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Treatment of Solid Tumors With Intratumoral Hiltonol® (Poly-ICLC) (Hiltonol)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01984892
Recruitment Status : Terminated (PI discretion, low enrollment)
First Posted : November 15, 2013
Results First Posted : January 23, 2018
Last Update Posted : January 23, 2018
Oncovir, Inc.
Information provided by (Responsible Party):
Nina Bhardwaj, Icahn School of Medicine at Mount Sinai

Brief Summary:

The purpose of this study is to test the safety of a course of injections containing Poly-ICLC in patients with advanced solid tumors that can be easily and safely reached with a needle.

Poly-ICLC is a compound that has been used to help the body in its fight against cancer.

Condition or disease Intervention/treatment Phase
Melanoma Squamous Cell Carcinoma of the Head and Neck Squamous Cell Carcinoma of the Skin Sarcoma of the Skin Basal Cell Cancer of the Skin Breast Cancer Drug: Poly-ICLC Phase 2

Detailed Description:
We hypothesize that this therapeutic in-situ autovaccination strategy is comprised of three immunomodulatory steps. The first is the innate immune local tumor killing induced by intratumoral Hiltonol (via NK, TNF, etc). A very close second step is optimal Th1-weighted priming through the in-situ combination of the poly-ICLC danger signal with the tumor antigens released in step 1 and further processed and cross-presented by poly-ICLC activated mDC, etc. The repeated administration of the Hiltonol danger signal IT in the context of the patient's own tumor antigens and in a way that mimics a natural viral infection may be critical to this step. Once the system is optimally primed, the third step is targeting and maintenance of the immune response and its facilitation at remote tumor sites with IM poly-ICLC through chemokine release, inflammasome activation and other costimulatory factors.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 8 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Treatment of Solid Tumors With Intratumoral Hiltonol® (Poly-ICLC): A Phase II Clinical Study
Study Start Date : November 2013
Actual Primary Completion Date : August 2014
Actual Study Completion Date : August 2014

Resource links provided by the National Library of Medicine

MedlinePlus Genetics related topics: Melanoma

Arm Intervention/treatment
Experimental: IT and IM injections Poly-ICLC
Enrolled patients will receive two cycles of Poly-ICLC treatment. Each priming (intratumoral injections - IT) and boosting (intramuscular injections - IM) treatment course will constitute one cycle.
Drug: Poly-ICLC

Cycle 1-Weeks 1 and 2: 1mg Poly-ICLC intratumoral (IT) injections (t=6) into same lesion over 2 weeks.

Weeks 3-9: 1mg Poly-ICLC 2x/week intramuscularly (IM) into thighs or upper arms.

Week 10: No treatment. CT scan of chest, abdomen, pelvis and extremities or neck; possible MRI brain scan.

Cycle 2-Weeks 11 and 12: 1mg Poly-ICLC IT injections (t=6) into same lesion over 2 weeks.

Weeks 13-19 - 1mg Poly-ICLC 2x/weekly IM in thighs or upper arms.

Weeks 20-26: no treatment. Week 26, evaluate response in absence of inflammation.

Maintenance - Weeks 27-36: For patients with stable disease or response; IM poly-ICLC injections; evaluation of clinical and immune response. Week 38 repeat tumor assessment, optional biopsy

Follow Up via phone every 3 months for 30months, after completion of treatments.

Other Name: Hiltonol®

Primary Outcome Measures :
  1. Progression-free Survival [ Time Frame: average 52 weeks ]

    Progression-free survival defined as the time in weeks from study entry until tumor progression defined using the Wolchok criteria or death. Patients who are alive and free from progression on the date of closing follow-up will be censored on that date.

    In order to minimize the potential for misdiagnosis of pseudoprogression, related to early inflammation, tumor measurement for determination of progression will be made at the earliest at 26 weeks.

Secondary Outcome Measures :
  1. Therapeutic Effect in Treated Patients [ Time Frame: 24 months ]
    Induction of innate and/or an adaptive, specific anti-tumor T cell immune response in the injected tumor lesion and also systemically.

Other Outcome Measures:
  1. Overall Survival in Treated Patients [ Time Frame: up to 30 months ]
    Patients who are alive on the date of closing follow-up, or 30 months after completing all study treatments, will be censored on that date

Information from the National Library of Medicine

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Ages Eligible for Study:   14 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Histologically confirmed diagnosis of melanoma, squamous head and neck cancer, sarcoma, squamous cell carcinoma of the skin, basal cell skin cancer, or breast cancer
  • Sarcoma Patients must be @ least14 yrs of age; all others 18 yrs of age or older.
  • Un-resectable disease. Patients with resectable disease may be enrolled after having refused surgery and documented consultation with a surgeon.
  • Disease progressed through @ least 1 systemic therapy or through local irradiation within the preceding 6 mos.
  • Radiologically or visually measurable recurrent or metastatic disease and @ least 10mm in longest dimension.
  • At least 1 accessible primary or metastatic tumor site that can be readily injected IT with poly-ICLC with or without ultrasound guidance. Lesion can be superficial cutaneous, subcutaneous or within a readily accessible lymph node & must measure @ least 10mm in longest dimension.
  • Tumor site injection cannot have been irradiated within 8 wks of C1D1
  • ECOG performance status ≤ 2.
  • Normal hematologic, renal & liver function. INR<2 if off of anticoagulation. Patients on anticoagulation therapy with an INR>2 may be enrolled at the discretion of the investigator.
  • Patients able to provide informed consent.
  • Must agree to follow acceptable birth control methods and continue for @ least 2 mos. after last poly-ICLC dose. Women of childbearing potential must have a (-) pregnancy test.

Exclusion Criteria:

  • Serious concurrent infection or medical illness.
  • Bulky intracranial metastatic disease with shift of midline structures or progressive brain metastasis. Administration of immunotherapy or conventional chemotherapy treatments for metastatic cancer within 4 wks of C1D1
  • Radiation treatments within 4 wks of C1D1
  • AIDS defined as a CD4 count < then 200 in the context of HIV sero-positivity or chronically is taking immunosuppressive medication such as steroids or transplant related medications.
  • Life expectancy of < than 6 mos.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01984892

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United States, New York
Icahn School of Medicine at Mount Sinai
New York, New York, United States, 10029
Sponsors and Collaborators
Nina Bhardwaj
Oncovir, Inc.
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Principal Investigator: Nina Bhardwaj, MD, PhD Icahn School of Medicine at Mount Sinai

Publications automatically indexed to this study by Identifier (NCT Number):
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Responsible Party: Nina Bhardwaj, Director, Immunotherapy Program, Icahn School of Medicine at Mount Sinai Identifier: NCT01984892    
Other Study ID Numbers: GCO 13-1687
BB-43984 ( Other Grant/Funding Number: Mount Sinai School of Medicine - Departmental Funds )
First Posted: November 15, 2013    Key Record Dates
Results First Posted: January 23, 2018
Last Update Posted: January 23, 2018
Last Verified: December 2017
Keywords provided by Nina Bhardwaj, Icahn School of Medicine at Mount Sinai:
Autologous Vaccination
Intratumoral Injections
Intramuscular Injections
Advanced Accessible Solid Tumors
Phase II Clinical Trial
In Situ
Host Targeted Strategy
Overall Survival
Additional relevant MeSH terms:
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Carcinoma, Squamous Cell
Squamous Cell Carcinoma of Head and Neck
Neoplasms, Basal Cell
Carcinoma, Basal Cell
Skin Neoplasms
Neoplasms, Glandular and Epithelial
Neoplasms by Histologic Type
Neoplasms, Squamous Cell
Head and Neck Neoplasms
Neoplasms by Site
Skin Diseases
Interferon Inducers
Immunologic Factors
Physiological Effects of Drugs