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NCT03066778

A Study of Pembrolizumab (MK-3475) in Combination With Etoposide/Platinum (Cisplatin or Carboplatin) for Participants With Extensive Stage Small Cell Lung Cancer (MK-3475-604/KEYNOTE-604)

Completed Phase 3 Results posted Last updated 3 October 2022
What this trial tests

Phase 3 trial testing Pembrolizumab in Small Cell Lung Cancer (SCLC) in 453 participants. Completed in 21 September 2021.

Timeline
2 May 2017
Primary endpoint
2 December 2019
21 September 2021

Quick facts

Lead sponsorMerck Sharp & Dohme LLC
PhasePhase 3
StatusCompleted
Study typeINTERVENTIONAL
Allocationrandomized
Designparallel
Maskingquadruple
Primary purposetreatment
Enrollment453
Start date2 May 2017
Primary completion2 December 2019
Estimated completion21 September 2021
Sites148 locations across Japan, Taiwan, Ireland, Poland, South Korea, New Zealand, Russia, United States

Drugs / interventions tested

Conditions studied

Sponsor

Merck Sharp & Dohme LLC — full company profile →

Who can join

18 and older, any sex, with Small Cell Lung Cancer (SCLC). 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.

Progression-free Survival (PFS) Per Response Evaluation Criteria in Solid Tumors Version 1.1 (RECIST 1.1) as Assessed by Blinded Independent Central Review (BICR) Primary · Up to approximately 30.5 months

PFS was defined as the time from randomization to the first documented progressive disease (PD) or death due to any cause, whichever occurred first. PD, as determined by RECIST 1.1, was defined as ≥20% increase in the sum of diameters of target lesions, taking as reference the smallest sum on study. In addition to the relative increase of 20%, the sum must also demonstrate an absolute increase of ≥5 mm. The appearance of one or more new lesions was also considered PD. For this study, RECIST 1.1 was modified to follow a maximum of 10 target lesions and a maximum of 5 target lesions per organ. T

GroupValue95% CI
Pembrolizumab+EP4.84.3 – 5.4
Placebo+EP4.34.2 – 4.5
Overall Survival (OS) Primary · Up to approximately 30.5 months

OS was defined as the time from randomization to death due to any cause. Participants without documented death at the time of the analysis were censored at the date of the last follow up. The OS was calculated using the non-parametric Kaplan-Meier method for censored data and presented for the first course of study treatment per protocol.

GroupValue95% CI
Pembrolizumab+EP10.89.2 – 12.9
Placebo+EP9.78.6 – 10.7
Objective Response Rate (ORR) Per Response Evaluation Criteria in Solid Tumors Version 1.1 (RECIST 1.1) as Assessed by Blinded Independent Central Review (BICR) Secondary · Up to approximately 30.5 months

ORR was defined as the percentage of participants who achieve a best objective response of complete response (CR) or partial response (PR) per RECIST 1.1. CR was defined as the disappearance of all target lesions. PR was defined as ≥30% decrease in the sum of diameters of target lesions taking as a reference the baseline sum diameters. In this study, RECIST 1.1 was modified to follow a maximum of 10 target lesions and a maximum of 5 target lesions per organ. The ORR was calculated using the Miettinen \& Nurminen method stratified by type of platinum therapy (carboplatin or cisplatin), baseline

GroupValue95% CI
Pembrolizumab+EP70.664.2 – 76.4
Placebo+EP61.855.1 – 68.2
Duration of Response (DOR) Per Response Evaluation Criteria in Solid Tumors Version 1.1 (RECIST 1.1) as Assessed by Blinded Independent Central Review (BICR) Secondary · Up to approximately 30.5 months

DOR was defined as the time from first documented evidence of a Complete Response (CR) or Partial Response (PR) per RECIST 1.1 until progressive disease (PD) per RECIST 1.1 or death due to any cause, whichever occurred first. CR was defined as the disappearance of all target lesions. PR was defined as ≥30% decrease in the sum of diameters of target lesions taking as a reference the baseline sum diameters. PD was defined as ≥20% increase in the sum of diameters of target lesions, taking as reference the smallest sum on study. In addition to the relative increase of 20%, the sum must also demons

GroupValue95% CI
Pembrolizumab+EPNANA – NA
Placebo+EPNANA – NA
Number of Participants Who Experienced an Adverse Event (AE) Secondary · Up to approximately 30.5 months

An adverse event was defined as any untoward medical occurrence in a patient or clinical investigation participant administered a pharmaceutical product and which did not necessarily have to have had a causal relationship with this treatment. An adverse event could therefore be any unfavourable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a medicinal product or protocol-specified procedure, whether or not considered related to the medicinal product or protocol-specified procedure. Any worsening (i.e., any

GroupValue95% CI
Pembrolizumab+EP223
Placebo+EP222
Number of Participants Discontinuing Study Treatment Due to an Adverse Event (AE) Secondary · Up to approximately 26 months

An adverse event was defined as any untoward medical occurrence in a patient or clinical investigation participant administered a pharmaceutical product and which did not necessarily have to have had a causal relationship with this treatment. An adverse event could therefore be any unfavourable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a medicinal product or protocol-specified procedure, whether or not considered related to the medicinal product or protocol-specified procedure. Any worsening (i.e., any

GroupValue95% CI
Pembrolizumab+EP33
Placebo+EP14
Number of Participants Experiencing Any Grade 3 to 5 Adverse Events (AE) as Assessed by National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events Version 4.03 (CTCAE 4.03) Secondary · Up to approximately 30.5 months

The CTCAE uses Grades 1 through 5 correlating to AE severity criteria. Grade 1=mild; asymptomatic or mild symptoms; clinical or diagnostic observations only; intervention not indicated. Grade 2=moderate; minimal, local or noninvasive intervention indicated; limiting age-appropriate instrumental Activities of Daily Living (ADL). Grade 3=severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self-care ADL. Grade 4=life-threatening consequences; urgent intervention indicated. Grade 5=death related to

GroupValue95% CI
Pembrolizumab+EP175
Placebo+EP172
Change From Baseline at Week 18 in European Organization for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire Core 30 (QLQ-C30) Global Health Status/Quality of Life Scale Secondary · Baseline (prior to first dose of study treatment in Cycle 1 [cycle length = 21 days]) and Week 18

EORTC QLQ-C30 is a questionnaire that rates the overall quality of life in cancer participants. The first 28 questions use a 4-point scale (1=not at all to 4=very much) for evaluating function (physical, role, social, cognitive, emotional), symptoms (diarrhea, fatigue, dyspnea, appetite loss, insomnia, nausea/vomiting, constipation, and pain) and financial difficulties. The last 2 questions use a 7-point scale (1=very poor to 7=excellent) to evaluate overall health and quality of life. Scores are transformed to a range of 0 to 100 using a standard EORTC algorithm. Change from baseline scores w

GroupValue95% CI
Pembrolizumab+EP8.665.26 – 12.06
Placebo+EP4.230.93 – 7.52
Time to True Deterioration (TTD) in the Composite Endpoint of Cough, Chest Pain, and Dyspnea Using the European Organization for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire Core 30 (QLQ-C30) and Lung Cancer Module 13 (QLQ-LC13) Secondary · Day 1 of Cycles 1-9, Day 1 of every other cycle for Cycles 10-17 and 30 days after last dose of study treatment (Up to approximately 27 months)

TTD in patient-reported lung cancer symptoms of cough (QLQ-LC-13 Item 1), chest pain (QLQ-LC-13 Item 10), and dyspnea (QLQ-C30 Item 8) was a composite endpoint defined as the time to first onset of ≥10 point deterioration from baseline in an item confirmed by a second adjacent ≥10 point deterioration. The QLQ-LC13 consists of 13 measures of lung cancer symptoms and side effects from chemotherapy and radiation. It is scored on a 4-point scale (1=none, 2=a little, 3=quite a bit, 4=very much). Scores were transformed to a range of 0 to 100 using a standard algorithm. Higher scores represented inc

GroupValue95% CI
Pembrolizumab+EPNANA – NA
Placebo+EP8.75.9 – NA

Adverse events — posted to ClinicalTrials.gov

Time frame: First Course: Up to 49.5 months Second Course: Up to 37.9 months First and second course dosing occurred concurrently. Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Pembrolizumab+EP
Serious: 111/223 (50%)
Deaths: 196/227
Placebo+EP
Serious: 89/223 (40%)
Deaths: 212/226
Pembrolizumab Second Course
Serious: 1/1 (100%)
Deaths: 1/1

Serious adverse events (137 terms)

ReactionSystemPembrolizumab+EPPlacebo+EPPembrolizumab Second Course
PneumoniaInfections and infestations
Febrile neutropeniaBlood and lymphatic system disorders
NeutropeniaBlood and lymphatic system disorders
AnaemiaBlood and lymphatic system disorders
ThrombocytopeniaBlood and lymphatic system disorders
HyponatraemiaMetabolism and nutrition disorders
Acute kidney injuryRenal and urinary disorders
PneumonitisRespiratory, thoracic and mediastinal disorders
Pulmonary embolismRespiratory, thoracic and mediastinal disorders
Atrial fibrillationCardiac disorders
DeathGeneral disorders
PyrexiaGeneral disorders
DiarrhoeaGastrointestinal disorders
AstheniaGeneral disorders
Lower respiratory tract infectionInfections and infestations
Neutropenic sepsisInfections and infestations
Upper respiratory tract infectionInfections and infestations
Urinary tract infectionInfections and infestations
DyspnoeaRespiratory, thoracic and mediastinal disorders
Pleural effusionRespiratory, thoracic and mediastinal disorders
Inappropriate antidiuretic hormone secretionEndocrine disorders
GastritisGastrointestinal disorders
NauseaGastrointestinal disorders
Clostridium difficile colitisInfections and infestations
Pleural infectionInfections and infestations
Other adverse events (50 terms — click to expand)

ReactionSystemPembrolizumab+EPPlacebo+EPPembrolizumab Second Course
NeutropeniaBlood and lymphatic system disorders
AnaemiaBlood and lymphatic system disorders
NauseaGastrointestinal disorders
AlopeciaSkin and subcutaneous tissue disorders
Decreased appetiteMetabolism and nutrition disorders
ConstipationGastrointestinal disorders
FatigueGeneral disorders
ThrombocytopeniaBlood and lymphatic system disorders
LeukopeniaBlood and lymphatic system disorders
DiarrhoeaGastrointestinal disorders
CoughRespiratory, thoracic and mediastinal disorders
AstheniaGeneral disorders
VomitingGastrointestinal disorders
DyspnoeaRespiratory, thoracic and mediastinal disorders
HeadacheNervous system disorders
PyrexiaGeneral disorders
DizzinessNervous system disorders
RashSkin and subcutaneous tissue disorders
ArthralgiaMusculoskeletal and connective tissue disorders
InsomniaPsychiatric disorders
HypothyroidismEndocrine disorders
Oedema peripheralGeneral disorders
Back painMusculoskeletal and connective tissue disorders
PruritusSkin and subcutaneous tissue disorders
Weight decreasedInvestigations
Chest painGeneral disorders
Alanine aminotransferase increasedInvestigations
HyponatraemiaMetabolism and nutrition disorders
Aspartate aminotransferase increasedInvestigations
Upper respiratory tract infectionInfections and infestations
HypotensionVascular disorders
Abdominal painGastrointestinal disorders
StomatitisGastrointestinal disorders
Blood creatinine increasedInvestigations
HypokalaemiaMetabolism and nutrition disorders
DysgeusiaNervous system disorders
Abdominal pain upperGastrointestinal disorders
PneumoniaInfections and infestations
Pain in extremityMusculoskeletal and connective tissue disorders
Dry skinSkin and subcutaneous tissue disorders

Most-reported serious reactions: Pneumonia, Febrile neutropenia, Neutropenia, Anaemia, Thrombocytopenia, Hyponatraemia, Acute kidney injury, Pneumonitis.

Data from ClinicalTrials.gov NCT03066778 adverse events section.

Sponsor's own description

The purpose of this study is to assess the safety and efficacy of pembrolizumab plus standard of care (SOC) chemotherapy (etoposide/platinum \[EP\]) in participants with newly diagnosed extensive stage small cell lung cancer (ES-SCLC) who have not previously received systemic therapy for this malignancy. The primary study hypotheses are that pembrolizumab+EP prolongs Progression-free Survival (PFS) per Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1 by blinded independent central review (BICR) and Overall Survival (OS) compared with placebo+EP in adult participants with ES-SCLC. In this study, RECIST 1.1 has been modified to follow a maximum of 10 target lesions and a maximum of 5 target lesions per organ. With protocol Amendment 07 (03-Oct-2018), the outcome measure of "Change from Baseline at Weeks 12 and 24 in European Organization for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire Core 30 (QLQ-C30) Global Health Status/Quality of Life Scale" was replaced with a single time point analysis at Week 18.

Publications & conference data

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

  1. Immune checkpoint inhibitors: recent progress and potential biomarkers.
    Darvin P, Toor SM, Sasidharan Nair V, Elkord E. · · 2018 · cited 1495× · PMID 30546008 · DOI 10.1038/s12276-018-0191-1
  2. Lung cancer immunotherapy: progress, pitfalls, and promises.
    Lahiri A, Maji A, Potdar PD, Singh N, et al · · 2023 · cited 737× · PMID 36810079 · DOI 10.1186/s12943-023-01740-y
  3. Pembrolizumab or Placebo Plus Etoposide and Platinum as First-Line Therapy for Extensive-Stage Small-Cell Lung Cancer: Randomized, Double-Blind, Phase III KEYNOTE-604 Study.
    Rudin CM, Awad MM, Navarro A, Gottfried M, et al · · 2020 · cited 594× · PMID 32468956 · DOI 10.1200/jco.20.00793
  4. Immune checkpoint therapy for solid tumours: clinical dilemmas and future trends.
    Sun Q, Hong Z, Zhang C, Wang L, et al · · 2023 · cited 387× · PMID 37635168 · DOI 10.1038/s41392-023-01522-4
  5. Improvement of the anticancer efficacy of PD-1/PD-L1 blockade via combination therapy and PD-L1 regulation.
    Wu M, Huang Q, Xie Y, Wu X, et al · · 2022 · cited 336× · PMID 35279217 · DOI 10.1186/s13045-022-01242-2
  6. Immunotherapeutic approaches for small-cell lung cancer.
    Iams WT, Porter J, Horn L. · · 2020 · cited 237× · PMID 32055013 · DOI 10.1038/s41571-019-0316-z
  7. Genetic, transcriptional and post-translational regulation of the programmed death protein ligand 1 in cancer: biology and clinical correlations.
    Zerdes I, Matikas A, Bergh J, Rassidakis GZ, et al · · 2018 · cited 223× · PMID 29765155 · DOI 10.1038/s41388-018-0303-3
  8. The Use of Immune Checkpoint Inhibitors in Oncology and the Occurrence of AKI: Where Do We Stand?
    Franzin R, Netti GS, Spadaccino F, Porta C, et al · · 2020 · cited 142× · PMID 33162990 · DOI 10.3389/fimmu.2020.574271

Verify or expand the search:

Other trials of Pembrolizumab

Trials testing the same drug.

Other recruiting trials for Small Cell Lung Cancer (SCLC)

Currently open trials in the same condition.

Other Merck Sharp & Dohme LLC trials

Trials by the same sponsor.

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