A Phase 1/2 interventional study of montelukast 4 mg granule and Placebo in Bronchopulmonary Dysplasia (BPD), Premature Births and Critical Illness, sponsored by Duke University. Recruiting at 5 sites in United States. Open to participants aged 7 Days to 28 Days. Per ClinicalTrials.gov, last updated 2026-07-14.
Sponsored by Duke University · Phase 1/2, Interventional, and Treatment
The purpose of the study is to learn how safe montelukast may be in premature infants at significant risk for Bronchopulmonary Dysplasia (BPD) and to determine how much and how quickly montelukast moves from the stomach into the bloodstream, and how quickly it is removed from the bloodstream.
Data supporting the prospect of montelukast benefit involved 6 previous studies involving 206 preterm infants. The dosing ranged from 0.5 to 2.5 mg/kg/day, which aligns with the proposed initial dose of 0.75 mg/kg/day. Though each previous study had a small population, collectively they reveal montelukast as a promising drug in populations of preterm infants developing BPD and for individual preterm infants who are "developing BPD." Thus, researchers expect clinical benefit for preterm infants in this study.
Despite the benefit-to-risk ratio presented by these previous studies, the optimal dose remains to be determined; thus, this study design and PK analysis will start with the lowest dose that is likely to provide direct benefit to participants.
Multi-center, Prospective, Randomized, Double-masked, Placebo-Controlled Trial Participants (n=28) will be enrolled into a randomized, double-blinded, placebo-controlled trial of once daily montelukast (0.75 mg/kg/day) or placebo (1:1 allotment) for 7 days in critically ill premature infants with developing BPD.
The overall aim is to characterize the pharmacokinetics (PK), short- and long-term adverse events (safety), and respiratory support changes (preliminary efficacy) with montelukast following once daily dosing for 7 days.
Primary: Characterize the PK of montelukast in critically ill premature infants with developing bronchopulmonary dysplasia (BPD).
Secondary: Describe the acute safety profile of montelukast and 2-year developmental progress in critically ill premature infants with developing BPD.
Tertiary: Determine preliminary efficacy of montelukast in critically ill premature infants with developing BPD.
Inpatient participation: Will vary based on gestational age and age at randomization; Up to approximately 60 days (7 days of study drug plus 30 days of post-drug safety monitoring or to 36 weeks postmenstrual age, whichever is longer).
Outpatient participation: Medical and neurodevelopmental follow-up assessments at 6, 12, 18 and 24 months old.
339 studies on the registry are indexed under Bronchopulmonary Dysplasia; 80 are open to participants now.
This study's planned enrollment of 28 is below the median of 70 across 228 interventional studies indexed under Bronchopulmonary Dysplasia.
Browse Bronchopulmonary Dysplasia studies →Duke University is the lead sponsor of 2,025 studies on the registry; 275 are open to participants now.
Of its 194 completed or terminated interventional studies of FDA-regulated products, 159 (82%) have results posted.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria
Once daily montelukast dosed at 0.75 mg/kg/day, maximum dose 4mg. 4mg of montelukast mixed in 5mlL breast milk/formula for a concentration of 0.8mg/mL.
Drug: montelukast 4 mg granule
Plain breast milk or formula
Drug: Placebo
Montelukast sodium (4 mg oral granules) dissolved into 5mL of breast milk/formula yielding a solution concentration of 0.8mg/mL. Dosed once daily by weight, montelukast (0.75 mg/kg/day) or placebo .
Also known as: montelukast sodium
Plain breast milk or formula
Apparent clearance (CL/F) of montelukast
The elimination of montelukast divided by the concentration of montelukast.
Time frame: From enrollment to 14 days post first dose.
Volume of distribution
The total amount of montelukast in the body to the concentration in the bloodstream.
Time frame: From first dose of study drug though 7 days post last dose.
Half-life
The time that it takes for the concentration of montelukast in blood plasma to reach one-half of its steady-state value (the "plasma half-life").
Time frame: From first dose of study drug though 7 days post last dose.
Area Under Curve (AUC)
The concentration of montelukast in blood plasma as a function of time.
Time frame: From first dose of study drug though 7 days post last dose.
Maximum Concentration (Cmax)
The maximum (or peak) serum concentration that montelukast achieves in a specified compartment or test area of the body after the drug has been administered and before the administration of a second dose.
Time frame: From first dose of study drug though 7 days post last dose.
Death- Safety
All infants who die at or before 30 days post treatment or 36 weeks PMA, whichever is longer, will be enumerated by treatment group. Researchers will also summarize by treatment group the percent and cumulative incidence of deaths through 24 months of follow-up.
Time frame: From 30 days post treatment or 36 weeks PMA, whichever is longer; through 24 months of follow-up.
Serious Adverse Events
Total SAEs by participants. Researchers will determine the incidence (rate per person-time) and prevalence (percent of each treatment group experiencing an SAE) at or before 30 days post treatment or 36 weeks PMA, whichever is longer.
Time frame: At or before 30 days post treatment or 36 weeks PMA, whichever is longer.
Total Neuropsychiatric Adverse Events (NPAE)
Researchers will collect safety events of special interest: neuropsychiatric events, including irritability, sedation, apnea, convulsion/seizure, sleep disturbance, tremor and neuropsychiatric event (other). 'Other' will be any event not fitting the previous categories but deemed to be neuropsychiatric in nature by the site principal investigator (PI). For total NPAEs, researchers will determine the incidence (rate per person-time) and prevalence (percent of each treatment arm experiencing an NPAE) for each treatment group. For all safety events researchers will calculate the incidence and prevalence for both the treatment period and for the period to 30 days post treatment or 36-weeks PMA, whichever is longer. Site investigators will determine seriousness and severity for all AEs.
Time frame: From first dose to 30 days post treatment or 36-weeks PMA, whichever is longer.
Neonatal Infections
Researchers will determine the prevalence (percent of each treatment group experiencing an infectious adverse event) at or before 30 days post treatment or 36 weeks PMA, whichever is longer. Specific infections will include (but not limited to): upper respiratory infections, pharyngitis, sinusitis, and otitis.
Time frame: From first dose till at or before 30 days post treatment or 36 weeks PMA, whichever is longer.
Neurodevelopmental outcomes (Bayley-4)
The Bayley Scales of Infant and Toddler Development, Fourth Edition, (often referred to as the Bayley-4) will be completed by a site-based specialist at 24 months of age (range 22 to 26 months). The Bayley-4 is a standardized assessment tool designed to evaluate the developmental functioning of infants and toddlers. Composite scores for each domain are standardized, with a mean of 100 and a standard deviation of 15. Composite scores are also converted into percentile ranks, which indicate the child's performance relative to a normative sample. For instance, a percentile rank of 50 means the child is performing at the average level for their age group.
Time frame: From first dose through 24 months of age.
Neurodevelopmental outcomes (Ages and Stages Questionnaires (ASQ))
The Ages and Stages Questionnaires (ASQ) is a developmental screening tool designed to assess the developmental progress of children from one month to 5 ½ years of age. For each of the 5 domains assessed by the ASQ, the minimum score is 0 and maximum score is 60. Higher scores mean better outcome.
Time frame: 6 months, 12 months, 18 months, and 24 months
Neurodevelopmental outcomes (Child Behavior Checklist (CBCL)
The Child Behavior Checklist (CBCL) is a widely used tool for assessing behavioral and emotional problems in children as young as infancy and toddlerhood.
Time frame: 18 months, 24 months
Oxygen saturation index (OSI)
Oxygen saturation index (OSI) is used to assess the severity of respiratory failure. A higher OSI value indicates worse respiratory compromise. The main OSI endpoint will be the change from randomization baseline to day 7 of treatment.
Time frame: From randomization baseline to day 7 of treatment.
Delirium Assessment
The Cornell Assessment of Pediatric Delirium (CAPD) will be completed in infants at 36 weeks PMA who remain hospitalized. This tool involves scoring based on observations over several hours, rather than a single point in time. A score of 9 or higher typically indicates the presence of delirium.
Time frame: From Enrollment until at 36 weeks PMA who remain hospitalized.
Clinical Bronchopulmonary Dysplasia (BPD)
Clinical BPD will be determined and graded based on clinical severity, which will be based on respiratory support (supplemental oxygen or positive airway pressure) needed at 36 weeks' PMA. This outcome will be measured on a 4-level ordinal scale (as a-d below) and on a dichotomous scale (moderate-to-severe BPD vs. no BPD/mild BPD). 1. No BPD - receiving no respiratory support. 2. Mild (grade 1) BPD - receiving ≤2 L/min nasal cannula. 3. Moderate (grade 2) BPD - receiving \>2 L/min or non-invasive positive airway pressure. 4. Severe (grade 3) BPD - receiving invasive mechanical ventilation.
Time frame: From first dose through 36 Week PMA.
Time on supplemental oxygen
Duration in days that participant requires supplemental oxygen from the time of randomization.
Time frame: From the time of randomization until 30 days post last dose or 36 week PMA, whichever is longer.
Time on positive pressure ventilation
Duration in days that participant requires positive airway pressure (any type of mechanical ventilation or continuous positive airway pressure (CPAP) or high-flow nasal cannula \>1 L/min flow) from the time of randomization. Researchers will also collect time specifically requiring mechanical ventilation (conventional ventilation or hi-frequency ventilation).
Time frame: From the time of randomization until 30 days post last dose or 36 week PMA, whichever is longer.
Death- Efficacy
All infants who died at or before 36 weeks PMA will be included
Time frame: From first dose till at or before 36 weeks PMA
Death or moderate (grade 2) to severe (grade 3) BPD
All infants who die during the course of the study.
Time frame: From first dose until 24 months of age. (+/- 2 months)
Plan to share: No
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