Montelukast for critically ill premature infants developing bronchopulmonary dysplasia: pharmacokinetics, safety, and early efficacy

Pharmacokinetics, Safety and Preliminary Efficacy Study of Montelukast in Critically Ill Infants With Developing Bronchopulmonary Dysplasia

PHASE1; PHASE2 · Duke University · NCT07101640

This study gives once-daily montelukast to critically ill premature infants developing bronchopulmonary dysplasia to test how the drug moves through the body, whether it is safe, and whether it helps breathing.

Quick facts

PhasePHASE1; PHASE2
Study typeInterventional
Enrollment28 (estimated)
AgesN/A to 28 Weeks
SexAll
SponsorDuke University (other)
Locations4 sites (Little Rock, Arkansas and 3 other locations)
Trial IDNCT07101640 on ClinicalTrials.gov

What this trial studies

This is a multicenter, randomized, double-masked, placebo-controlled Phase 1/2 trial enrolling 28 critically ill preterm infants to receive once-daily montelukast (0.75 mg/kg/day) or placebo for 7 days. The primary goal is to characterize montelukast pharmacokinetics in infants born <28 weeks' gestation and <1000 g who are 7–28 days old and require mechanical ventilation with FiO2 ≥30%. Secondary and tertiary aims include short- and long-term safety monitoring and preliminary measures of respiratory support needs, with inpatient monitoring up to about 60 days and neurodevelopmental follow-up out to 24 months. The design builds on several small prior pediatric studies but focuses on detailed PK and safety data in this high-risk population.

Who should consider this trial

Good fit: Ideal candidates are critically ill preterm infants born before 28 weeks' gestation and under 1000 g at birth, aged 7–28 days, receiving mechanical ventilation with FiO2 ≥30%, and able to tolerate enteral feeding volumes.

Not a fit: Infants with known montelukast allergy, recent montelukast exposure, major non-survivable conditions, acute intercurrent illnesses that increase respiratory support, or those not requiring significant respiratory support are unlikely to benefit from this protocol.

Why it matters

Potential benefit: If successful, montelukast could reduce lung injury and improve respiratory outcomes for premature infants developing bronchopulmonary dysplasia, potentially decreasing time on ventilators and improving longer-term respiratory and developmental outcomes.

How similar studies have performed: Six small prior studies totaling about 206 preterm infants reported promising signals with montelukast (doses 0.5–2.5 mg/kg/day), but those studies were small and the optimal dose remains uncertain.

Eligibility criteria

Show full inclusion / exclusion criteria
Inclusion Criteria

1. Documented informed consent from parent or guardian, prior to study activities
2. Receiving mechanical ventilation \[high frequency or conventional\] and requiring supplemental oxygen (FiO2 ≥ 30%) at time of randomization
3. \<28 weeks' gestational age and \<1000 g bodyweight at birth
4. 7 to 28 (inclusive) days postnatal age at the time of first study drug dose
5. Able to tolerate 5 mL of enteral volume

Exclusion Criteria

1. Previous enrollment and dosing in the current PRISM study (NICHD-2023-MON01)
2. Previous exposure to montelukast within 7 days prior to randomization
3. Known allergy to montelukast
4. PI deems infant - prior to enrollment - is not expected to survive
5. Has a disease complication that would preclude safe participation of the participant
6. Increased respiratory support due to intercurrent illness (e.g., sepsis, necrotizing enterocolitis, etc.). Infants should be excluded from the study until after resolution of the acute event
7. Congenital lung and diaphragmatic malformations

Where this trial is running

Little Rock, Arkansas and 3 other locations

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.

View on ClinicalTrials.gov →

Conditions: Bronchopulmonary Dysplasia, Premature Births, Critical Illness, PK, Pharmacokinetics, bronchopulmonary dysplasia, montelukast

Last reviewed 2026-05-15 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.