# Phase 1 trial of inhaled tobramycin in very preterm infants with bronchopulmonary dysplasia

> **NIH NIH R34** · CHILDREN'S HOSP OF PHILADELPHIA · 2022 · $220,000

## Abstract

PROJECT SUMMARY/ABSTRACT
Bronchopulmonary dysplasia (BPD), or chronic lung disease of prematurity, is among the most devastating
complications of preterm birth. It affects half of surviving extremely preterm infants, is associated with life-long
deficits in health and cognition, and carries enormous societal burden and cost. Strikingly, there are no drug
therapies shown to improve outcomes for infants with BPD. Our research seeks to resolve this care gap.
 An abundance of data support a causal link between pathologic microbial invasion of the lower respiratory
tract (LRT) and worsening of respiratory health in chronic lung illness. Our work, and others’, has shown that
the presence of pathogenic Gram-negative rod (GNR) bacteria in the lungs of ventilator dependent very
preterm infants with BPD is an independent risk factor for significant and enduring respiratory morbidity.
Systemically administered antibiotics do not adequately penetrate the lung epithelial lining fluid to eradicate
these bacteria. In contrast, inhaled antibiotics deliver drug directly to the site of infection, offering a safer and
more effective alternative. Inhaled tobramycin, an aminoglycoside with potent anti-GNR activity, is now a
benchmark therapy in cystic fibrosis, where lung infection by Pseudomonas aeruginosa (a common GNR
pathogen in BPD) accelerates parenchymal lung damage, promotes decline in lung function, and contributes to
early mortality. Emerging data also show benefit with inhaled tobramycin in other pediatric and adult chronic
respiratory conditions plagued by difficult to treated respiratory pathogens. These data provide strong
biological plausibility that inhaled tobramycin will benefit very preterm infants with BPD who have
pathogenic GNR organisms cultured from the LRT. However, inhaled tobramycin is only FDA approved for
use in patients 6 years and older. Whether the typical dose and duration of therapy used in older children is
appropriate for infants is uncertain.
 To rigorously characterize the risks and benefits of inhaled tobramycin in very preterm infants with BPD, we
must first identify a well-tolerated dose for investigation in this unique population. To obtain these
essential data, we propose to conduct a 3+3 inter-patient, ascending dose phase 1 trial of inhaled tobramycin
in ventilator dependent very preterm infants with BPD who have pathogenic GNR bacteria cultured from the
LRT. This trial will investigate up to 4 different doses of inhaled tobramycin: 78mg, 150mg, 216mg, and 300mg
(FDA approved dose), each administered every 12 hours for up to 14 days. This study will establish the
preliminary dose tolerability, pharmacokinetic, and exploratory efficacy data needed to design and conduct the
first, placebo-controlled, randomized trial of this promising drug therapy in very preterm infants with BPD.
Collectively, our proposed studies of inhaled tobramycin are poised to identify the first drug therapy that
improves long-term outcomes in this un...

## Key facts

- **NIH application ID:** 10472648
- **Project number:** 5R34HL155690-02
- **Recipient organization:** CHILDREN'S HOSP OF PHILADELPHIA
- **Principal Investigator:** ERIK ALLEN JENSEN
- **Activity code:** R34 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2022
- **Award amount:** $220,000
- **Award type:** 5
- **Project period:** 2021-08-20 → 2024-07-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10472648

## Citation

> US National Institutes of Health, RePORTER application 10472648, Phase 1 trial of inhaled tobramycin in very preterm infants with bronchopulmonary dysplasia (5R34HL155690-02). Retrieved via AI Analytics 2026-05-27 from https://api.ai-analytics.org/grant/nih/10472648. Licensed CC0.

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