A Phase II, Open-Label Trial Evaluating GV1001 in Advanced Hepatocellular Carcinoma.

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT00444782
• Date of registration: 07/03/2007
• Prospective Registration: No
• Primary sponsor: Pharmexa A/S
• Public title: A Phase II, Open-Label Trial Evaluating GV1001 in Advanced Hepatocellular Carcinoma.
• Scientific title: "Heptovax" - A Phase II, Open-Label Trial Evaluating the Safety and Efficacy of GV1001 in Advanced Hepatocellular Carcinoma.
• Date of first enrolment: November 2006
• Target sample size: 41
• Recruitment status: Completed
• URL: http://clinicaltrials.gov/show/NCT00444782
• Study type: Interventional
• Study design: Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: Phase 2

Countries of recruitment

France, Germany, Spain

Contacts

• Name: Lotte Rosendahl, Pharmacist (CTM)
• Affiliation: Pharmexa A/S

Key inclusion & exclusion criteria

Inclusion Criteria:

- Hepatocellular carcinoma diagnosis fulfilling one of the following criteria (as per
the American Association for the Study of Liver Diseases [AASLD] guidelines, see
Appendix 5):

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
excluded).

- 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.

Exclusion Criteria:

- 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
cyclophosphamide:

- 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).



Age minimum: 18 Years
Age maximum: N/A
Gender: Both

Health Condition(s) or Problem(s) studied

Carcinoma, Hepatocellular

Intervention(s)

Biological: GV1001

Primary Outcome(s)

Response rate (Partial and Complete Response) according to modified RECIST. [Time Frame: No]

Secondary Outcome(s)

Time to Symptomatic Progression (TTSP) [Time Frame: No]

Progression Free Survival (PFS) [Time Frame: No]

Immune Response [Time Frame: No]

Time to Progression (TTP) [Time Frame: No]

Secondary ID(s)

PX115.1.1-201

Source(s) of Monetary Support

Please refer to primary and secondary sponsors