Inhaled Tissue Plasminogen Activator for Acute Plastic Bronchitis
Status:
Recruiting
Trial end date:
2022-12-31
Target enrollment:
Participant gender:
Summary
Plastic bronchitis (PB) is a rare, most often pediatric disease characterized by the
formation of obstructive airway casts primarily composed of fibrin. There is presently no
FDA-approved pharmacotherapy for PB, but acute exacerbations of the illness are often treated
with inhaled tissue plasminogen activator (tPA). To date, this is done somewhat anecdotally
because there has been no safety or efficacy testing of this treatment. In addition, there is
presently no reliable surrogate marker of adverse drug events. Nevertheless, in the absence
of inhaled tPA treatment, PB-induced respiratory distress can be severe, often warranting
urgent or emergent bronchoscopy for cast removal, or can sometimes result in respiratory
failure. As such there is a significant unmet need for safety and efficacy testing of inhaled
tPA and for biomarkers of drug response.
Objectives and Endpoints: The objectives of this protocol are to: 1) test the safety and
efficacy of an inhaled tPA regimen in children with PB; and 2) identify potential candidate
biomarkers of inhaled tPA drug response. Safety endpoints will consist of the development of
new, active bleeding that is systemic and/or pulmonary and/or new hematuria (defined as gross
hematuria). Secondary endpoints of efficacy will also be measured (e.g., frequency of cast
production). Urine and blood will also be collected for the development of potential
biomarkers of inhaled tPA drug response.
Funding source- FDA OOPD