"Heptovax" - A Phase II, Open-Label Trial Evaluating the Safety and Efficacy of GV1001 in Advanced Hepatocellular Carcinoma.
- Hepatocellular carcinoma diagnosis fulfilling one of the following criteria (as per
the American Association for the Study of Liver Diseases [AASLD] guidelines, see
1. Nodule in a cirrhotic or non-cirrhotic liver with a biopsy showing HCC;
2. Nodule in cirrhotic liver where no biopsy is performed:
- Nodules between 1-2 cm in a cirrhotic liver with a typical coincidal
vascular pattern of HCC (i.e. hypervascular with washout in the
portal/venous phase) in two dynamic studies: either CT scan, contrast
ultrasound or MRI with contrast.
- Nodule larger than 2 cm in a cirrhotic liver with a typical vascular
pattern of HCC on a dynamic imaging technique.
Please note: HCC in a non-cirrhotic liver can only be diagnosed with a biopsy showing HCC.
- Measurable disease according to modified RECIST (see Appendix 7).
- At least one treatment-naïve target lesion (treatment-naïve being defined as not
having been treated with local therapy, such as surgery, radiation therapy, hepatic
arterial embolisation, chemoembolisation, radio-frequency ablation or cryo-ablation).
- Barcelona Clinic Liver Cancer (BCLC) stage A, B or C (see Appendix 6) (Stage D is
- Child-Pugh stage A (see Appendix 8).
- Male or female aged 18 years or older.
- Adequate haematological parameters, as demonstrated by:
- Haemoglobin greater than or equal to 9.0 g/dL (SI units: 5.6 mmol/L);
- WBC greater than or equal to 3.0 x 109/L;
- Platelets greater than or equal to 75 x 109/L.
- ALT and AST ≤ 5 times the upper limit of normal.
- Bilirubin < 2 mg/dL.
- Serum creatinine smaller than or equal to 1.5 mg/dL (SI units: 132 µmol/L).
- Performance status ECOG 0 or 1.
- Minimum life expectancy of 3 months at screening.
- Written informed consent given prior to any study specific procedures.
- HCC amenable to curative treatment or transplantation.
- History of other malignancies in the last 5 years (10 years in the case of breast
cancer), except for adequately treated non-melanoma skin cancers (Basal Cell
Carcinoma, Squamous Cell Carcinoma) and carcinoma in situ of the cervix.
- Known history of or co-existing autoimmune disease.
- Known Central Nervous System (CNS) metastases.
- Known history of human immunodeficiency virus (HIV).
- Any medical condition that, in the opinion of the Investigator, may compromise the
compliance of the patient to receive study treatment and follow study procedures.
- Treatment with any other IMP within 4 weeks prior to cyclophosphamide administration
at Day -3.
- Known sensitivity to any components of cyclophosphamide, GV1001 or GM-CSF.
- Concomitant treatment with the following within 4 weeks of pre-treatment with
- Anti-tumour treatment (including radiotherapy, chemotherapy, immunotherapy,
endocrine therapy, cytokines, interferons, protease inhibitors, and gene
therapy) and vaccines.
- Chronic corticosteroids (inhaled and topical steroids are permitted including
low dose steroids at non-immunosuppressive doses e.g. 15 mg prednisolone daily
for up to 7 days).
- Herbal medicine either containing hypericum perforacum (e.g., St Johns Wort) or
claiming to have anti-tumour effects (e.g., Iscador).
- Pregnancy or lactation.
- Women of childbearing potential not using reliable and adequate contraceptive
methods, defined as the use of oral, implanted, injectable, mechanical or barrier
products for the prevention of pregnancy; or women who are practising abstinence; or
where the partner is sterile, for example a vasectomy.
- Unable for any other reason to comply with the protocol (treatment or assessments).