Trial Outcomes & Findings for CMP-001 in Combination With Pembrolizumab in Subjects With Recurrent or Metastatic Head and Neck Squamous Cell Carcinoma (NCT NCT04633278)

Last Updated: 2025-05-11

Results Overview

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).

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

24 participants

Primary outcome timeframe

Up to approximately 109 weeks

Results posted on

2025-05-11

Participant Flow

Participant milestones

Participant milestones
Measure	CMP-001 IT + Pembrolizumab - Schedule A Participants received CMP-001 intratumorally (IT) and pembrolizumab intravenously (IV) according to treatment schedule A.	CMP-001 IT + Pembrolizumab - Schedule B Participants received CMP-001 IT and pembrolizumab IV according to treatment schedule B.
Overall Study STARTED	17	7
Overall Study COMPLETED	0	0
Overall Study NOT COMPLETED	17	7

Reasons for withdrawal

Reasons for withdrawal
Measure	CMP-001 IT + Pembrolizumab - Schedule A Participants received CMP-001 intratumorally (IT) and pembrolizumab intravenously (IV) according to treatment schedule A.	CMP-001 IT + Pembrolizumab - Schedule B Participants received CMP-001 IT and pembrolizumab IV according to treatment schedule B.
Overall Study Adverse Event	1	0
Overall Study Withdrawal by Subject	1	0
Overall Study Death	11	7
Overall Study Sponsor Decision	3	0
Overall Study Not Listed	1	0

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	CMP-001 IT + Pembrolizumab - Schedule A n=17 Participants Participants received CMP-001 intratumorally (IT) and pembrolizumab intravenously (IV) according to treatment schedule A.	CMP-001 IT + Pembrolizumab - Schedule B n=7 Participants Participants received CMP-001 IT and pembrolizumab IV according to treatment schedule B.	Total n=24 Participants Total of all reporting groups
Age, Continuous	64.1 Years STANDARD_DEVIATION 8.57 • n=5 Participants	71.0 Years STANDARD_DEVIATION 10.33 • n=7 Participants	66.1 Years STANDARD_DEVIATION 9.45 • n=5 Participants
Sex: Female, Male Female	4 Participants n=5 Participants	3 Participants n=7 Participants	7 Participants n=5 Participants
Sex: Female, Male Male	13 Participants n=5 Participants	4 Participants n=7 Participants	17 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	15 Participants n=5 Participants	7 Participants n=7 Participants	22 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) White	15 Participants n=5 Participants	7 Participants n=7 Participants	22 Participants n=5 Participants
Race (NIH/OMB) More than one race	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants

PRIMARY outcome

Timeframe: Up to approximately 109 weeks

Population: Treated Analysis Set (All participants who received at least 1 dose of CMP-001)

Outcome measures

Outcome measures
Measure	CMP-001 IT + Pembrolizumab - Schedule A n=17 Participants Participants received CMP-001 intratumorally (IT) and pembrolizumab intravenously (IV) according to treatment schedule A.	CMP-001 IT + Pembrolizumab - Schedule B n=7 Participants Participants received CMP-001 IT and pembrolizumab IV according to treatment schedule B.
Confirmed Objective Response Rate (ORR)	17.6 Percentage of participants Interval 3.8 to 43.43	0 Percentage of participants Interval 0.0 to 40.96

SECONDARY outcome

Timeframe: Up to approximately 109 weeks

Population: Treated Analysis Set (All participants who received at least 1 dose of CMP-001)

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.

Outcome measures

Outcome measures
Measure	CMP-001 IT + Pembrolizumab - Schedule A n=17 Participants Participants received CMP-001 intratumorally (IT) and pembrolizumab intravenously (IV) according to treatment schedule A.	CMP-001 IT + Pembrolizumab - Schedule B n=7 Participants Participants received CMP-001 IT and pembrolizumab IV according to treatment schedule B.
Number of Participants With Any Treatment-emergent Adverse Event (TEAE), Any Serious TEAE, and Any TEAE Leading to Discontinuation or Death Any TEAE	17 Participants	7 Participants
Number of Participants With Any Treatment-emergent Adverse Event (TEAE), Any Serious TEAE, and Any TEAE Leading to Discontinuation or Death Any serious TEAE	7 Participants	5 Participants
Number of Participants With Any Treatment-emergent Adverse Event (TEAE), Any Serious TEAE, and Any TEAE Leading to Discontinuation or Death Any TEAE leading to death	1 Participants	0 Participants

SECONDARY outcome

Timeframe: Up to approximately 109 weeks

Population: Treated Analysis Set (All participants who received at least 1 dose of CMP-001)

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

Outcome measures

Outcome measures
Measure	CMP-001 IT + Pembrolizumab - Schedule A n=17 Participants Participants received CMP-001 intratumorally (IT) and pembrolizumab intravenously (IV) according to treatment schedule A.	CMP-001 IT + Pembrolizumab - Schedule B n=7 Participants Participants received CMP-001 IT and pembrolizumab IV according to treatment schedule B.
Severity of TEAEs as Assessed by the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE), Version 5.0 Grade 1 - 2	7 Participants	1 Participants
Severity of TEAEs as Assessed by the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE), Version 5.0 Grade 3	7 Participants	6 Participants
Severity of TEAEs as Assessed by the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE), Version 5.0 Grade 4	2 Participants	0 Participants
Severity of TEAEs as Assessed by the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE), Version 5.0 Grade 5	1 Participants	0 Participants

SECONDARY outcome

Timeframe: Up to approximately 28 months (120 weeks)

Population: Number of participants analyzed for DOR includes only participants who have a confirmed best overall response (BOR) of complete response (CR) or partial response (PR).

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).

Outcome measures

Outcome measures
Measure	CMP-001 IT + Pembrolizumab - Schedule A n=3 Participants Participants received CMP-001 intratumorally (IT) and pembrolizumab intravenously (IV) according to treatment schedule A.	CMP-001 IT + Pembrolizumab - Schedule B Participants received CMP-001 IT and pembrolizumab IV according to treatment schedule B.
Duration of Response (DOR)	NA Months Interval 2.825 to Not evaluable due to low number of participants analyzed.	—

SECONDARY outcome

Timeframe: Up to approximately 28 months (120 weeks)

Population: Treated Analysis Set (All participants who received at least 1 dose of CMP-001)

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.

Outcome measures

Outcome measures
Measure	CMP-001 IT + Pembrolizumab - Schedule A n=17 Participants Participants received CMP-001 intratumorally (IT) and pembrolizumab intravenously (IV) according to treatment schedule A.	CMP-001 IT + Pembrolizumab - Schedule B n=7 Participants Participants received CMP-001 IT and pembrolizumab IV according to treatment schedule B.
Duration of Progression-free Survival (PFS)	2.103 Months Interval 1.183 to 6.472	1.807 Months Interval 1.478 to 3.45

SECONDARY outcome

Timeframe: From first dose up to approximately 28 months (120 weeks)

Population: Treated Analysis Set (All participants who received at least 1 dose of CMP-001)

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.

Outcome measures

Outcome measures
Measure	CMP-001 IT + Pembrolizumab - Schedule A n=17 Participants Participants received CMP-001 intratumorally (IT) and pembrolizumab intravenously (IV) according to treatment schedule A.	CMP-001 IT + Pembrolizumab - Schedule B n=7 Participants Participants received CMP-001 IT and pembrolizumab IV according to treatment schedule B.
Duration of Overall Survival (OS)	6.932 Months Interval 2.333 to 14.456	7.129 Months Interval 1.643 to 12.287

SECONDARY outcome

Timeframe: Up to approximately 109 weeks

Population: Treated Analysis Set (All participants who received at least 1 dose of CMP-001)

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.

Outcome measures

Outcome measures
Measure	CMP-001 IT + Pembrolizumab - Schedule A n=17 Participants Participants received CMP-001 intratumorally (IT) and pembrolizumab intravenously (IV) according to treatment schedule A.	CMP-001 IT + Pembrolizumab - Schedule B n=7 Participants Participants received CMP-001 IT and pembrolizumab IV according to treatment schedule B.
Immune Objective Response Rate (iORR)	0 Percentage of participants Interval 0.0 to 97.5	0 Percentage of participants Interval 0.0 to 84.19

SECONDARY outcome

Timeframe: Up to approximately 28 months (120 weeks)

Population: Number analyzed = Number of participants continuing study treatment beyond PD.

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.

Outcome measures

Outcome measures
Measure	CMP-001 IT + Pembrolizumab - Schedule A n=1 Participants Participants received CMP-001 intratumorally (IT) and pembrolizumab intravenously (IV) according to treatment schedule A.	CMP-001 IT + Pembrolizumab - Schedule B n=2 Participants Participants received CMP-001 IT and pembrolizumab IV according to treatment schedule B.
Immune Progression-free Survival (iPFS)	9.955 Months Interval 9.955 to 9.955	NA Months Interval 3.38 to 3.45 Data not available due to insufficient number of participants with events.

SECONDARY outcome

Timeframe: Up to approximately 109 weeks

Population: Subgroup of participants in Treated Analysis Set that completed HPV testing

Outcome measures

Outcome measures
Measure	CMP-001 IT + Pembrolizumab - Schedule A n=10 Participants Participants received CMP-001 intratumorally (IT) and pembrolizumab intravenously (IV) according to treatment schedule A.	CMP-001 IT + Pembrolizumab - Schedule B n=6 Participants Participants received CMP-001 IT and pembrolizumab IV according to treatment schedule B.
Confirmed ORR Based on Human Papillomavirus (HPV) Status HPV positive	16.7 Percentage of participants Interval 0.42 to 64.12	0 Percentage of participants Interval 0.0 to 97.5
Confirmed ORR Based on Human Papillomavirus (HPV) Status HPV negative	25.0 Percentage of participants Interval 0.63 to 80.59	0 Percentage of participants Interval 0.0 to 52.18

SECONDARY outcome

Timeframe: Up to approximately 109 weeks

Population: Subgroup of participants in Treated Analysis Set that had PL-1 expressions; For 1 participant, the site used a local, qualitative test (positive/negative). Therefore, the CPS (%) is missing for this 1 participant.

Outcome measures

Outcome measures
Measure	CMP-001 IT + Pembrolizumab - Schedule A n=17 Participants Participants received CMP-001 intratumorally (IT) and pembrolizumab intravenously (IV) according to treatment schedule A.	CMP-001 IT + Pembrolizumab - Schedule B n=6 Participants Participants received CMP-001 IT and pembrolizumab IV according to treatment schedule B.
Confirmed ORR Based on Programmed Death-ligand 1 (PD-L1) Expressions PD-L1 Expression: 1 to < 20	20.0 Percentage of participants Interval 2.52 to 55.61	0 Percentage of participants Interval 0.0 to 84.19
Confirmed ORR Based on Programmed Death-ligand 1 (PD-L1) Expressions PD-L1 Expression: >= 20	14.3 Percentage of participants Interval 0.36 to 57.87	0 Percentage of participants Interval 0.0 to 60.24

SECONDARY outcome

Timeframe: Up to approximately 28 months (120 weeks)

Population: Subgroup of participants in Treated Analysis Set that completed HPV testing

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

Outcome measures

Outcome measures
Measure	CMP-001 IT + Pembrolizumab - Schedule A n=10 Participants Participants received CMP-001 intratumorally (IT) and pembrolizumab intravenously (IV) according to treatment schedule A.	CMP-001 IT + Pembrolizumab - Schedule B n=6 Participants Participants received CMP-001 IT and pembrolizumab IV according to treatment schedule B.
Duration of PFS Based on HPV Status HPV positive	3.253 Months Interval 0.72 to 22.44	3.975 Months Interval 3.975 to 3.975
Duration of PFS Based on HPV Status HPV negative	1.183 Months Interval 0.03 to 6.9	1.807 Months Interval 1.48 to 3.45

SECONDARY outcome

Timeframe: Up to approximately 28 months (120 weeks)

Population: Number analyzed = Number of participants who had iBOR of immune complete response (iCR) or immune partial response (iPR). Here, 0 participants met the criteria.

iDOR, defined as the time from the date of the first immune response (iCR or iPR) to the date of immune confirmed progressive disease (iCPD) by IA.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to approximately 28 months (120 weeks)

Population: Number of participants analyzed for DOR based on HPV status includes only participants who have a confirmed best overall response (BOR) of complete response (CR) or partial response (PR) to date of documented PD and completed HPV testing.

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.

Outcome measures

Outcome measures
Measure	CMP-001 IT + Pembrolizumab - Schedule A n=2 Participants Participants received CMP-001 intratumorally (IT) and pembrolizumab intravenously (IV) according to treatment schedule A.	CMP-001 IT + Pembrolizumab - Schedule B Participants received CMP-001 IT and pembrolizumab IV according to treatment schedule B.
DOR Based on HPV Status HPV negative	NA Months Data not available due to insufficient number of participants with events.	—
DOR Based on HPV Status HPV positive	NA Months Data not available due to insufficient number of participants with events.	—

SECONDARY outcome

Timeframe: Up to approximately 28 months (120 weeks)

Population: Number of participants analyzed for DOR based on PD-L1 expressions included only participants with confirmed BOR of CR or PR and CPS \>=20. Here, 0 participants met the criteria.

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 PD-L1 expressions (combined positive score \[CPS\] 20).

Outcome measures

Outcome measures
Measure	CMP-001 IT + Pembrolizumab - Schedule A n=1 Participants Participants received CMP-001 intratumorally (IT) and pembrolizumab intravenously (IV) according to treatment schedule A.	CMP-001 IT + Pembrolizumab - Schedule B Participants received CMP-001 IT and pembrolizumab IV according to treatment schedule B.
DOR Based on PD-L1 Expressions	NA Months Data not available due to insufficient number of participants with events.	—

SECONDARY outcome

Timeframe: Up to approximately 28 months (120 weeks)

Population: Number of participants analyzed for PFS based on PD-L1 expressions included only participants with documented PD or death and CPS \>=20. Here, 0 participants met the criteria.

Outcome measures

Outcome data not reported

Adverse Events

CMP-001 IT + Pembrolizumab - Schedule A

Serious events: 7 serious events

Other events: 17 other events

Deaths: 11 deaths

CMP-001 IT + Pembrolizumab - Schedule B

Serious events: 5 serious events

Other events: 6 other events

Deaths: 7 deaths

Serious adverse events

Other adverse events

Additional Information

Clinical Trials Administrator

Regeneron Pharmaceuticals, Inc.

Phone: 844-734-6643

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee The investigator has the right to independently publish study results from the investigator's site after a multi-center publication, or a defined period after the completion of the study by all sites. The investigator must provide the Sponsor a copy of any such publication derived from the study for review and comment in advance of any submission, and delay publication, if requested, to allow the Sponsor to preserve its proprietary rights.
Publication restrictions are in place

Restriction type: OTHER

Measure	CMP-001 IT + Pembrolizumab - Schedule A n=17 participants at risk Participants received CMP-001 intratumorally (IT) and pembrolizumab intravenously (IV) according to treatment schedule A.	CMP-001 IT + Pembrolizumab - Schedule B n=7 participants at risk Participants received CMP-001 IT and pembrolizumab IV according to treatment schedule B.
Metabolism and nutrition disorders Hypercalcaemia	11.8% 2/17 • Number of events 2 • From signing of informed consent through end of study up to approximately 28 months (120 weeks)	0.00% 0/7 • From signing of informed consent through end of study up to approximately 28 months (120 weeks)
Metabolism and nutrition disorders Hypernatraemia	5.9% 1/17 • Number of events 1 • From signing of informed consent through end of study up to approximately 28 months (120 weeks)	0.00% 0/7 • From signing of informed consent through end of study up to approximately 28 months (120 weeks)
Immune system disorders Anaphylactic reaction	5.9% 1/17 • Number of events 1 • From signing of informed consent through end of study up to approximately 28 months (120 weeks)	0.00% 0/7 • From signing of informed consent through end of study up to approximately 28 months (120 weeks)
Immune system disorders Cytokine release syndrome	5.9% 1/17 • Number of events 1 • From signing of informed consent through end of study up to approximately 28 months (120 weeks)	0.00% 0/7 • From signing of informed consent through end of study up to approximately 28 months (120 weeks)
Gastrointestinal disorders Gastric haemorrhage	5.9% 1/17 • Number of events 1 • From signing of informed consent through end of study up to approximately 28 months (120 weeks)	0.00% 0/7 • From signing of informed consent through end of study up to approximately 28 months (120 weeks)
General disorders Multiple organ dysfunction syndrome	5.9% 1/17 • Number of events 1 • From signing of informed consent through end of study up to approximately 28 months (120 weeks)	0.00% 0/7 • From signing of informed consent through end of study up to approximately 28 months (120 weeks)
Infections and infestations COVID-19	5.9% 1/17 • Number of events 1 • From signing of informed consent through end of study up to approximately 28 months (120 weeks)	0.00% 0/7 • From signing of informed consent through end of study up to approximately 28 months (120 weeks)
Infections and infestations Bacterial sepsis	0.00% 0/17 • From signing of informed consent through end of study up to approximately 28 months (120 weeks)	14.3% 1/7 • Number of events 1 • From signing of informed consent through end of study up to approximately 28 months (120 weeks)
Infections and infestations Pneumonia	0.00% 0/17 • From signing of informed consent through end of study up to approximately 28 months (120 weeks)	14.3% 1/7 • Number of events 1 • From signing of informed consent through end of study up to approximately 28 months (120 weeks)
Infections and infestations Sepsis	0.00% 0/17 • From signing of informed consent through end of study up to approximately 28 months (120 weeks)	28.6% 2/7 • Number of events 2 • From signing of informed consent through end of study up to approximately 28 months (120 weeks)
Respiratory, thoracic and mediastinal disorders Laryngeal oedema	5.9% 1/17 • Number of events 1 • From signing of informed consent through end of study up to approximately 28 months (120 weeks)	0.00% 0/7 • From signing of informed consent through end of study up to approximately 28 months (120 weeks)
Respiratory, thoracic and mediastinal disorders Pulmonary embolism	5.9% 1/17 • Number of events 1 • From signing of informed consent through end of study up to approximately 28 months (120 weeks)	0.00% 0/7 • From signing of informed consent through end of study up to approximately 28 months (120 weeks)
Respiratory, thoracic and mediastinal disorders Haemoptysis	0.00% 0/17 • From signing of informed consent through end of study up to approximately 28 months (120 weeks)	14.3% 1/7 • Number of events 1 • From signing of informed consent through end of study up to approximately 28 months (120 weeks)
Vascular disorders Hypotension	5.9% 1/17 • Number of events 1 • From signing of informed consent through end of study up to approximately 28 months (120 weeks)	0.00% 0/7 • From signing of informed consent through end of study up to approximately 28 months (120 weeks)
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Tumour haemorrhage	0.00% 0/17 • From signing of informed consent through end of study up to approximately 28 months (120 weeks)	14.3% 1/7 • Number of events 2 • From signing of informed consent through end of study up to approximately 28 months (120 weeks)
Nervous system disorders Syncope	0.00% 0/17 • From signing of informed consent through end of study up to approximately 28 months (120 weeks)	14.3% 1/7 • Number of events 1 • From signing of informed consent through end of study up to approximately 28 months (120 weeks)