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Phase I Safety Study of Dendritic Cell Vaccine to Treat Patients With Hepatocellular Carcinoma

Phase 1
Completed
Conditions
Hepatocellular Carcinoma
Interventions
Biological: COMBIG-DC (ilixadencel)
Registration Number
NCT01974661
Lead Sponsor
Mendus
Brief Summary

The primary objective of this study is to answer the question "Is it possible to inject the COMBIG-DC vaccine in a hepatic tumor without getting unacceptable side effects"?

Detailed Description

Patients diagnosed with hepatocellular carcinoma will get COMBIG-DC vaccinations at three occasions with 2-3 weeks and 3-5 weeks between vaccination 2 and 3 respectively. Adverse events will be registered until 6 months after last vaccination, as well as changes in vital signs (heart rate, blood pressure and body temperature) and lab parameters. Immunologic response will be evaluated by measuring immunologic markers in blood. The size of the tumor/tumors will be evaluated after 3 and 6 months and thereafter every three months until tumor progression.

For patients included after approval of Amendment 3 (2015-12-10), COMBIG-DC will be given as add on to standard treatment; sorafenib or Transarterial Chemoembolization (TACE).

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
18
Inclusion Criteria
  • Be informed of the nature of the study and have provided written informed consent

  • At least 18 years of age.

  • Diagnosis of hepatocellular carcinoma according to EASL criteria or pathology.

  • Radiologically measurable liver tumor(s), i.e. at least 20 mm in longest uni-dimensional diameter as measured by CT/MRI

  • Not eligible for curatively aiming treatment or TACE. Tumor stage B or C according to BCLC.

  • For patients included according to Amendment 3: tumour stage A, B or C according to BCLC and

    1. eligible for sorafenib treatment or having ongoing sorafenib treatment for not more than 4 weeks ant the time for inclusion or
    2. eligible for TACE or having received not more than 1 previous TACE treatment.
Exclusion Criteria
  • Performance status > ECOG 2

  • Liver function according to Child-Pugh >7 points.

  • Known major reaction/adverse event in connection with previously made vaccination (e.g. asthma, anaphylaxia or other serious reaction).

  • Known major reaction/adverse event in connection with previous transfusions of blood products

  • Active autoimmune disease requiring treatment with systemic immunosuppressive agents, e.g. inflammatory bowel disease, multiple sclerosis, sarcoidosis, psoriasis, autoimmune hemolytic anemia, rheumatoid arthritis, SLE, vasculitis, Sjögren's syndrome, scleroderma, autoimmune hepatitis, and other rheumatological diseases.

  • Tested positive for HIV

  • Active disease (HBV and HCV) requiring antiviral treatment

  • Ongoing infection that requires treatment with antibiotics or antiviral medication

  • Treatment with immunosuppressive treatments like corticosteroids (Immunosuppression (within 28 days) prior to the first injection of COMBIG-DC. Inhaled, intranasal and local steroids accepted), or mTor inhibitors within 28 days before first vaccination.

  • Patients with prior history of malignancy other than HCC, within the preceding 3 years OR with relaps after complete response, except for 5 years follow-up of adequately treated in situ carcinoma without recurrences or non-melanoma skin cancer.

  • Inadequate laboratory parameters, i.e.:

    1. P-Prothrombincomplex (PK) >1.4,
    2. Platelet count <50 75 x109/L
    3. Leukocyte count <3.0 x 109/L
    4. P-APT time outside normal limit
  • Previous organ transplantation

  • Women of Childbearing Potential (WOCBP) refusing to use adequate contraception (oral or injectable contraceptives, hormone releasing intrauterine device) throughout the study period.

  • Pregnant or lactating women

  • Life expectancy less than 3 months.

  • Concomitant anti-tumor treatment (within 28 days) prior to the first injection of COMBIG-DC, except for sorafenib or TACE for patients included according to Amendment 3.

  • For patients included according to Amendment 3: Previous systemic anti-cancer treatment.

  • Investigational treatment (within 28 days) prior to the first injection of COMBIG-DC.

  • Known blood dyscrasia (bleeding complication).

  • Known malignancy in CNS

  • Any reason that, in the opinion of the investigator, contraindicates that the patient participates in the study.

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
COMBIG-DCCOMBIG-DC (ilixadencel)COMBIG-DC (allogeneic dendritic cells) Cancer Vaccine 3 vaccinations: 5, 10 or 20 million cells per injection
Primary Outcome Measures
NameTimeMethod
Registration of adverse events as a measure of safety and tolerabilityUp to 6 months after last patient's last vaccination

* Changes in vital signs from baseline (heart rate, blood pressure, body temperature)

* Changes in lab parameters from baseline

* Short term worsening in ECOG and/or Child Pugh and/or MELD score

* Local procedural injuries, assessed by MRI or ultrasound

Secondary Outcome Measures
NameTimeMethod
Change in body weight3 and 6 months after last vaccination
To evaluate systemic immunological responseUp to 3 months after last vaccination

* Vaccine cell tracking; PBMCs will be stained with antibodies specific for one HLA class I or one HLA-class II antigen that is selectively expressed on donor vaccine cells.

* vaccine-induced alloimmunization; screening of alloantibodies against HLA-A, B, C (MHC-class I) and HLA-DR, DQ, DP (MHC-class II) antigens

* autoimmune events; screening of autoantibodies against autoantigens, including nuclear antigens (ANA, SSA, SSB, Sm, RNP, Scl-70, Centromeres and Jo-1) and liver parenchyma-associated autoantigens (liver-kidney microsomal antigens and mitochondrial antigens)

* complement activation; classical/alternative complement function, C3, C4, C3d, and Factor-B.

* immune cell occurrence and activation state; CD3+ , CD3+4+ and CD3+8+ T cells, CD19+ B-cells CD3-16+56+ NK-cells, CD3-16+56+69+ NK cells, CD3+16+56+ NKT-cells, CD3+16+56+69+ NKT-cells and CD3+HLA-DR+ T cells.

To evaluate systemic inflammatory responseUntil 3 months after last vaccination

Potential systemic release of relevant cytokines, chemokines and other inflammatory parameters in blood;IL-1R, IL-2,IL-4, IL-5, IL-6, IL-7, IL-8, IL-10, IL-12p70, IL-13, IL-17A, G-CSF. GM-CSF, IFN-gamma, MCP-1, MIP-1 beta and TNF-alpha.

Long term changes in ECOG scores3 and 6 months after last vaccination
To study overall survival (OS)Up to 6 months after last patient's last vaccination

OS measured as survival time from first dose of COMBIG-DC until end of study or death (whichever comes first)

To evaluate tumor controlUntil 6 months after last patient's last vaccination

* CT/MRI evaluation 3 and 6 months after first vaccination. Patients with stable disease or tumor response will continue tumor evaluation every 3rd month until progress or until last study patient has had his/her 6 month visit.

* Measuring number of tumor specific T cells with flow cytometry after in vitro stimulation with different pools of HCC-associated tumor peptides (Alpha-feto protein (AFP), and hTERT)

* Measuring AFP (alpha-feto protein) levels in blood

* Measuring the level of circulating tumor cell, identified as tested positive for MICA, EpCAM, CD133, CD34, CK18

Long term changes in Quality of Life scores3 and 6 months after last vaccination
To evaluate immunological responseUp to 3 months after last vaccination

* complement activation; classical/alternative complement function, C3, C4, C3d, and Factor-B.

* immune cell occurrence and activation state; CD3+ , CD3+4+ and CD3+8+ T cells, CD19+ B-cells CD3-16+56+ NK-cells, CD3-16+56+69+ NK cells, CD3+16+56+ NKT-cells, CD3+16+56+69+ NKT-cells and CD3+HLA-DR+ T cells.

Changes in HBV, HCV virus titersDay 8 after each injection and at the 3 and 6 months visit

Changes in HBV, HCV virus titers vs baseline, for patients that are tested positive at screening

To study time to progress (TTP)Measured every 3 months until progression

TTP measured as time from first dose of COMBIG-DC until radiologically proven progress according to mRECIST.

Trial Locations

Locations (1)

Dept. of Transplantation and Liver Surgery, Sahlgrenska University Hospital

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Gothenburg, Sweden

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