This is a single-arm, open-label, multicenter, efficacy, and safety study of pembrolizumab in adult and pediatric participants with previously untreated advanced Merkel Cell Carcinoma (MCC). The primary objective of the trial is to assess the objective response rate, as assessed by blinded independent central review per Response Evaluation Criteria in Solid Tumors version 1.1 (RECIST 1.1) modified to follow a maximum of 10 target lesions and a maximum of 5 target lesions per organ, following administration of pembrolizumab.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
55
200 mg (adult participants) or 2 mg/kg (up to 200 mg; pediatric participants) on Day 1 of each 3-week cycle (Q3W) IV up to 35 administrations (approximately 2 years).
Laura and Isaac Perlmutter Cancer Center at NYU Langone Health ( Site 0006)
New York, New York, United States
Objective Response Rate (ORR)
ORR was defined as the percentage of participants with Complete Response (CR: disappearance of all target lesions) or Partial Response (PR: at least a 30% decrease in the sum of diameters of target lesions) per Response Evaluation Criteria in Solid Tumors version 1.1 (RECIST 1.1). The percentage of participants who experienced CR or PR as assessed by blinded independent central review (BICR) were presented.
Time frame: Up to ~34 months
Duration of Response (DOR)
For participants who demonstrated a confirmed Complete Response (CR: disappearance of all target lesions) or Partial Response (PR: At least a 30% decrease in the sum of diameters of target lesions) per RECIST 1.1, DOR was defined as the time from first documented evidence of a CR or PR until progressive disease (PD) or death. DOR for participants who had not progressed or died at the time of analysis was censored at the date of their last tumor assessment. Per RECIST 1.1, PD was defined as at least a 20% increase in the sum of diameters of target lesions as well as an absolute increase of at least a 5 mm in the sum of diameters. The appearance of one or more new lesions was also considered PD. DOR assessments were based on BICR with confirmation. The DOR as assessed using RECIST 1.1 for all participants who experienced a confirmed CR or PR was presented.
Time frame: Up to ~58 months
Progression-free Survival (PFS)
Progression-Free Survival was defined as the time from the first dose of study treatment to the first documented evidence of disease progression per RECIST 1.1 by BICR or death due to any cause, whichever occurred first. Per RECIST 1.1, PD was defined as ≥20% increase in the sum of diameters of target lesions. In addition to the relative increase of 20%, the sum must also have demonstrated an absolute increase of ≥5 mm. The appearance of one or more new lesions was also considered PD. PFS as assessed by BICR per RECIST 1.1 was presented.
Time frame: Up to ~58 months
Overall Survival (OS)
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Icahn School of Medicine at Mount Sinai ( Site 0004)
New York, New York, United States
Melanoma Institute Australia ( Site 0400)
North Sydney, New South Wales, Australia
Calvary Mater Newcastle ( Site 0402)
Waratah, New South Wales, Australia
Moncton Hospital - Horizon Health Network ( Site 0055)
Moncton, New Brunswick, Canada
Princess Margaret Cancer Centre ( Site 0051)
Toronto, Ontario, Canada
Hopital de la Cote de Nacre - Caen ( Site 0204)
Caen, Calvados, France
CHU de Bordeaux- Hopital Saint Andre ( Site 0203)
Bordeaux, Gironde, France
Hopital Ambroise Pare Boulogne ( Site 0201)
Boulogne-Billancourt, Hauts-de-Seine, France
C.H.R.U. de Tours. Hopital Trousseau ( Site 0202)
Chambray-lès-Tours, Indre-et-Loire, France
...and 12 more locations
OS was defined as the time from the first dose of study treatment until death from any cause.
Time frame: Up to ~58 months
Number of Participants With One or More Adverse Events (AEs)
An AE was any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of a study intervention. The number of participants with at least one AE was assessed.
Time frame: Up to ~58 months
Number of Participants Who Discontinued From Study Treatment Due to an AE
An AE was any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of a study intervention. The number of participants who discontinued from study treatment due to an AE was assessed.
Time frame: Up to ~27 months