A Study of Syngenon (BSG-001) for Inhalation in Subjects With Malignant Pleural Effusion and/or Malignant Ascites

Study Overview
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Brief Summary
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Malignant pleural effusion and/ or malignant ascites is generally defined by presence of malignant cells in the effusion fluid. The first-line therapies are mostly intrusive, medically demanding and inefficient, and therefore, it is important to study and develop new therapeutic option to address the unmet need. This protocol for BSG-001 is developed for the treatment of malignant pleural effusion and/ or malignant ascites. BSG-001 is an immune-modulator primarily exerts its effect via Toll-like receptor. The purpose of this study is to assess the safety and tolerability of BSG-001. All eligible subjects will receive BSG-001 for at least 12 weeks (3 cycles).

Detailed Description
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This study is a phase 1/2a, single arm study with main purpose to evaluate the safety, tolerability and efficacy of BSG-001 in subjects with cancer that causes an abnormal amount of fluid to collect between the thin layers of tissue (pleura) lining the outside of the lung and the wall of the chest cavity, namely malignant pleural effusion and/ or subjects with cancer that causes the accumulation of fluid in the peritoneal cavity, causing abdominal swelling, namely malignant ascites. The study aims to recruit 9 - 18 subjects in phase 1, and once the safety, tolerability and the preliminary efficacy of BSG-001 reach an optimal target exposure for recommended dose (RD), phase 2a will be opened for enrolment of approximately 40 subjects.

Official Title
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A Phase I/IIa Study of Syngenon (BSG-001) for Inhalation in Subjects With Malignant Pleural Effusion and/or Malignant Ascites

Conditions
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Malignant Pleural Effusion, Malignant Ascites

Intervention / Treatment
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* Drug: BSG-001


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Histologically or cytologically documented malignant pleural effusion and/or malignant ascites OR morphological diagnosis of malignant pleural effusion and/or malignant ascites by CT or ultrasound Histologically confirmed cancer Malignant pleural effusion and/or malignant ascites clinically judged as not responsive to conventional systemic therapy(ies) for primary malignancy Adequate liver and renal function as defined below: Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2 Life expectancy of > 12 weeks Willing and able to provide written, signed informed consent after the nature of the study has been explained, and prior to any research-related procedures Females of childbearing potential must have a negative serum pregnancy test at screening and be willing to have additional serum pregnancy tests during the study. Willing and able to comply with all study procedures Exclusion Criteria: Presence of > grade 3 active infection or gastric bleeding at the time of screening Change in chemotherapy regimen within 28 days before Day 1 of study drug administration Concurrent use of any investigational product (IP) or investigational medicine within 28 days before Day 1 of study drug administration Symptomatic interstitial lung disease or inflammatory pneumonitis Concurrent disease or condition which, in the opinion of the Investigator, would pose a risk to patient safety or interfere with study participation or interpretation of individual patient results Breastfeeding at screening or planning to become pregnant (self or partner) at any time during study participation

Ages Eligible for Study
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Minimum Age: 21 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: BSG-001<br>Inhalation route, daily | Drug: BSG-001<br>* BSG-001 is in solution form, 4mg per vial (2mg/mL) with Inhalation as route of administration using nebulizer. BSG-001 will be administrated as an approximately 5 - 30 minutes inhalation session daily for 3 cycles (in repeated 28 days cycle).<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Safety and Tolerability of BSG-001 assessed with incidence of Treatment-Emergent Adverse Events (AEs) and Serious AEs | Treatment-Emergent Adverse Events (AEs) and Serious AEs will be assessed by CTCAE v4.0 or higher version | From start of treatment throughout 12 weeks dosing | 
| Efficacy of BSG-001 assessed with change in fluid volume of Malignant Pleural Effusion and/or Malignant Ascites | Efficacy of BSG-001 will be assessed with change in frequency and total change volume (for subjects require paracentesis and/or thoracentesis) and assessed by radiographic imaging (CT/ ultrasound) (for subjects without catheter drainage) | From start of treatment throughout 12 weeks dosing, until disease progression and onward survival follow up up to 6 months or study completion, whichever came first. | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Recommended dose (RD) of BSG-001 | RD will be assessed with incidence of BSG-001-related Adverse Events Grading during the dose limiting toxicity (DLT) period | Start of treatment throughout 28-day DLT dosing period |