Bronchopulmonary DysplasiaPhase 1NCT04560179

Inhaled Tobramycin in BPD

A Phase 1 study testing Tobramycin solution for inhalation 78mg dose, Tobramycin solution for inhalation 150mg dose and Tobramycin solution for inhalation 216mg dose in people with Bronchopulmonary Dysplasia, run by Erik Allen Jensen at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

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… Read more about this study

Who is it for?: Ages 4 weeks and older
Conditions the study is for: Bronchopulmonary Dysplasia
What is being tested?: Tobramycin solution for inhalation 78mg dose, Tobramycin solution for inhalation 150mg dose and Tobramycin solution for inhalation 216mg dose
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 27 participants
Where: 1 siteUnited States
Study dates: 22 Mar 2022to 17 May 2024
Phase: Phase 1
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 4 weeks and older
All sexes
No healthy volunteers
Child, Adult, Older adult

Who may be able to join

Male or female infants born <32 weeks' gestation
Diagnosed with BPD (use of supplemental oxygen or respiratory support at 36 weeks postmenstrual age )
Postmenstrual age ≥36 weeks at study enrollment
Treatment with invasive mechanical ventilation at enrollment without planned tracheal extubation within 7 days after enrollment
Tracheal aspirate culture positive for one of the following pathogenic GNR bacteria within 7 days prior to enrollment: Pseudomonas aeruginosa, Klebsiella species, Enterobacter species, Stenotrophomonas maltophilia, Escherichia coli, Acinetobacter baumannii, or Serratia marcescens
Parental/guardian permission (informed consent).

Who cannot join

Serum creatinine >0.4mg/dL within 14 days prior to enrollment
Congenital or acquired disease of the kidney or renal collecting system that adversely affects renal function
Congenital or acquired hepatobiliary disease that adversely affects liver function
Treatment with a systemic antibiotic within 7 days prior to enrollment
Treatment with a nephrotoxic medication, excluding diuretics, within 48 hours prior to enrollment
Treatment with a neuromuscular blocker within 48 hours prior to enrollment

Show all 13Show fewer

Known intolerance to aminoglycoside antibiotics
Current treatment with high frequency or other oscillating mechanical ventilation
Presence of a cancer diagnosis
Maternal family history of early onset hearing loss defined as the need for an assistive hearing device prescribed before 30 years of age
Endotracheal tube leak >20%.
Any prior use of an investigational drug [as part of an FDA approved Investigational New Drug (IND) protocol].
A subject who, in the judgement of the Investigator, is not an appropriate candidate for this research study.

Trial locations and contacts

1 site in 1 country.

United States1 site

The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104

Other trials in United States

About this study

Official title: Phase 1 Feasibility Trial of Inhaled Tobramycin in Preterm Infants With Bronchopulmonary Dysplasia

Conditions: Bronchopulmonary Dysplasia
Being tested: Tobramycin solution for inhalation 78mg dose, Tobramycin solution for inhalation 150mg dose, Tobramycin solution for inhalation 216mg dose, Tobramycin solution for inhalation 300mg dose
Medical subject headings: Bronchopulmonary Dysplasia

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Tobramycin solution for inhalation 78mg doseDrug
Inhaled tobramycin - 78mg administered every 12 hours for 14 days.
Tobramycin solution for inhalation 150mg doseDrug
Inhaled tobramycin - 150mg administered every 12 hours for 14 days.
Tobramycin solution for inhalation 216mg doseDrug
Inhaled tobramycin - 216mg administered every 12 hours for 14 days.
Tobramycin solution for inhalation 300mg doseDrug
Inhaled tobramycin - 300mg administered every 12 hours for 14 days.

Study groups in NCT04560179
Study group	Role	What they receive
Treatment Arm - 78mg	Experimental	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. Receives: Drug: Tobramycin solution for inhalation 78mg dose
Treatment Arm - 150mg	Experimental	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. Receives: Drug: Tobramycin solution for inhalation 150mg dose
Treatment Arm - 216mg	Experimental	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. Receives: Drug: Tobramycin solution for inhalation 216mg dose
Treatment Arm - 300mg	Experimental	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. Receives: Drug: Tobramycin solution for inhalation 300mg dose
Observational Arm	No 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.

What is being measured

The primary outcome is the main question the study is designed to answer.

Elevated Serum Tobramycin Trough or Creatinine or Severe Adverse Event
Trough serum tobramycin level (measured 11 hours after the administered dose) ≥1mcg/mL; increase in serum creatinine level by ≥0.3mg/dL above pre-trial baseline; increase in serum creatinine level >1.5-fold above pre-trial baseline; urine output <0.5mL/kg/hr for 12 consecutive hours; or any serious adverse event possibly attributable to the study drug
Measured: Any time during the 14-day trial

Secondary outcomes (11)

New Onset or Worsened Coughing Associated With a Change in Respiratory Status (SpO2 <80% for >10 Seconds; Need for Increase in FiO2 by >20%)
Measured: Any time during the 14-day trial
Obstruction of the Endotracheal Tube Requiring Tube Replacement
Measured: Any time during the 14-day trial
Unplanned Tracheal Extubation
Measured: Any time during the 14-day trial
Desaturation (SpO2 <80% for >10 Seconds) During Administration of Inhaled Tobramycin
Measured: Any time during the 14 day trial
Pre-discharge Failed Audiology Examination
Measured: up to 1 year of age
New Intra-patient Microbial Resistance to Tobramycin During the Primary Hospitalization
Measured: up to 1 year of age
Change in Tracheal Aspirate Pathogenic Bacterial Colony Forming Unit (CFU) Counts Measured by Quantitative Culture
Measured: During the 14-day trial
Change in the Fraction of Inspired Oxygen (FiO2), Ventilator Mean Airway Pressure (MAP), and Respiratory Severity Score (MAP x FiO2)
Measured: During the 14-day trial
Change in Intermittent Hypoxemia (SpO2<80% Lasting >/=10s), Prolonged Hypoxemia (SpO2<80% Lasting >1min), and Daily Proportion of Time in Hypoxemia
Measured: During the 14-day trial
Change in Tracheal Aspirate Cytokine Levels, Neutrophil to Total WBC Ratio, and Patterns in the Airway Microbiome
Measured: During the 14-day trial
Change in Dynamic Lung Compliance, Airway Resistance, Peak Expiratory Flow, and Carbon Dioxide (CO2) Elimination
Measured: During the 14-day trial

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

22 Mar 202217 May 2024

First posted23 Sep 2020
Study start22 Mar 2022
Primary completion31 Dec 2023
Study completion17 May 2024

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for Bronchopulmonary DysplasiaStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 1
Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Sequential
Model description: 3+3 dose escalation trial.
Masking: None (open label)
Enrolment: 27 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT04560179
Sponsor's protocol ID: 19-016800
Status last verified: Dec 2024
First submitted: 17 Sep 2020
Last updated: 31 Jan 2025
Results posted: Yes, on ClinicalTrials.gov
FDA-regulated drug: Yes
FDA-regulated device: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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All Bronchopulmonary Dysplasia trials

Study at a glance

From the registry record, last updated by the sponsor on 31 Jan 2025. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Phase 1 Feasibility Trial of Inhaled Tobramycin in Preterm Infants With Bronchopulmonary Dysplasia
ClinicalTrials.gov ID: NCT04560179
Acronym / sponsor ID: 19-016800
Conditions: Bronchopulmonary Dysplasia
Phase: Phase 1
Study type: Interventional
Recruitment status: Completed
Enrolment: 27 participants (actual)
Lead sponsor: Erik Allen JensenUniversity, hospital or other
Collaborators: University of Florida
Study start: 22 Mar 2022 (actual)
Primary completion: 31 Dec 2023 (actual)
Study completion: 17 May 2024 (actual)
First posted: 23 Sep 2020 (actual)
Results: Posted 31 Jan 2025

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Sequential
Masking: None (open label)
Number of arms: 5
Model description: 3+3 dose escalation trial.

Arm	Type	Receives
Treatment Arm - 78mg	Experimental	Drug: Tobramycin solution for inhalation 78mg doseThe 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.
Treatment Arm - 150mg	Experimental	Drug: Tobramycin solution for inhalation 150mg doseIf 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.
Treatment Arm - 216mg	Experimental	Drug: Tobramycin solution for inhalation 216mg doseIf 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.
Treatment Arm - 300mg	Experimental	Drug: Tobramycin solution for inhalation 300mg doseIf 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.
Observational Arm	No 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 may qualify

Ages: 4 weeks and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult

Key inclusion criteria (6 listed)

Male or female infants born <32 weeks' gestation
Diagnosed with BPD (use of supplemental oxygen or respiratory support at 36 weeks postmenstrual age )
Postmenstrual age ≥36 weeks at study enrollment
Treatment with invasive mechanical ventilation at enrollment without planned tracheal extubation within 7 days after enrollment
Tracheal aspirate culture positive for one of the following pathogenic GNR bacteria within 7 days prior to enrollment: Pseudomonas aeruginosa, Klebsiella species, Enterobacter species, Stenotrophomonas maltophilia, Escherichia coli, Acinetobacter baumannii, or Serratia marcescens

Key exclusion criteria (13 listed)

Serum creatinine >0.4mg/dL within 14 days prior to enrollment
Congenital or acquired disease of the kidney or renal collecting system that adversely affects renal function
Congenital or acquired hepatobiliary disease that adversely affects liver function
Treatment with a systemic antibiotic within 7 days prior to enrollment
Treatment with a nephrotoxic medication, excluding diuretics, within 48 hours prior to enrollment

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
Tobramycin solution for inhalation 78mg doseInhaled tobramycin - 78mg administered every 12 hours for 14 days.	Drug	Treatment Arm - 78mg
Tobramycin solution for inhalation 150mg doseInhaled tobramycin - 150mg administered every 12 hours for 14 days.	Drug	Treatment Arm - 150mg
Tobramycin solution for inhalation 216mg doseInhaled tobramycin - 216mg administered every 12 hours for 14 days.	Drug	Treatment Arm - 216mg
Tobramycin solution for inhalation 300mg doseInhaled tobramycin - 300mg administered every 12 hours for 14 days.	Drug	Treatment Arm - 300mg

Primary outcome

Elevated Serum Tobramycin Trough or Creatinine or Severe Adverse Event
Time frame: Any time during the 14-day trial
Trough serum tobramycin level (measured 11 hours after the administered dose) ≥1mcg/mL; increase in serum creatinine level by ≥0.3mg/dL above pre-trial baseline; increase in serum creatinine level >1.5-fold above pre-trial baseline; urine output <0.5mL/kg/hr for 12 consecutive hours; or any serious adverse event possibly attributable to the study drug

11 secondary outcomes

New Onset or Worsened Coughing Associated With a Change in Respiratory Status (SpO2 <80% for >10 Seconds; Need for Increase in FiO2 by >20%)
Time frame: Any time during the 14-day trial
Obstruction of the Endotracheal Tube Requiring Tube Replacement
Time frame: Any time during the 14-day trial
Unplanned Tracheal Extubation
Time frame: Any time during the 14-day trial
Desaturation (SpO2 <80% for >10 Seconds) During Administration of Inhaled Tobramycin
Time frame: Any time during the 14 day trial
Pre-discharge Failed Audiology Examination
Time frame: up to 1 year of age
New Intra-patient Microbial Resistance to Tobramycin During the Primary Hospitalization
Time frame: up to 1 year of age
Change in Tracheal Aspirate Pathogenic Bacterial Colony Forming Unit (CFU) Counts Measured by Quantitative Culture
Time frame: During the 14-day trial
Change in the Fraction of Inspired Oxygen (FiO2), Ventilator Mean Airway Pressure (MAP), and Respiratory Severity Score (MAP x FiO2)
Time frame: During the 14-day trial
Change in Intermittent Hypoxemia (SpO2<80% Lasting >/=10s), Prolonged Hypoxemia (SpO2<80% Lasting >1min), and Daily Proportion of Time in Hypoxemia
Time frame: During the 14-day trial
Change in Tracheal Aspirate Cytokine Levels, Neutrophil to Total WBC Ratio, and Patterns in the Airway Microbiome
Time frame: During the 14-day trial
Change in Dynamic Lung Compliance, Airway Resistance, Peak Expiratory Flow, and Carbon Dioxide (CO2) Elimination
Time frame: During the 14-day trial

Results and safety

Results posted to the registry on 31 Jan 2025, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Treatment Arm - 78mg	4	3
Treatment Arm - 150mg	3	3
Treatment Arm - 216mg	3	3
Treatment Arm - 300mg	6	5
Observational Arm	0	0

Primary outcome: Elevated Serum Tobramycin Trough or Creatinine or Severe Adverse Event

Time frame: Any time during the 14-day trial. Count of participants. Unit: Participants

Measure	Treatment Arm - 78mg n = 3	Treatment Arm - 150mg n = 3	Treatment Arm - 216mg n = 3	Treatment Arm - 300mg n = 6	Observational Arm n = 0
Value	0	0	0	1	0

Population analysed: No infants were enrolled into the observational arm. 1 of the 16 enrolled infants (treatment arm - 78mg) was withdrawn early from the trial by the medical team and was not assessed for this outcome.

11 secondary outcomes are reported in the full results.

Adverse events

Time frame: During the 14 day drug trial and continuing until hospital discharge. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Treatment Arm - 78mg	0 of 4 (0%)	0 of 4 (0%)	2 of 4
Treatment Arm - 150mg	0 of 3 (0%)	1 of 3 (33.3%)	0 of 3
Treatment Arm - 216mg	0 of 3 (0%)	1 of 3 (33.3%)	1 of 3
Treatment Arm - 300mg	0 of 6 (0%)	1 of 6 (16.7%)	1 of 6
Observational Arm	0 of 0	0 of 0	0
Screened But Not Eligible	0 of 0	0 of 0	0

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 31 Jan 2025.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.

Related trials for Bronchopulmonary Dysplasia

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Strap To Reduce Apnea in Preterm Infants: A Randomized Crossover Trial
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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

The record lists no contact. Use the official record to reach the sponsor.

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 6 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 31 Jan 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 31 Jan 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Elevated Serum Tobramycin Trough or Creatinine or Severe Adverse Event (time frame: Any time during the 14-day trial). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04560179.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 31 Jan 2025.