Treatment for primary liver cancer using PTS100 injections

A Phase II Study to Assess the Safety and Efficacy of PTS100 in Primary Hepatocellular Carcinoma Patients Who Are Ineligible for Operation or Current Locoregional Therapy

This study is testing if PTS100 injections can help people with primary liver cancer who can't have surgery feel better and improve their condition.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 33 (estimated)
• Ages: 20 Years to 80 Years
• Sex: All
• Sponsor: Gongwin Biopharm Co., Ltd. (Industry-sponsored)
• Drugs / interventions: chemotherapy
• Locations: 3 sites (Taipei City and 2 other locations)
• Trial ID: NCT03447951 on ClinicalTrials.gov

What this trial studies

This study evaluates the efficacy and safety of PTS100, administered as an intratumoral injection therapy, in patients with primary Hepatocellular Carcinoma (HCC) who are not eligible for surgery or local regional therapies. Participants will receive 30% of their total tumor volume in PTS100 through multiple injections, with the treatment adjusted based on individual tolerability. The treatment course continues until the total dose is completed, and efficacy will be assessed 28 days post-treatment.

Who should consider this trial

Good fit: Ideal candidates are adults aged 20 to 80 with clinically confirmed primary HCC who are not suitable for surgical or local therapies.

Not a fit: Patients with advanced liver disease or those who do not meet the inclusion criteria for tumor size and performance status may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new therapeutic option for patients with inoperable primary liver cancer.

How similar studies have performed: While similar approaches have been explored, the specific use of PTS100 in this context is novel and has not been extensively tested in prior studies.

Eligibility criteria

Inclusion Criteria:

* 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:

  1. Cyto-histological evidence, or
  2. 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:

  1. Eastern Cooperative Oncology Group (ECOG) performance status of ≤ 1.
  2. 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:

  1. Hemoglobin \> 10.0 g/dl.
  2. Absolute neutrophil count (ANC) \> 1,500/mm3.
  3. Platelet count \> 80k/mm3 correctable by component therapy.
  4. Albumin ≥ 3 g/dl.
  5. Total bilirubin \< 2 mg/dL.
  6. Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) \< 5 x upper normal limit (UNL).
  7. Blood urea nitrogen (BUN) and serum creatinine \< 1.5 x UNL.
  8. International normalized ratio (INR) \< 1.5 or prothrombin time (PT) \< 15 seconds.
* Patients with life expectancy \> 3 months as judged by investigator.
* Patients who understand and comply to the study procedure and be willing to provide a written informed consent form.

Exclusion Criteria:

* 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.

Where this trial is running

Taipei City and 2 other locations

• Taipei Medical University Hospital — Taipei City, Taiwan (Recruiting)
• Taipei Veterans General Hospital — Taipei City, Taiwan (Recruiting)
• National Taiwan University Hospital — Taipei, Taiwan (Recruiting)

Study contacts

• Principal investigator: Ja Der Liang — National Taiwan University Hospital
• Study coordinator: Ben Liu

Conditions Hepatocellular Carcinoma