41 Clinical Trials for Various Conditions
The purpose of this study is to determine if an investigational drug, AT-100, can reduce the occurrence of Bronchopulmonary Dysplasia (BPD) in babies born premature, as compared to babies born premature who receive an air-sham alone.
This study will describe the safety of furosemide in premature infants at risk of bronchopulmonary dysplasia and determine the preliminary effectiveness and pharmacokinetics (PK) of furosemide. Funding Source - FDA OOPD
PNEUMOSTEM® consists of ex vivo cultured allogeneic, unrelated, human umbilical cord blood-derived mesenchymal stem cells (hUCB-MSCs) and it is intended for use as a cellular therapy product for prevention of Bronchopulmonary Dysplasia (BPD). This study is an open-label, single-center, dose escalation study to evaluate of safety and efficacy of PNEUMOSTEM® in premature infants at high risk for BPD.
This phase 3, multi-center, double blind, placebo-controlled, randomized clinical trial will attempt to demonstrate if preterm infants who require mechanical ventilation and/or positive pressure support at any point during days 5 to 14 after birth may benefit from treatment with iNO.
SURFAXIN® (lucinactant) treatment will be examined in very low birth weight infants to prevent development of chronic lung disease, commonly known as bronchopulmonary dysplasia (BPD), in premature infants who have required continued intubation and received surfactants for the prevention or treatment of respiratory distress syndrome (RDS).
This is a Phase 3, randomized, masked, active-controlled, multicenter trial designed to determine whether early intratracheal administration of a combination of budesonide with surfactant, as compared to surfactant alone, will reduce the incidence of physiologic bronchopulmonary dysplasia (BPD) or death by 36 weeks' post-menstrual age in extremely preterm infants.
This is a phase I/II open-label study to determine the lowest, safe, effective dose of budesonide given with calfactant as the vehicle.
A lung condition called bronchopulmonary dysplasia (BPD) is a major cause of poor outcomes and death for premature infants. Infants with BPD are also at high risk for pulmonary hypertension (PH)-an important contributor to their condition. Previous research has suggested that a protein in the blood, endothelin-1 (ET-1), is associated with pulmonary disease. This study aims to investigate the incidence of PH and levels of ET-1 among premature babies with BPD. It will also potentially allow us to focus further research efforts and treatment towards these infants, some of our sickest patients at LPCH.
The purpose of this study is to develop rapid MRI techniques for imaging the lung with hyperpolarized helium-3 gas as an inhaled contrast agent. These techniques will be piloted in adults and older children before testing them in younger children and infants. The purpose is to enable imaging of non-sedated infants by imaging so fast as to freeze motion.
The objective of the TOP trial is to determine whether higher hemoglobin thresholds for transfusing ELBW infants resulting in higher hemoglobin levels lead to improvement in the primary outcome of survival and rates of neurodevelopmental impairment (NDI) at 22-26 months of age, using standardized assessments by Bayley.
This observational study was conducted to design and test a physiologic definition for bronchopulmonary dysplasia at 36 weeks of life. Infants were studied in a supine position with the pulse oximeter in position with good signal prior to collecting baseline data. Feedings and medications were given 30 minutes before the evaluation. Baseline data was collected on infant's current oxygen. Then, the infants were weaned to room air for 30 minutes. If saturations remain ≥90%, the infant was considered to have passed the oxygen reduction challenge (to NOT have BPD). The infant should then be placed back in his/her baseline oxygen. If the infant has saturations \<90% for 5 continuous minutes or \<80% for 15 seconds, the infant should be immediately placed back in his/her baseline oxygen, and the infant was considered to have NOT passed the challenge (to have BPD).
This pilot study is a randomized, placebo-controlled, clinical trial to measure changes in blood and urine levels of inositol in premature infants at high risk for retinopathy of prematurity (ROP) following repeated doses of inositol. Based on previous studies, the premise is that maintaining inositol concentrations similar to those occurring naturally in utero will reduce the rates of ROP and bronchopulmonary dysplasia in premature infants. The objective is to evaluate pharmacokinetics, safety, and clinical outcomes of multiple doses of three different dose amounts of myo-inositol (provided by Abbott Laboratories) in very low birth weight premature infants. This study will enroll an estimated 96 infants at 17 NICHD Neonatal Research Network sites. Infants will be randomly assigned to receive either 10 mg/kg of 5% inositol, 40 mg/kg of 5% inositol, 80 mg/kg of 5% inositol, or 5% glucose given in the same volumes and timings as the inositol dosage to maintain masking. Enrollees will receive their assigned dose or placebo daily, starting within 72 hours of birth, and continuing until they reach 34 weeks post-menstrual age, 10 weeks chronologic age, or until the time of hospital discharge, whichever occurs first. The study drug will be administered first intravenously; as the infants progress to full feeding, the drug will be given enterally (orally or via feeding tube). Enrollees will be seen for a follow-up examination at 18-22 months corrected age. This pilot study is in preparation for a future Phase III multi-center randomized controlled trial.
This pilot study was a randomized, placebo-controlled, clinical trial to measure changes in blood and urine levels of inositol in premature infants at high risk for retinopathy of prematurity (ROP) following a single intravenous dose of inositol. Based on previous studies, the premise is that maintaining inositol concentrations similar to those occurring naturally in utero will reduce the rates of ROP and bronchopulmonary dysplasia in premature infants. The objective was to evaluate the single-dose pharmacokinetics and safety of different amounts of intravenous myo-inositol (provided by Ross Products Division, Abbott Laboratories) in very low birth weight neonates, in preparation for a future Phase III multi-center randomized controlled trial. This study enrolled 74 infants at high risk for retinopathy at 9 NICHD Neonatal Research Network sites, and randomly assigned them to receive either 60mg/kg of 5% inositol, 120 mg/kg of 5% inositol, 60 mg/kg of 5% glucose (the placebo), or 120 mg/kg of 5% glucose.
This study tested whether Neonatal Intensive Care Unit (NICU) teams trained in benchmarking -- comparing care practices between different NICUs to see which practices prevent bronchopulmonary dysplasia (BPD) -- and quality improvement would change practices and improve rates of survival without BPD in inborn neonates with birth weights of \<1250 grams. Benchmarking is a method involving detailed comparisons of processes between similar organizations. For this study, three NRN centers with the lowest rates of BPD have been identified as Benchmark centers. During a 6-month pre-intervention period, details of care practices and management style at these centers were carefully assessed. Based on practices at these Benchmarking sites, we developed a quality improvement program. For this study, 14 other NRN sites were randomized to either implement the benchmarking intervention (intervention sites) or continue with their usual care practices (control sites). After the 1-year intervention period, we compared changes in the rate of survival without BPD at 36 weeks corrected age between the intervention and control sites.
The purpose of this study is to evaluate the efficacy of fundoplication in premature infants with GERD and BPD.
Assess the impact of steroid, diuretic, and fluid practices on BPD outcomes in extreme premature infants in the Banner - University Medical Center Phoenix (BUMCP) neonatal intensive care unit (NICU).
Premature infants have high rates of bronchopulmonary dysplasia (BPD) due to prematurity of the participants' lungs and the need for prolonged respiratory support. These infants are at increased risk for gastroesophageal reflux and aspiration which may exacerbate lung injury. Transpyloric feeds, specifically duodenal feeds, may be used to bypass the stomach and directly feed the duodenum decreasing the amount of gastric reflux contributing to aspiration. Duodenal feeds are equivalent to gastric feeds with regards to nutritional outcomes, and have been shown to decrease events of apnea and bradycardia in premature infants. This study will evaluate the feasibility and safety of duodenal feeds in premature infants. The hypothesis is that duodenal feeds may be safely and successfully performed in premature very low birth weight infants.
Infants delivered weighing less than 1 kg at birth (ELBW) are at high risk for the development of bronchopulmonary dysplasia (BPD) and Ventilator-Induced Lung Injury (VILI), in part because of the need for mechanical ventilation utilizing an endotracheal tube (MVET). In spite of strategies to minimize the need for MVET, the incidence of BPD in ELBW infants continues to be 20-80%. The hypothesis is that synchronized NIPPV will decrease the need for MVET and reduce BPD in ELBW infants as compared to NIPPV.
This is a clinical trial which will investigate whether administration of caffeine, a respiratory stimulant, to preterm babies soon after birth can prevent the need for a breathing tube, or intubation. Many preterm babies who require intubation are intubated soon after birth, often within the first few hours. If caffeine is given early enough and is sufficient to stimulate effective breathing, perhaps these babies may not require intubation. Additionally, caffeine may improve blood flow in preterm babies when given soon after birth. Approximately half of babies in this study will receive caffeine within two hours after birth, and half will receive caffeine 12 hours after birth. The hypothesis is that preterm babies who receive caffeine within 2 hours after birth will have a lower incidence of intubation than preterm babies who receive caffeine 12 hours after birth. The main secondary hypothesis is that caffeine given soon after birth will enhance blood flow in preterm babies.
Despite significant improvement in preterm infant survival, the incidence of bronchopulmonary dysplasia (BPD) in infants born \< 28 weeks gestational age (GA) has been relatively stable at \~40%, with 10,000-15,000 new cases estimated annually. Delivery room (DR) management of preterm infants during the initial resuscitation has a significant impact on future development of BPD. Current DR practice as recommended by the Neonatal Resuscitation Program (NRP), focuses on providing positive pressure ventilation (PPV) for intubated infants based on pressure limited ventilation (PLV). But with rapidly changing pulmonary compliance during the early newborn period, PLV may lead to under or over inflation of the lungs and induce significant volutrauma, barotrauma and/or atelectotrauma, all of which are associated in the pathogenesis of BPD. No studies have specifically reported tidal volume (TV) provided in the DR in intubated infants with current PLV practices. Similarly, no study has evaluated the safety and efficacy of volume targeted ventilation (VTV) in the DR and its impact on BPD. With the proposed study, in Phase I, the investigators aim to demonstrate that measuring TV in intubated infants receiving PPV via PLV is feasible. The investigators also seek to demonstrate that with PLV, TV is highly variable in the first few hours of life, even with the same peak inspiratory pressures (PiP) due to rapidly changing pulmonary compliance. A successful Phase I will demonstrate that measuring TV is feasible in the DR, and with information on real time actual TV achieved during PPV, it is possible to target the TV for a goal TV by adjusting the PiP provided. Phase II will be a pilot randomized control trial to demonstrate feasibility of VTV compared to PLV. The investigators will also aim to understand the pulmonary mechanics and physiology during VTV. A successful Phase II will demonstrate VTV is feasible, is associated with stable TV, decreased peak inspiratory pressure and oxygen needs compared to PLV, and not associated with increased complications compared to PLV. It will thereby justify a larger randomized control trial with enough power to evaluate the efficacy of VTV in reducing BPD and other long term pulmonary morbidities for preterm infants.
Extubation failure is a significant problem in preterm neonates and prolonged intubation is a well-documented risk factor for development of chronic lung disease. Out of the respiratory modalities available to extubate a preterm neonate; high flow nasal canula, nasal continuous positive airway pressure (nCPAP) and noninvasive positive pressure ventilation (NIPPV) are the most commonly used. A recent Cochrane meta-analysis concluded that NIPPV has lower extubation failure as compared to nCPAP (30% vs. 40%) NAVA (neurally adjusted ventilatory assist), a relatively new mode of mechanical ventilation in which the diaphragmatic electrical activity initiates a ventilator breath and adjustment of a preset gain (NAVA level) determines the peak inspiratory pressure. It has been reported to improve patient - ventilator synchrony and minimize mean airway pressure and ability to wean an infant from a ventilator. However till date there has been no head to head comparison of extubation failure in infants managed on NAVA with conventional ventilator strategies. In this study the investigators aim to compare primary extubation failure rates in infants/participants managed by NIPPV vs. NI-NAVA (non invasive NAVA). Eligible infants/participants will be randomized to be extubated to predefined NIPPV or NI-NAVA ventilator settings and will be assessed for primary extubation failure (defined as reintubation within 5 days after an elective extubation).
Hospitalized infants with severe bronchopulmonary dysplasia (BPD) and feeding intolerance will be randomized to 2 weeks of continuous gastric feeding or continuous transpyoloric feeding. Subjects will crossover after 2 weeks and receive 4 weeks of each feeding mode. Respiratory status will be assessed to determine the optimal feeding mode for each infant.
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.
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?
This prospective, unblinded, pilot randomized cross-over trial of 2 modes of mechanical ventilation will compare measures of pulmonary mechanics, respiratory gas exchange, and patient comfort between conventional flow triggered mechanical ventilation and neurally adjusted ventilatory assist (NAVA) among 20 prematurely born infants and young children receiving invasive respiratory support for severe bronchopulmonary dysplasia (BPD).
This study is an open-label, phase 1, sequential dose escalation trial seeking to establish preliminary tolerability, efficacy, and pharmacokinetic data for up to 4 different doses of inhaled tobramycin administered to very preterm infants with BPD who are receiving invasive mechanical ventilation and have a pathogenic Gram-negative organism detected by tracheal aspirate culture.
Patients will be randomized to begin the study with either NAVA-synchronized or continuous HFNC. Each patient will receive two 15-minute trials at different levels of continuous HFNC and two 15-minute trials at corresponding levels of synchronized HFNC. In synchronized HFNC, using the NIV NAVA mode on the ventilator each subject will receive a constant minimum flow, but with each neurally triggered breath (as measured with an Edi catheter), an additional flow will be given to the patient. This differs from continuous HFNC in which the subject receives a constant flow without variation. Subjects will be observed during the entirety of these trials. Values for the primary and secondary outcomes will be monitored, recorded, and calculated.
Investigators want to learn the role of indoor environmental exposures on respiratory symptoms, and, separately, on lung function deficits in school-aged children with bronchopulmonary dysplasia (BPD).
Hyperpolarized (HP) gas magnetic resonance imaging (MRI) of the lungs offers additional information that cannot be obtained with CT scan, the current gold standard for imaging this disorder. As a nonionizing technique, MRI is an ideal modality for pulmonary imaging; in particular in the infant and pediatric population. Nevertheless, due to the low proton density of the lung parenchyma (only \~20% that of solid tissues), numerous air-tissue interfaces that lead to rapid signal decay, and cardiac and respiratory sources of motion that further degrade image quality , MRI has played a limited role in the evaluation of lung pathologies. In this setting, HP gas (using 129Xe) MRI may play a role in helping determine the regional distribution of alveolar sizes, partial pressure of oxygen, alveolar wall thickness, and gas transport efficiency of the microvasculature within the lungs of infants with a diagnosis of bronchopulmonary dysplasia (BPD).
A multicenter, placebo-controlled, randomized, dose escalation, safety, and tolerability study of UNEX-42 in infants born at \<27 weeks of gestational age (GA) at high risk for bronchopulmonary dysplasia (BPD).