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Bronchopulmonary DysplasiaPhase 1FFLOATNCT03041740

Fluid Filled Lung Oxygenation Assistance Trial

A Phase 1 study testing Perfluorooctyl Bromide in people with Bronchopulmonary Dysplasia, run by Children's Hospital of Philadelphia 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

The primary study objective is to assess the safety and feasibility of perfluorooctylbromide (PFOB) partial liquid ventilation (PLV) in infants with severe Bronchopulmonary Dysplasia (BPD). Read more about this study

Who is it for?
Ages Up to 6 months
Conditions the study is for
Bronchopulmonary Dysplasia
What is being tested?
Perfluorooctyl Bromide
Could I receive a placebo?
No, everyone receives an active treatment
How many people
16 participants
Where
1 siteUnited States
Study dates
27 Jun 2017to 10 Jun 2019
Phase
Phase 1
Are there results?
None posted in the registry

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 Up to 6 months
  • All sexes
  • No healthy volunteers
  • Child

Who may be able to join

  • Neonates with severe BPD as defined by 36 weeks post conception age and require positive pressure ventilation
  • Infants born at less than 32 weeks post conception age
  • Subjects may be up to 6 months corrected age
  • On conventional mechanical ventilation for chronic lung disease for at least two days prior to enrollment
  • On conventional mechanical ventilation at the time of enrollment and anticipated to continue for 14 days
  • Off systemic steroids for lung disease for 72 hours (3 days) prior to T=0
  • Hemoglobin value ≥8 g/dL: if less than 8 g/dL, transfusion is permitted. Clinical blood transfusion should be administered prior to enrollment.
  • Parental/guardian permission (informed consent)

Who cannot join

  • Mechanical ventilation for acute disease, such as for infection or for post-operative complications
  • Severe Pulmonary Hypertension (PAH) (pulmonary pressure greater than 2/3 systemic) as defined by either echocardiogram (ECHO), or cardiac catheterizations or a CT-Angiogram consistent with PAH within the last 3 weeks.
  • Pneumothorax (active air leak) requiring chest tube within 72 hours of T=0
  • Active pulmonary hemorrhage within 72 hours of T=0
  • History of Grade III/IV interventricular hemorrhage without resolution or stability within 3 weeks of verifying eligibility
  • Severe congenital heart disease compromising pulmonary circulation
Show all 9Show fewer
  • Other major congenital malformation (including but not limited to CDH) or known genetic syndromes at the discretion of the investigator
  • Use of an investigational drug within 7 days prior to confirmation of eligibility.
  • The clinical attending physician believes it is not in the subject's and/or parents/guardians best interest to participate in the trial.

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104

Other trials in United States

About this study

Official title: A Pilot Study to Evaluate the Safety of Perflurooctylbromide (PFOB) Partial Liquid Ventilation (PLV) for Up to 10 Days in Neonates With Severe Bronchopulmonary Dysplasia (BPD)

The primary study objective is to assess the safety and feasibility of perfluorooctylbromide (PFOB) partial liquid ventilation (PLV) in infants with severe Bronchopulmonary Dysplasia (BPD).

Read the detailed description

The primary study objective is to assess the safety and feasibility of perfluorooctylbromide (PFOB) partial liquid ventilation for up to ten days in infants with severe BPD as evaluated by: (1) no sustained oxygen desaturations (SpO2 ≤ 80%) for greater than ten minutes without response to increased oxygen therapy, (2) no persistent hypotension without response to volume expansion and/or inotropic therapy, (3) no major mucus plugging events (defined as events that are unresolved after two bronchoscopes), (4) no pneumothoraces or pleural effusion with PFOB, (5) and no evidence of increased carbon dioxide (CO2) retention, renal insufficiency, hyperkalemia, or metabolic acidosis.

Being tested
Perfluorooctyl Bromide
Keywords
Bronchopulmonary Dysplasia, Neonates
Medical subject headings
Bronchopulmonary Dysplasia

What participants receive

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

  • Perfluorooctyl BromideDrug

    Also called Perflubron

    Subjects in the PFOB group will receive PFOB partial liquid ventilation instilled via side port of endotracheal tube (2.5 mL/kg/day) across 10 treatment days.

Study groups in NCT03041740
Study groupRoleWhat they receive
Usual Care (Control) GroupNo interventionControl subjects will be treated as per standard of care for preterm infants with BPD.
Perfluorooctylbromide (PFOB) GroupActive comparatorSubjects in the PFOB group will be administered an initial PFOB treatment dose of 2.5 mL/kg and up to a total intra-pulmonary volume of 25 mL/kg for up to 10 days.
Receives: Drug: Perfluorooctyl Bromide

What is being measured

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

  1. Sustained oxygen desaturations for greater than ten minutes without response to increased oxygen therapy

    Incidence of sustained (greater than 10 minutes) oxygen desaturation events without response to increased oxygen therapy. These incidences will be measured by a continuous non-invasive percutaneous oxygen saturation monitor.

    Measured: Day 5, Day 10

  2. Persistent hypotension without response to volume expansion and/or inotropic therapy

    Incidence of persistent hypotension without response to volume expansion and/or inotropic therapy. Hypotension is a decrease in systolic blood pressure deemed significant by clinical staff. Measurement will be performed with a standard intensive care unit blood pressure cuff.

    Measured: Day 5, Day 10

  3. Change in number of major mucus plugging events

    Incidence of airway obstruction of the endotracheal tube, as indicated by decreased chest movement during mechanical ventilation, need for increased ventilator pressure, and/or elevation of carbon dioxide levels in the blood. Mucus plugs are confirmed by endotracheal suctioning.

    Measured: Day 5, Day 10

  4. Incidence of pneumothorax or pleural effusion with PFOB

    Incidence of pneumothorax in the child will be measured by transillumination of the chest and confirmed by chest x-ray.

    Measured: Day 5, Day 10

  5. Number of participants with sustained hypercapnia (elevated carbon dioxide in the blood, greater than 95 mmHg, for over four hours).

    Hypercapnia will be measured with blood tests and/or cutaneous carbon dioxide monitor.

    Measured: Day 5, Day 10

Secondary outcomes (3)

  1. Change in fraction of inspired oxygen (FiO2)

    Fraction of inspired oxygen is the percentage of oxygen that the patient is receiving for his or her lung disease. An FiO2 of 0.21 is room air oxygen and 1.00 is 100% oxygen. The higher the FiO2, the more severe the respiratory disease.

    Measured: Day 5, Day 10

  2. Change in ventilator mean airway pressure (MAP)

    Mean airway pressure (MAP) is the average amount of airway pressure supplied to the lungs throughout a breath. Higher MAP indicates more severe disease.

    Measured: Day 5, Day 10

  3. Change in Respiratory Severity Score (MAP x FiO2)

    Respiratory Severity Score (RSS) is a scale computed as the Mean Airway Pressure (MAP) multiplied by the Fraction of Inspired Oxygen (FiO2). Clinically, the scale range will often lie between 1 and 10, with higher numbers indicating more severe disease. The RSS can theoretically reach a score as high as 30-35.

    Measured: Day 5, Day 10

Timeline

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

  1. First posted3 Feb 2017 (estimated)
  2. Study start27 Jun 2017
  3. Primary completion10 Jun 2019
  4. Study completion10 Jun 2019

Resources

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
Randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
16 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03041740
Sponsor's protocol ID
12-008686
Acronym
FFLOAT
Status last verified
Oct 2022
First submitted
30 Jan 2017
Last updated
21 Oct 2022
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Publications

  • Nocentini G, MacLaren G, Bartlett R, De Luca D, Perdichizzi S, Stoppa F, Marano M, Cecchetti C, Biasucci DG, Polito A, AlGhobaishi A, Guner Y, Gowda SH, Hirschl RB, Di Nardo M. Perfluorocarbons in Research and Clinical Practice: A Narrative Review. ASAIO J. 2023 Dec 1;69(12):1039-1048. doi: 10.1097/MAT.0000000000002017. Epub 2023 Aug 7.

    PubMed 37549675

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Perfluorooctyl Bromide in people with Bronchopulmonary Dysplasia, run by Children's Hospital of Philadelphia at 1 site in United States.

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 8 inclusion and 9 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 21 Oct 2022. 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?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

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?

Sustained oxygen desaturations for greater than ten minutes without response to increased oxygen therapy (time frame: Day 5, Day 10). The study lists 5 primary outcomes in all. 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 NCT03041740, which also lists 1 linked publication.

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 21 Oct 2022.

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