Fluid Filled Lung Oxygenation Assistance Trial (FFLOAT)
Primary Purpose
Bronchopulmonary Dysplasia
Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
Perfluorooctyl Bromide
Sponsored by
About this trial
This is an interventional treatment trial for Bronchopulmonary Dysplasia focused on measuring Bronchopulmonary Dysplasia, Neonates
Eligibility Criteria
Inclusion Criteria
- 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)
Exclusion Criteria
- 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
- 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.
Sites / Locations
- Children's Hospital of Philadelphia
Arms of the Study
Arm 1
Arm 2
Arm Type
No Intervention
Active Comparator
Arm Label
Usual Care (Control) Group
Perfluorooctylbromide (PFOB) Group
Arm Description
Control subjects will be treated as per standard of care for preterm infants with BPD.
Subjects 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.
Outcomes
Primary Outcome Measures
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.
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.
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.
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.
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.
Secondary Outcome Measures
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.
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.
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.
Full Information
NCT ID
NCT03041740
First Posted
January 30, 2017
Last Updated
October 19, 2022
Sponsor
Children's Hospital of Philadelphia
Collaborators
Auto Dealers Caring for Kids Foundation
1. Study Identification
Unique Protocol Identification Number
NCT03041740
Brief Title
Fluid Filled Lung Oxygenation Assistance Trial
Acronym
FFLOAT
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)
Study Type
Interventional
2. Study Status
Record Verification Date
October 2022
Overall Recruitment Status
Completed
Study Start Date
June 27, 2017 (Actual)
Primary Completion Date
June 10, 2019 (Actual)
Study Completion Date
June 10, 2019 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Children's Hospital of Philadelphia
Collaborators
Auto Dealers Caring for Kids Foundation
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
Brief 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).
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Bronchopulmonary Dysplasia
Keywords
Bronchopulmonary Dysplasia, Neonates
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
16 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Usual Care (Control) Group
Arm Type
No Intervention
Arm Description
Control subjects will be treated as per standard of care for preterm infants with BPD.
Arm Title
Perfluorooctylbromide (PFOB) Group
Arm Type
Active Comparator
Arm Description
Subjects 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.
Intervention Type
Drug
Intervention Name(s)
Perfluorooctyl Bromide
Other Intervention Name(s)
Perflubron
Intervention Description
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.
Primary Outcome Measure Information:
Title
Sustained oxygen desaturations for greater than ten minutes without response to increased oxygen therapy
Description
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.
Time Frame
Day 5, Day 10
Title
Persistent hypotension without response to volume expansion and/or inotropic therapy
Description
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.
Time Frame
Day 5, Day 10
Title
Change in number of major mucus plugging events
Description
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.
Time Frame
Day 5, Day 10
Title
Incidence of pneumothorax or pleural effusion with PFOB
Description
Incidence of pneumothorax in the child will be measured by transillumination of the chest and confirmed by chest x-ray.
Time Frame
Day 5, Day 10
Title
Number of participants with sustained hypercapnia (elevated carbon dioxide in the blood, greater than 95 mmHg, for over four hours).
Description
Hypercapnia will be measured with blood tests and/or cutaneous carbon dioxide monitor.
Time Frame
Day 5, Day 10
Secondary Outcome Measure Information:
Title
Change in fraction of inspired oxygen (FiO2)
Description
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.
Time Frame
Day 5, Day 10
Title
Change in ventilator mean airway pressure (MAP)
Description
Mean airway pressure (MAP) is the average amount of airway pressure supplied to the lungs throughout a breath. Higher MAP indicates more severe disease.
Time Frame
Day 5, Day 10
Title
Change in Respiratory Severity Score (MAP x FiO2)
Description
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.
Time Frame
Day 5, Day 10
10. Eligibility
Sex
All
Maximum Age & Unit of Time
6 Months
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria
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)
Exclusion Criteria
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
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
William Fox, MD
Organizational Affiliation
Children's Hospital of Philadelphia
Official's Role
Principal Investigator
Facility Information:
Facility Name
Children's Hospital of Philadelphia
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
12. IPD Sharing Statement
Plan to Share IPD
No
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Fluid Filled Lung Oxygenation Assistance Trial
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