PD-1 in Patients With Advanced Basal Cell Carcinoma Who Experienced Progression of Disease on Hedgehog Pathway Inhibitor Therapy, or Were Intolerant of Prior Hedgehog Pathway Inhibitor Therapy

• ClinicalTrials.gov Identifier: NCT03132636
• Recruitment Status: Completed
• First Posted: April 28, 2017
• Results First Posted: July 26, 2022
• Last Update Posted: June 18, 2023
• Sponsor: Regeneron Pharmaceuticals
• Collaborator: Sanofi
• Information provided by (Responsible Party): Regeneron Pharmaceuticals

• Study Type: Interventional
• Study Design: Allocation: Non-Randomized; Intervention Model: Parallel Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Carcinoma, Basal Cell
• Intervention: Drug: cemiplimab
• Enrollment: 138

Participant Flow

Recruitment Details

Pre-assignment Details

138 of the 170 screened participants, were enrolled & treated. Eligible participants were enrolled into 2 groups. Group 1: participants with metastatic Basal Cell Carcinoma (mBCC). Group 2: participants with unresectable locally advanced BCC (laBCC) who experienced progression of disease on Hedgehog inhibitor (HHI) therapy, or response no better than stable disease for at least 9 months or were intolerant of prior HHI therapy. Results presented here based on primary analysis cut-off (20May2021).

Arm/Group Title	Group 1: Metastatic BCC (mBCC)	Group 2: Unresectable Locally Advanced BCC (laBCC)
Arm/Group Description	Participants with metastatic BCC received IV infusion of cemiplimab at a dose of 350 mg Q3W for up to 93 weeks of treatment cycles (cycles 1-5 [each cycle of 9 weeks] followed by cycles 6-9 [each cycle of 12 weeks]) or until PD, unacceptable toxicity, withdrawal of consent, or CR.	Participants with unresectable laBCC received IV infusion of cemiplimab at a dose of 350 mg Q3W for up to 93 weeks of treatment cycles (cycles 1-5 [each cycle of 9 weeks] followed by cycles 6-9 [each cycle of 12 weeks]) or until PD, unacceptable toxicity, withdrawal of consent, or confirmed CR.
Period Title: Overall Study
Started	54	84
Completed	1	9
Not Completed	53	75
Reason Not Completed
Adverse Event	1	2
Death	4	7
Lost to Follow-up	2	2
Protocol Violation	1	1
Participant Decision	1	9
Sponsor Decision	0	1
Progressive disease	32	36
Withdrawal of consent	2	5
Study ongoing	9	10
Other	1	2

Baseline Characteristics

Arm/Group Title		Group 1: Metastatic BCC (mBCC)	Group 2: Unresectable Locally Advanced BCC (laBCC)	Total
Arm/Group Description		Participants with metastatic BCC received IV infusion of cemiplimab at a dose of 350 mg Q3W for up to 93 weeks of treatment cycles (cycles 1-5 [each cycle of 9 weeks] followed by cycles 6-9 [each cycle of 12 weeks]) or until PD, unacceptable toxicity, withdrawal of consent, or CR.	Participants with unresectable laBCC received IV infusion of cemiplimab at a dose of 350 mg Q3W for up to 93 weeks of treatment cycles (cycles 1-5 [each cycle of 9 weeks] followed by cycles 6-9 [each cycle of 12 weeks]) or until PD, unacceptable toxicity, withdrawal of consent, or confirmed CR.	Total of all reporting groups
Overall Number of Baseline Participants		54	84	138
Baseline Analysis Population Description		The full analysis set (FAS) included all enrolled participants for each group who passed screening and were deemed to be eligible for this study.
Age, Continuous; Mean (Standard Deviation); Unit of measure: Years
	Number Analyzed	54 participants	84 participants	138 participants
		63.8 (11.09)	69.1 (12.84)	67.0 (12.42)
Sex: Female, Male; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	54 participants	84 participants	138 participants
	Female	16 29.6%	28 33.3%	44 31.9%
	Male	38 70.4%	56 66.7%	94 68.1%
Race/Ethnicity, Customized; Measure Type: Number; Unit of measure: Participants	Number Analyzed	54 participants	84 participants	138 participants
Race : White		47	57	104
Race : Not Reported		1	0	1
Race : Missing		6	27	33
Race/Ethnicity, Customized; Measure Type: Number; Unit of measure: Participants	Number Analyzed	54 participants	84 participants	138 participants
Ethnicity : Not Hispanic or Latino		46	56	102
Ethnicity : Hispanic or Latino		2	1	3
Ethnicity : Missing		6	27	33

Outcome Measures

1. Primary Outcome

Title	Objective Response Rates (ORR) as Assessed by Independent Central Review (ICR)
Description	ORR was defined as percentage of participants with best overall response of complete response (CR) or partial response (PR) according to RECIST v1.1 assessed as per ICR assessment. CR: Disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to less than (<) 10 millimeter (mm) (< 1 centimeter [cm]). PR: At least a 30 percent (%) decrease in the sum of the diameters of target lesions, taking as reference the baseline sum diameters. ORR was determined by Clopper-Pearson method.
Time Frame	Up to 1422 days

Outcome Measure Data

Analysis Population Description

The FAS included all enrolled participants for each group who passed screening and were deemed to be eligible for this study.

Arm/Group Title	Group 1: Metastatic BCC (mBCC)	Group 2: Unresectable Locally Advanced BCC (laBCC)
Arm/Group Description:	Participants with metastatic BCC received IV infusion of cemiplimab at a dose of 350 mg Q3W for up to 93 weeks of treatment cycles (cycles 1-5 [each cycle of 9 weeks] followed by cycles 6-9 [each cycle of 12 weeks]) or until PD, unacceptable toxicity, withdrawal of consent, or CR.	Participants with unresectable laBCC received IV infusion of cemiplimab at a dose of 350 mg Q3W for up to 93 weeks of treatment cycles (cycles 1-5 [each cycle of 9 weeks] followed by cycles 6-9 [each cycle of 12 weeks]) or until PD, unacceptable toxicity, withdrawal of consent, or confirmed CR.
Overall Number of Participants Analyzed	54	84
Measure Type: Number; Number (95% Confidence Interval); Unit of Measure: Percentage of Participants
	24.1; (13.5 to 37.6)	32.1; (22.4 to 43.2)

2. Secondary Outcome

Title	Duration of Response (DOR) Per ICR
Description	DOR per ICR was determined for participants with best overall response of CR or PR. DOR was measured from the time measurement criteria are first met for CR/PR (whichever was first recorded) until the first date of recurrent or progressive disease (PD) (photographic or radiographic), or death due to any cause. CR: Disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to <10 mm (<1 cm). PR: At least a 30% decrease in the sum of the diameters of target lesions, taking as reference the baseline sum diameters. PD: At least a 20% increase in the sum of the diameters of target lesions, taking as reference the smallest sum on study (this includes the baseline sum if that is the smallest on study). DOR was determined by Kaplan-Meier estimate.
Time Frame	Up to 40 months

Outcome Measure Data

Analysis Population Description

The FAS included all enrolled participants for each group who passed screening and were deemed to be eligible for this study. Here, "Overall number of participants analyzed" signifies those participants who were evaluable for this outcome measure.

Arm/Group Title	Group 1: Metastatic BCC (mBCC)	Group 2: Unresectable Locally Advanced BCC (laBCC)
Arm/Group Description:	Participants with metastatic BCC received IV infusion of cemiplimab at a dose of 350 mg Q3W for up to 93 weeks of treatment cycles (cycles 1-5 [each cycle of 9 weeks] followed by cycles 6-9 [each cycle of 12 weeks]) or until PD, unacceptable toxicity, withdrawal of consent, or CR.	Participants with unresectable laBCC received IV infusion of cemiplimab at a dose of 350 mg Q3W for up to 93 weeks of treatment cycles (cycles 1-5 [each cycle of 9 weeks] followed by cycles 6-9 [each cycle of 12 weeks]) or until PD, unacceptable toxicity, withdrawal of consent, or confirmed CR.
Overall Number of Participants Analyzed	13	27
Median (95% Confidence Interval); Unit of Measure: Months
	16.7 [1]; (9.8 to NA)	NA [1]; (15.5 to NA)

[1]

mBCC: The upper limit of the 95% confidence interval could not be estimated because 53.8% of patients were censored. laBCC: Because 70.4% of patients were censored and there was a greater than 50% chance of surviving at follow-up, the median survival and upper bounds of the 95% confidence interval could not be estimated.

3. Secondary Outcome

Title	Duration of Response (DOR) Per Investigator Assessment
Description	DOR per investigator assessment was determined for participants with best overall response of CR or PR. DOR was measured from the time measurement criteria are first met for CR/PR (whichever was first recorded) until the first date of recurrent or progressive disease (PD) (photographic or radiographic), or death due to any cause. CR: Disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to <10 mm (<1 cm). PR: At least a 30% decrease in the sum of the diameters of target lesions, taking as reference the baseline sum diameters. PD: At least a 20% increase in the sum of the diameters of target lesions, taking as reference the smallest sum on study (this includes the baseline sum if that is the smallest on study). DOR was determined by Kaplan-Meier estimate.
Time Frame	Up to 40 months

Outcome Measure Data

Analysis Population Description

The FAS included all enrolled participants for each group who passed screening and were deemed to be eligible for this study. Here, "Overall number of participants analyzed" signifies those participants who were evaluable for this outcome measure.

Arm/Group Title	Group 1: Metastatic BCC (mBCC)	Group 2: Unresectable Locally Advanced BCC (laBCC)
Arm/Group Description:	Participants with metastatic BCC received IV infusion of cemiplimab at a dose of 350 mg Q3W for up to 93 weeks of treatment cycles (cycles 1-5 [each cycle of 9 weeks] followed by cycles 6-9 [each cycle of 12 weeks]) or until PD, unacceptable toxicity, withdrawal of consent, or CR.	Participants with unresectable laBCC received IV infusion of cemiplimab at a dose of 350 mg Q3W for up to 93 weeks of treatment cycles (cycles 1-5 [each cycle of 9 weeks] followed by cycles 6-9 [each cycle of 12 weeks]) or until PD, unacceptable toxicity, withdrawal of consent, or confirmed CR.
Overall Number of Participants Analyzed	14	31
Median (95% Confidence Interval); Unit of Measure: Months
	19.3 [1]; (9.8 to NA)	19.6 [1]; (16.7 to NA)

[1]

Data could not be estimated due to higher number (>50%) of censored participants.

4. Secondary Outcome

Title	Progression Free Survival (PFS) Determined by ICR
Description	PFS was defined as the time from start of treatment until the first date of recurrent or PD (photographic or radiographic), or death due to any cause, whichever occurred first, was determined by IRC. PD: At least a 20% increase in the sum of the diameters of target lesions, taking as reference the smallest sum on study (this includes the baseline sum if that is the smallest on study). PFS was determined by Kaplan-Meier estimate.
Time Frame	Up to 40 months

Outcome Measure Data

Analysis Population Description

The FAS included all enrolled participants for each group who passed screening and were deemed to be eligible for this study. Here, "Overall number of participants analyzed" signifies those participants who were evaluable for this outcome measure.

Arm/Group Title	Group 1: Metastatic BCC (mBCC)	Group 2: Unresectable Locally Advanced BCC (laBCC)
Arm/Group Description:	Participants with metastatic BCC received IV infusion of cemiplimab at a dose of 350 mg Q3W for up to 93 weeks of treatment cycles (cycles 1-5 [each cycle of 9 weeks] followed by cycles 6-9 [each cycle of 12 weeks]) or until PD, unacceptable toxicity, withdrawal of consent, or CR.	Participants with unresectable laBCC received IV infusion of cemiplimab at a dose of 350 mg Q3W for up to 93 weeks of treatment cycles (cycles 1-5 [each cycle of 9 weeks] followed by cycles 6-9 [each cycle of 12 weeks]) or until PD, unacceptable toxicity, withdrawal of consent, or confirmed CR.
Overall Number of Participants Analyzed	34	46
Median (95% Confidence Interval); Unit of Measure: Months
	8.3; (4.2 to 15.9)	16.5; (8.6 to 21.4)

5. Secondary Outcome

Title	Progression Free Survival (PFS) Determined by Investigator Assessment
Description	PFS was defined as the time from start of treatment until the first date of recurrent or PD (photographic or radiographic), or death due to any cause, whichever occurred first, was determined by IRC. PD: At least a 20% increase in the sum of the diameters of target lesions, taking as reference the smallest sum on study (this includes the baseline sum if that is the smallest on study). PFS was determined by Kaplan-Meier estimate.
Time Frame	Up to 40 months

Outcome Measure Data

Analysis Population Description

The FAS included all enrolled participants for each group who passed screening and were deemed to be eligible for this study. Here, "Overall number of participants analyzed" signifies those participants who were evaluable for this outcome measure.

Arm/Group Title	Group 1: Metastatic BCC (mBCC)	Group 2: Unresectable Locally Advanced BCC (laBCC)
Arm/Group Description:	Participants with metastatic BCC received IV infusion of cemiplimab at a dose of 350 mg Q3W for up to 93 weeks of treatment cycles (cycles 1-5 [each cycle of 9 weeks] followed by cycles 6-9 [each cycle of 12 weeks]) or until PD, unacceptable toxicity, withdrawal of consent, or CR.	Participants with unresectable laBCC received IV infusion of cemiplimab at a dose of 350 mg Q3W for up to 93 weeks of treatment cycles (cycles 1-5 [each cycle of 9 weeks] followed by cycles 6-9 [each cycle of 12 weeks]) or until PD, unacceptable toxicity, withdrawal of consent, or confirmed CR.
Overall Number of Participants Analyzed	41	52
Median (95% Confidence Interval); Unit of Measure: Months
	6.6; (4.2 to 8.3)	17.1; (10.3 to 19.8)

6. Secondary Outcome

Title	Overall Survival (OS)
Description	OS was measured as time from the start of treatment until death due to any cause. Participants who do not die was censored at the last date that participant was documented to be alive. OS was calculated based on Kaplan-Meier method.
Time Frame	Up to 40 months

Outcome Measure Data

Analysis Population Description

The FAS included all enrolled participants for each group who passed screening and were deemed to be eligible for this study.

Arm/Group Title	Group 1: Metastatic BCC (mBCC)	Group 2: Unresectable Locally Advanced BCC (laBCC)
Arm/Group Description:	Participants with metastatic BCC received IV infusion of cemiplimab at a dose of 350 mg Q3W for up to 93 weeks of treatment cycles (cycles 1-5 [each cycle of 9 weeks] followed by cycles 6-9 [each cycle of 12 weeks]) or until PD, unacceptable toxicity, withdrawal of consent, or CR.	Participants with unresectable laBCC received IV infusion of cemiplimab at a dose of 350 mg Q3W for up to 93 weeks of treatment cycles (cycles 1-5 [each cycle of 9 weeks] followed by cycles 6-9 [each cycle of 12 weeks]) or until PD, unacceptable toxicity, withdrawal of consent, or confirmed CR.
Overall Number of Participants Analyzed	54	84
Median (95% Confidence Interval); Unit of Measure: Months
	NA [1]; (25.7 to NA)	NA [1]; (NA to NA)

[1]

mBCC: Because 70.4% of patients were censored and there was a greater than 50% chance of surviving at follow-up, the median overall survival and upper bound of the 95% confidence interval could not be estimated. laBCC: Because 79.8% of patients were censored and there was a greater than 50% chance of surviving at follow-up, the median overall survival and lower and upper bounds of the 95% confidence interval could not be estimated.

7. Secondary Outcome

Title	Percentage of Participants With Complete Response (CR) Rate Assessed by ICR
Description	CR rate by ICR was determined by the percentage of participants with best overall response of CR. CR: Disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to <10 mm (<1 cm).
Time Frame	Up to 1422 days

Outcome Measure Data

Analysis Population Description

The FAS included all enrolled participants for each group who passed screening and were deemed to be eligible for this study.

Arm/Group Title	Group 1: Metastatic BCC (mBCC)	Group 2: Unresectable Locally Advanced BCC (laBCC)
Arm/Group Description:	Participants with metastatic BCC received IV infusion of cemiplimab at a dose of 350 mg Q3W for up to 93 weeks of treatment cycles (cycles 1-5 [each cycle of 9 weeks] followed by cycles 6-9 [each cycle of 12 weeks]) or until PD, unacceptable toxicity, withdrawal of consent, or CR.	Participants with unresectable laBCC received IV infusion of cemiplimab at a dose of 350 mg Q3W for up to 93 weeks of treatment cycles (cycles 1-5 [each cycle of 9 weeks] followed by cycles 6-9 [each cycle of 12 weeks]) or until PD, unacceptable toxicity, withdrawal of consent, or confirmed CR.
Overall Number of Participants Analyzed	54	84
Measure Type: Number; Number (95% Confidence Interval); Unit of Measure: Percentage of Participants
	1.9; (0.0 to 9.9)	7.1; (2.7 to 14.9)

8. Secondary Outcome

Title	Change From Baseline of Patient-reported Outcomes in European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30)
Description	The EORTC QLQ-C30 is a 30-item questionnaire used to assess the overall QoL in cancer participants. It consists of 15 domains: 1 Global Health Status (GHS)/QoL scale, 5 functional scales (Physical, role, cognitive, emotional, social), 9 symptom scales/items (Fatigue, nausea and vomiting, pain, dyspnea, sleep disturbance, appetite loss, constipation, diarrhea, financial impact). Most items are scored 1 ("not at all") to 4 ("very much") except for the items contributing to the GHS/QoL, which are scored 1 ("very poor") to 7 ("excellent"). A linear transformation was applied to the raw scores so that all transformed scores lie between 0 to 100. For the GHS/QoL and 5 functional scales a higher score indicates higher ("better") quality of life/functioning and a positive change from baseline indicates improvement. For the symptom scales/items, a higher score indicates a higher ("worse") level of symptoms/problems, and a negative change from baseline indicates improvement.
Time Frame	Baseline (Day 1 of Cycle 1); Day 1 of Cycles 2 to 9 (Cycles 1-5 [Each cycle of 9 weeks], Cycles 6 to 9 [Each cycle of 12 weeks])

Outcome Measure Data

Analysis Population Description

The FAS included all enrolled participants for each group who passed screening and were deemed to be eligible for this study. Here, "Overall number of participants analyzed" signifies those participants who were evaluable for this outcome measure and "Number analyzed" signifies those participants who were evaluable at specific time points.

Arm/Group Title		Group 1: Metastatic BCC (mBCC)	Group 2: Unresectable Locally Advanced BCC (laBCC)
Arm/Group Description:		Participants with metastatic BCC received IV infusion of cemiplimab at a dose of 350 mg Q3W for up to 93 weeks of treatment cycles (cycles 1-5 [each cycle of 9 weeks] followed by cycles 6-9 [each cycle of 12 weeks]) or until PD, unacceptable toxicity, withdrawal of consent, or CR.	Participants with unresectable laBCC received IV infusion of cemiplimab at a dose of 350 mg Q3W for up to 93 weeks of treatment cycles (cycles 1-5 [each cycle of 9 weeks] followed by cycles 6-9 [each cycle of 12 weeks]) or until PD, unacceptable toxicity, withdrawal of consent, or confirmed CR.
Overall Number of Participants Analyzed		43	75
Mean (Standard Deviation); Unit of Measure: Score on a Scale
Physical Functioning: Change at Cycle 2 Day 1	Number Analyzed	43 participants	75 participants
		-4.88 (14.274)	-1.55 (12.101)
Physical Functioning: Change at Cycle 3 Day 1	Number Analyzed	29 participants	65 participants
		-1.78 (13.481)	-0.56 (15.947)
Physical Functioning: Change at Cycle 4 Day 1	Number Analyzed	26 participants	55 participants
		-6.60 (12.828)	-3.79 (17.814)
Physical Functioning: Change at Cycle 5 Day 1	Number Analyzed	20 participants	50 participants
		1.08 (10.033)	-2.86 (17.063)
Physical Functioning: Change at Cycle 6 Day 1	Number Analyzed	19 participants	40 participants
		-1.67 (9.734)	0.33 (12.073)
Physical Functioning: Change at Cycle 7 Day 1	Number Analyzed	13 participants	33 participants
		-0.38 (11.142)	-0.20 (15.410)
Physical Functioning: Change at Cycle 8 Day 1	Number Analyzed	8 participants	33 participants
		-3.33 (12.344)	-3.03 (13.549)
Physical Functioning: Change at Cycle 9 Day 1	Number Analyzed	10 participants	29 participants
		-1.83 (9.110)	-5.06 (20.444)
Role Functioning: Change at Cycle 2 Day 1	Number Analyzed	43 participants	75 participants
		-2.71 (27.442)	-3.11 (20.264)
Role Functioning: Change at Cycle 3 Day 1	Number Analyzed	29 participants	65 participants
		5.17 (27.854)	-4.87 (25.297)
Role Functioning: Change at Cycle 4 Day 1	Number Analyzed	26 participants	55 participants
		-3.21 (25.394)	-6.06 (26.326)
Role Functioning: Change at Cycle 5 Day 1	Number Analyzed	20 participants	50 participants
		5.00 (22.361)	-5.33 (26.393)
Role Functioning: Change at Cycle 6 Day 1	Number Analyzed	19 participants	40 participants
		6.14 (21.667)	-3.33 (16.963)
Role Functioning: Change at Cycle 7 Day 1	Number Analyzed	13 participants	33 participants
		12.82 (24.677)	-7.07 (16.682)
Role Functioning: Change at Cycle 8 Day 1	Number Analyzed	8 participants	33 participants
		10.42 (19.796)	-4.04 (19.557)
Role Functioning: Change at Cycle 9 Day 1	Number Analyzed	10 participants	29 participants
		10.00 (23.831)	-9.77 (30.052)
Emotional Functioning: Change at Cycle 2 Day 1	Number Analyzed	43 participants	74 participants
		3.17 (19.638)	1.95 (20.483)
Emotional Functioning: Change at Cycle 3 Day 1	Number Analyzed	29 participants	64 participants
		1.15 (18.730)	1.56 (18.538)
Emotional Functioning: Change at Cycle 4 Day 1	Number Analyzed	26 participants	54 participants
		3.53 (22.007)	1.39 (21.277)
Emotional Functioning: Change at Cycle 5 Day 1	Number Analyzed	20 participants	49 participants
		5.83 (24.046)	-4.59 (19.622)
Emotional Functioning: Change at Cycle 6 Day 1	Number Analyzed	19 participants	40 participants
		7.02 (20.650)	1.04 (19.809)
Emotional Functioning: Change at Cycle 7 Day 1	Number Analyzed	13 participants	33 participants
		3.85 (18.199)	-4.63 (19.576)
Emotional Functioning: Change at Cycle 8 Day 1	Number Analyzed	9 participants	33 participants
		20.37 (26.389)	3.11 (19.810)
Emotional Functioning: Change at Cycle 9 Day 1	Number Analyzed	10 participants	29 participants
		5.83 (18.023)	2.11 (17.246)
Cognitive Functioning: Change at Cycle 2 Day 1	Number Analyzed	43 participants	74 participants
		0.78 (13.586)	-2.48 (26.985)
Cognitive Functioning: Change at Cycle 3 Day 1	Number Analyzed	29 participants	64 participants
		-1.72 (16.871)	-4.17 (23.382)
Cognitive Functioning: Change at Cycle 4 Day 1	Number Analyzed	26 participants	54 participants
		-0.64 (15.261)	-4.94 (21.385)
Cognitive Functioning: Change at Cycle 5 Day 1	Number Analyzed	20 participants	49 participants
		-4.17 (14.178)	-6.46 (25.188)
Cognitive Functioning: Change at Cycle 6 Day 1	Number Analyzed	19 participants	40 participants
		-0.88 (12.998)	-1.25 (19.017)
Cognitive Functioning: Change at Cycle 7 Day 1	Number Analyzed	13 participants	33 participants
		-2.56 (11.479)	-5.56 (18.942)
Cognitive Functioning: Change at Cycle 8 Day 1	Number Analyzed	9 participants	33 participants
		-7.41 (12.108)	1.52 (17.362)
Cognitive Functioning: Change at Cycle 9 Day 1	Number Analyzed	10 participants	29 participants
		-11.67 (26.117)	-2.30 (23.873)
Social Functioning: Change at Cycle 2 Day 1	Number Analyzed	43 participants	74 participants
		0.39 (19.412)	4.50 (21.422)
Social Functioning: Change at Cycle 3 Day 1	Number Analyzed	29 participants	64 participants
		3.45 (16.891)	0.78 (21.088)
Social Functioning: Change at Cycle 4 Day 1	Number Analyzed	26 participants	54 participants
		3.85 (24.179)	1.85 (23.272)
Social Functioning: Change at Cycle 5 Day 1	Number Analyzed	20 participants	49 participants
		3.33 (22.685)	0.34 (23.445)
Social Functioning: Change at Cycle 6 Day 1	Number Analyzed	19 participants	40 participants
		10.53 (16.860)	0.00 (24.749)
Social Functioning: Change at Cycle 7 Day 1	Number Analyzed	13 participants	33 participants
		12.82 (22.724)	0.00 (18.162)
Social Functioning: Change at Cycle 8 Day 1	Number Analyzed	9 participants	33 participants
		12.96 (16.197)	2.02 (23.848)
Social Functioning: Change at Cycle 9 Day 1	Number Analyzed	10 participants	29 participants
		10.00 (14.055)	-2.30 (27.359)
Fatigue: Change at Cycle 2 Day 1	Number Analyzed	43 participants	75 participants
		5.17 (22.919)	6.44 (24.542)
Fatigue: Change at Cycle 3 Day 1	Number Analyzed	29 participants	65 participants
		1.92 (18.322)	6.58 (24.901)
Fatigue: Change at Cycle 4 Day 1	Number Analyzed	26 participants	55 participants
		-2.56 (21.154)	7.58 (25.912)
Fatigue: Change at Cycle 5 Day 1	Number Analyzed	20 participants	50 participants
		-5.56 (24.048)	9.67 (23.404)
Fatigue: Change at Cycle 6 Day 1	Number Analyzed	19 participants	40 participants
		-5.85 (19.376)	7.78 (17.649)
Fatigue: Change at Cycle 7 Day 1	Number Analyzed	13 participants	33 participants
		-1.71 (19.692)	14.14 (20.838)
Fatigue: Change at Cycle 8 Day 1	Number Analyzed	8 participants	33 participants
		-12.50 (22.567)	9.09 (19.534)
Fatigue: Change at Cycle 9 Day 1	Number Analyzed	10 participants	29 participants
		-1.11 (16.932)	12.64 (20.080)
Nausea/Vomiting: Change at Cycle 2 Day 1	Number Analyzed	43 participants	75 participants
		-0.78 (8.095)	0.22 (12.703)
Nausea/Vomiting: Change at Cycle 3 Day 1	Number Analyzed	29 participants	65 participants
		0.00 (13.363)	-1.79 (12.884)
Nausea/Vomiting: Change at Cycle 4 Day 1	Number Analyzed	26 participants	55 participants
		0.00 (16.330)	0.30 (13.414)
Nausea/Vomiting: Change at Cycle 5 Day 1	Number Analyzed	20 participants	50 participants
		0.83 (10.080)	1.00 (13.640)
Nausea/Vomiting: Change at Cycle 6 Day 1	Number Analyzed	19 participants	40 participants
		-0.88 (3.824)	1.67 (13.503)
Nausea/Vomiting: Change at Cycle 7 Day 1	Number Analyzed	13 participants	33 participants
		-1.28 (14.372)	3.03 (13.472)
Nausea/Vomiting: Change at Cycle 8 Day 1	Number Analyzed	8 participants	33 participants
		0.00 (0.000)	0.51 (12.832)
Nausea/Vomiting: Change at Cycle 9 Day 1	Number Analyzed	10 participants	29 participants
		0.00 (0.000)	-2.30 (13.890)
Pain: Change at Cycle 2 Day 1	Number Analyzed	43 participants	75 participants
		-2.33 (30.338)	-0.22 (26.351)
Pain: Change at Cycle 3 Day 1	Number Analyzed	29 participants	65 participants
		-9.77 (22.056)	-1.54 (30.151)
Pain: Change at Cycle 4 Day 1	Number Analyzed	26 participants	55 participants
		0.00 (29.439)	-4.85 (25.795)
Pain: Change at Cycle 5 Day 1	Number Analyzed	20 participants	50 participants
		-4.17 (25.291)	-4.00 (25.098)
Pain: Change at Cycle 6 Day 1	Number Analyzed	19 participants	40 participants
		-14.04 (29.535)	-2.92 (24.427)
Pain: Change at Cycle 7 Day 1	Number Analyzed	13 participants	33 participants
		-21.79 (29.174)	-4.04 (24.661)
Pain: Change at Cycle 8 Day 1	Number Analyzed	9 participants	33 participants
		-14.81 (28.191)	-7.58 (25.716)
Pain: Change at Cycle 9 Day 1	Number Analyzed	10 participants	29 participants
		-21.67 (28.382)	-5.17 (24.030)
Dyspnoea: Change at Cycle 2 Day 1	Number Analyzed	42 participants	75 participants
		2.38 (17.097)	1.33 (24.162)
Dyspnoea: Change at Cycle 3 Day 1	Number Analyzed	28 participants	65 participants
		3.57 (16.578)	-0.51 (23.930)
Dyspnoea: Change at Cycle 4 Day 1	Number Analyzed	25 participants	55 participants
		0.00 (16.667)	2.42 (24.724)
Dyspnoea: Change at Cycle 5 Day 1	Number Analyzed	19 participants	49 participants
		3.51 (21.928)	0.00 (28.054)
Dyspnoea: Change at Cycle 6 Day 1	Number Analyzed	18 participants	40 participants
		1.85 (13.873)	-1.67 (19.900)
Dyspnoea: Change at Cycle 7 Day 1	Number Analyzed	12 participants	33 participants
		2.78 (30.011)	2.02 (21.952)
Dyspnoea: Change at Cycle 8 Day 1	Number Analyzed	8 participants	33 participants
		8.33 (23.570)	2.02 (21.952)
Dyspnoea: Change at Cycle 9 Day 1	Number Analyzed	9 participants	29 participants
		7.41 (27.778)	2.30 (17.663)
Insomnia: Change at Cycle 2 Day 1	Number Analyzed	43 participants	75 participants
		-2.33 (26.622)	0.44 (24.195)
Insomnia: Change at Cycle 3 Day 1	Number Analyzed	29 participants	65 participants
		-6.90 (24.200)	-0.51 (26.016)
Insomnia: Change at Cycle 4 Day 1	Number Analyzed	26 participants	55 participants
		-10.26 (29.468)	-1.82 (19.687)
Insomnia: Change at Cycle 5 Day 1	Number Analyzed	20 participants	50 participants
		-15.00 (31.484)	1.33 (30.087)
Insomnia: Change at Cycle 6 Day 1	Number Analyzed	19 participants	40 participants
		-17.54 (32.142)	-4.17 (24.093)
Insomnia: Change at Cycle 7 Day 1	Number Analyzed	13 participants	33 participants
		-7.69 (41.172)	1.01 (28.241)
Insomnia: Change at Cycle 8 Day 1	Number Analyzed	8 participants	33 participants
		-25.00 (52.705)	3.03 (25.500)
Insomnia: Change at Cycle 9 Day 1	Number Analyzed	10 participants	29 participants
		-6.67 (40.976)	5.75 (25.306)
Appetite loss: Change at Cycle 2 Day 1	Number Analyzed	43 participants	74 participants
		4.65 (26.807)	-2.25 (27.215)
Appetite loss: Change at Cycle 3 Day 1	Number Analyzed	29 participants	62 participants
		-2.30 (15.252)	-4.30 (22.163)
Appetite loss: Change at Cycle 4 Day 1	Number Analyzed	26 participants	54 participants
		-1.28 (30.523)	-3.09 (26.117)
Appetite loss: Change at Cycle 5 Day 1	Number Analyzed	20 participants	49 participants
		3.33 (21.357)	-1.36 (30.398)
Appetite loss: Change at Cycle 6 Day 1	Number Analyzed	19 participants	39 participants
		-1.75 (17.476)	-2.56 (29.004)
Appetite loss: Change at Cycle 7 Day 1	Number Analyzed	13 participants	33 participants
		-5.13 (12.518)	-1.01 (30.601)
Appetite loss: Change at Cycle 8 Day 1	Number Analyzed	8 participants	32 participants
		-4.17 (11.785)	-2.08 (31.609)
Appetite loss: Change at Cycle 9 Day 1	Number Analyzed	10 participants	28 participants
		-3.33 (18.922)	1.19 (30.741)
Constipation: Change at Cycle 2 Day 1	Number Analyzed	43 participants	74 participants
		1.55 (19.181)	2.25 (21.603)
Constipation: Change at Cycle 3 Day 1	Number Analyzed	29 participants	63 participants
		0.00 (21.822)	1.06 (20.712)
Constipation: Change at Cycle 4 Day 1	Number Analyzed	26 participants	54 participants
		-2.56 (18.674)	1.23 (21.440)
Constipation: Change at Cycle 5 Day 1	Number Analyzed	20 participants	49 participants
		5.00 (16.312)	-0.68 (22.037)
Constipation: Change at Cycle 6 Day 1	Number Analyzed	19 participants	40 participants
		0.00 (15.713)	-1.67 (18.413)
Constipation: Change at Cycle 7 Day 1	Number Analyzed	13 participants	33 participants
		5.13 (18.490)	4.04 (13.838)
Constipation: Change at Cycle 8 Day 1	Number Analyzed	8 participants	33 participants
		8.33 (15.430)	-2.02 (16.540)
Constipation: Change at Cycle 9 Day 1	Number Analyzed	10 participants	29 participants
		0.00 (15.713)	1.15 (22.683)
Diarrhea: Change at Cycle 2 Day 1	Number Analyzed	43 participants	74 participants
		3.88 (14.924)	0.45 (24.360)
Diarrhea: Change at Cycle 3 Day 1	Number Analyzed	29 participants	64 participants
		1.15 (14.037)	-1.04 (20.547)
Diarrhea: Change at Cycle 4 Day 1	Number Analyzed	26 participants	54 participants
		3.85 (23.715)	-1.85 (19.870)
Diarrhea: Change at Cycle 5 Day 1	Number Analyzed	20 participants	49 participants
		6.67 (20.520)	-0.68 (23.064)
Diarrhea: Change at Cycle 6 Day 1	Number Analyzed	19 participants	40 participants
		3.51 (18.904)	-1.67 (18.413)
Diarrhea: Change at Cycle 7 Day 1	Number Analyzed	13 participants	33 participants
		0.00 (13.608)	1.01 (19.516)
Diarrhea: Change at Cycle 8 Day 1	Number Analyzed	9 participants	33 participants
		3.70 (11.111)	-1.01 (19.516)
Diarrhea: Change at Cycle 9 Day 1	Number Analyzed	10 participants	29 participants
		3.33 (10.541)	-2.30 (21.696)
Financial Problems: Change at Cycle 2 Day 1	Number Analyzed	43 participants	74 participants
		-3.10 (17.539)	-3.15 (25.385)
Financial Problems: Change at Cycle 3 Day 1	Number Analyzed	28 participants	63 participants
		0.00 (18.144)	-4.23 (24.313)
Financial Problems: Change at Cycle 4 Day 1	Number Analyzed	26 participants	54 participants
		-5.13 (22.494)	-4.94 (23.710)
Financial Problems: Change at Cycle 5 Day 1	Number Analyzed	20 participants	49 participants
		0.00 (18.732)	-6.80 (22.546)
Financial Problems: Change at Cycle 6 Day 1	Number Analyzed	19 participants	40 participants
		-1.75 (23.501)	-5.83 (27.099)
Financial Problems: Change at Cycle 7 Day 1	Number Analyzed	13 participants	33 participants
		-2.56 (21.350)	1.01 (19.516)
Financial Problems: Change at Cycle 8 Day 1	Number Analyzed	9 participants	33 participants
		-3.70 (20.031)	1.01 (19.516)
Financial Problems: Change at Cycle 9 Day 1	Number Analyzed	10 participants	29 participants
		-10.00 (16.102)	-2.30 (23.454)
Global health status/QoL: Change at Cycle 2 Day 1	Number Analyzed	43 participants	72 participants
		-3.68 (21.845)	2.55 (15.298)
Global health status/QoL: Change at Cycle 3 Day 1	Number Analyzed	29 participants	62 participants
		3.74 (14.362)	-2.55 (19.823)
Global health status/QoL: Change at Cycle 4 Day 1	Number Analyzed	26 participants	51 participants
		-2.24 (14.636)	-0.49 (18.136)
Global health status/QoL: Change at Cycle 5 Day 1	Number Analyzed	20 participants	48 participants
		7.50 (14.023)	-1.91 (21.210)
Global health status/QoL: Change at Cycle 6 Day 1	Number Analyzed	19 participants	38 participants
		10.96 (11.802)	4.17 (19.447)
Global health status/QoL: Change at Cycle 7 Day 1	Number Analyzed	13 participants	32 participants
		16.03 (22.428)	-3.13 (19.715)
Global health status/QoL: Change at Cycle 8 Day 1	Number Analyzed	9 participants	31 participants
		7.41 (14.096)	2.15 (21.727)
Global health status/QoL: Change at Cycle 9 Day 1	Number Analyzed	10 participants	28 participants
		6.67 (20.713)	-7.14 (28.211)

9. Secondary Outcome

Title	Change From Baseline of Patient-reported Outcomes in Skindex-16 Questionnaire
Description	Skindex-16 questionnaire contains 16 questions related to quality of life in cancer participants. It consisted of a short 16-item assessment completed by the participant, with each item rated on a 7-point Likert scale (0=never bothered to 6=always bothered). Each raw score is multiplied by 16.667 to transform all responses to a linear scale from 0 (no effect) to 100 (effect experienced all the time). Responses to the Skindex-16 are categorized into 3 subscales: symptom, emotional & functional; their respective scores are expressed in a linear scale from 0 to 100. Symptoms scale score was an average of items 1 to 4 expressed in a linear scale from 0 to 100, Emotions scale score was an average of items 5 to 11 expressed in a linear scale from 0 to 100 and Functioning scale score was an average of items 12 to 16 expressed in a linear scale from 0 to 100. A negative change from baseline indicates an improvement in the participants condition compared to the baseline.
Time Frame	Baseline (Day 1 of Cycle 1); Day 1 of Cycles 2 to 9 (Cycles 1-5 [Each cycle of 9 weeks], Cycles 6 to 9 [Each cycle of 12 weeks])

Outcome Measure Data

Analysis Population Description

The FAS included all enrolled participants for each group who passed screening and were deemed to be eligible for this study. Here, "Overall number of participants analyzed" signifies those participants who were evaluable for this outcome measure and "Number analyzed" signifies those participants who were evaluable at specific time points.

Arm/Group Title		Group 1: Metastatic BCC (mBCC)	Group 2: Unresectable Locally Advanced BCC (laBCC)
Arm/Group Description:		Participants with metastatic BCC received IV infusion of cemiplimab at a dose of 350 mg Q3W for up to 93 weeks of treatment cycles (cycles 1-5 [each cycle of 9 weeks] followed by cycles 6-9 [each cycle of 12 weeks]) or until PD, unacceptable toxicity, withdrawal of consent, or CR.	Participants with unresectable laBCC received IV infusion of cemiplimab at a dose of 350 mg Q3W for up to 93 weeks of treatment cycles (cycles 1-5 [each cycle of 9 weeks] followed by cycles 6-9 [each cycle of 12 weeks]) or until PD, unacceptable toxicity, withdrawal of consent, or confirmed CR.
Overall Number of Participants Analyzed		42	71
Mean (Standard Deviation); Unit of Measure: Score on a Scale
Emotions: Change at Cycle 2 Day 1	Number Analyzed	42 participants	69 participants
		-6.90 (24.422)	-8.93 (27.767)
Emotions: Change at Cycle 3 Day 1	Number Analyzed	26 participants	63 participants
		-7.92 (18.033)	-8.60 (25.641)
Emotions: Change at Cycle 4 Day 1	Number Analyzed	22 participants	51 participants
		-3.97 (17.175)	-11.45 (24.215)
Emotions: Change at Cycle 5 Day 1	Number Analyzed	17 participants	46 participants
		-0.14 (12.835)	-10.25 (24.646)
Emotions: Change at Cycle 6 Day 1	Number Analyzed	16 participants	35 participants
		-9.08 (22.824)	-19.73 (27.304)
Emotions: Change at Cycle 7 Day 1	Number Analyzed	12 participants	30 participants
		6.55 (17.053)	-13.65 (27.132)
Emotions: Change at Cycle 8 Day 1	Number Analyzed	8 participants	30 participants
		2.38 (5.247)	-13.97 (25.001)
Emotions: Change at Cycle 9 Day 1	Number Analyzed	10 participants	28 participants
		8.17 (12.868)	-15.08 (31.843)
Symptoms: Change at Cycle 2 Day 1	Number Analyzed	42 participants	71 participants
		0.99 (18.788)	-1.31 (21.607)
Symptoms: Change at Cycle 3 Day 1	Number Analyzed	26 participants	64 participants
		3.85 (16.580)	-0.26 (24.158)
Symptoms: Change at Cycle 4 Day 1	Number Analyzed	22 participants	52 participants
		5.30 (18.464)	-6.62 (23.917)
Symptoms: Change at Cycle 5 Day 1	Number Analyzed	17 participants	47 participants
		-0.98 (15.205)	-4.11 (18.062)
Symptoms: Change at Cycle 6 Day 1	Number Analyzed	16 participants	36 participants
		-1.56 (16.307)	-1.85 (21.419)
Symptoms: Change at Cycle 7 Day 1	Number Analyzed	12 participants	30 participants
		4.17 (23.233)	0.69 (24.518)
Symptoms: Change at Cycle 8 Day 1	Number Analyzed	8 participants	31 participants
		6.77 (18.222)	-2.96 (24.395)
Symptoms: Change at Cycle 9 Day 1	Number Analyzed	10 participants	28 participants
		10.42 (21.178)	-3.42 (24.455)
Functioning: Change at Cycle 2 Day 1	Number Analyzed	42 participants	71 participants
		-3.49 (19.183)	-4.98 (23.650)
Functioning: Change at Cycle 3 Day 1	Number Analyzed	26 participants	63 participants
		-5.00 (18.166)	-4.76 (20.203)
Functioning: Change at Cycle 4 Day 1	Number Analyzed	22 participants	51 participants
		-8.18 (24.119)	-5.82 (23.275)
Functioning: Change at Cycle 5 Day 1	Number Analyzed	17 participants	47 participants
		-7.06 (20.746)	-3.76 (16.369)
Functioning: Change at Cycle 6 Day 1	Number Analyzed	16 participants	35 participants
		-12.08 (27.022)	-11.14 (18.113)
Functioning: Change at Cycle 7 Day 1	Number Analyzed	12 participants	30 participants
		-3.89 (19.583)	-6.00 (15.767)
Functioning: Change at Cycle 8 Day 1	Number Analyzed	8 participants	30 participants
		-8.33 (26.367)	-7.00 (17.926)
Functioning: Change at Cycle 9 Day 1	Number Analyzed	10 participants	28 participants
		-9.67 (22.192)	-5.60 (20.965)

10. Secondary Outcome

Title	Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious TEAEs
Description	An adverse event (AE) was defined as any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with this treatment. TEAEs are defined as AEs that developed or worsened during the on-treatment period and treatment-related AEs that occur during post-treatment period. A serious adverse event (SAE) was defined as any untoward medical occurrence that resulted in any of the following outcomes: death, life-threatening, required initial or prolonged in-patient hospitalization, persistent or significant disability/incapacity, congenital anomaly/birth defect, or considered as medically important event. Any TEAE included participants with both serious and non-serious TEAEs.
Time Frame	Up to 1422 days

Outcome Measure Data

Analysis Population Description

The safety analysis set (SAF) included all enrolled participants who received any study drug for each group.

Arm/Group Title	Group 1: Metastatic BCC (mBCC)	Group 2: Unresectable Locally Advanced BCC (laBCC)
Arm/Group Description:	Participants with metastatic BCC received IV infusion of cemiplimab at a dose of 350 mg Q3W for up to 93 weeks of treatment cycles (cycles 1-5 [each cycle of 9 weeks] followed by cycles 6-9 [each cycle of 12 weeks]) or until PD, unacceptable toxicity, withdrawal of consent, or CR.	Participants with unresectable laBCC received IV infusion of cemiplimab at a dose of 350 mg Q3W for up to 93 weeks of treatment cycles (cycles 1-5 [each cycle of 9 weeks] followed by cycles 6-9 [each cycle of 12 weeks]) or until PD, unacceptable toxicity, withdrawal of consent, or confirmed CR.
Overall Number of Participants Analyzed	54	84
Measure Type: Count of Participants; Unit of Measure: Participants
Participants with any TEAEs	51 94.4%	83 98.8%
Participants with any Serious TEAEs	16 29.6%	31 36.9%

11. Secondary Outcome

Title	Serum Concentration at End of Infusion (Cmax) of Cemiplimab
Description	Cmax of cemiplimab was reported.
Time Frame	At end-of-infusion (within 10 minutes after the end of infusion) on Cycle 1 Day 1 and Cycle 3 Day 1 (Each cycle of 9 weeks)

Outcome Measure Data

Analysis Population Description

The pharmacokinetics (PK) analysis set included all participants who received any cemiplimab and had at least one non-missing post-baseline measurement of cemiplimab concentration in serum. Here, "Overall number of participants analyzed" signifies those participants who were evaluable for this outcome measure.

Arm/Group Title	Group 1: Metastatic BCC (mBCC)	Group 2: Unresectable Locally Advanced BCC (laBCC)
Arm/Group Description:	Participants with metastatic BCC received IV infusion of cemiplimab at a dose of 350 mg Q3W for up to 93 weeks of treatment cycles (cycles 1-5 [each cycle of 9 weeks] followed by cycles 6-9 [each cycle of 12 weeks]) or until PD, unacceptable toxicity, withdrawal of consent, or CR.	Participants with unresectable laBCC received IV infusion of cemiplimab at a dose of 350 mg Q3W for up to 93 weeks of treatment cycles (cycles 1-5 [each cycle of 9 weeks] followed by cycles 6-9 [each cycle of 12 weeks]) or until PD, unacceptable toxicity, withdrawal of consent, or confirmed CR.
Overall Number of Participants Analyzed	30	61
Mean (Standard Deviation); Unit of Measure: Milligram per Liter (mg/L)
	160 (52.7)	192 (91.6)

12. Secondary Outcome

Title	Serum Concentration at Pre-infusion (Ctrough) of Cemiplimab
Description	Ctrough of cemiplimab was reported.
Time Frame	At pre-infusion on Cycle 1 Day 22 and Cycle 3 Day 1 (Each cycle of 9 weeks)

Outcome Measure Data

Analysis Population Description

The PK analysis set included all participants who received any cemiplimab and had at least one non-missing post-baseline measurement of cemiplimab concentration in serum. Here, "Overall number of participants analyzed" signifies those participants who were evaluable for this outcome measure.

Arm/Group Title	Group 1: Metastatic BCC (mBCC)	Group 2: Unresectable Locally Advanced BCC (laBCC)
Arm/Group Description:	Participants with metastatic BCC received IV infusion of cemiplimab at a dose of 350 mg Q3W for up to 93 weeks of treatment cycles (cycles 1-5 [each cycle of 9 weeks] followed by cycles 6-9 [each cycle of 12 weeks]) or until PD, unacceptable toxicity, withdrawal of consent, or CR.	Participants with unresectable laBCC received IV infusion of cemiplimab at a dose of 350 mg Q3W for up to 93 weeks of treatment cycles (cycles 1-5 [each cycle of 9 weeks] followed by cycles 6-9 [each cycle of 12 weeks]) or until PD, unacceptable toxicity, withdrawal of consent, or confirmed CR.
Overall Number of Participants Analyzed	32	66
Mean (Standard Deviation); Unit of Measure: mg/L
	60.6 (28.2)	68.6 (32.8)

13. Secondary Outcome

Title	Number of Participants With Anti-Drug Antibody (ADA) Status
Description	Immunogenicity was characterized by ADA responses & titers. Responses categories: Negative - ADA negative response at all time points, regardless of missing samples; Pre-existing immunoreactivity - ADA positive response at baseline with all post first dose negative results or positive response at baseline with all post first dose ADA responses < 9-fold over baseline titer levels; Treatment-boosted response - positive response in the assay post first dose, ≥ 9-fold over baseline titer levels, when baseline results are positive; Treatment-emergent response - ADA positive response in the cemiplimab ADA assay post first dose when baseline results = negative or missing.
Time Frame	Cycle 1: Days 1 and 43; Cycles 3 and 5: Day 1 (Each cycle of 9 weeks)

Outcome Measure Data

Analysis Population Description

The anti-drug antibody set included all participants who received cemiplimab and who had at least 1 non-missing result in the ADA assay after the first dose of the study drug.

Arm/Group Title	Group 1: Metastatic BCC (mBCC)	Group 2: Unresectable Locally Advanced BCC (laBCC)
Arm/Group Description:	Participants with metastatic BCC received IV infusion of cemiplimab at a dose of 350 mg Q3W for up to 93 weeks of treatment cycles (cycles 1-5 [each cycle of 9 weeks] followed by cycles 6-9 [each cycle of 12 weeks]) or until PD, unacceptable toxicity, withdrawal of consent, or CR.	Participants with unresectable laBCC received IV infusion of cemiplimab at a dose of 350 mg Q3W for up to 93 weeks of treatment cycles (cycles 1-5 [each cycle of 9 weeks] followed by cycles 6-9 [each cycle of 12 weeks]) or until PD, unacceptable toxicity, withdrawal of consent, or confirmed CR.
Overall Number of Participants Analyzed	52	81
Measure Type: Count of Participants; Unit of Measure: Participants
Negative ADA	50 96.2%	75 92.6%
Pre-Existing ADA	2 3.8%	2 2.5%
Treatment-emergent ADA	0 0.0%	4 4.9%

Adverse Events

Time Frame	All Adverse Events (AEs) were collected from signature of the informed consent form up to 1422 days.
Adverse Event Reporting Description	[Not Specified]
Arm/Group Title	Group 1: Metastatic BCC (mBCC)		Group 2: Unresectable Locally Advanced BCC (laBCC)
Arm/Group Description	Participants with metastatic BCC received IV infusion of cemiplimab at a dose of 350 mg Q3W for up to 93 weeks of treatment cycles (cycles 1-5 [each cycle of 9 weeks] followed by cycles 6-9 [each cycle of 12 weeks]) or until PD, unacceptable toxicity, withdrawal of consent, or CR.		Participants with unresectable laBCC received IV infusion of cemiplimab at a dose of 350 mg Q3W for up to 93 weeks of treatment cycles (cycles 1-5 [each cycle of 9 weeks] followed by cycles 6-9 [each cycle of 12 weeks]) or until PD, unacceptable toxicity, withdrawal of consent, or confirmed CR.
All-Cause Mortality
	Group 1: Metastatic BCC (mBCC)		Group 2: Unresectable Locally Advanced BCC (laBCC)
	Affected / at Risk (%)		Affected / at Risk (%)
Total	16/54 (29.63%)		17/84 (20.24%)
Serious Adverse Events
	Group 1: Metastatic BCC (mBCC)		Group 2: Unresectable Locally Advanced BCC (laBCC)
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	16/54 (29.63%)		32/84 (38.10%)
Blood and lymphatic system disorders
Anaemia † 1	0/54 (0.00%)	0	2/84 (2.38%)	3
Pancytopenia † 1	1/54 (1.85%)	1	0/84 (0.00%)	0
Lymphadenopathy mediastinal † 1	1/54 (1.85%)	1	0/84 (0.00%)	0
Cardiac disorders
Cardiac failure † 1	0/54 (0.00%)	0	1/84 (1.19%)	1
Autoimmune pericarditis † 1	1/54 (1.85%)	1	0/84 (0.00%)	0
Myocardial infarction † 1	0/54 (0.00%)	0	2/84 (2.38%)	2
Supraventricular tachycardia † 1	0/54 (0.00%)	0	1/84 (1.19%)	1
Acute coronary syndrome † 1	1/54 (1.85%)	1	0/84 (0.00%)	0
Atrial fibrillation † 1	1/54 (1.85%)	1	0/84 (0.00%)	0
Autoimmune myocarditis † 1	1/54 (1.85%)	1	0/84 (0.00%)	0
Immune-mediated myocarditis † 1	1/54 (1.85%)	1	0/84 (0.00%)	0
Ear and labyrinth disorders
Ear disorder † 1	0/54 (0.00%)	0	1/84 (1.19%)	1
Endocrine disorders
Adrenal insufficiency † 1	0/54 (0.00%)	0	2/84 (2.38%)	2
Hypophysitis † 1	0/54 (0.00%)	0	1/84 (1.19%)	1
Gastrointestinal disorders
Colitis † 1	2/54 (3.70%)	2	2/84 (2.38%)	2
Autoimmune colitis † 1	0/54 (0.00%)	0	1/84 (1.19%)	1
Constipation † 1	0/54 (0.00%)	0	1/84 (1.19%)	1
Erosive oesophagitis † 1	0/54 (0.00%)	0	1/84 (1.19%)	1
General disorders
Pyrexia † 1	1/54 (1.85%)	1	0/84 (0.00%)	0
Fatigue † 1	0/54 (0.00%)	0	1/84 (1.19%)	1
General physical health deterioration † 1	1/54 (1.85%)	1	0/84 (0.00%)	0
Hepatobiliary disorders
Autoimmune hepatitis † 1	1/54 (1.85%)	1	0/84 (0.00%)	0
Immune-mediated hepatitis † 1	0/54 (0.00%)	0	1/84 (1.19%)	1
Immune system disorders
Sarcoidosis † 1	0/54 (0.00%)	0	1/84 (1.19%)	2
Infections and infestations
Urinary tract infection † 1	1/54 (1.85%)	1	4/84 (4.76%)	4
Pneumonia † 1	1/54 (1.85%)	1	0/84 (0.00%)	0
Infection † 1	1/54 (1.85%)	1	0/84 (0.00%)	0
Skin infection † 1	1/54 (1.85%)	1	0/84 (0.00%)	0
Clostridium difficile infection † 1	1/54 (1.85%)	1	0/84 (0.00%)	0
Hepatitis C † 1	0/54 (0.00%)	0	1/84 (1.19%)	1
Influenza † 1	0/54 (0.00%)	0	1/84 (1.19%)	1
Lower respiratory tract infection † 1	0/54 (0.00%)	0	1/84 (1.19%)	1
Oral candidiasis † 1	0/54 (0.00%)	0	1/84 (1.19%)	1
Soft tissue infection † 1	0/54 (0.00%)	0	1/84 (1.19%)	1
Subcutaneous abscess † 1	0/54 (0.00%)	0	1/84 (1.19%)	1
Wound infection staphylococcal † 1	0/54 (0.00%)	0	1/84 (1.19%)	1
Arthritis bacterial † 1	1/54 (1.85%)	1	0/84 (0.00%)	0
Atypical pneumonia † 1	1/54 (1.85%)	1	0/84 (0.00%)	0
Clostridium difficile colitis † 1	1/54 (1.85%)	1	0/84 (0.00%)	0
Pneumonia staphylococcal † 1	1/54 (1.85%)	1	0/84 (0.00%)	0
Injury, poisoning and procedural complications
Radial head dislocation † 1	0/54 (0.00%)	0	1/84 (1.19%)	1
Upper limb fracture † 1	0/54 (0.00%)	0	1/84 (1.19%)	1
Fall † 1	1/54 (1.85%)	1	0/84 (0.00%)	0
Infusion related reaction † 1	1/54 (1.85%)	1	0/84 (0.00%)	0
Multiple fractures † 1	1/54 (1.85%)	1	0/84 (0.00%)	0
Procedural pain † 1	1/54 (1.85%)	1	0/84 (0.00%)	0
Tibia fracture † 1	1/54 (1.85%)	1	0/84 (0.00%)	0
Wound haemorrhage † 1	1/54 (1.85%)	1	0/84 (0.00%)	0
Investigations
Weight decreased † 1	0/54 (0.00%)	0	1/84 (1.19%)	1
Metabolism and nutrition disorders
Cachexia † 1	0/54 (0.00%)	0	1/84 (1.19%)	2
Musculoskeletal and connective tissue disorders
Back pain † 1	0/54 (0.00%)	0	1/84 (1.19%)	1
Dupuytren's contracture † 1	0/54 (0.00%)	0	1/84 (1.19%)	1
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Infected neoplasm † 1	0/54 (0.00%)	0	2/84 (2.38%)	2
Basal cell carcinoma † 1	0/54 (0.00%)	0	1/84 (1.19%)	1
Brain neoplasm malignant † 1	0/54 (0.00%)	0	1/84 (1.19%)	1
Meningioma † 1	0/54 (0.00%)	0	1/84 (1.19%)	1
Lymphoproliferative disorder † 1	1/54 (1.85%)	1	0/84 (0.00%)	0
Nervous system disorders
Cerebrovascular accident † 1	0/54 (0.00%)	0	1/84 (1.19%)	1
Brain oedema † 1	0/54 (0.00%)	0	1/84 (1.19%)	1
Cerebrospinal fluid leakage † 1	0/54 (0.00%)	0	1/84 (1.19%)	1
Dizziness † 1	0/54 (0.00%)	0	1/84 (1.19%)	1
Haemorrhage intracranial † 1	0/54 (0.00%)	0	1/84 (1.19%)	1
Somnolence † 1	1/54 (1.85%)	2	1/84 (1.19%)	1
Facial paralysis † 1	1/54 (1.85%)	1	0/84 (0.00%)	0
Headache † 1	1/54 (1.85%)	1	0/84 (0.00%)	0
Psychiatric disorders
Delirium † 1	0/54 (0.00%)	0	1/84 (1.19%)	1
Renal and urinary disorders
Acute kidney injury † 1	0/54 (0.00%)	0	2/84 (2.38%)	2
Urinary retention † 1	1/54 (1.85%)	1	0/84 (0.00%)	0
Respiratory, thoracic and mediastinal disorders
Pneumonitis † 1	1/54 (1.85%)	1	0/84 (0.00%)	0
Haemoptysis † 1	1/54 (1.85%)	1	0/84 (0.00%)	0
Respiratory failure † 1	1/54 (1.85%)	1	0/84 (0.00%)	0
Pulmonary oedema † 1	0/54 (0.00%)	0	1/84 (1.19%)	1
Pleural effusion † 1	1/54 (1.85%)	1	0/84 (0.00%)	0
Skin and subcutaneous tissue disorders
Dermal cyst † 1	0/54 (0.00%)	0	1/84 (1.19%)	1
Vascular disorders
Hypotension † 1	0/54 (0.00%)	0	1/84 (1.19%)	1
Hypertensive crisis † 1	0/54 (0.00%)	0	1/84 (1.19%)	2
Peripheral ischaemia † 1	0/54 (0.00%)	0	1/84 (1.19%)	1
Phlebitis † 1	0/54 (0.00%)	0	1/84 (1.19%)	1
1 Term from vocabulary, MedDRA 23.1; † Indicates events were collected by systematic assessment
Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events	5%
	Group 1: Metastatic BCC (mBCC)		Group 2: Unresectable Locally Advanced BCC (laBCC)
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	50/54 (92.59%)		75/84 (89.29%)
Blood and lymphatic system disorders
Anaemia † 1	6/54 (11.11%)	7	13/84 (15.48%)	19
Leukocytosis † 1	1/54 (1.85%)	1	7/84 (8.33%)	8
Endocrine disorders
Hypothyroidism † 1	4/54 (7.41%)	4	8/84 (9.52%)	9
Hyperthyroidism † 1	5/54 (9.26%)	7	2/84 (2.38%)	2
Eye disorders
Cataract † 1	0/54 (0.00%)	0	5/84 (5.95%)	6
Gastrointestinal disorders
Nausea † 1	6/54 (11.11%)	9	13/84 (15.48%)	19
Vomiting † 1	7/54 (12.96%)	9	6/84 (7.14%)	10
Constipation † 1	12/54 (22.22%)	14	5/84 (5.95%)	6
Diarrhoea † 1	20/54 (37.04%)	27	20/84 (23.81%)	33
Abdominal pain † 1	4/54 (7.41%)	4	6/84 (7.14%)	7
Dry mouth † 1	3/54 (5.56%)	3	3/84 (3.57%)	3
General disorders
Pyrexia † 1	7/54 (12.96%)	9	5/84 (5.95%)	7
Asthenia † 1	5/54 (9.26%)	6	17/84 (20.24%)	27
Fatigue † 1	23/54 (42.59%)	31	25/84 (29.76%)	34
Oedema peripheral † 1	6/54 (11.11%)	8	5/84 (5.95%)	5
Influenza like illness † 1	2/54 (3.70%)	2	5/84 (5.95%)	7
Pain † 1	3/54 (5.56%)	4	2/84 (2.38%)	2
Infections and infestations
Urinary tract infection † 1	4/54 (7.41%)	5	10/84 (11.90%)	11
Bronchitis † 1	0/54 (0.00%)	0	6/84 (7.14%)	7
Upper respiratory tract infection † 1	3/54 (5.56%)	3	6/84 (7.14%)	6
Conjunctivitis † 1	0/54 (0.00%)	0	5/84 (5.95%)	5
Injury, poisoning and procedural complications
Fall † 1	4/54 (7.41%)	7	5/84 (5.95%)	5
Infusion related reaction † 1	3/54 (5.56%)	3	0/84 (0.00%)	0
Investigations
Alanine aminotransferase increased † 1	3/54 (5.56%)	3	4/84 (4.76%)	6
Blood creatinine increased † 1	4/54 (7.41%)	4	8/84 (9.52%)	9
Weight decreased † 1	5/54 (9.26%)	6	7/84 (8.33%)	7
Blood creatine phosphokinase increased † 1	4/54 (7.41%)	5	6/84 (7.14%)	8
Weight increased † 1	8/54 (14.81%)	12	2/84 (2.38%)	2
Metabolism and nutrition disorders
Decreased appetite † 1	6/54 (11.11%)	6	14/84 (16.67%)	18
Hypoalbuminaemia † 1	1/54 (1.85%)	2	5/84 (5.95%)	11
Hypokalaemia † 1	4/54 (7.41%)	4	4/84 (4.76%)	6
Hyperglycaemia † 1	6/54 (11.11%)	8	2/84 (2.38%)	2
Musculoskeletal and connective tissue disorders
Back pain † 1	5/54 (9.26%)	5	5/84 (5.95%)	6
Arthralgia † 1	9/54 (16.67%)	10	16/84 (19.05%)	24
Pain in extremity † 1	6/54 (11.11%)	10	4/84 (4.76%)	5
Muscle spasms † 1	3/54 (5.56%)	3	5/84 (5.95%)	5
Myalgia † 1	3/54 (5.56%)	4	3/84 (3.57%)	3
Neck pain † 1	4/54 (7.41%)	4	2/84 (2.38%)	4
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Tumour haemorrhage † 1	1/54 (1.85%)	1	9/84 (10.71%)	12
Basal cell carcinoma † 1	3/54 (5.56%)	5	7/84 (8.33%)	8
Seborrhoeic keratosis † 1	1/54 (1.85%)	1	5/84 (5.95%)	5
Nervous system disorders
Headache † 1	6/54 (11.11%)	7	12/84 (14.29%)	14
Dizziness † 1	5/54 (9.26%)	5	7/84 (8.33%)	8
Psychiatric disorders
Anxiety † 1	3/54 (5.56%)	3	3/84 (3.57%)	3
Renal and urinary disorders
Haematuria † 1	4/54 (7.41%)	5	3/84 (3.57%)	3
Respiratory, thoracic and mediastinal disorders
Cough † 1	4/54 (7.41%)	5	10/84 (11.90%)	14
Dyspnoea † 1	4/54 (7.41%)	5	11/84 (13.10%)	17
Skin and subcutaneous tissue disorders
Rash † 1	4/54 (7.41%)	4	6/84 (7.14%)	6
Pruritus † 1	8/54 (14.81%)	13	20/84 (23.81%)	24
Dry skin † 1	5/54 (9.26%)	6	7/84 (8.33%)	7
Actinic keratosis † 1	3/54 (5.56%)	3	6/84 (7.14%)	9
Rash maculo-papular † 1	5/54 (9.26%)	6	6/84 (7.14%)	7
Dermatitis † 1	0/54 (0.00%)	0	5/84 (5.95%)	6
Eczema † 1	5/54 (9.26%)	7	5/84 (5.95%)	5
Vascular disorders
Hypertension † 1	11/54 (20.37%)	29	8/84 (9.52%)	13
1 Term from vocabulary, MedDRA 23.1; † Indicates events were collected by systematic assessment

Limitations and Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.

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.

Results Point of Contact

• Name/Title: Clinical Trials Administrator
• Organization: Regeneron Pharmaceuticals, Inc.
• Phone: 844-734-6643
• EMail: clinicaltrials@regeneron.com

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Stratigos AJ, Sekulic A, Peris K, Bechter O, Prey S, Kaatz M, Lewis KD, Basset-Seguin N, Chang ALS, Dalle S, Orland AF, Licitra L, Robert C, Ulrich C, Hauschild A, Migden MR, Dummer R, Li S, Yoo SY, Mohan K, Coates E, Jankovic V, Fiaschi N, Okoye E, Bassukas ID, Loquai C, De Giorgi V, Eroglu Z, Gutzmer R, Ulrich J, Puig S, Seebach F, Thurston G, Weinreich DM, Yancopoulos GD, Lowy I, Bowler T, Fury MG. Cemiplimab in locally advanced basal cell carcinoma after hedgehog inhibitor therapy: an open-label, multi-centre, single-arm, phase 2 trial. Lancet Oncol. 2021 Jun;22(6):848-857. doi: 10.1016/S1470-2045(21)00126-1. Epub 2021 May 14.

Li R, Lee G, Huang M, El-Sherief A. Rare basal cell metastasis of a basal-squamous skin collision tumour to the lung and axillary lymph node. BMJ Case Rep. 2019 Oct 3;12(10):e231487. doi: 10.1136/bcr-2019-231487.

• Responsible Party: Regeneron Pharmaceuticals
• ClinicalTrials.gov Identifier: NCT03132636
• Other Study ID Numbers: R2810-ONC-1620; 2016-003122-16 ( EudraCT Number )
• First Submitted: April 24, 2017
• First Posted: April 28, 2017
• Results First Submitted: May 17, 2022
• Results First Posted: July 26, 2022
• Last Update Posted: June 18, 2023