A Parallel Group, 1:1 Randomised, Pilot Study Comparing the Clinical Efficacy and Bleeding Risk Between Low Dose and...

Area of Science:

• Pulmonology
• Thoracic Surgery
• Clinical Trials

Background:

• Unresolved pleural infection poses a significant clinical challenge.
• Intrapleural fibrinolysis with tissue plasminogen activator (tPA) and deoxyribonucleases (DNase) is a treatment option.
• Dosing strategies for intrapleural tPA require further investigation.

Purpose of the Study:

• To assess the feasibility of a randomized controlled trial protocol comparing low-dose (2.5 mg) versus standard-dose (10 mg) intrapleural tPA with DNase.
• To evaluate the acceptability and potential for future implementation of the proposed trial design.
• To gather preliminary data on patient recruitment, retention, and adherence for a larger study.

Main Methods:

• A single-center, two-arm, double-blinded, randomized controlled feasibility study.
• Inclusion of patients with unresolved pleural infection eligible for intrapleural tPA/DNase.
• Randomization to receive a maximum of 6 doses of intrapleural tPA (2.5 mg or 10 mg) with DNase, with an option to convert to open-label 10 mg doses.
• Follow-up for 3 months post-hospital discharge.

Main Results:

• The study aims to determine feasibility based on enrollment, retention, and follow-up completion rates.
• Key outcomes include survival, need for surgery, additional pleural interventions, and clinical/radiographic response.
• Safety outcomes, particularly bleeding complications, will be assessed descriptively.

Conclusions:

• The proposed randomization trial protocol for comparing low-dose and standard-dose intrapleural tPA with DNase is feasible and acceptable.
• Findings will inform the design of future definitive trials in unresolved pleural infection.
• This pilot study provides essential data for optimizing treatment strategies and trial methodology.