Clinical trial NCT03041740: Fluid Filled Lung Oxygenation Assistance Trial
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
Overall status: COMPLETED
Phase: PHASE1
Conditions: Bronchopulmonary Dysplasia
Keywords: Bronchopulmonary Dysplasia
Neonates
Lead sponsor: Children's Hospital of Philadelphia
Sponsor class: OTHER
Collaborators: name: Auto Dealers Caring for Kids Foundation
First posted: 2017-02-03
Enrollment: 16
Sex: ALL
Maximum age (years): 6
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

Eligibility criteria: Inclusion Criteria

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

Exclusion Criteria

1. Mechanical ventilation for acute disease, such as for infection or for post-operative complications
2. 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.
3. Pneumothorax (active air leak) requiring chest tube within 72 hours of T=0
4. Active pulmonary hemorrhage within 72 hours of T=0
5. History of Grade III/IV interventricular hemorrhage without resolution or stability within 3 weeks of verifying eligibility
6. Severe congenital heart disease compromising pulmonary circulation
7. Other major congenital malformation (including but not limited to CDH) or known genetic syndromes at the discretion of the investigator
8. Use of an investigational drug within 7 days prior to confirmation of eligibility.
9. The clinical attending physician believes it is not in the subject's and/or parents/guardians best interest to participate in the trial.

Primary outcomes:
measure: Sustained oxygen desaturations for greater than ten minutes without response to increased oxygen therapy
timeFrame: Day 5, Day 10
measure: Persistent hypotension without response to volume expansion and/or inotropic therapy
timeFrame: Day 5, Day 10
measure: Change in number of major mucus plugging events
timeFrame: Day 5, Day 10
measure: Incidence of pneumothorax or pleural effusion with PFOB
timeFrame: Day 5, Day 10
measure: Number of participants with sustained hypercapnia (elevated carbon dioxide in the blood, greater than 95 mmHg, for over four hours).
timeFrame: Day 5, Day 10
Secondary outcomes:
measure: Change in fraction of inspired oxygen (FiO2)
timeFrame: Day 5, Day 10
measure: Change in ventilator mean airway pressure (MAP)
timeFrame: Day 5, Day 10
measure: Change in Respiratory Severity Score (MAP x FiO2)
timeFrame: Day 5, Day 10
Other outcomes:
Locations:
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
Study officials:
affiliation: Children's Hospital of Philadelphia
name: William Fox, MD
role: PRINCIPAL_INVESTIGATOR
References: