Merkel Cell CarcinomaPhase 2NCT04916002
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 Phase 2 study testing vidutolimod and cemiplimab in people with Merkel Cell Carcinoma and Cutaneous Squamous Cell Carcinoma, run by Regeneron Pharmaceuticals at 24 sites in 2 countries.
Trial summary
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… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Merkel Cell Carcinoma, Cutaneous Squamous Cell Carcinoma, Basal Cell Carcinoma
- What is being tested?
- vidutolimod and cemiplimab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 77 participants
- Where
- 24 sitesin 2 countries
- Study dates
- 30 Nov 2021to 31 Oct 2024
- Phase
- Phase 2
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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Participants enrolled in the study must meet all of the following inclusion criteria to be eligible.
- Histopathologically-confirmed diagnosis of cancer, as defined by the protocol.
- 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.
- 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.
- Eastern Cooperative Oncology Group (ECOG) Performance Status of 0 to 1 at Screening.
Who cannot join
- Participants presenting with any of the following will not qualify for entry into the study:
- 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.
- 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.
- 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.
- History of immune-mediated AE leading to permanent discontinuation due to prior PD-1-blocking antibody.
- 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.
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- Active pneumonitis or history of noninfectious pneumonitis that required steroids.
- 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.
- Known history of immunodeficiency.
- Known additional malignancy that is progressing or required active treatment within the past 3 years, as defined in the protocol.
- Active autoimmune disease that required systemic treatment in past 2 years; replacement therapy is not considered a form of systemic treatment.
- 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
Trial locations and contacts
24 sites in 2 countries.
24 sites in 2 countries
United States20 sites
VA Maryland Health Care System
Baltimore, Maryland, 20814
University of Alabama
Birmingham, Alabama, 35294
Dana-Farber Cancer Institute
Boston, Massachusetts, 02215
Ohio State University
Columbus, Ohio, 43210
Karmanos Cancer Institute
Detroit, Michigan, 48201
City of Hope
Duarte, California, 91010
East Carolina University
Greenville, North Carolina, 27858
Oncology Consultants
Houston, Texas, 77030
University of Iowa
Iowa City, Iowa, 52242
GenesisCare USA
Jacksonville, Florida, 32204
UC San Diego Moores Cancer Center
La Jolla, California, 92037-0845
Dartmouth Clinical Trial Office
Lebanon, New Hampshire, 03756
University of California
Los Angeles, California, 90095
Norton Cancer Institute
Louisville, Kentucky, 40202
Memorial Sloan Kettering Cancer Center
New York, New York, 10065
OU Health Stephenson Cancer Center
Oklahoma City, Oklahoma, 73104
Orlando Health Cancer Institute
Orlando, Florida, 32806
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, 15232
University of Utah Huntsman Cancer Center
Salt Lake City, Utah, 84112
University of Washington
Seattle, Washington, 98109
Australia4 sites
St. Vincent's Hospital
Darlinghurst, New South Wales, 2010
Peter MacCallum Cancer Centre (PMCC) and The Royal Melbourne Hospital
Melbourne, Victoria, 3050
Fiona Stanley Hospital
Murdoch, Western Australia, 6150
Princess Alexandra Hospital
Woolloongabba, Queensland, 4102
About this study
Official title: 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.
Read the detailed description
Former Sponsor Checkmate Pharmaceuticals Note: Early termination planned on 31Oct2024 due to study drug supply
- Conditions
- Merkel Cell Carcinoma, Cutaneous Squamous Cell Carcinoma, Basal Cell Carcinoma, Triple Negative Breast Cancer, Non-Small Cell Lung Cancer, Oropharynx Squamous Cell Carcinoma
- Being tested
- vidutolimod, cemiplimab
- Keywords
- vidutolimod, Metastatic Cancer, Advanced Cancer, MCC, CSCC, BCC, TNBC, NSCLC, OPSCC
- Medical subject headings
- Carcinoma, Merkel Cell, Carcinoma, Basal Cell, Triple Negative Breast Neoplasms, Carcinoma, Non-Small-Cell Lung, Squamous Cell Carcinoma of Head and Neck, Neoplasm Metastasis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
vidutolimodDrug
Also called CMP-001
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.
cemiplimabDrug
Also called Libtayo, REGN2810
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.
| Study group | Role | What they receive |
|---|---|---|
| Vidutolimod and cemiplimab for cutaneous squamous cell carcinoma (CSCC) (A1) | Experimental | 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. Receives: Drug: vidutolimod; Drug: cemiplimab |
| Vidutolimod and cemiplimab for CSCC (A2) | Experimental | 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. Receives: Drug: vidutolimod; Drug: cemiplimab |
| Vidutolimod and cemiplimab for Merkel cell carcinoma (MCC) (B1) | Experimental | 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. Receives: Drug: vidutolimod; Drug: cemiplimab |
| Vidutolimod and cemiplimab for MCC (B2) | Experimental | 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. Receives: Drug: vidutolimod; Drug: cemiplimab |
| Vidutolimod and cemiplimab for triple negative breast cancer (TNBC) (C1) | Experimental | 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. Receives: Drug: vidutolimod; Drug: cemiplimab |
| Vidutolimod and cemiplimab for TNBC (C2) | Experimental | 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. Receives: Drug: vidutolimod; Drug: cemiplimab |
| Vidutolimod and cemiplimab for basal cell carcinoma (BCC) (D) | Experimental | 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. Receives: Drug: vidutolimod; Drug: cemiplimab |
| Vidutolimod and cemiplimab for non-small cell lung cancer (NSCLC) (E) | Experimental | 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 Receives: Drug: vidutolimod; Drug: cemiplimab |
| Vidutolimod and cemiplimab for recurrent/metastatic Oropharynx Squamous Cell Carcinoma (OPSCC) (F) | Experimental | 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. Receives: Drug: vidutolimod; Drug: cemiplimab |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: Up to 31.5 Months
Secondary outcomes (5)
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.
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: Up to 31.5 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Jun 2021
- Study start30 Nov 2021
- Primary completion31 Oct 2024
- Study completion31 Oct 2024
Why it stopped: Sponsor Decision related to study drug supply
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Merkel Cell CarcinomaStudies of the same condition, open and under way.
- More trials run by Regeneron PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 77 participants (actual)
Sponsor and investigators
- Lead sponsor
- Regeneron Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Trial Management, Study director, Regeneron Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04916002
- Sponsor's protocol ID
- CMP-001-009
- Other IDs
- 2023-507344-36-01, 2023
- Status last verified
- May 2026
- First submitted
- 1 Jun 2021
- Last updated
- 9 Jun 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: All Individual Patient Data (IPD) that underlie publicly available results will be considered for sharing