Study of PDS0101 and Pembrolizumab Combination I/O in Subjects With HPV16 + Recurrent and/or Metastatic HNSCC

NCT04260126 | Completed Phase 2 DMC FDA Drug

• 4 other identifiers: PDS0101-HNC-201KEYNOTE 643MK-3475-6432021-004046-38
• Study Type: interventional
• Target: 95
• Locations: 4 countries
• Sites: 23

Brief Summary

VERSATILE-002 is a Phase 2, open-label, multicenter study of the efficacy and safety of PDS0101 administered in combination with pembrolizumab in adults with HPV16 and PD-L1 positive recurrent or metastatic head and neck squamous cell carcinoma (HNSCC).

Conditions

Metastatic Head and Neck Cancer; Recurrent Head and Neck Cancer; HPV Positive Oropharyngeal Squamous Cell Carcinoma; Neoplasms, Head and Neck

Keywords

Head and Neck Squamous Cell Carcinoma; Human Papillomavirus; Immunotherapy; PDS0101; Vaccine; Human papillomavirus 16; Keynote 643; MK3475-643

Outcome Measures

Primary Outcomes (1)

• Best Overall Response (BOR) of confirmed complete response (CR) or confirmed partial response (PR) of the combination of pembrolizumab and PDS0101.

  RECIST 1.1

  24 Months

Secondary Outcomes (3)

• Progression-Free Survival (PFS) in all patients

  12 and 24 months

• Overall Survival in all patients

  24 months

• Incidence of Treatment-Emergent Adverse Events using NCI CTCAE, version 50 (v.11.27.17) [Safety and Tolerability] of Pembrolizumab and PDS0101 combination treatment

  24 months

Other Outcomes (2)

• Duration of response for all patients

  Up to 35 months

• Evaluate anti-HPV-16 E6 and E7 immune responses elicited by treatment with pembrolizumab and PDS0101

  Baseline (Cycle 1) to Day 253 (Cycle 13)

Study Arms (1)

Pembrolizumab and PDS0101

EXPERIMENTAL

Pembrolizumab will be administered via IV Infusion followed by subcutaneous injections of PDS0101 five times throughout the course of the study. Pembrolizumab monotherapy will be administered every cycle there is not a combination treatment until disease progression or up to Cycle 35.

Combination Product: Pembrolizumab (KEYTRUDA®) and PDS0101

Interventions

Pembrolizumab (KEYTRUDA®) and PDS0101 COMBINATION_PRODUCT

IV infusion of pembrolizumab 200 mg + two 0.5mL sub-cutaneous injections of PDS0101 administered on Cycle 1, 2, 3, 4 and 12. IV infusion of Pembrolizumab 200 mg monotherapy will be administered Cycles 5 - 11 and 13 - 35.

Pembrolizumab and PDS0101

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• The participant (or legally acceptable representative if applicable) provides written informed consent for the study.
• Be ≥18 years of age on the day of signing the informed consent.
• Checkpoint-naïve subjects: Have a history of histologically- confirmed diagnosis of squamous cell cancer of the head and neck (HNSCC) that is recurrent, metastatic, or persistent with:
• Confirmed HPV16 infection
• Confirmed tumor PDL1 expression defined as a combined positive score (CPS) ≥1 using the FDA-approved Dako PD-L1 immunohistochemistry (IHC) 22C3 PharmDx Assay.
• No prior receipt of any immunological therapy for metastatic disease.
• Checkpoint experienced subjects have a history of histologically-confirmed diagnosis of HNSCC that is recurrent, metastatic, or persistent with:
• Confirmed HPV16 infection
• Characterization of tumor PDL1 expression using the FDA-approved PD-L1 IHC 22C3 PharmDx Assay.
• Receipt of prior treatment with checkpoint inhibitors as a single agent or in combination, and have received at least 2 doses of the agent or a minimum of 6 weeks on treatment
• Have documented clinical progression or recurrence that has been radiologically confirmed
• Have recurrent and/or metastatic measurable disease based on RECIST 1.1 as assessed by the local PI/radiology. There must be confirmation that the subject's imaging shows at least 1 lesion that is appropriate for selection as a target lesion per RECIST 1.1. Lesions situated in a previously irradiated area are considered measurable if progression has been demonstrated in such lesions.
• Have adequate organ function as defined in Hematological: ANC ≥1500/μL, Platelets ≥100 000/μL; Hemoglobin ≥9.0 g/dL or ≥5.6 mmol/L; Renal: Creatinine ≤1.5 × ULN measured or calculated creatinine clearance \>30mL/min with creatinine levels or \>1.5 × institutional ULN; Hepatic: Total bilirubin ≤1.5 × ULN or direct bilirubin ≤ULN for subjects with total bilirubin levels \>1.5 × ULN, AST and ALT ≤2.5 ULN (5 × ULN for subjects with liver metastases); Coagulation: INR, PTT ≤1.5 × ULN. Specimens must be collected within 10 days prior to the start of study combination treatment.
• If subject received major surgery or radiation therapy of \>30 Gy, they must have recovered from the toxicity and/or complications from the intervention.
• For female subjects defined as women of childbearing potential (WOCBP), a negative urine pregnancy test must be obtained during screening. Women who are surgically sterile or at least 2 years postmenopausal do not require pregnancy testing.

You may not qualify if:

• Has received prior therapy with an anti-PD-1, anti-PD- L1, or anti-PD-L2 agent or with an agent directed to another stimulatory or co-inhibitory T-cell receptor (e.g. CTLA-4, OX40, CD137) and was discontinued from that treatment due to a Grade 3 or higher AE.
• Has received prior systemic anti-cancer therapy including investigational agents within 30 days prior to treatment.
• Note: Subjects must have recovered from all AEs due to previous therapies to ≤Grade 1 or baseline. Subjects with ≤Grade 2 neuropathy and ≤Grade 2 alopecia are an exception to this criterion and may qualify for therapy.
• Note: If participant received major surgery, they must have recovered adequately from the toxicity and/or complications from the intervention prior to starting treatment.
• Has received prior radiotherapy within 2 weeks of start of study treatment. Subjects must have recovered from all-radiation-related toxicities, not require corticosteroids and not have had radiation pneumonitis. A 1-week washout is permitted for palliative radiation (\<2 weeks of radiotherapy) to non- CNS disease.
• Coordination and timing of coronavirus disease 2019 (COVID-19) vaccination should be based on local investigator clinical assessment and judgment.
• Note: Whenever possible, it is recommended to avoid COVID vaccination on the day of PDS0101 and/or pembrolizumab dosing because it may be difficult to attribute certain AEs (eg, fever, infusion reaction) to the study drug(s) or the COVID vaccine if they are both administered on the same day.
• Has received a live vaccine within 30 days prior to the first dose of treatment. Examples of live vaccines include, but are not limited to, the following: measles, mumps, rubella, varicella/zoster (chicken pox), yellow fever, rabies, Bacillus Calmette-Guerin (BCG), and typhoid vaccine. Seasonal influenza vaccines for injection are generally killed virus vaccines and are allowed; however, intranasal influenza vaccines (e.g. FluMist®) are live attenuated vaccines and are not allowed.
• Received immunotherapy/immunomodulatory or immunosuppressive agents (e.g. IFNs, tumor necrosis factor, interleukins, immunoglobulins or other biological response modifiers \[GM-CSF, granulocyte colony-stimulating factor, macrophage colony- stimulating factor\]) within 6 weeks prior to administration of the first study combination treatment.
• Is currently participating in or has participated in a study of an investigational agent or has used an investigational device within 30 days prior to the first dose of study treatment.
• Note: Subjects who entered the follow-up phase of an investigational study may participate as long as it has been 30 days after the last dose of the previous investigational agent.
• Has undergone prior allogeneic hematopoietic stem cell transplantation within the last 5 years. (Subjects eligible as long as there are no symptoms of graft- versus-host disease (GVHD).
• Has a diagnosis of immunodeficiency or is receiving chronic systemic steroid therapy (in dosing exceeding 10 mg daily of prednisone equivalent) or any other form of immunosuppressive therapy within 7 days prior to the first dose of study drug. Current or recent use of physiologic doses of intra-articular, topical, or inhaled corticosteroids is acceptable.
• Has a known additional malignancy that is progressing or has required active treatment within the past 3 years. Note: Subjects with basal cell carcinoma of the skin, squamous cell carcinoma of the skin or carcinoma in situ (eg, breast carcinoma, cervical cancer in situ) or other malignant tumors that have undergone potentially curative therapy are not excluded.
• Has known active central nervous system (CNS) metastases and/or carcinomatosis meningitis. Subjects with previously treated brain metastases may participate provided they are radiologically stable, i.e. without evidence of progression for at least 4 weeks by repeat imaging (note that repeat imaging should be performed during study screening clinical stable and without requirement of steroid treatment for at least 14 days prior to first dose of study treatment).

Sponsors & Collaborators

• PDS Biotechnology Corp.: lead
• Merck Sharp & Dohme LLC: collaborator

Study Sites (23)

Marin Cancer Center

Greenbrae, California, 94904, United States

Location

University of California San Francisco

San Francisco, California, 94158, United States

Location

Mayo Clinic

Jacksonville, Florida, 32224, United States

Location

Cleveland Clinic Florida

Weston, Florida, 33331, United States

Location

University of Kentucky Chandler Medical Center - Markey Cancer Center

Lexington, Kentucky, 40536, United States

Location

University of Maryland Greenebaum Comprehensive Cancer Center

Baltimore, Maryland, 21201, United States

Location

University of Michigan

Ann Arbor, Michigan, 48109, United States

Location

Barbara Ann Karmanos Cancer Institute

Detroit, Michigan, 48201, United States

Location

Mayo Clinic

Rochester, Minnesota, 55902, United States

Location

Atlantic Health

Morristown, New Jersey, 07962, United States

Location

University of North Carolina

Chapel Hill, North Carolina, 27599, United States

Location

Ohio State University

Columbus, Ohio, 43210, United States

Location

Fox Chase Cancer Center

Philadelphia, Pennsylvania, 19111, United States

Location

Medical University of South Carolina Hollings Cancer Center

Charleston, South Carolina, 29425, United States

Location

University of Tennessee

Knoxville, Tennessee, 37920, United States

Location

Texas Oncology - Sammons Cancer Center

Dallas, Texas, 75246, United States

Location

The University of Virginia

Charlottesville, Virginia, 22908, United States

Location

West Virginia University

Morgantown, West Virginia, 26506, United States

Location

St. James Hospital

Dublin, D08NHY1, Ireland

Location

FDI Clinical Research

San Juan, Puerto Rico, 00927, Puerto Rico

Location

The Royal Marsden NHS Foundation Trust (Chelsea)

Chelsea, London, SW3 6JJ, United Kingdom

Location

The Royal Marsden NHS Foundation Trust (Sutton)

Sutton, Surrey, SM2 5PT, United Kingdom

Location

Edinburgh Cancer Centre

Edinburgh, UK EH4 2XU, United Kingdom

Location

Related Publications (1)

• Gandhapudi SK, Ward M, Bush JPC, Bedu-Addo F, Conn G, Woodward JG. Antigen Priming with Enantiospecific Cationic Lipid Nanoparticles Induces Potent Antitumor CTL Responses through Novel Induction of a Type I IFN Response. J Immunol. 2019 Jun 15;202(12):3524-3536. doi: 10.4049/jimmunol.1801634. Epub 2019 May 3.

  PMID: 31053626 BACKGROUND

MeSH Terms

Conditions

Head and Neck Neoplasms; Squamous Cell Carcinoma of Head and Neck

Interventions

pembrolizumab

Condition Hierarchy (Ancestors)

Neoplasms by Site; Neoplasms; Carcinoma, Squamous Cell; Carcinoma; Neoplasms, Glandular and Epithelial; Neoplasms by Histologic Type

Study Officials

• David T Schaaf, MD

  PDS Biotechnology Corporation

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 5, 2020
• First Posted: February 7, 2020
• Study Start: March 29, 2021
• Primary Completion: May 14, 2025
• Study Completion: May 14, 2025
• Last Updated: October 1, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

US (18); GB (3); IE (1); PR (1)