Inhaled Tobramycin for Bronchopulmonary Dysplasia

Overseen By Erik A Jensen, MD MSCE

Age: Any Age

Sex: Any

Trial Phase: Phase 1

Sponsor: Erik Allen Jensen

Must not be taking: Systemic antibiotics, Nephrotoxic drugs

Disqualifiers: Kidney disease, Liver disease, Cancer, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

Duration: 2 weeks

27 Participants Needed

Trial Summary

What is the purpose of this trial?

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.

Eligibility Criteria

This trial is for very preterm infants born before 32 weeks with Bronchopulmonary Dysplasia (BPD) on mechanical ventilation and a recent tracheal culture positive for certain bacteria. Infants must be at least 36 weeks postmenstrual age, without cancer, significant hearing loss in the family, kidney or liver disease, large endotracheal tube leaks, known drug intolerance or recent use of systemic antibiotics.

Inclusion Criteria

I was diagnosed with BPD and needed extra oxygen or help breathing at 36 weeks after conception.

I am on a ventilator and not expected to be taken off it within a week.

My baby was born before 32 weeks of pregnancy.

Exclusion Criteria

I haven't taken any kidney-damaging medications, except for water pills, in the last 2 days.

I am currently on a high-frequency ventilator.

A close female relative needed a hearing aid before age 30.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive inhaled tobramycin at varying doses every 12 hours for up to 14 days

2 weeks

Continuous monitoring

Observational

Enrolled infants in the observational cohort undergo data collection for 14 days without receiving the study drug

2 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• Tobramycin solution for inhalation

Trial Overview The study tests four escalating doses of inhaled tobramycin in these infants to check tolerability and effectiveness against Gram-negative bacterial infections. It's an open-label phase 1 trial where each infant receives one dose level to gather initial data on how the drug works and its safety.

Participant Groups

Group I: Treatment Arm - 78mg Experimental Treatment 1 Intervention

The phase 1 trial will begin with a dose of 78mg. All treatment arms will administer study drug every 12 hours for up to 14 days (28 doses). Up to 6 infants in this arm will receive the 78mg dose of tobramycin solution for inhalation administered via vibrating mesh nebulizer. During the trial, infants in each treatment arm will undergo blood and tracheal aspirate sampling and respiratory mechanics measurements at pre-specified time points to assess dose safety and potential efficacy. Continuous pulse oximetry monitoring for the duration of the trial will also occur. Clinical data will also be recorded daily throughout the trial in all participants.

Group II: Treatment Arm - 300mg Experimental Treatment 1 Intervention

If tolerability is demonstrated in the 216mg treatment arm following the 3+3 trial design, up to 6 infants will then be enrolled and receive 150mg of tobramycin solution for inhalation every 12 hours. The same monitoring procedures will be performed in this treatment arm as in the prior.

Group III: Treatment Arm - 216mg Experimental Treatment 1 Intervention

If tolerability is demonstrated in the 150mg treatment arm following the 3+3 trial design, up to 6 infants will then be enrolled and receive 150mg of tobramycin solution for inhalation every 12 hours. The same monitoring procedures will be performed in this treatment arm as in the prior.

Group IV: Treatment Arm - 150mg Experimental Treatment 1 Intervention

If tolerability is demonstrated in the 78mg treatment arm following the 3+3 trial design, up to 6 infants will then be enrolled and receive 150mg of tobramycin solution for inhalation every 12 hours. The same monitoring procedures will be performed in this treatment arm as in the prior.

Group V: Observational Arm Active Control 1 Intervention

Enrolled infants who are eligible to participate in the phase-1 trial may be enrolled in an untreated observational cohort at parental discretion. This cohort will undergo collection of clinical and respiratory mechanics data for 14 days after enrollment but will not receive the study drug.

Who Is Running the Clinical Trial?

Erik Allen Jensen

Lead Sponsor

Trials

1

Recruited

30+

University of Florida

Collaborator

Trials

1,428

Recruited

987,000+