A Study of PTS100 in Primary HCC Patients

NCT03447951 | Unknown Phase 2

• 1 other identifier: GW-020202
• Study Type: interventional
• Target: 33
• Locations: 1 country
• Sites: 3

Brief Summary

This study is being performed to evaluate the efficacy and safety of 30% PTS100 of total tumor volume as intra-tumoral injection therapy in primary Hepatocellular Carcinoma patients who are ineligible for operation or local regional therapy.

Conditions

Hepatocellular Carcinoma

Outcome Measures

Primary Outcomes (1)

• Objective Response Rate (ORR)

  To evaluate the efficacy, measured by objective response rate (ORR) of pooled treatment groups based on the modified RECIST (mRECIST) criteria at concluding visit.

  4-week post treatment

Secondary Outcomes (10)

• Local disease control rate (LDCR)

  4-week post treatment

• Time to treated tumor progression (TTTTP)

  4-week post treatment, then every 2 months follow up with imaging will be continued for 12 months

• Three-year overall survival (OS)

  Time from first IP administration to patient death or to the date of 3 years after first IP administration

• Time to tumor stage progression (TSP)

  Time from first IP administration to every 2 months follow up with imaging will be continued for 12 months

• Difference in ORR between group 1 and group 2

  4-week post treatment

Study Arms (1)

PTS100 (Para-Toluenesulfonamide): 30%TTV

EXPERIMENTAL

Total dose = 30% total tumor volume

Drug: Para-Toluenesulfonamide

Interventions

Para-Toluenesulfonamide DRUG

A total dose of PTS100 in this study, the expected accumulated dose in the treatment duration, depends on the patient's tumour size. For each patient, up to 4 tumors, each not exceeding 8.8 cm in diameter, are selected for treatment by the joint decision of the investigator and the radiologist. The total dose is defined as the accumulated dose in treatment duration. The total dose is assigned as 30% of TTV for each patient and maximum TTV should be less than 366 cm3.

Also known as: PTS100

PTS100 (Para-Toluenesulfonamide): 30%TTV

Eligibility Criteria

• Age: 20 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female, ≥ 20 years and ≤ 80 years of age.
• Patients with clinically confirmed primary HCC following American Association for the Study of Liver Diseases (AASLD, Appendix 1) guidance:
• Cyto-histological evidence, or
• Coincident imaging evidence using computerized tomography (CT) or magnetic resonance imaging (MRI)
• Based on investigator discretion, patients who are diagnosed at Barcelona Clinic Liver Cancer (BCLC) stage B and are ineffective or unsuitable for resection, immediate liver transplantation, Trans Arterial Chemoembolization (TACE), or current local ablative treatment and meet all of the following conditions at study entry:
• Eastern Cooperative Oncology Group (ECOG) performance status of ≤ 1.
• Child Pugh score class A or B.
• Patients with at least one measurable lesion with size ≥ 1 cm.
• Patients with cumulative total treated tumor volume ≤ 366 cm3 and ≤ 4 target tumors.
• Patients with adequate bone marrow, liver and renal function within 28 days prior to study entry, as defined by the following:
• Hemoglobin \> 10.0 g/dl.
• Absolute neutrophil count (ANC) \> 1,500/mm3.
• Platelet count \> 80k/mm3 correctable by component therapy.
• Albumin ≥ 3 g/dl.
• Total bilirubin \< 2 mg/dL.

You may not qualify if:

• Infiltrative HCC or tumor burden ≥ 50% of liver parenchyma.
• Patients with cumulative total treated tumor volume \> 366 cm3 or more than 4 target tumors.
• Any target tumor exceeds 8.8 cm in diameter.
• The treated tumor is located in close proximity to another organ (e.g. gall bladder, liver capsule, diaphragm ) or major blood vessel and less than 0.5 cm in distance .
• Presence of metastasis or vascular invasion.
• Systemic chemotherapy treatment for HCC within 12 weeks prior to study entry.
• Major surgery within 4 weeks prior to study entry (e.g. thoracolaparotomy is not allowed, but noninvasive surgery, e.g. biopsy, is allowed).
• Use of any investigational drugs, biologics, or devices within 4 weeks prior to study entry or planned use during the course of study.
• Any other severe disease (e.g. active infection, uncontrolled diabetes mellitus, severe heart dysfunction or angina, gastric ulcer, active auto-immune disease) judged by the investigator to limit subject participation in the study.
• Female subjects who are pregnant or lactating. Women of childbearing potential must have a negative urine pregnancy test performed within seven days prior to the start of study drug and agree to practice medically acceptable contraceptive regimen from screening until at least 28 days after the study treatment. Patients who are postmenopausal for at least 1 year (\> 12 months since the last menstrual cycle) or were surgically sterilized do not require the pregnancy test.
• Known or suspected allergy and/or hypersensitivity to any of the ingredients of PTS100.
• Any target lesion blocked by bile ducts or important blood vessels, judged by investigator, that is difficult to conduct intratumoral injection.
• Any condition, judged by investigator, that shows subjects are not suitable for participation.

Sponsors & Collaborators

• Gongwin Biopharm Co., Ltd.: lead

Study Sites (3)

National Taiwan University Hospital

Taipei, Taiwan

RECRUITING

Taipei Medical University Hospital

Taipei, Taiwan

RECRUITING

Taipei Veterans General Hospital

Taipei, Taiwan

RECRUITING

MeSH Terms

Conditions

Carcinoma, Hepatocellular

Interventions

4-toluenesulfonamide

Condition Hierarchy (Ancestors)

Adenocarcinoma; Carcinoma; Neoplasms, Glandular and Epithelial; Neoplasms by Histologic Type; Neoplasms; Liver Neoplasms; Digestive System Neoplasms; Neoplasms by Site; Digestive System Diseases; Liver Diseases

Study Officials

• Ja Der Liang

  National Taiwan University Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Ben Liu

CONTACT

+886-2-2503-5282; ben.liu@gongwinbiopharm.com

Eric Tu

CONTACT

+886-2-2503-5282; erictu@gongwinbiopharm.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 13, 2018
• First Posted: February 27, 2018
• Study Start: June 19, 2018
• Primary Completion: December 1, 2024
• Study Completion: December 1, 2025
• Last Updated: September 13, 2023

Record last verified: 2023-09

Locations

TW (3)