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Hepatocellular CarcinomaPhase 2NCT02089763

Efficacy Study of Pegylated Recombinant Human Arginase 1 as a Second-line Therapy in Patients With Advanced Liver Cancer

A Phase 2 study testing pegylated recombinant human arginase in people with Hepatocellular Carcinoma, run by Bio-Cancer Treatment International Limited at 1 site in Hong Kong.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

The purpose of the study is to evaluate the efficacy and safety of PEG-BCT-100 as the second-line therapy following sorafenib in advanced HCC patients. Another objective of the study is to explore whether the expression of OTC and ASS are predictive biomarkers for drug response and prognosis. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Hepatocellular Carcinoma
What is being tested?
pegylated recombinant human arginase
Could I receive a placebo?
No, everyone receives an active treatment
How many people
27 participants
Where
1 siteHong Kong
Study dates
Apr 2014to Mar 2017
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 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Clinical diagnosis of hepatocellular carcinoma (HCC) according to the European Association for the Study of the Liver (EASL) criteria
  • Patients with advanced HCC defined as unresectable disease which is not amenable OR refractory to local-regional therapy OR with extra-hepatic involvement
  • Patients have received prior systemic treatment with sorafenib for at least 14 days (not necessarily consecutive), and resulted in either disease progression or intolerance with sorafenib treatment
  • Sorafenib must be the last antineoplastic treatment before enrollment
  • Patients who are suitable for percutaneous tissue biopsy
  • ECOG Performance Status 0-2
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  • Adequate hematological, renal and hepatic function as assessed by the following blood tests sampled at screening visit
  • Life expectancy longer than 12 weeks
  • Subjects with at least one measurable lesion assessed by CT scan or other imaging within 4 weeks prior to the first dose of PEG-BCT-100
  • Normal ECG
  • Patients who give written informed consent prior to any study specific screening procedures with the understanding that the patient has the right to withdraw from the study at any time, without prejudice.

Who cannot join

  • Has received any surgery, loco-ablative, transarterial therapy or radiotherapy ≤ 2 weeks prior to the first dose of PEG-BCT-100
  • Has received systemic cancer therapy, e.g. chemotherapy, targeted biologic or enzymes, either approved or investigational, ≤ 2 weeks prior to the first dose of PEG-BCT-100.
  • Any toxic effects (except hair loss) of the prior therapy have not been resolved to Grade 2 or less according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events.
  • Prior malignancy except cervical carcinoma in situ or treated basal cell carcinoma. Any cancers treated curatively > 5 years prior to study entry are permitted.
  • Child-Pugh class of B or C
  • Patients with ascites uncontrolled by medication
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  • Known human immunodeficiency virus (HIV) or acquired immunodeficiency syndrome (AIDS) related illness.
  • Prior treatment with arginine depleting agent.
  • Female patients who are pregnant or lactating, or men and women of reproductive potential not willing or not able to employ an effective method of birth control/contraception to prevent pregnancy during treatment and for 6 months after discontinuing study treatment. The definition of effective contraception should be in agreement with local regulation and based on the judgment of the principal investigator or a designated associate.
  • A serious uncontrolled medical disorder or active infection that would impair their ability to receive study treatment.
  • Dementia or significantly altered mental status that would prohibit the understanding or rendering of informed consent and compliance with the requirements of this protocol.
  • Other severe acute or chronic medical or psychiatric condition, or laboratory abnormality that may increase the risk associated with study participation or study drug administration, or may interfere with the interpretation of study results, and in the judgment of the investigator would make the patient inappropriate for entry into this study.
  • Subjects, who in the opinion of the Investigator, are unable to comply with the trial treatment and the related trial procedures.

Trial locations and contacts

1 site in 1 country.

Hong Kong1 site

  • Prince of Wales Hospital, the Chinese University of Hong Kong

    Hong Kong

Other trials in Hong Kong

About this study

Official title: A Phase II Trial of PEG-BCT-100 as the Second-line Therapy Following Sorafenib in Patients With Advanced Hepatocellular Carcinoma

The purpose of the study is to evaluate the efficacy and safety of PEG-BCT-100 as the second-line therapy following sorafenib in advanced HCC patients. Another objective of the study is to explore whether the expression of OTC and ASS are predictive biomarkers for drug response and prognosis.

Read the detailed description

This is a phase II, single-arm clinical trial. Approximately 35 subjects will be enrolled in the study. All subjects will be treated with PEG-BCT-100 2.7 mg/kg weekly (day1, 8 and 15). Three week treatment of PEG-BCT-100 is considered as 1 cycle. All subjects will receive PEG-BCT-100 till progressive disease, intolerable toxicity or patients withdraw consent.

The clinical effects of PEG-BCT-100 on disease response will be evaluated every 6 weeks until disease progression. Disease response evaluation will be based on RECIST 1.1 criteria. The disease response based on modified RECIST criteria will also be evaluated and documented for reference only.

Safety parameters will be evaluated throughout the study. Adverse event (AE) will be graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events version 4.0 (NCI CTC AE v4).

All subjects will undergo a tumor tissue biopsy at baseline for evaluation of the biomarkers of ornithine transcarbamylase (OTC) and arginine succinate synthetase (ASS) except for those subjects whose tumor tissue blocks have been obtained within 1 year and are available for the biomarkers evaluation. The association between the levels of the 2 biomarkers and disease response to PEG-BCT-100 treatment will be explored in the study.

The effects on patients' quality of life will be evaluated every 2 cycles of the PEG-BCT-100 treatment.

Being tested
pegylated recombinant human arginase
Keywords
Hepatocellular carcinoma, HCC, Pegylated recombinant human arginase, PEG-BCT-100, ASS, OTC
Medical subject headings
Carcinoma, Hepatocellular

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • pegylated recombinant human arginaseBiological

    Also called PEG-BCT-100

Study groups in NCT02089763
Study groupRoleWhat they receive
PEG-BCT-100Experimentalpegylated recombinant human arginase 1
Receives: Biological: pegylated recombinant human arginase

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Time to progression (TTP)

    TTP is defined as the time from start of study treatment (Cycle 1 Day 1) to first documentation of objective tumor progression.

    TTP data will be censored on the day following the date of the last tumor assessment documenting absence of progressive disease under the following conditions:

    • Patients who die for causes other than progressive disease
    • Patients who do not have objective tumor progression and are still on study at the time of an analysis
    • Patients who are given antitumor treatment other than the study treatment
    • Patients who are removed from study follow-up prior to documentation of objective tumor progression.

    Patients lacking an evaluation of tumor response after their first dose will have their event time censored at Day 1.

    Measured: 3 years

Secondary outcomes (7)

  1. Progression-free Survival (PFS)

    PFS is defined as the time from start of study treatment (Cycle 1 Day 1) to first documentation of objective tumor progression or to death due to any cause.

    PFS data will be censored on the day following the date of the last tumor assessment documenting absence of progressive disease under the following conditions:

    • Patients who do not have objective tumor progression and are still on study at the time of an analysis
    • Patients who are given antitumor treatment other than the study treatment
    • Patients who are removed from study follow-up prior to documentation of objective tumor progression.

    Patients lacking an evaluation of tumor response after their first dose will have their event time censored at Day 1.

    Measured: 3 years

  2. Overall Survival (OS)

    OS is defined as the time from start of study treatment to date of death due to any cause. In the absence of confirmation of death, survival time will be censored at the last date the patient is known to be alive. Patients lacking data beyond the day of first dose will have their survival times censored at 1 day.

    Measured: 3 years

  3. Overall Response Rate (ORR)

    ORR is defined as the proportion of patients with confirmed complete response (CR) or confirmed partial response (PR), relative to the total evaluable patient population.

    Measured: 3 years

  4. Disease Control Rate (DCR)

    DCR is defined as the percent of patients with confirmed CR, PR, or stable disease for at least 12 weeks on study, relative to the total evaluable patient population.

    Measured: 3 years

  5. Duration of Response (DR)

    DR is defined as the time from the first documentation of objective tumor response to the first documentation of objective tumor progression or to death due to any cause.

    DR data will be censored on the day following the date of the last tumor assessment documenting absence of progressive disease under the following conditions:

    • Patients who do not have objective tumor progression and are still on study at the time of an analysis;
    • Patients who are given antitumor treatment other than the study treatment;
    • Patients who are removed from study follow-up prior to documentation of objective tumor progression.

    DR will only be calculated for the subgroup of patients with a tumor response (PR or CR).

    Measured: 3 years

  6. Quality of Life (QoL)

    The quality of life will be assessed at within 5 days prior to the start of trial treatment, every 2 cycles and at the End of Treatment. Subjects are required to complete the questionnaires: EORTC QLQ-C30 and EORTC QLQ-HCC18

    Measured: 3 years

  7. Safety profile of PEG-BCT-100 treatment

    Safety parameters will be evaluated throughout the study. Adverse event (AE) will be graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events version 4.0 (NCI CTC AE v4).

    Measured: 3 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted18 Mar 2014 (estimated)
  2. Study startApr 2014
  3. Primary completionMar 2017
  4. Study completionMar 2017

Resources

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
27 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02089763
Sponsor's protocol ID
BCT-100-006
Status last verified
Aug 2021
First submitted
14 Mar 2014
Last updated
9 Aug 2021
Results posted
No
Data monitoring committee
No

Publications

  • Chan SL, Cheng PNM, Liu AM, Chan LL, Li L, Chu CM, Chong CCN, Lau YM, Yeo W, Ng KKC, Yu SCH, Mok TSK, Chan AWH. A phase II clinical study on the efficacy and predictive biomarker of pegylated recombinant arginase on hepatocellular carcinoma. Invest New Drugs. 2021 Oct;39(5):1375-1382. doi: 10.1007/s10637-021-01111-8. Epub 2021 Apr 15.

    PubMed 33856599

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing pegylated recombinant human arginase in people with Hepatocellular Carcinoma, run by Bio-Cancer Treatment International Limited at 1 site in Hong Kong.

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 11 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: finished, as of the sponsor's last update on 9 Aug 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in Hong Kong. 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?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Time to progression (TTP) (time frame: 3 years). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02089763, which also lists 1 linked publication.

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 9 Aug 2021.

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