Hepatocellular CarcinomaPhase 2OASESNCT03447951
A Study of PTS100 in Primary HCC Patients
A Phase 2 study testing Para-Toluenesulfonamide in people with Hepatocellular Carcinoma, run by Gongwin Biopharm Co., Ltd. at 3 sites in Taiwan.
Trial 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. Read more about this study
- Who is it for?
- Ages 20 to 80 years
- Conditions the study is for
- Hepatocellular Carcinoma
- What is being tested?
- Para-Toluenesulfonamide
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 33 participants
- Where
- 3 sitesTaiwan
- Study dates
- 19 Jun 2018to Dec 2025
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 20 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
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- 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.
- Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) < 5 x upper normal limit (UNL).
- Blood urea nitrogen (BUN) and serum creatinine < 1.5 x UNL.
- 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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
3 sites in 1 country.
Taiwan3 sites, 3 recruiting
- Recruiting
National Taiwan University Hospital
Taipei
Ja Der Liang
- Recruiting
Taipei Medical University Hospital
Taipei
Wei-Yu Kao, MD
Wei-Yu Kao, MD
- Recruiting
Taipei Veterans General Hospital
Taipei
Yi-Hsiang Huang, MD
Yi-Hsiang Huang, MD
About this study
Official title: 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 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.
Read the detailed description
BCLC stage B patients who are ineligible for operational or current locoregional therapy. Enrolled patients are assigned to 30% of total tumor volume. PTS100 is administrated via intratumoral injection. The required total dose is given in separate injection days, with maximal daily dose up to 10 mL at first IP administration, and is adjusted based on each patient's tolerability along the treatment. The treatment course lasts up to completion of the total dose, and the efficacy is evaluated 28 days afterwards.
- Conditions
- Hepatocellular Carcinoma
- Being tested
- Para-Toluenesulfonamide
- Medical subject headings
- Carcinoma, Hepatocellular
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Para-ToluenesulfonamideDrug
Also called PTS100
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.
| Study group | Role | What they receive |
|---|---|---|
| PTS100 (Para-Toluenesulfonamide): 30%TTV | Experimental | Total dose = 30% total tumor volume Receives: Drug: Para-Toluenesulfonamide |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 4-week post treatment
Secondary outcomes (10)
Local disease control rate (LDCR)
Local disease control rate (LDCR) of pooled treatment groups based on the modified RECIST (mRECIST) criteria at concluding visit.
Measured: 4-week post treatment
Time to treated tumor progression (TTTTP)
Time to treated tumor progression (TTTTP) of pooled treatment groups
Measured: 4-week post treatment, then every 2 months follow up with imaging will be continued for 12 months
Three-year overall survival (OS)
Three-year overall survival of pooled treatment groups
Measured: 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 to tumor stage progression of pooled treatment groups
Measured: 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
Difference in ORR between 20% group and 30% group at concluding visit
Measured: 4-week post treatment
Difference in LDCR between group 1 and group 2
Difference in LDCR between 20% group and 30% group at concluding visit
Measured: 4-week post treatment
Difference in TTTTP between group 1 and group 2
Difference in TTTTP between 20% group and 30% group at concluding visit
Measured: 4-week post treatment to every 2 months follow up with imaging will be continued for 12 months
Incidence of Treatment-Emergent Adverse Events
Incidence of Treatment-Emergent Adverse Events as assessed by CTCAE v4.0
Measured: Enrollment to 4-week post treatment
FACT-Hep quality of life score
To evaluate the health-related Quality of life, assessed by change in Functional Assessment of Cancer Therapy-Hepatobiliary (FACT-Hep, version 4, Traditional Chinese). The FACT-Hep, version 4, Traditional Chinese Scale is a patient completed questionnaire consisting 45 items that assesses health related quality of life (HRQL) in HCC patients. The FACT-Hep, version 4, Traditional Chinese consists of the 27-item FACT-G assessing generic HRQL concerns and 18-item Hepatobiliary Subscale assessing disease-specific issues. Instrument scoring yields a range from 0 to 180 with higher scores representing better patient status. This questionnaire should be completed by patient prior to any procedures being performed at the visit, if possible. The form should then be checked by site staff for completeness.
Measured: 4-week post treatment and every 2 months follow up will be continued for 12 months
EQ-5D-5L quality of life score
To evaluate the health-related Quality of life, assessed by change in EuroQol 5 dimensions 5 level (EQ-5D-5L). It comprises the same 5 dimensions (mobility, self care, usual activities, pain/discomfort, anxiety/depression). Each dimension has 5 levels. However, this results have no arithmetic properties and should not be used as a cardinal score. This questionnaire should be completed by the patient prior to any procedure being performed at the visit, if possible. The form should then be checked by site staff for completeness.
Measured: 4-week post treatment and every 2 months follow up will be continued for 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Feb 2018
- Study start19 Jun 2018
- Primary completionDec 2024 (estimated)
- Study completionDec 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hepatocellular CarcinomaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 33 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Gongwin Biopharm Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Ja Der Liang, Principal investigator, National Taiwan University Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03447951
- Sponsor's protocol ID
- GW-020202
- Acronym
- OASES
- Status last verified
- Sep 2023
- First submitted
- 13 Feb 2018
- Last updated
- 13 Sep 2023
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Sep 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT03447951
- Acronym / sponsor ID
- OASES / GW-020202
- Conditions
- Hepatocellular Carcinoma
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 33 participants (estimated)
- Lead sponsor
- Gongwin Biopharm Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 19 Jun 2018 (actual)
- Primary completion
- Dec 2024 (estimated)
- Study completion
- Dec 2025 (estimated)
- First posted
- 27 Feb 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
BCLC stage B patients who are ineligible for operational or current locoregional therapy. Enrolled patients are assigned to 30% of total tumor volume. PTS100 is administrated via intratumoral injection. The required total dose is given in separate injection days, with maximal daily dose up to 10 mL at first IP administration, and is adjusted based on each patient's tolerability along the treatment. The treatment course lasts up to completion of the total dose, and the efficacy is evaluated 28 days afterwards.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| PTS100 (Para-Toluenesulfonamide): 30%TTV | Experimental | Drug: Para-ToluenesulfonamideTotal dose = 30% total tumor volume |
Who may qualify
- Ages
- 20 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (20 listed)
- 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:
- 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:
- Patients with at least one measurable lesion with size ≥ 1 cm.
- Patients with cumulative total treated tumor volume ≤ 366 cm3 and ≤ 4 target tumors.
Key exclusion criteria (13 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Para-ToluenesulfonamideAlso: PTS100A 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. | Drug | PTS100 (Para-Toluenesulfonamide): 30%TTV |
Primary outcome
Objective Response Rate (ORR)
Time frame: 4-week post treatment
To evaluate the efficacy, measured by objective response rate (ORR) of pooled treatment groups based on the modified RECIST (mRECIST) criteria at concluding visit.
10 secondary outcomes
Local disease control rate (LDCR)
Time frame: 4-week post treatment
Time to treated tumor progression (TTTTP)
Time frame: 4-week post treatment, then every 2 months follow up with imaging will be continued for 12 months
Three-year overall survival (OS)
Time frame: 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 frame: 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
Time frame: 4-week post treatment
Difference in LDCR between group 1 and group 2
Time frame: 4-week post treatment
Difference in TTTTP between group 1 and group 2
Time frame: 4-week post treatment to every 2 months follow up with imaging will be continued for 12 months
Incidence of Treatment-Emergent Adverse Events
Time frame: Enrollment to 4-week post treatment
FACT-Hep quality of life score
Time frame: 4-week post treatment and every 2 months follow up will be continued for 12 months
EQ-5D-5L quality of life score
Time frame: 4-week post treatment and every 2 months follow up will be continued for 12 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Sep 2023.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Ja Der Liang principal investigator
National Taiwan University Hospital
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Para-Toluenesulfonamide in people with Hepatocellular Carcinoma, run by Gongwin Biopharm Co., Ltd. at 3 sites in Taiwan.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 20 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 13 Sep 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in Taiwan. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Ben Liu (+886-2-2503-5282 ext. 304, ben.liu@gongwinbiopharm.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Objective Response Rate (ORR) (time frame: 4-week post treatment). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Sep 2023.