Recombinant Human Arginase I (rhArgI) for Patients With Advanced Hepatocellular Carcinoma (HCC): An Adaptive Design Dose Escalation Trial With Addition of Standard Doxorubicin Treatment
The primary objectives of this study are:
- To establish a safe dose level for weekly intravenous administration of PEG- BCT-100
(rhArgIpeg5000) based on clinical and laboratory safety assessments following a range
of doses (from 500 to >5000 U/kg).
- To define the optimal biological dose (OBD) of PEG-BCT-100 based on the
pharmacodynamics (PD) of arginine depletion (ADD) relative to plasma PK of PEG-
- To evaluate any objective tumor responses to PEG-BCT-100 in HCC patients receiving
weekly doses of PEG-BCT-100 alone and in combination with standard doses of doxorubicin
Secondary objectives of this study are:
- To define any toxicities associated with the metabolic and cellular alterations of ADD
relative to dose and PK of PEG-BCT-100 (rhArgIpeg5000).
- To develop a safe and biologically active dose and schedule for PEG-BCT-100 treatment
in phase 2 trials, either as monotherapy or in combination with best standard of care
- To confirm the safety and initial anti-tumor activity of the preferred dose and regimen
of PEG-BCT-100 in 18 additional patients with advanced HCC
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Plasma arginase and arginine levels,as well as tumour response, i.e. an effect on growth in the milieu of arginine depletion.
Ronnie TP Poon, Prof
Division of Hepatobiliary and Pancreatic Surgery, Department of Surgery, The University of Hong Kong
Hong Kong: Department of Health