A Trial To Find Out If Vidutolimod Together With Cemiplimab Is Safe And If It Works In Adult Participants With Advanced Cancer Or Metastatic Cancer

A Multicenter, Open-label, Phase 2 Study of Intratumoral Vidutolimod (CMP-001) in Combination With Intravenous Cemiplimab in Subjects With Selected Types of Advanced or Metastatic Cancer

The goal of this study is to learn if giving cemiplimab and vidutolimod together could be effective in treating advanced cancer. The main questions it aims to answer are:

• How many participants' cancers respond to vidutolimod together with cemiplimab?
• Is vidutolimod together with cemiplimab safe and well-tolerated?
• How well does vidutolimod together with cemiplimab treat participants' cancer?

Participants will receive trial treatment for up to 2 years. 30 days after stopping treatment, participants will have a follow-up visit. After that visit, the trial staff will continue to follow up with participants about every 3 months, until the trial ends.

Study Overview

• Status: Terminated
• Conditions: Non-Small Cell Lung Cancer; Triple Negative Breast Cancer; Merkel Cell Carcinoma; Basal Cell Carcinoma; Oropharynx Squamous Cell Carcinoma; Cutaneous Squamous Cell Carcinoma
• Intervention / Treatment: Drug: vidutolimod; Drug: cemiplimab

Detailed Description

Former Sponsor Checkmate Pharmaceuticals Note: Early termination planned on 31Oct2024 due to study drug supply

• Study Type: Interventional
• Enrollment (Actual): 77
• Phase: Phase 2

Contacts and Locations

Study Locations

• Australia
  • New South Wales
    • Darlinghurst, New South Wales, Australia, 2010
      • St. Vincent's Hospital
  • Queensland
    • Woolloongabba, Queensland, Australia, 4102
      • Princess Alexandra Hospital
  • Victoria
    • Melbourne, Victoria, Australia, 3050
      • Peter MacCallum Cancer Centre (PMCC) and The Royal Melbourne Hospital
  • Western Australia
    • Murdoch, Western Australia, Australia, 6150
      • Fiona Stanley Hospital
• United States
  • Alabama
    • Birmingham, Alabama, United States, 35294
      • University Of Alabama
  • California
    • Duarte, California, United States, 91010
      • City of Hope
    • La Jolla, California, United States, 92037-0845
      • UC San Diego Moores Cancer Center
    • Los Angeles, California, United States, 90095
      • University of California
  • Florida
    • Jacksonville, Florida, United States, 32204
      • GenesisCare USA
    • Orlando, Florida, United States, 32806
      • Orlando Health Cancer Institute
  • Iowa
    • Iowa City, Iowa, United States, 52242
      • University of Iowa
  • Kentucky
    • Louisville, Kentucky, United States, 40202
      • Norton Cancer Institute
  • Maryland
    • Baltimore, Maryland, United States, 20814
      • VA Maryland Health Care System
  • Massachusetts
    • Boston, Massachusetts, United States, 02215
      • Dana-Farber Cancer Institute
  • Michigan
    • Detroit, Michigan, United States, 48201
      • Karmanos Cancer Institute
  • New Hampshire
    • Lebanon, New Hampshire, United States, 03756
      • Dartmouth Clinical Trial Office
  • New York
    • New York, New York, United States, 10065
      • Memorial Sloan Kettering Cancer Center
  • North Carolina
    • Greenville, North Carolina, United States, 27858
      • East Carolina University
  • Ohio
    • Columbus, Ohio, United States, 43210
      • Ohio State University
  • Oklahoma
    • Oklahoma City, Oklahoma, United States, 73104
      • OU Health Stephenson Cancer Center
  • Pennsylvania
    • Pittsburgh, Pennsylvania, United States, 15232
      • University of Pittsburgh Medical Center
  • Texas
    • Houston, Texas, United States, 77030
      • Oncology Consultants
  • Utah
    • Salt Lake City, Utah, United States, 84112
      • University of Utah Huntsman Cancer Center
  • Washington
    • Seattle, Washington, United States, 98109
      • University of Washington

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Key Inclusion Criteria:

Participants enrolled in the study must meet all of the following inclusion criteria to be eligible.

1. Histopathologically-confirmed diagnosis of cancer, as defined by the protocol.
2. Measurable disease, as defined by RECIST v1.1 and as defined in the protocol. Note: CSCC, MCC and BCC subjects without radiographically measurable disease are not excluded if there is at least 1 lesion ≥ 10 mm in at east 1 dimension documented by color photography.
3. Adequate organ function based on most recent laboratory values within 3 weeks before first dose of study treatment on Week 1 Day 1 (W1D1), as defined in the protocol.
4. Eastern Cooperative Oncology Group (ECOG) Performance Status of 0 to 1 at Screening.

Key Exclusion Criteria:

Participants presenting with any of the following will not qualify for entry into the study:

1. Received radiation therapy (or other non-systemic therapy) within 2 weeks before first dose of study treatment on W1D1. Participants should have recovered (i.e. Grade ≤ 1 or at baseline) from radiation-related toxicities.
2. Had major surgeries (including complete oncologic resection) within last 4 weeks prior to enrollment, and/or have not recovered adequately from the toxicities and/or complications from the intervention. Minor surgeries (including routine resections of early stage CSCCs and BCCs that may be due to field cancerization) require a 7-day washout.
3. Received systemic pharmacologic doses of corticosteroids > 10 mg/day prednisone within 15 days before first dose of study treatment on W1D1, as defined in the protocol.
4. History of immune-mediated AE leading to permanent discontinuation due to prior PD-1-blocking antibody.
5. Not fully recovered from AEs due to prior treatment (to Grade 1 or less, per Common Terminology Criteria for Adverse Events (CTCAE), with the exception of persistent vitiligo, alopecia, hypothyroidism, diabetes mellitus, and adrenal and/or pituitary insufficiency.
6. Active pneumonitis or history of noninfectious pneumonitis that required steroids.
7. Severe uncontrolled medical disease within 12 months of screening, including but not limited to poorly controlled hypertension, unstable angina, myocardial infarction, congestive heart failure (New York Heart Association Class II or greater), pericarditis, cerebrovascular accident, or implanted or continuous use of a pacemaker or defibrillator, or emphysema with FEV1 ≤ 50% predicted.
8. Known history of immunodeficiency.
9. Known additional malignancy that is progressing or required active treatment within the past 3 years, as defined in the protocol.
10. Active autoimmune disease that required systemic treatment in past 2 years; replacement therapy is not considered a form of systemic treatment.
11. Untreated, symptomatic, or enlarging central nervous system metastases or carcinomatous meningitis (including leptomeningeal metastases from solid tumors).

NOTE: Other protocol defined Inclusion/Exclusion Criteria apply

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

9

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Vidutolimod and cemiplimab for cutaneous squamous cell carcinoma (CSCC) (A1); Participants who have not received prior systemic therapy for metastatic or locally and/or regionally advanced unresectable CSCC and who are not eligible for curative radiation will receive vidutolimod intratumoral(ly) (IT) and cemiplimab intravenous (IV) according to the treatment schedule until a reason for treatment discontinuation is reached.	Drug: vidutolimod; Participants will receive vidutolimod for up to 2 years as follows: 10 mg IT weekly for 7 doses after which vidutolimod will be administered every 3 weeks (Q3W). The first dose of vidutolimod may be administered subcutaneously (SC) or IT at the discretion of Investigator. All subsequent doses will be IT. The initial 7 doses of vidutolimod, delivered on a weekly dosing schedule, must be completed before starting the Q3W vidutolimod dosing schedule.; Other Names: CMP-001; Drug: cemiplimab; Participants will receive cemiplimab for up to 2 years as follows: 350 mg IV infusion over 30 minutes at week 1 dose 1 (W1D1) and Q3W thereafter.; Other Names: REGN2810; Libtayo
Experimental: Vidutolimod and cemiplimab for CSCC (A2); Participants who have progressed while receiving a programmed cell death protein 1 (PD-1)-blocking antibody or within 12 weeks of discontinuation of PD-1 blocking antibody for metastatic or locally and/or regionally advanced unresectable CSCC, will receive vidutolimod IT and cemiplimab IV according to the treatment schedule until a reason for treatment discontinuation is reached.	Drug: vidutolimod; Participants will receive vidutolimod for up to 2 years as follows: 10 mg IT weekly for 7 doses after which vidutolimod will be administered every 3 weeks (Q3W). The first dose of vidutolimod may be administered subcutaneously (SC) or IT at the discretion of Investigator. All subsequent doses will be IT. The initial 7 doses of vidutolimod, delivered on a weekly dosing schedule, must be completed before starting the Q3W vidutolimod dosing schedule.; Other Names: CMP-001; Drug: cemiplimab; Participants will receive cemiplimab for up to 2 years as follows: 350 mg IV infusion over 30 minutes at week 1 dose 1 (W1D1) and Q3W thereafter.; Other Names: REGN2810; Libtayo
Experimental: Vidutolimod and cemiplimab for Merkel cell carcinoma (MCC) (B1); Participants who have not received prior systemic therapy for metastatic or locally and/or regionally advanced unresectable MCC will receive vidutolimod IT and cemiplimab IV according to the treatment schedule until a reason for treatment discontinuation is reached.	Drug: vidutolimod; Participants will receive vidutolimod for up to 2 years as follows: 10 mg IT weekly for 7 doses after which vidutolimod will be administered every 3 weeks (Q3W). The first dose of vidutolimod may be administered subcutaneously (SC) or IT at the discretion of Investigator. All subsequent doses will be IT. The initial 7 doses of vidutolimod, delivered on a weekly dosing schedule, must be completed before starting the Q3W vidutolimod dosing schedule.; Other Names: CMP-001; Drug: cemiplimab; Participants will receive cemiplimab for up to 2 years as follows: 350 mg IV infusion over 30 minutes at week 1 dose 1 (W1D1) and Q3W thereafter.; Other Names: REGN2810; Libtayo
Experimental: Vidutolimod and cemiplimab for MCC (B2); Participants who have progressed while receiving a PD-1-blocking antibody or within 12 weeks of discontinuation of the PD-1 blocking antibody, will receive vidutolimod IT and cemiplimab IV according to the treatment schedule until a reason for treatment discontinuation is reached.	Drug: vidutolimod; Participants will receive vidutolimod for up to 2 years as follows: 10 mg IT weekly for 7 doses after which vidutolimod will be administered every 3 weeks (Q3W). The first dose of vidutolimod may be administered subcutaneously (SC) or IT at the discretion of Investigator. All subsequent doses will be IT. The initial 7 doses of vidutolimod, delivered on a weekly dosing schedule, must be completed before starting the Q3W vidutolimod dosing schedule.; Other Names: CMP-001; Drug: cemiplimab; Participants will receive cemiplimab for up to 2 years as follows: 350 mg IV infusion over 30 minutes at week 1 dose 1 (W1D1) and Q3W thereafter.; Other Names: REGN2810; Libtayo
Experimental: Vidutolimod and cemiplimab for triple negative breast cancer (TNBC) (C1); Participants who have not received prior therapy with immune checkpoint inhibitors (iCPIs) and who must have previously received treatment with sacituzumab govitecan (all advanced or metastatic TNBC participants), with trastuzumab deruxtecan [human epidermal growth factor receptor 2 (HER2)-low participants] and with adenosine diphosphate ribose polymerase (PARP) inhibitor [for BReast CAncer gene (BRCA)] for TNBC will receive vidutolimod IT and cemiplimab IV according to the treatment schedule until a reason for treatment discontinuation is reached.	Drug: vidutolimod; Participants will receive vidutolimod for up to 2 years as follows: 10 mg IT weekly for 7 doses after which vidutolimod will be administered every 3 weeks (Q3W). The first dose of vidutolimod may be administered subcutaneously (SC) or IT at the discretion of Investigator. All subsequent doses will be IT. The initial 7 doses of vidutolimod, delivered on a weekly dosing schedule, must be completed before starting the Q3W vidutolimod dosing schedule.; Other Names: CMP-001; Drug: cemiplimab; Participants will receive cemiplimab for up to 2 years as follows: 350 mg IV infusion over 30 minutes at week 1 dose 1 (W1D1) and Q3W thereafter.; Other Names: REGN2810; Libtayo
Experimental: Vidutolimod and cemiplimab for TNBC (C2); Participants who have previously received treatment with sacituzumab govitecan (all advanced or metastatic TNBC participants), with trastuzumab deruxtecan (HER2-low participants) and with PARP inhibitor (for BRCA) and who have progressed while receiving a PD-1-blocking antibody or within 12 weeks of discontinuation of a PD-1 blocking antibody for advanced or metastatic TNBC, will receive vidutolimod IT and cemiplimab IV according to the treatment schedule until a reason for treatment discontinuation is reached.	Drug: vidutolimod; Participants will receive vidutolimod for up to 2 years as follows: 10 mg IT weekly for 7 doses after which vidutolimod will be administered every 3 weeks (Q3W). The first dose of vidutolimod may be administered subcutaneously (SC) or IT at the discretion of Investigator. All subsequent doses will be IT. The initial 7 doses of vidutolimod, delivered on a weekly dosing schedule, must be completed before starting the Q3W vidutolimod dosing schedule.; Other Names: CMP-001; Drug: cemiplimab; Participants will receive cemiplimab for up to 2 years as follows: 350 mg IV infusion over 30 minutes at week 1 dose 1 (W1D1) and Q3W thereafter.; Other Names: REGN2810; Libtayo
Experimental: Vidutolimod and cemiplimab for basal cell carcinoma (BCC) (D); Participants who have not received prior hedgehog pathway inhibitor therapy, or prior anti-PD-1/programmed cell death ligand 1 (PD-L1) therapy and who do not wish to receive or who are not candidates for a hedgehog inhibitor, for metastatic or locally and/or regionally advanced unresectable BCC and will receive vidutolimod IT and cemiplimab IV according to the treatment schedule until a reason for treatment discontinuation is reached.	Drug: vidutolimod; Participants will receive vidutolimod for up to 2 years as follows: 10 mg IT weekly for 7 doses after which vidutolimod will be administered every 3 weeks (Q3W). The first dose of vidutolimod may be administered subcutaneously (SC) or IT at the discretion of Investigator. All subsequent doses will be IT. The initial 7 doses of vidutolimod, delivered on a weekly dosing schedule, must be completed before starting the Q3W vidutolimod dosing schedule.; Other Names: CMP-001; Drug: cemiplimab; Participants will receive cemiplimab for up to 2 years as follows: 350 mg IV infusion over 30 minutes at week 1 dose 1 (W1D1) and Q3W thereafter.; Other Names: REGN2810; Libtayo
Experimental: Vidutolimod and cemiplimab for non-small cell lung cancer (NSCLC) (E); Participants with advanced NSCLC (locally advanced who are not candidates for surgical resection or definitive chemoradiation or metastatic) whose tumors have high PD-L1 expression [tumor proportion score (TPS) ≥50%] based on a prior PD-L1 result as determined by College of American Pathologists (CAP)/Clinical Laboratory Improvement Amendments (CLIA) (or equivalently licensed) lab, with no epidermal growth factor receptor (EGFR), anaplastic lymphoma kinase (ALK) or ros oncogene 1 (ROS1) aberrations, and who have not received prior anti-PD-1/PD-L1 therapy and are amenable to IT therapy and do not wish to receive chemotherapy. Note: this cohort is not conducted in Europe	Drug: vidutolimod; Participants will receive vidutolimod for up to 2 years as follows: 10 mg IT weekly for 7 doses after which vidutolimod will be administered every 3 weeks (Q3W). The first dose of vidutolimod may be administered subcutaneously (SC) or IT at the discretion of Investigator. All subsequent doses will be IT. The initial 7 doses of vidutolimod, delivered on a weekly dosing schedule, must be completed before starting the Q3W vidutolimod dosing schedule.; Other Names: CMP-001; Drug: cemiplimab; Participants will receive cemiplimab for up to 2 years as follows: 350 mg IV infusion over 30 minutes at week 1 dose 1 (W1D1) and Q3W thereafter.; Other Names: REGN2810; Libtayo
Experimental: Vidutolimod and cemiplimab for recurrent/metastatic Oropharynx Squamous Cell Carcinoma (OPSCC) (F); Participants with PD-L1 combined positive score (CPS) ≥ 1, based on a prior PD-L1 result and human papillomavirus (HPV)-positive disease based on a prior result, who have not received prior systemic therapy for recurrent/metastatic disease. Participants will receive vidutolimod IT and cemiplimab IV according to the treatment schedule until a reason for treatment discontinuation is reached.	Drug: vidutolimod; Participants will receive vidutolimod for up to 2 years as follows: 10 mg IT weekly for 7 doses after which vidutolimod will be administered every 3 weeks (Q3W). The first dose of vidutolimod may be administered subcutaneously (SC) or IT at the discretion of Investigator. All subsequent doses will be IT. The initial 7 doses of vidutolimod, delivered on a weekly dosing schedule, must be completed before starting the Q3W vidutolimod dosing schedule.; Other Names: CMP-001; Drug: cemiplimab; Participants will receive cemiplimab for up to 2 years as follows: 350 mg IV infusion over 30 minutes at week 1 dose 1 (W1D1) and Q3W thereafter.; Other Names: REGN2810; Libtayo

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Objective Response Rate (ORR)	ORR is defined as the Percent of participants with a confirmed objective response of complete response (CR) or partial response (PR) based on Response Evaluation Criteria in Solid Tumors version 1.1 (RECIST v1.1) by investigator assessment	Up to 31.5 Months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants With Any Treatment-emergent Adverse Event (TEAE), Any Serious TEAE, and Any TEAE Leading to Discontinuation or Death	Number of participants with any treatment-emergent adverse event (TEAE), any serious TEAE, and any TEAE leading to discontinuation or death reported.	Up to 31.5 months
Severity of TEAEs as Assessed by the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE)	Number of participants per NCI CTCAE version 5.0 Adverse Event Grade reported: Grade 1 Mild; asymptomatic or mild symptoms; clinical or diagnostic observations only; intervention not indicated Grade 2 Moderate; minimal, local, or non-invasive intervention indicated; limiting age-appropriate instrumental activities of daily living Grade 3 Severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limited self-care activities of daily living Grade 4 Life-threatening consequences: urgent intervention indicated Grade 5 Death related to adverse event	Up to 31.5 months
Duration of Response (DOR)	DOR is defined as the time from date of first documented response (CR or PR) to date of documented progressive disease (PD), based on RECIST v1.1, per investigator	Up to 31.5 months
Progression Free Survival (PFS)	PFS is defined as the time from date of first dose of study treatment to date of documented PD based on RECIST v1.1 or death, whichever occurs first	Up to 31.5 months
Overall Survival (OS)	OS is defined as the time from date of first dose of study treatment to date of death	Up to 31.5 months

Collaborators and Investigators

• Sponsor: Regeneron Pharmaceuticals
• Investigators: Study Director: Clinical Trial Management, Regeneron Pharmaceuticals

Study record dates

Study Major Dates

• Study Start (Actual): November 30, 2021
• Primary Completion (Actual): October 31, 2024
• Study Completion (Actual): October 31, 2024

Study Registration Dates

• First Submitted: June 1, 2021
• First Submitted That Met QC Criteria: June 1, 2021
• First Posted (Actual): June 7, 2021

Study Record Updates

• Last Update Posted (Estimated): December 5, 2025
• Last Update Submitted That Met QC Criteria: November 20, 2025
• Last Verified: November 1, 2025

More Information

Terms related to this study

• Keywords: NSCLC; TNBC; Advanced Cancer; Metastatic Cancer; BCC; OPSCC; CSCC; vidutolimod; MCC
• Additional Relevant MeSH Terms: Pathologic Processes; Neoplasms by Site; Neoplasms; Infections; Virus Diseases; Respiratory Tract Diseases; Neoplasms by Histologic Type; Lung Diseases; Head and Neck Neoplasms; Neoplasms, Glandular and Epithelial; Adenocarcinoma; Respiratory Tract Neoplasms; Thoracic Neoplasms; Neoplastic Processes; DNA Virus Infections; Lung Neoplasms; Skin Diseases; Breast Diseases; Carcinoma; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms, Nerve Tissue; Carcinoma, Bronchogenic; Bronchial Neoplasms; Neuroendocrine Tumors; Tumor Virus Infections; Carcinoma, Squamous Cell; Polyomavirus Infections; Neoplasms, Basal Cell; Carcinoma, Neuroendocrine; Breast Neoplasms; Pathological Conditions, Signs and Symptoms; Skin and Connective Tissue Diseases; Squamous Cell Carcinoma of Head and Neck; Neoplasm Metastasis; Carcinoma, Non-Small-Cell Lung; Triple Negative Breast Neoplasms; Carcinoma, Basal Cell; Carcinoma, Merkel Cell; Antineoplastic Agents, Immunological; Antineoplastic Agents; cemiplimab
• Other Study ID Numbers: CMP-001-009; 2023-507344-36-01 (Registry Identifier: EUCT Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: All Individual Patient Data (IPD) that underlie publicly available results will be considered for sharing
• IPD Sharing Time Frame: When Regeneron has received marketing authorization from major health authorities (e.g., FDA, European Medicines Agency (EMA), Pharmaceuticals and Medical Devices Agency (PMDA), etc.) for the product and indication, has made the study results publicly available (e.g., scientific publication, scientific conference, clinical trial registry), has the legal authority to share the data, and has ensured the ability to protect participant privacy.
• IPD Sharing Access Criteria: Qualified researchers can submit a proposal for access to individual patient or aggregate level data from a Regeneron-sponsored clinical trial through Vivli. Regeneron's Independent Research Request Evaluation Criteria can be found at: https://www.regeneron.com/sites/default/files/Regeneron-External-Data-Sharing-Policy-and-Independent-Research-Request-Evaluation-Criteria.pdf
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP; ICF; ANALYTIC_CODE; CSR

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No