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NCT04005716

Study of Platinum Plus Etoposide With or Without Tislelizumab in Participants With Untreated Extensive-Stage Small Cell Lung Cancer

Completed Phase 3 Results posted Last updated 28 February 2025
What this trial tests

Phase 3 trial testing Tislelizumab in Small Cell Lung Cancer in 457 participants. Completed in 29 December 2023.

Timeline
22 July 2019
Primary endpoint
19 April 2023
29 December 2023

Quick facts

Lead sponsorBeiGene
PhasePhase 3
StatusCompleted
Study typeINTERVENTIONAL
Allocationrandomized
Designparallel
Maskingdouble
Primary purposetreatment
Enrollment457
Start date22 July 2019
Primary completion19 April 2023
Estimated completion29 December 2023
Sites50 locations across China

Drugs / interventions tested

Conditions studied

Sponsor

BeiGene — full company profile →

Who can join

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

Overall Survival (OS) Primary · From randomization to the primary completion cut-off on April 19, 2023, the median follow-up was 15.44 months (range: 0.2-44.9) for Arm A and 13.22 months (range: 0.1-40.8) for Arm B.

Defined as the time from the date of randomization to the date of death due to any cause. Median OS was estimated using Kaplan-Meier methodology.

GroupValue95% CI
Arm A: Tislelizumab + Chemotherapy15.513.5 – 17.1
Arm B: Placebo + Chemotherapy13.512.1 – 14.9
Progression Free Survival (PFS) Secondary · From randomization to the primary completion cut-off on April 19, 2023, the median follow-up was 15.44 months (range: 0.2-44.9) for Arm A and 13.22 months (range: 0.1-40.8) for Arm B.

Defined as the time from randomization to the first objectively documented disease progression, or death from any cause, whichever occurred first, as assessed by the investigator per the Response Evaluation Criteria in Solid Tumors (RECIST) v1.1. Kaplan-Meier methodology was used to estimate the median PFS. Progressive Disease (PD): At least a 20% increase in the size of target lesions, with an absolute increase of at least 5 mm, or unequivocal progression of existing non-target lesions, or any new lesions.

GroupValue95% CI
Arm A: Tislelizumab + Chemotherapy4.74.3 – 5.5
Arm B: Placebo + Chemotherapy4.34.2 – 4.4
Overall Response Rate (ORR) Secondary · From randomization to the primary completion cut-off on April 19, 2023, the median follow-up was 15.44 months (range: 0.2-44.9) for Arm A and 13.22 months (range: 0.1-40.8) for Arm B.

Orr is defined as the percentage of participants who had partial response or complete response as determined by the investigator per RECIST v1.1 in all randomized patients with measurable disease at baseline. Complete Response (CR): Disappearance of all target and non-target lesions and no new lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to \< 10 mm. Partial Response (PR): At least a 30% decrease in the sum of diameters of target lesions with no progression of non-target lesions and no new lesions, or disappearance of all target lesio

GroupValue95% CI
Arm A: Tislelizumab + Chemotherapy68.361.8 – 74.3
Arm B: Placebo + Chemotherapy61.755.1 – 68.0
Disease Control Rate (DCR) Secondary · From randomization to the primary completion cut-off on April 19, 2023, the median follow-up was 15.44 months (range: 0.2-44.9) for Arm A and 13.22 months (range: 0.1-40.8) for Arm B.

Defined as the percentage of participants whose best overall response (BOR) is complete response, partial response, or stable disease per RECIST v1.1. Stable Disease: Neither sufficient shrinkage of target lesions to qualify for PR nor sufficient increase to qualify for progressive disease, persistence of one or more non-target lesion(s) and/or maintenance of tumor marker level above the normal limits, and no new lesions.

GroupValue95% CI
Arm A: Tislelizumab + Chemotherapy88.583.7 – 92.4
Arm B: Placebo + Chemotherapy88.383.4 – 92.1
Clinical Benefit Rate (CBR) Secondary · From randomization to the primary completion cut-off on April 19, 2023, the median follow-up was 15.44 months (range: 0.2-44.9) for Arm A and 13.22 months (range: 0.1-40.8) for Arm B.

Defined as the percentage of participants whose best overall response (BOR) is complete response, partial response, or durable stable disease (stable disease for at least 24 weeks) per RECIST v1.1.

GroupValue95% CI
Arm A: Tislelizumab + Chemotherapy69.262.7 – 75.1
Arm B: Placebo + Chemotherapy65.258.7 – 71.4
Duration of Response (DOR) Secondary · From randomization to the primary completion cut-off on April 19, 2023, the median follow-up was 15.44 months (range: 0.2-44.9) for Arm A and 13.22 months (range: 0.1-40.8) for Arm B.

Defined as the time from the first occurrence of a documented objective response to the time of relapse, as determined by the investigator per RECIST v1.1, or death from any cause, whichever comes first. Median DOR was estimated using Kaplan-Meier methodology.

GroupValue95% CI
Arm A: Tislelizumab + Chemotherapy4.34.1 – 5.6
Arm B: Placebo + Chemotherapy3.73.0 – 4.1
Number of Participants With Adverse Events Secondary · From the first dose to 30 days after the last dose or final cutoff on December 29, 2023, maximum treatment exposure was 232 weeks for Arm A and 157 weeks for Arm B.

Number of participants with treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) graded according to the National Cancer Institute-Common Terminology Criteria for Adverse Events (NCI-CTCAE) version 5.0.

TEAEs
GroupValue95% CI
Arm A: Tislelizumab + Chemotherapy226
Arm B: Placebo + Chemotherapy228
SAEs
GroupValue95% CI
Arm A: Tislelizumab + Chemotherapy94
Arm B: Placebo + Chemotherapy70
Change From Baseline in the European Organization for the Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) Global Health Status and Physical Function Score. Secondary · Baseline to Cycles 4 and 6 ( Each cycle was 21 days)

The EORTC QLQ-30 contains 30 questions that incorporate 5 functional scales (physical functioning, role functioning, emotional functioning, cognitive functioning, and social functioning), 1 global health status scale, 3 symptom scales (fatigue, nausea and vomiting, and pain), and 6 single items (dyspnea, insomnia, appetite loss, constipation, diarrhea, and financial difficulties). The participant answers questions about their health during the past week. There are 28 questions answered on a 4-point scale where 1 = Not at all (best) and 4 = Very Much (worst) and 2 global health quality of life

Global Health Status/Quality of Life (Cycle 4)
GroupValue95% CI
Arm A: Tislelizumab + Chemotherapy8.9± 22.79
Arm B: Placebo + Chemotherapy4.5± 19.46
Global Health Status/Quality of Life (Cycle 6)
GroupValue95% CI
Arm A: Tislelizumab + Chemotherapy11.3± 21.30
Arm B: Placebo + Chemotherapy4.2± 19.04
Physical Functioning (Cycle 4)
GroupValue95% CI
Arm A: Tislelizumab + Chemotherapy0.6± 14.82
Arm B: Placebo + Chemotherapy0.4± 14.57
Physical Functioning (Cycle 6)
GroupValue95% CI
Arm A: Tislelizumab + Chemotherapy1.9± 15.18
Arm B: Placebo + Chemotherapy1.8± 12.36
Change From Baseline in European Organization for the Research and Treatment of Cancer Quality of Life Questionnaire-Lung Cancer (EORTC QLQ-LC13) Symptom Scores Secondary · Baseline to Cycles 4 and 6 ( Each cycle was 21 days)

Change from baseline in EORTC QLQ-CL13 scores for coughing, dysphagia, and chest pain. The EORTC QLQ-LC13 is a questionnaire that measures lung cancer-specific disease and treatment symptoms. It includes questions about specific symptoms in which participants respond based on a 4-point scale, where 1 is "not at all" and 4 is "very much". Raw scores are transformed into a 0 to 100 scale via linear transformation. A lower score indicates an improvement in symptoms.

Coughing (Cycle 4)
GroupValue95% CI
Arm A: Tislelizumab + Chemotherapy-19.5± 26.58
Arm B: Placebo + Chemotherapy-16.0± 22.85
Coughing (Cycle 6)
GroupValue95% CI
Arm A: Tislelizumab + Chemotherapy-18.6± 27.86
Arm B: Placebo + Chemotherapy-16.7± 25.08
Dysphagia (Cycle 4)
GroupValue95% CI
Arm A: Tislelizumab + Chemotherapy-2.5± 12.12
Arm B: Placebo + Chemotherapy-3.1± 14.72
Dysphagia (Cycle 6)
GroupValue95% CI
Arm A: Tislelizumab + Chemotherapy-2.9± 11.47
Arm B: Placebo + Chemotherapy-3.5± 15.23
Chest Pain (Cycle 4)
GroupValue95% CI
Arm A: Tislelizumab + Chemotherapy-7.5± 22.65
Arm B: Placebo + Chemotherapy-5.9± 20.97
Chest Pain (Cycle 6)
GroupValue95% CI
Arm A: Tislelizumab + Chemotherapy-7.0± 25.34
Arm B: Placebo + Chemotherapy-6.9± 21.51
Time to Deterioration (TTD) Secondary · From randomization to the primary completion data cut-off on April 19, 2023, the median follow-up was 15.44 months (range: 0.2-44.9) for Arm A and 13.22 months (range: 0.1-40.8) for Arm B.

Time to deterioration is defined as the time from randomization to the first confirmed worsening score or death. Clinically meaningful deterioration is defined as a ≥10-point decrease from baseline in QLQ-C30 physical functioning and a ≥10-point increase in QLQ-LC13 coughing and chest pain scores. If a participant did not have an event (death or deterioration), they were censored at their last clinic visit at which corresponding score was measured. Median TTD was estimated using Kaplan-Meier methodology.

QLQ-C30: Physical Functioning
GroupValue95% CI
Arm A: Tislelizumab + ChemotherapyNA20.5 – NA
Arm B: Placebo + ChemotherapyNA10.3 – NA
QLQ-LC13: Coughing
GroupValue95% CI
Arm A: Tislelizumab + ChemotherapyNANA – NA
Arm B: Placebo + ChemotherapyNANA – NA
QLQ-LC13: Chest Pain
GroupValue95% CI
Arm A: Tislelizumab + ChemotherapyNANA – NA
Arm B: Placebo + ChemotherapyNANA – NA

Adverse events — posted to ClinicalTrials.gov

Time frame: All-cause mortality is reported from randomization to the end of study (up to 53 months). Adverse events are reported from from the first dose to 30 days after the last dose or final cutoff on December 29, 2023, maximum treatment exposure was 232 weeks for Arm A and 157 weeks for Arm B.. Reporting threshold: 3%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Arm A: Tislelizumab + Chemotherapy
Serious: 94/227 (41%)
Deaths: 175/227
Arm B: Placebo + Chemotherapy
Serious: 70/229 (31%)
Deaths: 196/230

Serious adverse events (102 terms)

ReactionSystemArm A: Tislelizumab + Chem…Arm B: Placebo + Chemother…
ThrombocytopeniaBlood and lymphatic system disorders
NeutropeniaBlood and lymphatic system disorders
Febrile neutropeniaBlood and lymphatic system disorders
PneumoniaInfections and infestations
Neutrophil count decreasedInvestigations
Platelet count decreasedInvestigations
White blood cell count decreasedInvestigations
HyponatraemiaMetabolism and nutrition disorders
HaemoptysisRespiratory, thoracic and mediastinal disorders
AnaemiaBlood and lymphatic system disorders
Alanine aminotransferase increasedInvestigations
PneumonitisRespiratory, thoracic and mediastinal disorders
LeukopeniaBlood and lymphatic system disorders
Immune-mediated myocarditisCardiac disorders
IleusGastrointestinal disorders
Aspartate aminotransferase increasedInvestigations
HypothyroidismEndocrine disorders
Abdominal painGastrointestinal disorders
DiarrhoeaGastrointestinal disorders
Intestinal obstructionGastrointestinal disorders
DeathGeneral disorders
FatigueGeneral disorders
PyrexiaGeneral disorders
Immune-mediated hepatitisHepatobiliary disorders
COVID-19 pneumoniaInfections and infestations
Other adverse events (78 terms — click to expand)

ReactionSystemArm A: Tislelizumab + Chem…Arm B: Placebo + Chemother…
AnaemiaBlood and lymphatic system disorders
AlopeciaSkin and subcutaneous tissue disorders
NeutropeniaBlood and lymphatic system disorders
White blood cell count decreasedInvestigations
ThrombocytopeniaBlood and lymphatic system disorders
NauseaGastrointestinal disorders
Decreased appetiteMetabolism and nutrition disorders
ConstipationGastrointestinal disorders
LeukopeniaBlood and lymphatic system disorders
Alanine aminotransferase increasedInvestigations
VomitingGastrointestinal disorders
Neutrophil count decreasedInvestigations
HyponatraemiaMetabolism and nutrition disorders
HypoalbuminaemiaMetabolism and nutrition disorders
Aspartate aminotransferase increasedInvestigations
HypokalaemiaMetabolism and nutrition disorders
InsomniaPsychiatric disorders
CoughRespiratory, thoracic and mediastinal disorders
DiarrhoeaGastrointestinal disorders
Platelet count decreasedInvestigations
ArthralgiaMusculoskeletal and connective tissue disorders
RashSkin and subcutaneous tissue disorders
MalaiseGeneral disorders
HyperuricaemiaMetabolism and nutrition disorders
PyrexiaGeneral disorders
HypothyroidismEndocrine disorders
Lymphocyte count decreasedInvestigations
HyperglycaemiaMetabolism and nutrition disorders
FatigueGeneral disorders
Gamma-glutamyltransferase increasedInvestigations
DyspnoeaRespiratory, thoracic and mediastinal disorders
Blood creatine phosphokinase increasedInvestigations
Pain in extremityMusculoskeletal and connective tissue disorders
HaemoptysisRespiratory, thoracic and mediastinal disorders
Weight decreasedInvestigations
DizzinessNervous system disorders
HeadacheNervous system disorders
HypertriglyceridaemiaMetabolism and nutrition disorders
Back painMusculoskeletal and connective tissue disorders
Productive coughRespiratory, thoracic and mediastinal disorders

Most-reported serious reactions: Thrombocytopenia, Neutropenia, Febrile neutropenia, Pneumonia, Neutrophil count decreased, Platelet count decreased, White blood cell count decreased, Hyponatraemia.

Data from ClinicalTrials.gov NCT04005716 adverse events section.

Sponsor's own description

This Phase 3 study was a randomized, double-blind, placebo-controlled, multicenter trial designed to evaluate the efficacy of tislelizumab in combination with either cisplatin or carboplatin and etoposide (Arm A), compared to placebo combined with either cisplatin or carboplatin and etoposide (Arm B), as a first-line treatment for participants with previously untreated extensive-stage small cell lung cancer (ES-SCLC).

Publications & conference data

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

  1. Antibodies to watch in 2020.
    Kaplon H, Muralidharan M, Schneider Z, Reichert JM. · · 2020 · cited 332× · PMID 31847708 · DOI 10.1080/19420862.2019.1703531
  2. Signal pathways and precision therapy of small-cell lung cancer.
    Yuan M, Zhao Y, Arkenau HT, Lao T, et al · · 2022 · cited 72× · PMID 35705538 · DOI 10.1038/s41392-022-01013-y
  3. CD8<sup>+</sup> T cell-based cancer immunotherapy.
    Chen Y, Yu D, Qian H, Shi Y, et al · · 2024 · cited 63× · PMID 38685033 · DOI 10.1186/s12967-024-05134-6
  4. First-line immune checkpoint inhibitors for extensive stage small-cell lung cancer: clinical developments and future directions.
    Ortega-Franco A, Ackermann C, Paz-Ares L, Califano R. · · 2021 · cited 29× · PMID 33450659 · DOI 10.1016/j.esmoop.2020.100003
  5. Current and future therapies for small cell lung carcinoma.
    Zhai X, Zhang Z, Chen Y, Wu Y, et al · · 2025 · cited 15× · PMID 40170056 · DOI 10.1186/s13045-025-01690-6
  6. Advances in research on malignant tumors and targeted agents for TOP2A (Review).
    Zhou T, Niu Y, Li Y. · · 2025 · cited 4× · PMID 39670307 · DOI 10.3892/mmr.2024.13415
  7. Immunotherapy for small cell lung cancer: the current state and future trajectories.
    Qiang M, Liu H, Yang L, Wang H, et al · · 2024 · cited 4× · PMID 39152301 · DOI 10.1007/s12672-024-01119-5
  8. Immune checkpoint inhibitors for the treatment of solid tumors and lymphoma in the past 26 years (2000-2025).
    Huang L, Zhu H, Shi Y. · · 2025 · cited 3× · PMID 41310835 · DOI 10.1186/s13045-025-01734-x

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Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT04005716.

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