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

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

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).

Study Overview

• Status: Unknown
• Conditions: Malignant Ascites; Malignant Pleural Effusion
• Intervention / Treatment: Drug: BSG-001

Detailed Description

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.

• Study Type: Interventional
• Enrollment (Anticipated): 58
• Phase: Phase 2; Phase 1

Contacts and Locations

• Study Contact: Name: Grace Khoo Koay; Phone Number: (65) 6926 0818; Email: clinicaltrials@biosg.com

Study Locations

• Australia
  • South Australia
    • Adelaide, South Australia, Australia, 5042
      • Flinders Medical Centre
      • Contact: Kathrina Bartolay; Phone Number: +61 8 8404 2201; Email: Kathrina.Bartolay@sa.gov.au
      • Principal Investigator: Anand Rose, MD

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 19 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

1. 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
2. Histologically confirmed cancer
3. Malignant pleural effusion and/or malignant ascites clinically judged as not responsive to conventional systemic therapy(ies) for primary malignancy
4. Adequate liver and renal function as defined below:
5. Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2
6. Life expectancy of > 12 weeks
7. 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
8. 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.
9. Willing and able to comply with all study procedures

Exclusion Criteria:

1. Presence of > grade 3 active infection or gastric bleeding at the time of screening
2. Change in chemotherapy regimen within 28 days before Day 1 of study drug administration
3. Concurrent use of any investigational product (IP) or investigational medicine within 28 days before Day 1 of study drug administration
4. Symptomatic interstitial lung disease or inflammatory pneumonitis
5. 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
6. Breastfeeding at screening or planning to become pregnant (self or partner) at any time during study participation

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: BSG-001; Inhalation route, daily	Drug: BSG-001; 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).

What is the study measuring?

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

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Subjects' reported quality of life [European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire (EORTCQLQ-30), version 3]	Reported quality of life will be assessed through EORTC QLQ-30 questionnaire score change from baseline, by a four-point scale (first 28 questions) and a 7-point scale (last 2 questions) total of 30 questions, where higher score represents higher (better) response level. Calculation is based on linear transformation to standardise the raw score, so that scores range from 0 to 100.	From screening/ baseline, followed by Day 1 of every cycle prior to other procedures (each cycle is 28 days) for 3 cycles.

Collaborators and Investigators

• Sponsor: BioSyngen Pte Ltd
• Investigators: Study Director: Lietao Li, MD, BioSyngen Australia Pty Ltd

Study record dates

Study Major Dates

• Study Start (Anticipated): January 1, 2019
• Primary Completion (Anticipated): January 1, 2022
• Study Completion (Anticipated): January 1, 2022

Study Registration Dates

• First Submitted: October 30, 2018
• First Submitted That Met QC Criteria: November 7, 2018
• First Posted (Actual): November 8, 2018

Study Record Updates

• Last Update Posted (Actual): November 8, 2018
• Last Update Submitted That Met QC Criteria: November 7, 2018
• Last Verified: October 1, 2018

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Respiratory Tract Diseases; Neoplasms; Neoplasms by Site; Pleural Diseases; Respiratory Tract Neoplasms; Thoracic Neoplasms; Pleural Neoplasms; Ascites; Pleural Effusion, Malignant; Pleural Effusion
• Other Study ID Numbers: BSG-001-001

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No