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NCT04633278

CMP-001 in Combination With Pembrolizumab in Subjects With Recurrent or Metastatic Head and Neck Squamous Cell Carcinoma

Terminated Phase 2 Results posted Last updated 11 May 2025
What this trial tests

Phase 2 trial testing CMP-001 in Squamous Cell Carcinoma of Head and Neck in 24 participants. Terminated before completion.

Timeline
21 January 2021
Primary endpoint
19 January 2024
19 January 2024

Quick facts

Lead sponsorRegeneron Pharmaceuticals
PhasePhase 2
StatusTerminated
Study typeINTERVENTIONAL
Allocationna
Designsingle group
Maskingnone
Primary purposetreatment
Enrollment24
Start date21 January 2021
Primary completion19 January 2024
Estimated completion19 January 2024
Sites19 locations across United States

Drugs / interventions tested

Conditions studied

Sponsor

Regeneron Pharmaceuticals — full company profile →

Who can join

18 and older, any sex, with Squamous Cell Carcinoma of Head and Neck. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Confirmed Objective Response Rate (ORR) Primary · Up to approximately 109 weeks

Confirmed ORR is defined as the percentage of participants with a confirmed best overall response (BOR) of complete response (CR) or partial response (PR) based on Response Evaluation Criteria in Solid Tumors version 1.1 (RECIST v1.1) as determined by Investigator assessment (IA).

GroupValue95% CI
CMP-001 IT + Pembrolizumab - Schedule A17.63.80 – 43.43
CMP-001 IT + Pembrolizumab - Schedule B00.00 – 40.96
Number of Participants With Any Treatment-emergent Adverse Event (TEAE), Any Serious TEAE, and Any TEAE Leading to Discontinuation or Death Secondary · Up to approximately 109 weeks

TEAE is defined as an AE that started or worsened in severity on or after the date that study drug was first administered (W1D1) until 30 days after the last dose of study treatment.

Any TEAE
GroupValue95% CI
CMP-001 IT + Pembrolizumab - Schedule A17
CMP-001 IT + Pembrolizumab - Schedule B7
Any serious TEAE
GroupValue95% CI
CMP-001 IT + Pembrolizumab - Schedule A7
CMP-001 IT + Pembrolizumab - Schedule B5
Any TEAE leading to death
GroupValue95% CI
CMP-001 IT + Pembrolizumab - Schedule A1
CMP-001 IT + Pembrolizumab - Schedule B0
Severity of TEAEs as Assessed by the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE), Version 5.0 Secondary · Up to approximately 109 weeks

NI CTCAE Adverse Event Grades: 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

Grade 1 - 2
GroupValue95% CI
CMP-001 IT + Pembrolizumab - Schedule A7
CMP-001 IT + Pembrolizumab - Schedule B1
Grade 3
GroupValue95% CI
CMP-001 IT + Pembrolizumab - Schedule A7
CMP-001 IT + Pembrolizumab - Schedule B6
Grade 4
GroupValue95% CI
CMP-001 IT + Pembrolizumab - Schedule A2
CMP-001 IT + Pembrolizumab - Schedule B0
Grade 5
GroupValue95% CI
CMP-001 IT + Pembrolizumab - Schedule A1
CMP-001 IT + Pembrolizumab - Schedule B0
Duration of Response (DOR) Secondary · Up to approximately 28 months (120 weeks)

DOR, defined as the time from date of first documented response (CR or PR) to date of documented PD, based on RECIST v1.1 by Investigator assessment (IA).

GroupValue95% CI
CMP-001 IT + Pembrolizumab - Schedule ANA2.825 – NA
Duration of Progression-free Survival (PFS) Secondary · Up to approximately 28 months (120 weeks)

Progression-free Survival (PFS), defined as the time from date of first dose of study drug to date of documented PD based on RECIST v1.1 by IA or death, whichever occurs first.

GroupValue95% CI
CMP-001 IT + Pembrolizumab - Schedule A2.1031.183 – 6.472
CMP-001 IT + Pembrolizumab - Schedule B1.8071.478 – 3.450
Duration of Overall Survival (OS) Secondary · From first dose up to approximately 28 months (120 weeks)

Overall survival (OS) is defined as the time from the first dose date of the study treatment to the date of death from any cause.

GroupValue95% CI
CMP-001 IT + Pembrolizumab - Schedule A6.9322.333 – 14.456
CMP-001 IT + Pembrolizumab - Schedule B7.1291.643 – 12.287
Immune Objective Response Rate (iORR) Secondary · Up to approximately 109 weeks

Immune objective response rate (iORR), defined as the percentage of participants who have an immune best overall response (iBOR) of immune complete response (iCR) or immune partial response (iPR) based on the immunotherapy Response Evaluation Criteria in Solid Tumors (iRECIST) as determined by Investigator.

GroupValue95% CI
CMP-001 IT + Pembrolizumab - Schedule A00.00 – 97.50
CMP-001 IT + Pembrolizumab - Schedule B00.00 – 84.19
Immune Progression-free Survival (iPFS) Secondary · Up to approximately 28 months (120 weeks)

iPFS, defined as the time from date of first dose of study drug to date of immune Confirmed Progressive Disease (iCPD) based on immunotherapy Response Evaluation Criteria in Solid Tumors (iRECIST) by IA or death, whichever occurs first.

GroupValue95% CI
CMP-001 IT + Pembrolizumab - Schedule A9.9559.955 – 9.955
CMP-001 IT + Pembrolizumab - Schedule BNA3.38 – 3.45
Confirmed ORR Based on Human Papillomavirus (HPV) Status Secondary · Up to approximately 109 weeks

Confirmed ORR is defined as the percentage of participants with a confirmed best overall response (BOR) of complete response (CR) or partial response (PR) based on Response Evaluation Criteria in Solid Tumors version 1.1 (RECIST v1.1) as determined by Investigator assessment (IA) based on HPV status.

HPV positive
GroupValue95% CI
CMP-001 IT + Pembrolizumab - Schedule A16.70.42 – 64.12
CMP-001 IT + Pembrolizumab - Schedule B00.00 – 97.50
HPV negative
GroupValue95% CI
CMP-001 IT + Pembrolizumab - Schedule A25.00.63 – 80.59
CMP-001 IT + Pembrolizumab - Schedule B00.00 – 52.18
Confirmed ORR Based on Programmed Death-ligand 1 (PD-L1) Expressions Secondary · Up to approximately 109 weeks

Confirmed ORR is defined as the percentage of participants with a confirmed best overall response (BOR) of complete response (CR) or partial response (PR) based on Response Evaluation Criteria in Solid Tumors version 1.1 (RECIST v1.1) as determined by Investigator assessment (IA) based on PD-L1 expressions (combined positive score \[CPS\] ≥ 1 and CPS ≥ 20)

PD-L1 Expression: 1 to < 20
GroupValue95% CI
CMP-001 IT + Pembrolizumab - Schedule A20.02.52 – 55.61
CMP-001 IT + Pembrolizumab - Schedule B00.00 – 84.19
PD-L1 Expression: >= 20
GroupValue95% CI
CMP-001 IT + Pembrolizumab - Schedule A14.30.36 – 57.87
CMP-001 IT + Pembrolizumab - Schedule B00.00 – 60.24
Duration of PFS Based on HPV Status Secondary · Up to approximately 28 months (120 weeks)

Progression-free Survival (PFS), defined as the time from date of first dose of study drug to date of documented PD based on RECIST v1.1 by IA or death, whichever occurs first. PFS based on HPV status

HPV positive
GroupValue95% CI
CMP-001 IT + Pembrolizumab - Schedule A3.2530.72 – 22.44
CMP-001 IT + Pembrolizumab - Schedule B3.9753.975 – 3.975
HPV negative
GroupValue95% CI
CMP-001 IT + Pembrolizumab - Schedule A1.1830.03 – 6.90
CMP-001 IT + Pembrolizumab - Schedule B1.8071.48 – 3.45
DOR Based on HPV Status Secondary · Up to approximately 28 months (120 weeks)

DOR, defined as the time from date of first documented response (CR or PR) to date of documented PD, based on RECIST v1.1 by Investigator assessment (IA). DOR based on HPV status.

HPV negative
GroupValue95% CI
CMP-001 IT + Pembrolizumab - Schedule ANANA – NA
HPV positive
GroupValue95% CI
CMP-001 IT + Pembrolizumab - Schedule ANANA – NA

Adverse events — posted to ClinicalTrials.gov

Time frame: From signing of informed consent through end of study up to approximately 28 months (120 weeks). Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

CMP-001 IT + Pembrolizumab - Schedule A
Serious: 7/17 (41%)
Deaths: 11/17
CMP-001 IT + Pembrolizumab - Schedule B
Serious: 5/7 (71%)
Deaths: 7/7

Serious adverse events (16 terms)

ReactionSystemCMP-001 IT + Pembrolizumab…CMP-001 IT + Pembrolizumab…
HypercalcaemiaMetabolism and nutrition disorders——
SepsisInfections and infestations——
HypernatraemiaMetabolism and nutrition disorders——
Anaphylactic reactionImmune system disorders——
Cytokine release syndromeImmune system disorders——
Gastric haemorrhageGastrointestinal disorders——
Multiple organ dysfunction syndromeGeneral disorders——
COVID-19Infections and infestations——
Bacterial sepsisInfections and infestations——
PneumoniaInfections and infestations——
Laryngeal oedemaRespiratory, thoracic and mediastinal disorders——
Pulmonary embolismRespiratory, thoracic and mediastinal disorders——
HaemoptysisRespiratory, thoracic and mediastinal disorders——
HypotensionVascular disorders——
Tumour haemorrhageNeoplasms benign, malignant and unspecified (incl cysts and polyps)——
SyncopeNervous system disorders——
Other adverse events (109 terms — click to expand)

ReactionSystemCMP-001 IT + Pembrolizumab…CMP-001 IT + Pembrolizumab…
ChillsGeneral disorders——
PyrexiaGeneral disorders——
NauseaGastrointestinal disorders——
ConstipationGastrointestinal disorders——
HeadacheNervous system disorders——
HypothyroidismEndocrine disorders——
FatigueGeneral disorders——
Injection site painGeneral disorders——
StomatitisGastrointestinal disorders——
DyspnoeaRespiratory, thoracic and mediastinal disorders——
Decreased appetiteMetabolism and nutrition disorders——
HypotensionVascular disorders——
Blood thyroid stimulating hormone increasedInvestigations——
Facial painGeneral disorders——
DiarrhoeaGastrointestinal disorders——
DysphagiaGastrointestinal disorders——
Oral painGastrointestinal disorders——
VomitingGastrointestinal disorders——
HaemoptysisRespiratory, thoracic and mediastinal disorders——
Pneumonia aspirationRespiratory, thoracic and mediastinal disorders——
HypercalcaemiaMetabolism and nutrition disorders——
HyperglycaemiaMetabolism and nutrition disorders——
HyponatraemiaMetabolism and nutrition disorders——
HypophosphataemiaMetabolism and nutrition disorders——
DizzinessNervous system disorders——
MyalgiaMusculoskeletal and connective tissue disorders——
SARS-CoV-2 test positiveInvestigations——
Weight decreasedInvestigations——
Tumour painNeoplasms benign, malignant and unspecified (incl cysts and polyps)——
RashSkin and subcutaneous tissue disorders——
TachycardiaCardiac disorders——
LymphopeniaBlood and lymphatic system disorders——
AnaemiaBlood and lymphatic system disorders——
Face oedemaGeneral disorders——
Injection site oedemaGeneral disorders——
Injection site swellingGeneral disorders——
Injection site vesiclesGeneral disorders——
Localised oedemaGeneral disorders——
MalaiseGeneral disorders——
PainGeneral disorders——

Most-reported serious reactions: Hypercalcaemia, Sepsis, Hypernatraemia, Anaphylactic reaction, Cytokine release syndrome, Gastric haemorrhage, Multiple organ dysfunction syndrome, COVID-19.

Data from ClinicalTrials.gov NCT04633278 adverse events section.

Sponsor's own description

CMP-001-007 is a Phase 2 study of CMP-001 intratumoral (IT) and pembrolizumab intravenous (IV) administered to participants with head and neck squamous cell carcinoma (HNSCC) who have not been previously treated with a programmed cell death protein 1 (PD-1) blocking antibody. The primary objective of the study is to determine the Investigator-assessed confirmed objective response with CMP-001 in combination with pembrolizumab in subjects with head and neck squamous cell carcinoma (HNSCC) The secondary objectives are to: * To evaluate the safety and tolerability of CMP-001 administered by intratumoral (IT) injection in combination with pembrolizumab in subjects with HNSCC * To evaluate the efficacy of CMP-001 in combination with pembrolizumab in subjects with HNSCC * To evaluate the effect of human papillomavirus (HPV) infection and programmed death-ligand 1 (PD-L1) expressions on the efficacy of CMP-001 in combination with pembrolizumab Participants will continue to receive treatment of CMP-001 and pembrolizumab according to the treatment schedule until a reason for treatment discontinuation is reached.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

  1. Type I interferon-mediated tumor immunity and its role in immunotherapy.
    Yu R, Zhu B, Chen D. · · 2022 · cited 229× · PMID 35292881 · DOI 10.1007/s00018-022-04219-z
  2. Tumor-Associated Macrophages Regulate PD-1/PD-L1 Immunosuppression.
    Pu Y, Ji Q. · · 2022 · cited 212× · PMID 35592338 · DOI 10.3389/fimmu.2022.874589
  3. Harnessing innate immune pathways for therapeutic advancement in cancer.
    Hu A, Sun L, Lin H, Liao Y, et al · · 2024 · cited 150× · PMID 38523155 · DOI 10.1038/s41392-024-01765-9
  4. Applications and clinical trial landscape using Toll-like receptor agonists to reduce the toll of cancer.
    Rolfo C, Giovannetti E, Martinez P, McCue S, et al · · 2023 · cited 119× · PMID 36890302 · DOI 10.1038/s41698-023-00364-1
  5. Toll-like receptor 9 agonists and combination therapies: strategies to modulate the tumour immune microenvironment for systemic anti-tumour immunity.
    Dongye Z, Li J, Wu Y. · · 2022 · cited 92× · PMID 35902641 · DOI 10.1038/s41416-022-01876-6
  6. Kickstarting Immunity in Cold Tumours: Localised Tumour Therapy Combinations With Immune Checkpoint Blockade.
    Appleton E, Hassan J, Chan Wah Hak C, Sivamanoharan N, et al · · 2021 · cited 46× · PMID 34733287 · DOI 10.3389/fimmu.2021.754436
  7. Trial watch: intratumoral immunotherapy.
    Humeau J, Le Naour J, Galluzzi L, Kroemer G, et al · · 2021 · cited 45× · PMID 34676147 · DOI 10.1080/2162402x.2021.1984677
  8. Toll-like receptor-targeted anti-tumor therapies: Advances and challenges.
    Yang Y, Li H, Fotopoulou C, Cunnea P, et al · · 2022 · cited 44× · PMID 36479129 · DOI 10.3389/fimmu.2022.1049340

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Other trials of CMP-001

Trials testing the same drug.

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Currently open trials in the same condition.

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Trials by the same sponsor.

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Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing