Inhaled Tissue Plasminogen Activator for Acute Plastic Bronchitis
Completed · Phase 2
Conditions studied: Plastic Bronchitis, Protein-Losing Enteropathies, Healthy
In brief
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
Key facts
- Study ID
- NCT02315898
- Run by
- University of Michigan
- People needed
- 40
- Starts
- 2018-03-19
- Expected to finish
- 2023-06-30
- Last updated by the study team
- 2025-08-19
Who can join
Age: 5 and older, up to 24. Sex: any. Healthy volunteers: accepted.
Where it is running
- Lucile Packard Children's Hospital, Stanford University — Palo Alto, California, United States
- Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois, United States
- University of Michigan Mott Children's Hospital — Ann Arbor, Michigan, United States
- Cincinnati Children's Hospital — Cincinnati, Ohio, United States
- Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
- Medical University of South Caroline — Charleston, South Carolina, United States
Full record on ClinicalTrials.gov
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