A Clinical Study to Investigate the Efficacy of Intratumoral Tigilanol Tiglate in Soft Tissue Sarcoma

A Phase IIa Open Label Study Evaluating the Preliminary Efficacy of Intratumoural Tigilanol Tiglate in Advanced and/or Metastatic Soft Tissue Sarcoma of the Extremities and Body Wall.

A Phase IIa open label study evaluating the preliminary efficacy of intratumoural tigilanol tiglate in advanced and/or metastatic soft tissue sarcoma of the extremities and body wall.

Study Overview

• Status: Recruiting
• Conditions: Soft Tissue Sarcoma
• Intervention / Treatment: Drug: Tigilanol Tiglate

Detailed Description

Primary Objective

1. To evaluate tumour ablation in tumours and/or tumour segments following one or more treatments with tigilanol tiglate; and
2. To evaluate the effect of tigilanol tiglate on overall disease control (not limited to injected tumours) (Stage 2 only).

Secondary Objective

1. To assess the safety and tolerability of intratumoural injections with tigilanol tiglate; and
2. To evaluate systemic exposure through pharmacokinetic (PK) assessment after a single intratumoural injection of tigilanol tiglate.

Exploratory Objectives

1. To evaluate the microenvironment of injected tumours +/- non-injected tumours;
2. To evaluate the degree of immune response elicited with intratumoural tigilanol tiglate;
3. To evaluate local recurrence rate;
4. To evaluate Progression Free Survival (PFS) (Stage 2 only); and
5. To evaluate metabolites after a single intratumoural injection of tigilanol tiglate (Stage 2 only).

• Study Type: Interventional
• Enrollment (Estimated): 40
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Head of Human Clinical Operations; Phone Number: +61 (0) 738 708 933; Email: enquiries@qbiotics.com

Study Locations

• United States
  • New York
    • New York, New York, United States, 10065
      • Recruiting
      • Memorial Sloan Kettering Cancer Centre
      • Principal Investigator: Edmund Bartlett

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Are willing and able to provide written informed consent for the study prior to any protocol-specific procedures and to comply with all local and study requirements.
2. Are ≥ 18 years of age on the day of providing informed consent.
3. Have advanced and/or metastatic disease of the body wall or extremities that is amenable to intratumoural injection either by palpation or under ultrasound guided injection, that has been histologically or pathologically confirmed as an STS. STS located on the scalp may also be considered for treatment.
4. Have an Eastern Cooperative Oncology Group (ECOG) performance status of ≤ 2.
5. Have life expectancy of more than 12 weeks.
6. Have adequate renal and hepatic function as assessed by the Investigator.
7. Female participants who are Women Of Child-Bearing Potential (WOCBP) must have a negative serum pregnancy test at Screening (within 14 days of the first study drug administration), must be willing to use a highly effective contraception from date of consent, throughout the study period and up to 30 days after the last study drug administration, and must not be breastfeeding.
8. Male participants with a potentially fertile female partner are eligible if they have had a vasectomy or are willing to use adequate contraception from prior to commencement of study drug administration, throughout the study period and up to 30 days after the last study drug administration, and must not donate sperm throughout the study period and up to 30 days after the last study drug administration.

Exclusion Criteria:

1. Are planning to receive intratumoural treatment or radiotherapy to any of the tumours intended for injection within 28 days prior to Screening, or during treatment with tigilanol tiglate.
2. Have a tumour intended for injection that is immediately adjacent to, or with infiltration into, any major artery or vein (e.g., if the tumour for injection is located adjacent to the jugular vein).
3. Are receiving or have received other investigational agents or have used an investigational device without undergoing a 28-day (or 5 half-lives, whichever is shorter) wash-out period prior to their first treatment with tigilanol tiglate. These patients must have recovered from all AEs due to previous investigational therapies to ≤ Grade 1 at baseline.
4. Are receiving or have received systemic anticancer therapy, without undergoing a 28-day (or 5 half-lives, whichever is shorter) wash-out period prior to their first treatment with tigilanol tiglate. These patients must have recovered from all AEs due to previous therapies to ≤ Grade 1 at baseline. Patients with ≤ Grade 2 neuropathy may be eligible following discussion with Sponsor Medical Monitor.
5. Have had major surgery within 28 days of their first treatment with tigilanol tiglate or anticipate the need for major surgery during the study period. Minor surgical procedures are permitted, but with sufficient time for wound healing.
6. Have known, active brain metastases and/or carcinomatous meningitis. Participants who have previously treated brain metastases and are neurologically stable can be included.
7. Have any bleeding diathesis or coagulopathy that would make intratumoural injection or biopsy unsafe, or if they are on therapeutic warfarin therapy.
8. Have a history of allergic reactions or severe hypersensitivity (Grade ≥ 3) attributed to tigilanol tiglate or compounds of similar chemical or biologic composition to tigilanol tiglate, any of its excipients or other agents used in the study.
9. In the opinion of the treating Investigator, they are not an appropriate candidate for the study for any reason (e.g., they have known psychiatric or substance abuse disorder that would interfere with their ability to cooperate with the requirements of the study.

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: Single Arm, Open Label; Single or multiple Intratumoural injections of tigilanol tiglate at up to a fixed dose of 3.6 mg/m2 (Body Surface Area [BSA]) per treatment.	Drug: Tigilanol Tiglate; Tigilanol tiglate is a novel, short-chain diterpene ester in clinical development for intratumoural treatment of a wide range of solid tumours.; Other Names: EBC-46

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Tumour Response	Proportion of participants who have achieved partial or complete ablation of treated tumour(s) and/or tumour segment(s) following injection(s) with tigilanol tiglate.	6 months
ORR as determined by RECIST	Objective Response Rate (ORR) as Determined by Response Evaluation Criteria in Solid Tumours (RECIST) V1.1	3 and 6 months post initial treatment (Stage 2 only)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs)	Total number of Adverse Events (AEs) and Serious Adverse Events (SAEs) and number of AEs and SAEs deemed related to tigilanol tiglate.	6 months
Systemic Exposure	Evaluation of how much tigilanol tiglate is circulating in the blood after a single injection.	Up to 24 hours after the first dose

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Local Recurrence Rate at injection site(s)	Percentage of participants with local recurrence at injection site(s) at 6-months after first treatment.	6 months
Tumour Microenvironment	To assess changes in the tumour microenvironment by looking at the change from baseline of immune cell infiltration in tumour biopsy tissue collected from injected and/or non-injected tumours after injection with tigilanol tiglate.	7 days before and between 28 and 56 days after standard of care (Stage 2).
Metabolite analysis	Metabolite analysis following a single intratumoural injection of tigilanol tiglate (Stage 2 only).	24hrs following first injection.
Evaluation of Peripheral Blood Mononucleocytes (PBMCs)	Evaluation of Peripheral Blood Mononucleocytes (PBMCs).	14 days after first treatment; as well as 7 days before and 14, 28 and 56 days after (only for participants who commence a systemic anti-cancer treatment after treatment)
Progression Free Survival	Assessing number of participants/treated tumours who remain disease free at pre-defined timepoints. (Stage 2 only).	12, 24, 36 and 48 weeks

Collaborators and Investigators

• Sponsor: QBiotics Group Limited
• Investigators: Principal Investigator: Edmund Bartlett, MD, Memorial Sloan Kettering Cancer Center

Study record dates

Study Major Dates

• Study Start (Actual): April 13, 2023
• Primary Completion (Estimated): January 1, 2027
• Study Completion (Estimated): June 1, 2027

Study Registration Dates

• First Submitted: February 16, 2023
• First Submitted That Met QC Criteria: March 2, 2023
• First Posted (Actual): March 6, 2023

Study Record Updates

• Last Update Posted (Actual): March 12, 2026
• Last Update Submitted That Met QC Criteria: March 10, 2026
• Last Verified: February 1, 2026

More Information

Terms related to this study

• Keywords: metastatic; advanced; extremeties; body wall
• Additional Relevant MeSH Terms: Pathologic Processes; Neoplasms; Neoplasms by Histologic Type; Neoplastic Processes; Neoplasms, Connective and Soft Tissue; Pathological Conditions, Signs and Symptoms; Neoplasm Metastasis; Sarcoma; EBC-46
• Other Study ID Numbers: QB46C-H07; U1111-1282-2967 (Other Identifier: [WHO Universal Trial Number (UTN)])

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No