This study aims to evaluate the safety of intrapleural injection of TolueneSulfonamide in patients with malignant pleural effusion and to explore its potential effectiveness.
This study is a first-in-human (FIM) expansion cohort study designed to evaluate the safety and clinical activity of intrapleural injection of PTS in patients with malignant pleural effusion (MPE). Patients with MPE will be prospectively enrolled, and the study will be conducted in two parts: a safety and dose-exploration phase of intrapleural PTS administration (Part 1, P1), followed by an efficacy evaluation phase of intrapleural PTS for the treatment of MPE (Part 2, P2). Part 2 will be initiated after the safety of PTS has been established in Part 1. Part 1 is designed as a prospective, single-arm, open-label study. Part 2 is designed as a prospective, multicenter, double-blind, randomized controlled trial with two groups: an experimental group and a control group. The experimental group will receive catheter drainage plus intrapleural PTS injection, while the control group will receive catheter drainage plus intrapleural normal saline injection.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
105
PTS Administration (One Treatment Cycle): One treatment cycle is defined as one week. PTS is administered three times per week on Days 1, 3, and 5. Prior to each administration, pleural effusion is drained as completely as possible until no further fluid can be obtained. The study drug is then administered via the indwelling drainage catheter, followed by an additional flush of 20 mL of normal saline. The drainage catheter is clamped for at least 24 hours after administration. After each injection, patients are instructed to maintain prone, lateral, and supine positions for approximately 30 minutes each to facilitate even distribution of the drug within the pleural cavity. During the same treatment cycle, the drainage catheter remains clamped until the next scheduled administration. After completion of treatment, catheter drainage and standard care are continued.
Normal Saline Administration (One Treatment Cycle): One treatment cycle is defined as one week. Normal saline is administered three times per week on Days 1, 3, and 5. Prior to each administration, pleural effusion is drained as completely as possible until no further fluid can be obtained. Normal saline is then administered via the indwelling drainage catheter, followed by an additional flush of 20 mL of normal saline. The drainage catheter is clamped for at least 24 hours after administration. After each administration, patients are instructed to maintain prone, lateral, and supine positions for approximately 30 minutes each. During the same treatment cycle, the drainage catheter remains clamped until the next scheduled administration. After completion of treatment, catheter drainage and standard care are continued.
China-Japan Friendship Hospital
Beijing, Beijing Municipality, China
Objective Response Rate
Objective Response Rate (ORR) is defined as the proportion of patients achieving complete response (CR) or partial response (PR) among all enrolled patients, calculated as (CR + PR) / total number of patients × 100%, based on WHO criteria and assessed by imaging methods (ultrasound or computed tomography).
Time frame: 4 weeks, 12 weeks, 6 months, and 12 months after treatment completion
Percentage Change in Pleural Effusion Volume
The rate of change in pleural effusion volume is calculated as (baseline pleural effusion volume - post-treatment pleural effusion volume) / baseline pleural effusion volume, with pleural effusion volume assessed by ultrasound and computed tomography (CT)
Time frame: Baseline; after one treatment cycle (each cycle is 7 days); and at 4 weeks, 12 weeks, 6 months, and 12 months of follow-up
Overall Survival (OS)
the time from enrollment to death from any cause
Time frame: through study completion, an average of 1 year
Incidence of Grade ≥3 treatment-emergent adverse events
Incidence of Grade ≥3 treatment-emergent adverse events
Time frame: through study completion, an average of 1 year
mMRC Dyspnea Score
Dyspnea will be assessed using the modified Medical Research Council (mMRC) Dyspnea Scale.
Time frame: At baseline, after one treatment cycle (each cycle is 7 days), and at 4 weeks, 12 weeks, 6 months, and 12 months during follow-up
Quality of Life score
Quality of life will be assessed using the EORTC QLQ-C30 (core questionnaire) together with the QLQ-LC13 (lung cancer-specific module), or the EuroQol five-dimension questionnaire (EQ-5D).
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Time frame: At baseline, after one treatment cycle (each cycle is 7 days), and at 4 weeks, 12 weeks, 6 months, and 12 months during follow-up