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Malignant Pleural MesotheliomaPhase 1NCT04525859

Poly-ICLC (Hiltonol®) Vaccine In Malignant Pleural Mesothelioma

A Phase 1 study testing Safety and Expansion Cohort in people with Malignant Pleural Mesothelioma, run by Oncovir, Inc. at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

This study will examine the safety and potential effectiveness of poly-ICLC directly injected into malignant pleural mesothelioma at the time of biopsy up to 21 days prior to the cancer being removed by the surgeon Read more about this study

Who is it for?
Any age
Conditions the study is for
Malignant Pleural Mesothelioma
What is being tested?
Safety and Expansion Cohort
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
19 participants
Where
1 siteUnited States
Study dates
19 Aug 2020to 18 Jun 2025
Phase
Phase 1
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages Any age
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • Biopsy proven MPM
  • If biopsied at an outside institution, must have a tissue block sample available
  • Deemed to be surgically resectable by a dedicated thoracic surgeon.
  • Acceptable hematologic, renal and liver function as follows:
  • Absolute neutrophil count > 1000/mm3
  • Platelets > 50,000/mm3,
Show all 12Show fewer
  • Creatinine ≤ 2.5 mg/dl,
  • Total bilirubin ≤ 1.5 mg/dl, unless patient has known Gilberts syndrome
  • Transaminases ≤ 2 times above the upper limits of the institutional normal.
  • INR<1.6 if off of anticoagulation. Patients on anticoagulation therapy with an INR>1.6 may be enrolled at the discretion of the investigator if they have not had any episodes of severe hemorrhage and if the site to be injected is fully surrounded by pleura where achieving homeostasis would be complicated.
  • Patient must be able to provide informed consent
  • Subject is willing to adhere to the study visit schedule and other protocol requirements.

Who cannot join

  • Serious concurrent infection or medical illness, which would jeopardize the ability of the patient to receive Poly-ICLC with reasonable safety.
  • History of any pulmonary process that precludes a biopsy to be done safely.
  • Known severe pulmonary hypertension; having a history of pulmonary hypertension or an estimated PA systolic pressure of >60mmHg as measured by tricuspid regurgitation on preoperative echocardiogram.
  • Subject unable to cooperate in terms of maintaining position during the biopsy procedure.
  • AIDS defined as a CD4 count less than 200 in the context of HIV seropositivity or chronically is taking immunosuppressive medication such as steroids or transplant related medications.
  • Persistent toxicity from recent therapy that has not sufficiently resolved in the judgment of the study physician.
Show all 13Show fewer
  • Subject has an active infection requiring therapy.
  • Subject has had an allogeneic tissue/solid organ transplant.
  • Subject has active autoimmune disease that has required systemic treatment within the past 2 years (eg, with use of disease modifying agents, corticosteroids or immunosuppressive drugs). Replacement therapy (eg, thyroxine, insulin, or physiologic corticosteroid replacement therapy for adrenal or pituitary insufficiency, etc.) is not considered a form of systemic treatment.
  • Subject has known active Hepatitis B, Hepatitis C or tuberculosis. Active Hepatitis B is defined as a known positive HBsAg result. Active Hepatitis C is defined by a known positive Hep C Ab result and known quantitative HCV ribonucleic acid (RNA) results greater than the lower limits of detection of the assay.
  • Concomitant comorbidities that are uncontrolled that would preclude the patient from being a surgical candidate including uncontrolled CHF, diabetes or heart disease
  • Women with a positive serum or urine pregnancy test at baseline, or are pregnant or breastfeeding.
  • -

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Icahn School of Medicine Mount Sinai

    New York, New York, 10029

Other trials in United States

About this study

Official title: Direct Injection of Poly-ICLC (Hiltonol®) Vaccine In Malignant Pleural Mesothelioma

This study will examine the safety and potential effectiveness of poly-ICLC directly injected into malignant pleural mesothelioma at the time of biopsy up to 21 days prior to the cancer being removed by the surgeon

Read the detailed description
  • To evaluate the safety and toxicity of IT Poly-ICLC, Hiltonol® prior to surgical resection for patients with MPM.
  • To determine objective response rate by RECIST 1.1 using CT imaging.
  • To determine recurrence free survival of subjects treated with IT Poly-ICLC followed by surgical resection defined as the time of injection until the first date that recurrent disease is confirmed or date of documented death.
  • To evaluate IT Poly-ICLC induced immune changes in the tumor microenvironment by comparing pre-injection biopsy to surgically resected tissue for immune cell infiltration and T cell receptor (TCR) diversity.
  • To characterize additional immune parameters in IT Poly-ICLC injected tumors including in-depth phenotypic and functional characterization of immune infiltrating cells.
  • To evaluate IT Poly-ICLC induced serological changes and changes of circulating immune cells, including regulatory T cells and NK cells, by comparing pre-injection to post-surgical resection blood samples.
Being tested
Safety, Expansion Cohort
Keywords
Malignant Pleural Mesothelioma (MPMV, Vaccine, Poly-ICLC
Medical subject headings
Mesothelioma, Malignant

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • SafetyBiological

    See previous Safety group description

  • Expansion CohortBiological

    See previous Expansion Cohort

Study groups in NCT04525859
Study groupRoleWhat they receive
SafetyExperimentalSix patients will be enrolled in the Phase 1 safety cohort. Patients will have an IR guided biopsy and FNA. Up to four core biopsies and FNAs at one site will be performed prior to intratumoral (IT) administration of Poly-ICLC. Pleural fluid will be collected for research analysis if available. Poly-ICLC will be injected in 2 locations within the pleura. Patients will undergo surgery 21±7 days after the biopsy and Poly-ICLC intratumoral (IT) injection. The type of surgery that will be performed is at the discretion of the thoracic surgeon and per the standard of care. This includes pleurectomy/decortication or extrapleural pneumonectomy. Patients will be evaluated per the standard of care post-operatively. On day 7±4 days a final toxicity assessment, physical exam and research blood will be collected. All post-operative care and monitoring thereafter is as per standard of care.
Receives: Biological: Safety
Expansion CohortExperimentalIf at most one (1) patient in the Phase 1 safety cohort experiences a DLT then a total of thirteen (13) additional patients will be enrolled into the Phase 1b Expansion Cohort. Patients in the Expansion Cohort will receive the same dose and schedule of Poly-ICLC as in the Phase 1 safety cohort. Patients will be followed for safety and tolerability, as well as efficacy. If a total of 4 or more patients experience DLTs then the study will be closed due to excessive toxicity.
Receives: Biological: Expansion Cohort

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. The probability of rejecting the investigational treatment is at least 81%, if the DLT rate is greater than 33% and the probability of accepting the treatment is at least 71% if the DLT rate is less than a safe level of 17%.

    Safety will be assessed by the frequency and severity of toxicities by use of NCI-CTCAE 5.0 criteria.

    Measured: up to 27 days

Secondary outcomes (1)

  1. Objective response rate by RECIST 1.1 using CT imaging.

    Local Recurrence-free survival from time of first injection until first date that locally recurrent disease is confirmed or date of documented death.

    Measured: up to 27days

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted25 Aug 2020
  2. Study start19 Aug 2020
  3. Primary completion18 Jun 2025
  4. Study completion18 Jun 2025

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Parallel groups
Model description
Study Subjects will receive poly-ICLC once within the pleural mesothelioma. Up to twenty one days later he/she will undergo surgery per the standard of care by the thoracic surgeon. The type of surgery will be decided upon by the treating thoracic surgeon. Follows up visits are scheduled every three months for the first year and then every 6 months thereafter. CT scans or surveillance scans to look for recurrences will be ordered by the treating surgeon and/or medical oncologist per the standard of care. If there is a need for adjuvant (after surgery) therapy, such as chemotherapy or radiation, this will be discussed with the study subject per the standard of care by the surgeon and/or a medical oncologist or radiation oncologist.
Masking
None (open label)
Enrolment
19 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04525859
Sponsor's protocol ID
GCO#19-2701
Status last verified
Jun 2025
First submitted
19 Aug 2020
Last updated
24 Jun 2025
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: Rebecca Hernandez is the Administrative Assistant for this study and should have access to the PRS for this study. Contact Information: email: rebecca.hernandez@mssm.edu Phone: 212-824-9472.

Publications

  • Sterman DH, Alley E, Stevenson JP, Friedberg J, Metzger S, Recio A, Moon EK, Haas AR, Vachani A, Katz SI, Sun J, Heitjan DF, Hwang WT, Litzky L, Yearley JH, Tan KS, Papasavvas E, Kennedy P, Montaner LJ, Cengel KA, Simone CB 2nd, Culligan M, Langer CJ, Albelda SM. Pilot and Feasibility Trial Evaluating Immuno-Gene Therapy of Malignant Mesothelioma Using Intrapleural Delivery of Adenovirus-IFNalpha Combined with Chemotherapy. Clin Cancer Res. 2016 Aug 1;22(15):3791-800. doi: 10.1158/1078-0432.CCR-15-2133. Epub 2016 Mar 11.

    PubMed 26968202

  • Hawkins MJ, Levin M, Borden EC. An Eastern Cooperative Oncology Group phase I-II pilot study of polyriboinosinic-polyribocytidylic acid poly-L-lysine complex in patients with metastatic malignant melanoma. J Biol Response Mod. 1985 Dec;4(6):664-8.

    PubMed 2418164

  • Longhi MP, Trumpfheller C, Idoyaga J, Caskey M, Matos I, Kluger C, Salazar AM, Colonna M, Steinman RM. Dendritic cells require a systemic type I interferon response to mature and induce CD4+ Th1 immunity with poly IC as adjuvant. J Exp Med. 2009 Jul 6;206(7):1589-602. doi: 10.1084/jem.20090247. Epub 2009 Jun 29.

    PubMed 19564349

  • Ammi R, De Waele J, Willemen Y, Van Brussel I, Schrijvers DM, Lion E, Smits EL. Poly(I:C) as cancer vaccine adjuvant: knocking on the door of medical breakthroughs. Pharmacol Ther. 2015 Feb;146:120-31. doi: 10.1016/j.pharmthera.2014.09.010. Epub 2014 Oct 2.

    PubMed 25281915

  • Uematsu S, Akira S. Toll-like receptors and Type I interferons. J Biol Chem. 2007 May 25;282(21):15319-23. doi: 10.1074/jbc.R700009200. Epub 2007 Mar 29.

    PubMed 17395581

  • Caskey M, Lefebvre F, Filali-Mouhim A, Cameron MJ, Goulet JP, Haddad EK, Breton G, Trumpfheller C, Pollak S, Shimeliovich I, Duque-Alarcon A, Pan L, Nelkenbaum A, Salazar AM, Schlesinger SJ, Steinman RM, Sekaly RP. Synthetic double-stranded RNA induces innate immune responses similar to a live viral vaccine in humans. J Exp Med. 2011 Nov 21;208(12):2357-66. doi: 10.1084/jem.20111171. Epub 2011 Nov 7.

    PubMed 22065672

  • Geiss G, Jin G, Guo J, Bumgarner R, Katze MG, Sen GC. A comprehensive view of regulation of gene expression by double-stranded RNA-mediated cell signaling. J Biol Chem. 2001 Aug 10;276(32):30178-82. doi: 10.1074/jbc.c100137200.

    PubMed 11487589

  • Huang CC, Duffy KE, San Mateo LR, Amegadzie BY, Sarisky RT, Mbow ML. A pathway analysis of poly(I:C)-induced global gene expression change in human peripheral blood mononuclear cells. Physiol Genomics. 2006 Jul 12;26(2):125-33. doi: 10.1152/physiolgenomics.00002.2006. Epub 2006 Mar 22.

    PubMed 16554548

  • Stahl-Hennig C, Eisenblatter M, Jasny E, Rzehak T, Tenner-Racz K, Trumpfheller C, Salazar AM, Uberla K, Nieto K, Kleinschmidt J, Schulte R, Gissmann L, Muller M, Sacher A, Racz P, Steinman RM, Uguccioni M, Ignatius R. Synthetic double-stranded RNAs are adjuvants for the induction of T helper 1 and humoral immune responses to human papillomavirus in rhesus macaques. PLoS Pathog. 2009 Apr;5(4):e1000373. doi: 10.1371/journal.ppat.1000373. Epub 2009 Apr 10.

    PubMed 19360120

  • Flynn BJ, Kastenmuller K, Wille-Reece U, Tomaras GD, Alam M, Lindsay RW, Salazar AM, Perdiguero B, Gomez CE, Wagner R, Esteban M, Park CG, Trumpfheller C, Keler T, Pantaleo G, Steinman RM, Seder R. Immunization with HIV Gag targeted to dendritic cells followed by recombinant New York vaccinia virus induces robust T-cell immunity in nonhuman primates. Proc Natl Acad Sci U S A. 2011 Apr 26;108(17):7131-6. doi: 10.1073/pnas.1103869108. Epub 2011 Apr 5.

    PubMed 21467219

  • Zhu X, Nishimura F, Sasaki K, Fujita M, Dusak JE, Eguchi J, Fellows-Mayle W, Storkus WJ, Walker PR, Salazar AM, Okada H. Toll like receptor-3 ligand poly-ICLC promotes the efficacy of peripheral vaccinations with tumor antigen-derived peptide epitopes in murine CNS tumor models. J Transl Med. 2007 Feb 12;5:10. doi: 10.1186/1479-5876-5-10.

    PubMed 17295916

  • Zhu X, Fallert-Junecko BA, Fujita M, Ueda R, Kohanbash G, Kastenhuber ER, McDonald HA, Liu Y, Kalinski P, Reinhart TA, Salazar AM, Okada H. Poly-ICLC promotes the infiltration of effector T cells into intracranial gliomas via induction of CXCL10 in IFN-alpha and IFN-gamma dependent manners. Cancer Immunol Immunother. 2010 Sep;59(9):1401-9. doi: 10.1007/s00262-010-0876-3. Epub 2010 Jun 12.

    PubMed 20549206

  • Salazar AM, Levy HB, Ondra S, Kende M, Scherokman B, Brown D, Mena H, Martin N, Schwab K, Donovan D, Dougherty D, Pulliam M, Ippolito M, Graves M, Brown H, Ommaya A. Long-term treatment of malignant gliomas with intramuscularly administered polyinosinic-polycytidylic acid stabilized with polylysine and carboxymethylcellulose: an open pilot study. Neurosurgery. 1996 Jun;38(6):1096-103; discussion 1103-4.

    PubMed 8727138

  • Butowski N, Chang SM, Junck L, DeAngelis LM, Abrey L, Fink K, Cloughesy T, Lamborn KR, Salazar AM, Prados MD. A phase II clinical trial of poly-ICLC with radiation for adult patients with newly diagnosed supratentorial glioblastoma: a North American Brain Tumor Consortium (NABTC01-05). J Neurooncol. 2009 Jan;91(2):175-82. doi: 10.1007/s11060-008-9693-3. Epub 2008 Sep 17.

    PubMed 18797818

  • Rosenfeld MR, Chamberlain MC, Grossman SA, Peereboom DM, Lesser GJ, Batchelor TT, Desideri S, Salazar AM, Ye X. A multi-institution phase II study of poly-ICLC and radiotherapy with concurrent and adjuvant temozolomide in adults with newly diagnosed glioblastoma. Neuro Oncol. 2010 Oct;12(10):1071-7. doi: 10.1093/neuonc/noq071. Epub 2010 Jul 8.

    PubMed 20615924

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Safety and Expansion Cohort in people with Malignant Pleural Mesothelioma, run by Oncovir, Inc. at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 12 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 24 Jun 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

The probability of rejecting the investigational treatment is at least 81%, if the DLT rate is greater than 33% and the probability of accepting the treatment is at least 71% if the DLT rate is less than a safe level of 17%. (time frame: up to 27 days). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04525859, which also lists 29 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Jun 2025.

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