First-in-Human Expanded Cohort Study of Intrapleural Administration of TolueneSulfonamide in Patients With Malignant Pleural Effusion

NCT07332858 | Not Yet Recruiting Phase 1 DMC

• 1 other identifier: 2025-HX-183
• Study Type: interventional
• Target: 105
• Locations: 1 country
• Sites: 1

Brief Summary

This study aims to evaluate the safety of intrapleural injection of TolueneSulfonamide in patients with malignant pleural effusion and to explore its potential effectiveness.

Conditions

Malignant Pleural Effusions (Mpe); Non Small Cell Lung Cancer

Keywords

Malignant Pleural Effusion; Para-toluenesulfonamide

Outcome Measures

Primary Outcomes (1)

• 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).

  4 weeks, 12 weeks, 6 months, and 12 months after treatment completion

Secondary Outcomes (5)

• Percentage Change in Pleural Effusion Volume

  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)

  through study completion, an average of 1 year

• Incidence of Grade ≥3 treatment-emergent adverse events

  through study completion, an average of 1 year

• mMRC Dyspnea Score

  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

  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

Study Arms (2)

Intrapleural PTS injection plus catheter drainage group

EXPERIMENTAL

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.

Drug: Intrapleural administration of TolueneSulfonamide

catheter drainage alone group

PLACEBO COMPARATOR

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.

Drug: Intrapleural administration of normal saline injection

Interventions

Intrapleural administration of TolueneSulfonamide DRUG

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.

Intrapleural PTS injection plus catheter drainage group

Intrapleural administration of normal saline injection DRUG

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.

catheter drainage alone group

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age between 18 and 75 years (inclusive) at the time of signing the informed consent form.
• Histologically or cytologically confirmed malignant pleural effusion, with the primary tumor diagnosed as non-small cell lung cancer (NSCLC).
• Eastern Cooperative Oncology Group (ECOG) performance status of 0-2, with an expected survival of at least 3 months.
• Presence of dyspnea symptoms.
• No prior local thoracic treatment within 1 month before enrollment, including intrapleural drug administration or thoracic radiotherapy (diagnostic thoracentesis is permitted).
• Except for the subject's current stable systemic anti-tumor therapy, any other ongoing tumor-related treatments must be suspended or discontinued after evaluation by the investigator to ensure they do not interfere with the assessment of PTS treatment.
• Fully understands the study objectives and procedures, agrees to participate in the study, and provides written informed consent.

You may not qualify if:

• History of allergy or hypersensitivity to PTS or any of its excipients.
• Presence of uncontrolled intrapleural infection or severe loculated pleural effusion that is difficult to manage.
• Participation in another interventional clinical study within 3 months prior to enrollment (diagnostic studies are excluded).
• Severe organ dysfunction, including but not limited to coagulation disorders, congestive heart failure, malignant arrhythmias, coronary artery disease requiring long-term medication, valvular heart disease, myocardial infarction, or refractory hypertension; severe cardiac, hepatic, or renal insufficiency, or active bleeding or thrombotic risk.
• Presence of uncontrolled infectious wounds.
• Pregnant or breastfeeding women.
• Any other condition that, in the investigator's judgment, makes the subject unsuitable for participation in this study.
• Criteria for withdrawal from the study.

Sponsors & Collaborators

• Gang Hou: lead
• The Affiliated Hospital of Xuzhou Medical University: collaborator
• Anhui Chest Hospital: collaborator
• The Second Affiliated Hospital of Harbin Medical University: collaborator

Study Sites (1)

China-Japan Friendship Hospital

Beijing, Beijing Municipality, 100029, China

Location

Related Publications (8)

• Guan WJ, Li SY, Zhong NS. Effects of para-toluenesulfonamide intratumoral injection on pulmonary adenoid cystic carcinoma complicating with severe central airway obstruction: a 5-year follow-up study. J Thorac Dis. 2018 Apr;10(4):2448-2455. doi: 10.21037/jtd.2018.03.70.

  PMID: 29850151 BACKGROUND

• Li SY, Li Q, Guan WJ, Huang J, Yang HP, Wu GM, Jin FG, Hu CP, Chen LA, Xu GL, Liu SZ, Wu CG, Han BH, Xiang Y, Zhao JP, Wang J, Zhou X, Li HP, Zhong NS. Effects of para-toluenesulfonamide intratumoral injection on non-small cell lung carcinoma with severe central airway obstruction: A multi-center, non-randomized, single-arm, open-label trial. Lung Cancer. 2016 Aug;98:43-50. doi: 10.1016/j.lungcan.2016.05.012. Epub 2016 May 17.

  PMID: 27393505 BACKGROUND

• Xu YF, Chen YR, Bu FL, Huang YB, Sun YX, Li CY, Sellick J, Liu JP, Qin DM, Liu ZL. Chinese herbal injections versus intrapleural cisplatin for lung cancer patients with malignant pleural effusion: A Bayesian network meta-analysis of randomized controlled trials. Front Oncol. 2022 Sep 20;12:942941. doi: 10.3389/fonc.2022.942941. eCollection 2022.

  PMID: 36203451 BACKGROUND

• Dong X, Huang Y, Yi T, Hu C, Gao Q, Chen Y, Zhang J, Chen J, Liu L, Meng R, Zhang S, Dai X, Fei S, Jin Y, Yin P, Hu Y, Wu G. Intrapleural infusion of tumor cell-derived microparticles packaging methotrexate or saline combined with pemetrexed-cisplatin chemotherapy for the treatment of malignant pleural effusion in advanced non-squamous non-small cell lung cancer: A double-blind, randomized, placebo-controlled study. Front Immunol. 2022 Oct 5;13:1002938. doi: 10.3389/fimmu.2022.1002938. eCollection 2022.

  PMID: 36275698 BACKGROUND

• Feller-Kopman DJ, Reddy CB, DeCamp MM, Diekemper RL, Gould MK, Henry T, Iyer NP, Lee YCG, Lewis SZ, Maskell NA, Rahman NM, Sterman DH, Wahidi MM, Balekian AA. Management of Malignant Pleural Effusions. An Official ATS/STS/STR Clinical Practice Guideline. Am J Respir Crit Care Med. 2018 Oct 1;198(7):839-849. doi: 10.1164/rccm.201807-1415ST.

  PMID: 30272503 BACKGROUND

• Porcel JM, Cordovilla R, Tazi-Mezalek R, Barrios-Barreto D, Perez-Pallares J, Novais E Bastos H, Martinez-Tomas R, Flandes-Aldeyturriaga J, Cases-Viedma E, Recalde B, Botana-Rial M. Efficacy and Safety of Indwelling Catheter for Malignant Pleural Effusions Related to Timing of Cancer Therapy: A Systematic Review. Arch Bronconeumol. 2023 Sep;59(9):566-574. doi: 10.1016/j.arbres.2023.06.007. Epub 2023 Jun 23. English, Spanish.

  PMID: 37429748 BACKGROUND

• 北京肿瘤学会临床研究专委会，中国医院协会肿瘤医院分会，王书航，等。恶性胸腔积液干预性临床研究设计专家共识[J]. 中华肿瘤防治杂志, 2025,32(8): 455-461.

  BACKGROUND

• Kumar A, Xu B, Srinivasan D, Potter AL, Raman V, Lanuti M, Yang CJ, Auchincloss HG. Long-Term Survival of American Joint Committee on Cancer 8th Edition Staging Descriptors for Clinical M1a Non-Small Cell Lung Cancer. Chest. 2024 Mar;165(3):725-737. doi: 10.1016/j.chest.2023.07.4220. Epub 2023 Aug 5.

  PMID: 37544427 BACKGROUND

MeSH Terms

Conditions

Pleural Effusion, Malignant; Myeloproliferative Disorder, Chronic, with Eosinophilia; Carcinoma, Non-Small-Cell Lung

Condition Hierarchy (Ancestors)

Pleural Neoplasms; Respiratory Tract Neoplasms; Thoracic Neoplasms; Neoplasms by Site; Neoplasms; Pleural Effusion; Pleural Diseases; Respiratory Tract Diseases; Carcinoma, Bronchogenic; Bronchial Neoplasms; Lung Neoplasms; Lung Diseases

Study Officials

• Gang Hou, PhD

  China-Japan Friendship Hospital

  STUDY DIRECTOR

Central Study Contacts

Gang Hou, PhD

CONTACT

＋86 13840065481; hougangcmu@163.com

Liwei Liao, PhD

CONTACT

＋86 18090027855; 291633325@qq.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Chief Physician

Model Details: This study consists of two parts. Part 1 is a first-in-human study enrolling 15 patients with malignant pleural effusion (MPE) to assess the safety of intrapleural PTS. Part 2 will enroll 90 patients with MPE and, after safety is confirmed, randomize them 2:1 to receive PTS plus catheter drainage or normal saline plus catheter drainage. Treatment is given in weekly cycles with dosing on Days 1, 3, and 5. Pleural effusion is drained before each dose, followed by intrapleural administration and catheter clamping. After treatment, standard drainage and supportive care are continued.

Study Record Dates

• First Submitted: December 19, 2025
• First Posted: January 12, 2026
• Study Start: January 20, 2026
• Primary Completion (Estimated): October 30, 2028
• Study Completion (Estimated): October 30, 2028
• Last Updated: January 14, 2026

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will share

Locations

CN (1)