Tremelimumab With Chemoembolization or Ablation for Liver Cancer

NCT ID: NCT01853618

Last Updated: 2019-12-10

Study Results

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

61 participants

Study Classification

INTERVENTIONAL

Study Start Date

2013-05-02

Study Completion Date

2017-06-07

Brief Summary

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Background:

\- Tremelimumab is a cancer treatment drug that helps the immune system recognize and destroy cancer cells. Researchers want to see if it can be used to treat advanced liver cancer. The drug will be given with one of two types of treatment for liver cancer. The first type, transarterial catheter chemoembolization (TACE), injects chemotherapy drugs into the tumor through the main blood vessel that is feeding it. That blood vessel is then closed off to help keep the drugs in the tumor longer. The second type, radiofrequency ablation (RFA), uses a heated probe to destroy the tumor tissue. Researchers want to study how safe and effective these treatments are with the study drug.

Objectives:

\- To test the safety and effectiveness of Tremelimumab with TACE or RFA for advanced liver cancer.

Eligibility:

\- Individuals at least 18 years of age who have advanced liver cancer that has not responded to other treatments.

Detailed Description

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Background:

Worldwide, hepatocellular carcinoma (HCC) is the fifth most common malignancy with a median survival of 6-9 months. For patients with advanced disease sorafenib is the only approved drug and this has limited benefit.

Tremelimumab is a monoclonal antibody against cytotoxic T-lymphocyte-associated protein 4 (CTLA4). Anti-CTLA4 therapy has been shown to enhance anti-tumor immunity by blocking tumor-induced immune suppression of cytotoxic T cells.

Various tumor ablative procedures and techniques have been shown to result in immunogenic cell death and induction of a peripheral immune response. Both transarterial catheter chemoembolization (TACE) and radiofrequency ablation (RFA) have been shown to do this, as well as cryoablation and external beam radiation.

The underlying hypothesis of this study is that the effect of anti-CTLA4 treatment can be enhanced by TACE or RFA in patients with advanced hepatocellular carcinoma. We will also evaluate this in the context of cryoablation and radiation in hepatocellular carcinoma (HCC) and RFA in cholangiocarcinoma.

Objective:

To assess the safety and feasibility of combining Tremelimumab with trans-arterial catheter chemoembolization (TACE) radiofrequency ablation (RFA), or cryoablation in patients with advanced HCC.

Eligibility:

Histologically or cytologically confirmed diagnosis of HCC.

Childs-Pugh A/B7 cirrhosis only is allowed. If patient does not have cirrhosis, this limitation does not apply.

Barcelona Clinic Liver Cancer (BCLC) Stage B and C patients.

Patients must have disease that is not amenable to potentially curative resection, radiofrequency ablation, or liver transplantation.

Conditions

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Heptocellular Cancer Biliary Tract Neoplasms Liver Cancer Hepatocellular Carcinoma Biliary Cancer

Keywords

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Blocking of Immune Suppression Monoclonal Antibody Immunotherapy Ablative Therapy

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Pilot 1/Arm A1-Tremelimumab + RFA or TACE

Escalating doses of Tremelimumab + Radiofrequency Ablation (RFA) or Transarterial Catheter Chemoembolization (TACE)

Group Type EXPERIMENTAL

Tremelimumab

Intervention Type DRUG

3.5 mg/kg or 10 mg /kg intravenous (IV) every 4 weeks times 6 doses and then every 12 weeks for 2 years

RFA

Intervention Type PROCEDURE

Performed on Day 36

TACE

Intervention Type PROCEDURE

Performed on Day 36 and may be repeated (as per standard of care) on months 3, 7, and 13, and every (q)6 months thereafter (if indicated)

2/Arm A2 - Tremelimumab + RFA or TACE

Tremelimumab + Radiofrequency Ablation (RFA) or Transarterial Catheter Chemoembolization (TACE)

Group Type EXPERIMENTAL

Tremelimumab

Intervention Type DRUG

3.5 mg/kg or 10 mg /kg intravenous (IV) every 4 weeks times 6 doses and then every 12 weeks for 2 years

RFA

Intervention Type PROCEDURE

Performed on Day 36

TACE

Intervention Type PROCEDURE

Performed on Day 36 and may be repeated (as per standard of care) on months 3, 7, and 13, and every (q)6 months thereafter (if indicated)

3/Arm B - Tremelimumab + TACE

Tremelimumab + Transarterial Catheter Chemoembolization (TACE)

Group Type EXPERIMENTAL

Tremelimumab

Intervention Type DRUG

3.5 mg/kg or 10 mg /kg intravenous (IV) every 4 weeks times 6 doses and then every 12 weeks for 2 years

TACE

Intervention Type PROCEDURE

Performed on Day 36 and may be repeated (as per standard of care) on months 3, 7, and 13, and every (q)6 months thereafter (if indicated)

4/Arm C (never opened)

Tremelimumab + Radiofrequency Ablation (RFA) or Transarterial Catheter Chemoembolization (TACE)

Group Type EXPERIMENTAL

Tremelimumab

Intervention Type DRUG

3.5 mg/kg or 10 mg /kg intravenous (IV) every 4 weeks times 6 doses and then every 12 weeks for 2 years

TACE

Intervention Type PROCEDURE

Performed on Day 36 and may be repeated (as per standard of care) on months 3, 7, and 13, and every (q)6 months thereafter (if indicated)

Cryoablation

Intervention Type PROCEDURE

Performed on Day 36

5/Arm D - Tremelimumab + Cryoablation

Tremelimumab + Cryoablation

Group Type EXPERIMENTAL

Tremelimumab

Intervention Type DRUG

3.5 mg/kg or 10 mg /kg intravenous (IV) every 4 weeks times 6 doses and then every 12 weeks for 2 years

Cryoablation

Intervention Type PROCEDURE

Performed on Day 36

6/Arm E - Tremelimumab + RFA

Tremelimumab + Radiofrequency Ablation (RFA)

Group Type EXPERIMENTAL

Tremelimumab

Intervention Type DRUG

3.5 mg/kg or 10 mg /kg intravenous (IV) every 4 weeks times 6 doses and then every 12 weeks for 2 years

RFA

Intervention Type PROCEDURE

Performed on Day 36

Interventions

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Tremelimumab

3.5 mg/kg or 10 mg /kg intravenous (IV) every 4 weeks times 6 doses and then every 12 weeks for 2 years

Intervention Type DRUG

RFA

Performed on Day 36

Intervention Type PROCEDURE

TACE

Performed on Day 36 and may be repeated (as per standard of care) on months 3, 7, and 13, and every (q)6 months thereafter (if indicated)

Intervention Type PROCEDURE

Cryoablation

Performed on Day 36

Intervention Type PROCEDURE

Other Intervention Names

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Ticilimumab Radiofrequency Ablation Transarterial Catheter Chemoembolization

Eligibility Criteria

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Inclusion Criteria

1. Patients must have histopathological confirmation of hepatocellular carcinoma (HCC) or (Cohort E only) biliary tract carcinoma (BTC) by the Laboratory of Pathology of the National Cancer Institute (NCI) prior to entering this study OR histopathological confirmation of carcinoma in the setting of clinical and radiological characteristics which, together with the pathology, are highly suggestive of a diagnosis of HCC (or biliary tract carcinoma in Cohort E). Fibrolammelar variant is also allowed. For cohort E, the term BTC includes intraor extrahepatic cholangiocarcinoma, gallbladder cancer or ampullary cancer, as long as there is an intrahepatic component amenable to radiofrequency ablation (RFA).
2. Patients must have disease that is not amenable to potentially curative resection, transplantation or ablation. For Cohorts A, C and D patients must have progressed on, been intolerant to, or refused prior sorafenib therapy. Cohort E patients must have received at least one line of chemotherapy for BTC.
3. Disease must be technically amenable to transhepatic arterial chemoembolization (TACE), radiofrequency ablation (RFA) or cryoablation. Each case will be discussed at gastrointestinal (GI) tumor board with interventional radiology. Patients must have evaluable disease.
4. If liver cirrhosis is present, patient must have a Child-Pugh A/B7 classification.
5. Age greater than or equal to 18 years
6. Life expectancy of greater than 3 months.
7. Eastern Cooperative Oncology Group (ECOG) performance status 0-2.
8. Patients must have normal organ and marrow function as defined below:

* leukocytes greater than or equal to 3,000/mcL
* absolute neutrophil count greater than or equal to 1,000/mcL
* platelets greater than or equal to 60,000/mcL
* total bilirubin, If cirrhosis present: Part of Child Pugh requirement. If no cirrhosis: Bili should be less than or equal to 2 times upper limit of normal (ULN)
* Serum albumin, If cirrhosis present: Part of Child Pugh requirement. If no cirrhosis: albumin should be greater than or equal to 2.5g/dl
* Patients are eligible with alanine aminotransferase (ALT) or aspartate aminotransferase (AST) up to 5 times ULN.
* creatinine, less than 1.5 times institution upper limit of normal OR
* creatinine clearance greater than or equal to 45 mL/min/1.73 m(2), as calculated below, for patients with creatinine levels above institutional normal
9. Patients must have recovered from any acute toxicity related to prior therapy, including surgery. Toxicity should be less than or equal to grade 1 or returned to baseline.
10. Patients must not have other invasive malignancies within the past 5 years (with the exception of non-melanoma skin cancers, non-invasive bladder cancer or localized prostate cancer for whom systemic therapy is not required).
11. Patient must be able to understand and willing to sign a written informed consent document.

Exclusion Criteria

1. Patients who have had standard of care chemotherapy, large field radiotherapy, or major surgery must wait 2 weeks prior to entering the study. For recent experimental therapies a 28 day period of time must elapse before treatment.
2. Patients who have undergone prior liver transplantation are ineligible.
3. Patients with known brain metastases will be excluded from this clinical trial because of their poor prognosis and because they often develop progressive neurologic dysfunction that would confound the evaluation of neurologic and other adverse events.
4. Uncontrolled intercurrent illness including, but not limited to, ongoing or active systemic infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia (excluding insignificant sinus bradycardia and sinus tachycardia) or psychiatric illness/social situations that would limit compliance with study requirements.
5. History of chronic autoimmune disease (e.g., Addison's disease, multiple sclerosis, Graves disease, Hashimoto's thyroiditis, rheumatoid arthritis, hypophysitis, etc.) with symptomatic disease within the 3 years before randomization. Note: Active vitiligo or a history of vitiligo will not be a basis for exclusion.
6. Dementia or significantly altered mental status that would prohibit the understanding or rendering of Information and Consent and compliance with the requirements of the protocol.
7. Diverticulitis (either active or history of) within the past 2 years. Note that diverticulosis is permitted.
8. Active or history of inflammatory bowel disease (colitis, Crohn's), irritable bowel disease, celiac disease, or other serious, chronic, gastrointestinal conditions associated with diarrhea. Active or history of systemic lupus erythematosus or Wegener's granulomatosis.
9. Currently receiving immunosuppressive doses of steroids or other immunosuppressive medications (inhaled and topical steroids are permitted)
10. History of sarcoidosis syndrome
11. Patients should not be vaccinated with live attenuated vaccines within 1 month of starting Tremelimumab treatment.

12 Has a known history of Human Immunodeficiency Virus (HIV) (HIV 1/2 antibodies)

13\. HIV-positive patients receiving anti-retroviral therapy are excluded from this study due to the possibility of pharmacokinetic interactions between antiretroviral medications and Tremelimumab. HIV positive patients not receiving antiretroviral therapy are excluded due to the possibility that Tremelimumab may worsen their condition and the likelihood that the underlying condition may obscure the attribution of adverse events.

14\. History of hypersensitivity reaction to human or mouse antibody products.

15\. Pregnancy and breast feeding are exclusion factors. The effects of Tremelimumab on the developing human fetus are unknown. Enrolled patients must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry, the duration of study participation and 3 months after the end of the treatment. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately.

16\. Patients with unhealed surgical wounds for more than 30 days.
Minimum Eligible Age

18 Years

Maximum Eligible Age

99 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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National Cancer Institute (NCI)

NIH

Sponsor Role lead

Responsible Party

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Tim Greten, M.D.

Principal Investigator

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Tim F Greten, M.D.

Role: PRINCIPAL_INVESTIGATOR

National Cancer Institute (NCI)

Locations

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National Institutes of Health Clinical Center, 9000 Rockville Pike

Bethesda, Maryland, United States

Site Status

Countries

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United States

References

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Parkin DM, Bray F, Ferlay J, Pisani P. Estimating the world cancer burden: Globocan 2000. Int J Cancer. 2001 Oct 15;94(2):153-6. doi: 10.1002/ijc.1440. No abstract available.

Reference Type BACKGROUND
PMID: 11668491 (View on PubMed)

Llovet JM, Di Bisceglie AM, Bruix J, Kramer BS, Lencioni R, Zhu AX, Sherman M, Schwartz M, Lotze M, Talwalkar J, Gores GJ; Panel of Experts in HCC-Design Clinical Trials. Design and endpoints of clinical trials in hepatocellular carcinoma. J Natl Cancer Inst. 2008 May 21;100(10):698-711. doi: 10.1093/jnci/djn134. Epub 2008 May 13.

Reference Type BACKGROUND
PMID: 18477802 (View on PubMed)

Kantoff PW, Higano CS, Shore ND, Berger ER, Small EJ, Penson DF, Redfern CH, Ferrari AC, Dreicer R, Sims RB, Xu Y, Frohlich MW, Schellhammer PF; IMPACT Study Investigators. Sipuleucel-T immunotherapy for castration-resistant prostate cancer. N Engl J Med. 2010 Jul 29;363(5):411-22. doi: 10.1056/NEJMoa1001294.

Reference Type BACKGROUND
PMID: 20818862 (View on PubMed)

Xie C, Duffy AG, Mabry-Hrones D, Wood B, Levy E, Krishnasamy V, Khan J, Wei JS, Agdashian D, Tyagi M, Gangalapudi V, Fioravanti S, Walker M, Anderson V, Venzon D, Figg WD, Sandhu M, Kleiner DE, Morelli MP, Floudas CS, Brar G, Steinberg SM, Korangy F, Greten TF. Tremelimumab in Combination With Microwave Ablation in Patients With Refractory Biliary Tract Cancer. Hepatology. 2019 May;69(5):2048-2060. doi: 10.1002/hep.30482. Epub 2019 Mar 10.

Reference Type RESULT
PMID: 30578687 (View on PubMed)

Agdashian D, ElGindi M, Xie C, Sandhu M, Pratt D, Kleiner DE, Figg WD, Rytlewski JA, Sanders C, Yusko EC, Wood B, Venzon D, Brar G, Duffy AG, Greten TF, Korangy F. The effect of anti-CTLA4 treatment on peripheral and intra-tumoral T cells in patients with hepatocellular carcinoma. Cancer Immunol Immunother. 2019 Apr;68(4):599-608. doi: 10.1007/s00262-019-02299-8. Epub 2019 Jan 28.

Reference Type RESULT
PMID: 30688989 (View on PubMed)

Duffy AG, Ulahannan SV, Makorova-Rusher O, Rahma O, Wedemeyer H, Pratt D, Davis JL, Hughes MS, Heller T, ElGindi M, Uppala A, Korangy F, Kleiner DE, Figg WD, Venzon D, Steinberg SM, Venkatesan AM, Krishnasamy V, Abi-Jaoudeh N, Levy E, Wood BJ, Greten TF. Tremelimumab in combination with ablation in patients with advanced hepatocellular carcinoma. J Hepatol. 2017 Mar;66(3):545-551. doi: 10.1016/j.jhep.2016.10.029. Epub 2016 Nov 2.

Reference Type RESULT
PMID: 27816492 (View on PubMed)

Greten TF, Sangro B. Targets for immunotherapy of liver cancer. J Hepatol. 2017 Sep 18:S0168-8278(17)32287-0. doi: 10.1016/j.jhep.2017.09.007. Online ahead of print.

Reference Type RESULT
PMID: 28923358 (View on PubMed)

Provided Documents

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Document Type: Study Protocol and Statistical Analysis Plan

View Document

Document Type: Informed Consent Form

View Document

Related Links

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Other Identifiers

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13-C-0120

Identifier Type: -

Identifier Source: secondary_id

130120

Identifier Type: -

Identifier Source: org_study_id