NCT05929664

Brief Summary

A non-randomized two-cohort study of neoadjuvant Cemiplimab or neoadjuvant Cemiplimab plus Fianlimab (CF) in patients with basal cell carcinoma of the head and neck. Enrollment in the dual-therapy cohort will begin after completion of enrollment in the monotherapy cohort. Patients will undergo at least 2 and up to 6 infusions of immunotherapy prior to surgical resection. If patients have progression on neoadjuvant treatment, they may switch to standard of care surgical resection or hedgehog inhibitors prior to surgery. The primary endpoints are objective response rate and disease control rate. Safety and surgical benefit rate (de-escalation of surgery) with preservation of key anatomic structures are secondary endpoints. Correlative endpoints include analysis of pre and post treatment primary tumor and blood samples compared for histology, tumor genetics and immune cell composition.

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
70

participants targeted

Target at P50-P75 for phase_2

Timeline
62mo left

Started Jul 2023

Longer than P75 for phase_2

Geographic Reach
1 country

3 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress36%
Jul 2023Jul 2031

First Submitted

Initial submission to the registry

February 7, 2023

Completed
5 months until next milestone

First Posted

Study publicly available on registry

July 3, 2023

Completed
2 days until next milestone

Study Start

First participant enrolled

July 5, 2023

Completed
8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 5, 2031

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

July 5, 2031

Last Updated

March 24, 2026

Status Verified

March 1, 2026

Enrollment Period

8 years

First QC Date

February 7, 2023

Last Update Submit

March 19, 2026

Conditions

Outcome Measures

Primary Outcomes (2)

  • Objective response rate (ORR)

    Defined by responders at surgery using clinical assessment and RECIST v1.1. Results will be summarized with frequency counts, percentages, and exact Clopper-Pearson 95% CIs. Tumor response will follow RECIST v1.1: up to five target lesions (max two per organ) measured by longest diameter (non-nodal) or short axis (nodal). Complete Response is disappearance of all target lesions and lymph nodes \<10 mm. Partial Response is ≥30% decrease from baseline. Progressive Disease is ≥20% increase (and ≥5 mm growth) from the smallest on-study sum or the appearance of new lesions. Stable Disease applies when changes do not meet PR or PD. Reasons for unevaluable cases will be documented.

    Up to 6 months post surgery (up to Day 309 +/- 3 days)

  • Disease control rate (DCR)

    Defined responders plus stable disease using clinical assessment and RECIST v1.1. Results will be summarized by frequency counts, percentages, and exact Clopper-Pearson 95% CIs. Tumor response will follow RECIST v1.1: up to five target lesions (no more than two per organ) measured by longest diameter (non-nodal) or short axis (nodal). Complete Response is disappearance of all target lesions and lymph nodes \<10 mm. Partial Response is ≥30% decrease from baseline. Progressive Disease is ≥20% increase (and ≥5 mm growth) from the smallest on-study sum or new lesions. Stable Disease applies when changes do not meet PR or PD. Reasons for unevaluable cases will be documented.

    Up to 6 months post surgery (up to Day 309 +/- 3 days)

Secondary Outcomes (10)

  • Surgical/Clinical Benefit Rate (SBR)

    At surgery (Day 129 +/- 3 days)

  • Pathologic complete response (pCR)

    Up to 6 months post surgery (up to Day 309 +/- 3 days)

  • Major pathologic response (mPR)

    Up to 6 months post surgery (up to Day 309 +/- 3 days)

  • Number of adverse events

    Up to 6 months post surgery (up to Day 309 +/- 3 days)

  • Changes in quality of life and functional organ preservation (FHNSI)

    At baseline (Day 1) to 6 months post-surgery (up to Day 309 +/- 3 days)

  • +5 more secondary outcomes

Study Arms (2)

Cohort 1: Cemiplimab

EXPERIMENTAL

Patients will undergo 2 infusions of Cemiplimab (Cemi), 350 mg every 3 weeks. Patients undergo CT or MRI scans and collection of blood samples throughout the trial. Patients also undergo biopsies during screening and on study. The recommended dose of Cemiplimab is 350 mg as an intravenous infusion over 30 minutes every 3 weeks (constituting one cycle). Dosing will occur in this manner for a single dose of 350mg Cemiplimab every 3 weeks constituting 1 cycle of therapy. Patients will undergo at least 2 cycles of Cemiplimab and at most, 4 additional cycles dependent upon treatment response.

Biological: CemiplimabProcedure: Computed TomographyProcedure: Biospecimen CollectionOther: Quality-of-Life AssessmentProcedure: Magnetic Resonance ImagingProcedure: Biopsy

Cohort 2: Fianlimab + Cemiplimab

EXPERIMENTAL

Patients will undergo infusions of Cemiplimab (C) and Fianlimab (F), 350 mg Cemiplimab + 1600 mg Fianlimab every 3 weeks. Fianlimab in combination with cemiplimab can be administered to patients in a sequential order through co-administration, or concurrently via a fixed-dose combination (FDC). When the FDC is used, the drug product containing co-formulated drugs in a vial is injected into the IV bag and delivered intravenously to the patient. FDC of fianlimab 1600 mg + cemiplimab 350 mg will be administered as a single infusion over 30 to 40 minutes every 3 weeks (constituting one cycle). Patients will undergo at least 2 cycles of the FDC and at most, 4 additional cycles dependent upon treatment response.

Biological: CemiplimabProcedure: Computed TomographyProcedure: Biospecimen CollectionOther: Quality-of-Life AssessmentProcedure: Magnetic Resonance ImagingProcedure: BiopsyBiological: Fianlimab

Interventions

CemiplimabBIOLOGICAL

Given IV

Also known as: 1801342-60-8, Cemiplimab RWLC, Cemiplimab-rwlc, Immunoglobulin G4, Anti-(Human Programmed Cell Death Protein 1) (Human Monoclonal REGN2810 Heavy Chain), Disulfide with Human Monoclonal REGN2810 kappa-chain, Dimer, Libtayo, REGN2810
Cohort 1: CemiplimabCohort 2: Fianlimab + Cemiplimab

Undergo CT scan

Also known as: CAT, CAT Scan, Computed Axial Tomography, computerized axial tomography, Computerized Tomography, CT, CT SCAN, tomography
Cohort 1: CemiplimabCohort 2: Fianlimab + Cemiplimab
BiopsyPROCEDURE

Undergo biopsy

Also known as: BIOPSY_TYPE, Bx
Cohort 1: CemiplimabCohort 2: Fianlimab + Cemiplimab

Undergo collection of blood samples

Also known as: Biological Sample Collection, Biospecimen Collected, Specimen Collection
Cohort 1: CemiplimabCohort 2: Fianlimab + Cemiplimab

Ancillary studies

Also known as: Quality of Life Assessment
Cohort 1: CemiplimabCohort 2: Fianlimab + Cemiplimab

Undergo MRI

Also known as: Magnetic Resonance, Magnetic Resonance Imaging (MRI), Magnetic resonance imaging (procedure), Magnetic Resonance Imaging Scan, Medical Imaging, Magnetic Resonance / Nuclear Magnetic Resonance, MR, MR Imaging, MRI, MRI Scan, MRIs, NMR Imaging, NMRI, nuclear magnetic resonance imaging, sMRI, Structural MRI
Cohort 1: CemiplimabCohort 2: Fianlimab + Cemiplimab
FianlimabBIOLOGICAL

Fianlimab (REGN3767) administered intravenously in combination with Cemiplimab.

Also known as: REGN3767, Anti-LAG-3 MoAb
Cohort 2: Fianlimab + Cemiplimab

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Pathologically confirmed, locally-advanced BCC of the head and neck of any stage which is not resectable without major morbidity or unresectable, defined as requiring greater than 30% auriculectomy, rhinectomy, upper or lower lip resection, orbital exenteration (due to lid or orbital involvement), facial nerve sacrifice, or any Brigham and Women's stage 2b or 3 disease of head and neck (see Table 5).
  • Male or female, aged ≥18 years of age
  • Performance status 0-1.
  • Must have a life expectancy of at least 6 months as judged by the treating physician.
  • Adequate organ function:
  • Absolute neutrophil count 1500/μl or more;
  • Platelets 100,000/μl or more,
  • Hemoglobin 9 g/dl or more;
  • Bilirubin less than or equal to 1.5 x the upper limit of normal (except subjects with Gilbert syndrome, who can have total bilirubin \<3 mg/dl);
  • AST and ALT less than or equal to 2.5 x the upper limit of normal,
  • GFR greater than or equal to 40 ml/min using the Cockcroft-Gault formula or measured creatinine clearance using 24 hours urine collection
  • Women of reproductive potential should have a negative serum or urine pregnancy test (minimum sensitivity 25 IU/L or equivalent units of HCG), which must also be confirmed as negative within 28 days of the start of study drugs.
  • Women of reproductive potential must use highly effective contraception methods to avoid pregnancy for 120 days after the last dose of study drugs. "Women of reproductive potential" is defined as any female who has experienced menarche and who has not undergone surgical sterilization (hysterectomy or bilateral oophorectomy) or who is not postmenopausal. Menopause is defined clinically as 12 months of amenorrhea in a woman over 45 in the absence of other biological or physiological causes. In addition, women under the age of 55 must have a documented serum follicle stimulating hormone (FSH) level less than 40 mIU/mL.
  • Men of reproductive potential who are sexually active with women of reproductive potential must use any contraceptive method with a failure rate of less than 1% per year. Men who are receiving the study medications will be instructed to adhere to contraception for 120 days after the last dose of study drugs. Men who are azoospermic do not require contraception.
  • Informed Consent: All subjects must be able to comprehend and sign a written informed consent document.

You may not qualify if:

  • An individual who meets any of the following criteria will be excluded from participation in this study:
  • Prior radiation therapy within the past 6 months for this target cancer documented by surgeon at Visit 1, Day 0 initial assessment. (Prior surgical resection to area/tumor is acceptable.)
  • Any history of allergy to the study drug components.
  • Any concurrent malignancies: exceptions include- basal cell carcinoma of the skin at another site, chronic lymphocytic leukemia, melanoma in situ, squamous cell carcinoma of the skin of a secondary location, superficial bladder cancer or in situ cervical cancer that has undergone potentially curative therapy. Patients with a history of other prior malignancy must have been treated with curative intent and must have remained disease-free for 2 years post-diagnosis.
  • Any Subjects with a condition requiring systemic treatment with either corticosteroids (\> 10 mg daily prednisone equivalents) or other immunosuppressive medications within 28 days of study drug administration., or a prior history of allogenic organ transplantation.
  • Any diagnosis of a significant connective tissue disorder as determined by the treating surgeon or medical team.
  • Patients must not be receiving any other investigational agents.
  • Receipt of a live attenuated vaccine within 30 days prior to the first dose of drug on trial.
  • Uncontrolled intercurrent illness, including but not limited to, ongoing or active infection, symptomatic congestive heart failure, uncontrolled hypertension, unstable angina pectoris, cardiac arrhythmia, interstitial lung disease, serious chronic gastrointestinal conditions associated with diarrhea, or psychiatric illness/social situations that would limit compliance with study requirement, substantially increase risk of incurring AEs or compromise the ability of the patient to give written informed consent.
  • Patients must not be pregnant or breastfeeding.
  • Active infection including tuberculosis (clinical evaluation that includes clinical history, physical examination and radiographic findings, and TB testing in line with local practice), hepatitis B (known positive HBV surface antigen (HBsAg) result), hepatitis C, or human immunodeficiency virus (positive HIV 1/2 antibodies). Patients with a past or resolved HBV infection (defined as the presence of hepatitis B core antibody \[anti-HBc\]and absence of HBsAg) are eligible. Patients positive for hepatitis C (HCV)antibody are eligible only if polymerase chain reaction is negative for HCVRNA.
  • Any patient with prior immunotherapy or HHI for malignancy treatment.
  • Any untreated metastasis(es) to the brain that may be considered active.
  • History of pneumonitis with the past 5 years.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (3)

University of Miami Health System

Miami, Florida, 33136, United States

RECRUITING

Thomas Jefferson University Hospital

Philadelphia, Pennsylvania, 19107, United States

RECRUITING

Vanderbilt University

Nashville, Tennessee, 37232, United States

RECRUITING

MeSH Terms

Conditions

Carcinoma, Basal Cell

Interventions

cemiplimabImmunoglobulin GDisulfidesSpecimen HandlingMagnetic Resonance SpectroscopyX-RaysBiopsy

Condition Hierarchy (Ancestors)

CarcinomaNeoplasms, Glandular and EpithelialNeoplasms by Histologic TypeNeoplasmsNeoplasms, Basal Cell

Intervention Hierarchy (Ancestors)

Immunoglobulin IsotypesAntibodiesImmunoglobulinsImmunoproteinsBlood ProteinsProteinsAmino Acids, Peptides, and ProteinsSerum GlobulinsGlobulinsSulfidesAnionsIonsElectrolytesInorganic ChemicalsHydrogen SulfideSulfur CompoundsOrganic ChemicalsClinical Laboratory TechniquesDiagnostic Techniques and ProceduresDiagnosisInvestigative TechniquesSpectrum AnalysisChemistry Techniques, AnalyticalElectromagnetic RadiationElectromagnetic PhenomenaMagnetic PhenomenaPhysical PhenomenaRadiationRadiation, IonizingCytodiagnosisCytological TechniquesDiagnostic Techniques, SurgicalSurgical Procedures, Operative

Study Officials

  • Joseph Curry, MD

    Thomas Jefferson University

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

February 7, 2023

First Posted

July 3, 2023

Study Start

July 5, 2023

Primary Completion (Estimated)

July 5, 2031

Study Completion (Estimated)

July 5, 2031

Last Updated

March 24, 2026

Record last verified: 2026-03

Locations