Preterm Infants

This Phase II, parallel-group, masked, randomized clinical trial aims to evaluate whether a DHA/ARA-enriched, fortified human milk diet administered during the first 14 days of life reduces respiratory morbidity and improves lung function in extremely preterm (EPT) infants (born at ≤28 weeks gestation).

Predictors of Prolonged Mechanical Ventilation in Preterm Infants

This prospective cohort study aims to identify the independent predictors of prolonged mechanical ventilation in preterm infants admitted to the Neonatal Intensive Care Unit of Assiut University Children Hospital.

Prospective Multicentre Mixed Methods Study to Explore Extubation Practices and Respiratory Outcomes in Extremely Preterm Neonates.

The purpose of this observational study is to learn about neonatologists' perceptions of extubation readiness and extubation and reintubation practices in extremely preterm infants in the first 2 weeks of life using prospective qualitative and quantitative data. Actual extubation readiness is defined as successful extubation, defined as no reintubation in the 7 days following extubation. Key research questions are: How do clinicians assess extubation readiness in this population? Does this assessment correlate with actual extubation success? What factors (reasons, clinical status, ventilatory parameters) are associated with extubation readiness? Patients born before 28 weeks gestational age and admitted to the neonatal intensive care unit (NICU) within the first 24 hours are be included. The attending physician will complete a prospectively administered questionnaire with open-ended and multiple-choice questions to daily assess the decision and rationale for extubation or non-extubation of patients mechanically ventilated during the first 15 days of life. Patient characteristics, respiratory outcomes, and mortality will be recorded until the end of hospitalisation and/or definitive weaning from any ventilatory support or supplemental oxygen.

Inhaled Ciclesonide Study in Preterm Infants

Our overall objective is to conduct a safety study with inhaled ciclesonide to evaluate known glucocorticoids (sGC)-related acute and intermediate toxic effects while measuring for the first time in neonates its systemic absorption and potential bioactivity (i.e. activation of primary target, the GR, in blood cells).

Pharmacokinetics, Pharmacodynamics, and Safety Profile of Understudied Drugs Administered to Children Per Standard of Care (POPS)

The study investigators are interested in learning more about how drugs, that are given to children by their health care provider, act in the bodies of children and young adults in hopes to find the most safe and effective dose for children. The primary objective of this study is to evaluate the PK of understudied drugs currently being administered to children per SOC as prescribed by their treating provider.

Early Predictors of Bronchopulmonary Dysplasia in Extremely Preterm Infants

Bronchopulmonary dysplasia (BPD) remains one of the most common complications in extremely preterm infants despite advances in neonatal intensive care. Early identification of infants at high risk for BPD could facilitate individualized treatment strategies and improve long-term respiratory outcomes. However, current prediction models rely primarily on conventional clinical variables and have limited predictive accuracy. This prospective observational study aims to evaluate the predictive value of novel physiological, imaging, and biomarker-based indicators for the development of BPD in infants born before 26 weeks of gestation. Participants admitted to the neonatal intensive care units of the Medical University of Vienna will undergo non-invasive assessments during routine clinical care. These include respiratory function monitoring during neonatal transition, lung ultrasound, diaphragmatic ultrasound, targeted neonatal echocardiography, forced oscillation technique, neurally adjusted ventilatory assist-derived diaphragmatic electrical activity, electrical impedance tomography, and proteomic analyses of plasma and tracheal aspirate samples. The association between these novel indicators and respiratory disease severity will be assessed using the Respiratory Severity Score (RSS). Their ability to predict BPD will be evaluated using receiver operating characteristic (ROC) analysis and uni- and multivariable logistic regression models. Predictive performance will be assessed using the C-statistics. The primary objectives are to determine the correlation between the novel indicators and the RSS and to evaluate their diagnostic accuracy for predicting BPD. Secondary objectives include identifying the combination of indicators that provides the best prediction of BPD, evaluating longitudinal changes in respiratory and cardiovascular parameters during the neonatal period, assessing the effects of respiratory interventions and treatments on these indicators, and investigating associations with survival and major neonatal morbidities. A total of 140 extremely preterm infants is planned for inclusion in the study. The results are expected to improve early risk stratification and contribute to the development of individualized strategies for preventing and managing BPD.

Maternal Omega-3 Supplementation to Reduce Bronchopulmonary Dysplasia

The aim of this randomized controlled trial is to determine whether docosahexaenoic acid (or DHA, an omega-3 lipid) supplementation in lactating mothers providing breast-milk to their infant born below 29 0/7 weeks of gestational age (GA) improves BPD-free survival at 36 weeks post-menstrual age (PMA). Half of participants will receive docosahexaenoic acid (DHA), an omega-3 lipid, while the other half will receive a placebo.

Lipid Infusions to Optimize Nutrition Trial

The purpose of this study is to identify survival free of bronchopulmonary dysplasia (BPD), fatty acid profiles, and early biochemical measures for oxidative stress comparing mixed oil lipid emulsion (MOLE) vs soybean oil-based lipid emulsion (SOLE) and to establish whether MOLE or SOLE is more effective in minimizing pulmonary outcomes, neonatal morbidities, long-term morbidity and mortality, and improving discharge growth and Bayley Scales of Infant Development Fourth Edition (BSID-IV) neurodevelopmental assessment at two years

A Clinical Efficacy and Safety Study of OHB-607 in Preventing Bronchopulmonary Dysplasia in Extremely Premature Infants

The purpose of this study is to determine if an investigational drug can prevent Bronchopulmonary Dysplasia, reducing the burden of chronic lung disease in extremely premature infants, as compared to extremely premature infants receiving standard neonatal care alone.

Effects of a Physical Therapy Intervention on Motor Delay in Infants Admitted to a Neonatal Intensive Care Unit

Study Aims Pilot study: Due to the large recruitment goal and length of the project, the study team/PIs will evaluate the first cohort of 6-10 participants to refine study procedures and study-related materials. If no major modifications are made to the protocol as a result of this evaluation, data from these participants will be included for analysis. Aim 1: Evaluate the efficacy of an early, evidence-based, clinical experience-based therapeutic intervention (from the NICU to 12-months corrected age) on improving motor function and reducing severity of motor delays in infants at 12-months corrected age. The investigators hypothesize that the intervention group will demonstrate an average 8-point difference (0.5 standard deviation) compared to the standard of care group. \[an 8-point difference is considered a clinically meaningful difference\] Aim 2: Evaluate the early effects (i.e., before 12 months) of a therapeutic intervention, provided from NICU to 12-months corrected age, on motor function and severity of motor delay. The Investigators hypothesize that a statistically significant higher percentage of infants in the intervention group will demonstrate improved motor function and reduced severity of motor delays, compared to the standard of care group-assessed using sensors, the NSMDA and TIMP-as early as 3-months corrected age. Aim 3: Evaluate whether an early intervention that focuses on caregiver engagement improves caregiver well-being. The invetigators hypothesize that an intervention that focuses on supporting and addressing the individual needs of the caregiver will improve caregiver well-being. The investigators will evaluate these effects using the PedsQL (Family Impact Module).

Multidimensional Phenotype Classification in Grade 3 Bronchopulmonary Dysplasia

Bronchopulmonary Dysplasia (BPD), or chronic lung disease of prematurity, is the most consequential complication of preterm birth and is strong predictor of childhood pulmonary and neurodevelopmental disability, particularly in infants diagnosed with grade 3 BPD (ventilator dependence at 36 weeks' postmenstrual age), the most severe disease form. This study aims to (1) generate the first empirically defined phenotype classification system for grade 3 BPD developed using a rich array of objective and quantitative cardiopulmonary diagnostic, clinical, and biological data; and (2) define the association between phenotype subgroups and neurodevelopmental and respiratory outcomes through 2 years' corrected age.

Lung Ultrasound Score for Early Prediction of Bronchopulmonary Dysplasia in Preterm Newborns

Bronchopulmonary dysplasia (BPD) is one of the most common and severe complications of extreme prematurity, affecting approximately 40% of infants born before 28 weeks of gestation. Despite advances in neonatal care and improved survival rates for extremely preterm infants, the incidence of BPD remains high. BPD is associated with significant short- and long-term morbidity, including chronic respiratory impairment, pulmonary hypertension, recurrent respiratory infections, and neurodevelopmental sequelae. Current diagnosis of BPD is based on the need for respiratory support at 36 weeks postmenstrual age, limiting opportunities for early therapeutic intervention. Since structural lung injury may become irreversible within the first weeks of life, the identification of reliable early predictors of BPD is a major clinical priority. Lung ultrasound (LUS) is a non-invasive, radiation-free, bedside imaging technique increasingly used in neonatal intensive care units. The Lung Ultrasound Score (LUS) provides a quantitative assessment of lung aeration and has demonstrated utility in predicting several neonatal respiratory outcomes. Recent studies suggest that both LUS and pleural line abnormalities detected during the first weeks of life may be associated with the subsequent development of BPD, although evidence remains heterogeneous and no universally validated predictive method is currently available.

Effects of IVIG for BPD in Preterm Infant Born at 28 Weeks and Below

It is intended to examine the short-term and long-term efficacy and safety of intravenous immunoglobulin(IVIG) for the bronchopulmonary dysplasia in preterms born at 28 weeks and below. Participants will received continuous infusion IVIG 1 g/kg/day for the first 2 days, 0.5 g/kg/day for next 3 days (total does 3.5 g/kg), repeatable if necessary.

Transpyloric Versus Gastric Feeding in Bronchopulmonary Dysplasia

The goal of this clinical trial is to learn if transpyloric tube feeding (feeding directly into the small intestine) versus gastric tube feeding tolerably and effectively reduces gastroesophageal reflux in infants born premature who have been diagnosed with bronchopulmonary dysplasia. The main questions this trial aims to answer are: Does transpyloric as compared to gastric tube feeding result in differences in the amount of experienced hypoxemia (low oxygen level in the blood) or serious adverse events? Does transpyloric as compared to gastric tube feeding reduce the frequency and severity of gastroesophageal reflux (GER) measured using 24 hour esophageal pH-multichannel intraluminal impedance (pH-MII) monitoring? Participants will: Undergo pre-trial 24 hour pH-MII monitoring to determine baseline severity of GER. Be randomly assigned to receive transpyloric or gastric tube feeding for 2 weeks. Undergo repeat pH-MII at the end of the 2 week trial to assess for change in GER. Undergo continuous pulse oximetry to record level of hypoxemia during the 2 week trial. Undergo saliva and airway (if supported by a breathing tube) fluid collection to measure biomarkers of GER. Be monitored clinically for possible adverse events.

Reversibility of Bronchial Obstruction in Children Born Preterm

This observational study aims to compare responses to different, commonly used inhaled bronchodilators in children born preterm with bronchial obstruction at spirometry. All children were diagnosed with Chronic Lung Disease of Immaturity (CLDI). The main questions are: * Is any inhaled bronchodilator or their combination generally superior in children with CLDI when assessing the reversibility of bronchial obstruction? * Is there an individual difference in the effect of betamimetic, anticholinergic or their combination between children with CLDI? Participants will: * Come to our clinic in a stable state without acute infection and they will be randomly assigned to the first inhaled bronchodilator. * They will then perform a spirometry test before and after the inhalation of the drug. * This visit will repeat 3 times, each with a different bronchodilator (beta2agonist, anticholinergic and their combination).

Pragmatic Research on Diuretic Management in Early BPD Pilot

Babies who are born prematurely often develop a chronic lung disease called bronchopulmonary dysplasia (BPD). BPD puts babies at higher risk for problems with growth and development. Diuretics, such as furosemide, are frequently used in the management of early BPD). Many clinicians use informal trials of therapy to see if a baby responds to diuretics in the short-term before starting chronic diuretic therapy. Despite frequent use of diuretics, it is unclear how many babies truly respond to therapy and if there are long-term benefits of diuretic treatment. Designing research studies to figure this out has been challenging. The Pragmatic Research on Diuretic Management in Early BPD (PRIMED) study is a feasibility pilot study to help us get information to design a larger trial of diuretic management for BPD. Key questions this study will answer include: (1) Can we use an N-of-1 trial to determine whether a particular baby responds to furosemide? In an N-of-1 trial, a baby is switched between furosemide and placebo to compare that particular infant's response on and off diuretics. It is a more rigorous approach to the informal trials of therapy that are often conducted in clinical care. We hope to learn how many babies have a short-term response to furosemide ("responders"); (2) how many babies will still be on respiratory support at the end of the N-of-1 trial? This will help us determine how many patients would be eligible to randomize to chronic diuretic therapy in the second phase of the larger trail, and (3) if a baby is identified as a short-term responder, how many parents and physicians would be willing to randomize the baby to chronic diuretics (3 months) versus placebo in the longer trial?

Early Prediction of Bronchopulmonary Dysplasia in Preterm Infants Using Clinical Data

Early Prediction of Bronchopulmonary Dysplasia in Preterm Infants Using Clinical Data from the First Three Postnatal Weeks with Large Language Models: A Retrospective Study This retrospective, observational study aims to evaluate the early prediction of bronchopulmonary dysplasia (BPD) in preterm infants using clinical data from the first, second, and third postnatal weeks. The study includes infants born before 32 weeks of gestation or weighing less than 1,500 grams, followed at the Neonatal Intensive Care Unit of Konya City Hospital. The study will compare the performance of different large language models (LLMs), including ChatGPT, Gemini, and Claude, in predicting BPD development. Clinical variables such as gestational age, birth weight, respiratory support, oxygen requirement, mechanical ventilation duration, and infection status will be used. Primary outcome: Accuracy of BPD risk prediction by each AI model compared to actual clinical outcomes. Secondary outcomes: Sensitivity and specificity of predictions, weekly prediction performance, and comparative performance among AI models. The results will provide insight into the potential clinical utility of AI-based approaches for early BPD risk assessment in preterm infants.

Frequent Standardized Oral Care Using Human Milk in the Neonatal Intensive Care Unit

Premature infants are susceptible to complications related to infrequent and non-standardized oral care. Although the benefits of frequent standardized oral care are known to reduce oral dysbiosis (increased level of potentially pathogenic bacteria) and its associated complications in critically ill adults leading to established evidence-based guidelines, no such information exists for VLBW infants. The proposed study will prospectively follow 168 VLBW infants for 4 weeks following birth.

Delivery Room CPAP in Extremely Low Birth Weight Infants

This pilot study was designed to determine the feasibility of randomizing extremely low birth weight (ELBW) infants \<28 weeks' gestation who required resuscitation to one of two resuscitation methods, either: (a) 100% oxygen by facemask and continuous positive airway pressure (CPAP) or positive pressure ventilation (PPV) with positive end-expiratory pressure (PEEP), if the infant required PPV (the intervention group); or (b) 100% oxygen and no CPAP and no PEEP if the infant required PPV (the control group).

Randomized Controlled Crossover Trial of Postpyloric Feedings to Improve Pulmonary Outcomes in High-risk Preterm Infants

The purpose of this study is to determine if postpyloric feedings effectively improve objective measures of pulmonary health in preterm infants with chronic lung disease when compared with nasogastric (NG) feedings. This research will (1) determine the optimal nutritional management to prevent a common and costly complication of prematurity, and (2) use a novel crossover design that examines outcomes of clinical endpoints alongside biomarkers.