Fluid Filled Lung Oxygenation Assistance Trial

NCT03041740 | Completed Phase 1 DMC FDA Drug

• 1 other identifier: 12-008686
• Study Type: interventional
• Target: 16
• Locations: 1 country
• Sites: 1

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).

Conditions

Bronchopulmonary Dysplasia

Keywords

Bronchopulmonary Dysplasia; Neonates

Outcome Measures

Primary Outcomes (5)

• 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.

  Day 5, Day 10

• 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.

  Day 5, Day 10

• 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.

  Day 5, Day 10

• 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.

  Day 5, Day 10

• 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.

  Day 5, Day 10

Secondary Outcomes (3)

• Change in fraction of inspired oxygen (FiO2)

  Day 5, Day 10

• Change in ventilator mean airway pressure (MAP)

  Day 5, Day 10

• Change in Respiratory Severity Score (MAP x FiO2)

  Day 5, Day 10

Study Arms (2)

Usual Care (Control) Group

NO INTERVENTION

Control subjects will be treated as per standard of care for preterm infants with BPD.

Perfluorooctylbromide (PFOB) Group

ACTIVE COMPARATOR

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.

Drug: Perfluorooctyl Bromide

Interventions

Perfluorooctyl Bromide DRUG

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.

Also known as: Perflubron

Perfluorooctylbromide (PFOB) Group

Eligibility Criteria

• Age: Up to 6 Months
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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)

You may not qualify if:

• 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.

Sponsors & Collaborators

• Children's Hospital of Philadelphia: lead
• Auto Dealers Caring for Kids Foundation: collaborator

Study Sites (1)

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Related Publications (1)

• 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.

  PMID: 37549675 DERIVED

MeSH Terms

Conditions

Bronchopulmonary Dysplasia

Interventions

perflubron

Condition Hierarchy (Ancestors)

Ventilator-Induced Lung Injury; Lung Injury; Lung Diseases; Respiratory Tract Diseases; Infant, Premature, Diseases; Infant, Newborn, Diseases; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• William Fox, MD

  Children's Hospital of Philadelphia

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Attending Physician

Study Record Dates

• First Submitted: January 30, 2017
• First Posted: February 3, 2017
• Study Start: June 27, 2017
• Primary Completion: June 10, 2019
• Study Completion: June 10, 2019
• Last Updated: October 21, 2022

Record last verified: 2022-10

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)