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Clinical Trials/NCT03783442
NCT03783442
Completed
Phase 3

A Randomized, Placebo-Controlled, Double-Blind Phase 3 Study to Evaluate the Efficacy and Safety of Tislelizumab (BGB-A317) in Combination With Chemotherapy as First-Line Treatment in Patients With Unresectable, Locally Advanced Recurrent or Metastatic Esophageal Squamous Cell Carcinoma

BeiGene124 sites in 4 countries649 target enrollmentDecember 11, 2018

Overview

Phase
Phase 3
Intervention
Cisplatin
Conditions
Not specified
Sponsor
BeiGene
Enrollment
649
Locations
124
Primary Endpoint
Overall Survival (OS)
Status
Completed
Last Updated
9 months ago

Overview

Brief Summary

The purpose of this study is to evaluate the efficacy and safety of tislelizumab as first line treatment in combination with chemotherapy in participants with advanced unresectable/metastatic esophageal squamous cell carcinoma (ESCC).

Registry
clinicaltrials.gov
Start Date
December 11, 2018
End Date
August 22, 2024
Last Updated
9 months ago
Study Type
Interventional
Study Design
Parallel
Sex
All

Investigators

Sponsor
BeiGene
Responsible Party
Sponsor

Eligibility Criteria

Inclusion Criteria

  • Pathologically (histologically) confirmed diagnosis of ESCC
  • Stage IV unresectable ESCC at first diagnosis OR unresectable, locally advanced recurrent or metastatic disease (per American Joint Committee on Cancer 7th Edition), if there is prior neoadjuvant/adjuvant therapy with platinum-based chemotherapy, a treatment-free interval of at least 6 months is required.

Exclusion Criteria

  • Palliative radiation treatment for ESCC within 4 weeks of study treatment initiation
  • Prior systemic therapy for unresectable, locally advanced recurrent or metastatic ESCC
  • Received prior therapies targeting programmed cell death protein-1 (PD-1), programmed cell death protein ligand-1 (PD-L1) or PD-L2
  • Participants with evidence of fistula (either esophageal/bronchial or esophageal/aorta)
  • Uncontrollable pleural effusion, pericardial effusion, or ascites requiring frequent drainage or medical intervention (clinically significant recurrence requiring an additional intervention within 2 weeks of intervention)
  • Evidence of complete esophageal obstruction not amenable to treatment
  • Unintentional weight loss ≥ 5% within one month prior to randomization or Nutritional Risk Index (NRI) \< 83.5 per investigator's choice
  • Locally advanced esophageal carcinoma that is resectable or potentially curable with radiation therapy per local investigator.
  • Participants with untreated chronic hepatitis B or chronic hepatitis B virus (HBV) carriers whose HBV DNA is ≥ 500 IU/mL or participants with active hepatitis C virus (HCV)
  • NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.

Arms & Interventions

Tislelizumab + Chemotherapy

Participants received tislelizumab 200 milligrams (mg) administered intravenously (IV) on Day 1 of each 3-week treatment cycle together with an investigator-chosen chemotherapy doublet until unacceptable toxicity, disease progression or withdrawal for other reasons. Chemotherapy options were a platinum agent (cisplatin 60-80 mg/m² intravenously on Day 1 or oxaliplatin 130 mg/m² intravenously on Day 1) combined with a fluoropyrimidine (fluorouracil \[750-800 mg/m² intravenously on Days 1-5\] or capecitabine \[1000 mg/m² orally twice daily on Days 1-14\]) or paclitaxel (175 mg/m² intravenously on Day 1).

Intervention: Cisplatin

Tislelizumab + Chemotherapy

Participants received tislelizumab 200 milligrams (mg) administered intravenously (IV) on Day 1 of each 3-week treatment cycle together with an investigator-chosen chemotherapy doublet until unacceptable toxicity, disease progression or withdrawal for other reasons. Chemotherapy options were a platinum agent (cisplatin 60-80 mg/m² intravenously on Day 1 or oxaliplatin 130 mg/m² intravenously on Day 1) combined with a fluoropyrimidine (fluorouracil \[750-800 mg/m² intravenously on Days 1-5\] or capecitabine \[1000 mg/m² orally twice daily on Days 1-14\]) or paclitaxel (175 mg/m² intravenously on Day 1).

Intervention: Oxaliplatin

Tislelizumab + Chemotherapy

Participants received tislelizumab 200 milligrams (mg) administered intravenously (IV) on Day 1 of each 3-week treatment cycle together with an investigator-chosen chemotherapy doublet until unacceptable toxicity, disease progression or withdrawal for other reasons. Chemotherapy options were a platinum agent (cisplatin 60-80 mg/m² intravenously on Day 1 or oxaliplatin 130 mg/m² intravenously on Day 1) combined with a fluoropyrimidine (fluorouracil \[750-800 mg/m² intravenously on Days 1-5\] or capecitabine \[1000 mg/m² orally twice daily on Days 1-14\]) or paclitaxel (175 mg/m² intravenously on Day 1).

Intervention: Fluorouracil (5-FU)

Tislelizumab + Chemotherapy

Participants received tislelizumab 200 milligrams (mg) administered intravenously (IV) on Day 1 of each 3-week treatment cycle together with an investigator-chosen chemotherapy doublet until unacceptable toxicity, disease progression or withdrawal for other reasons. Chemotherapy options were a platinum agent (cisplatin 60-80 mg/m² intravenously on Day 1 or oxaliplatin 130 mg/m² intravenously on Day 1) combined with a fluoropyrimidine (fluorouracil \[750-800 mg/m² intravenously on Days 1-5\] or capecitabine \[1000 mg/m² orally twice daily on Days 1-14\]) or paclitaxel (175 mg/m² intravenously on Day 1).

Intervention: Capecitabine

Tislelizumab + Chemotherapy

Participants received tislelizumab 200 milligrams (mg) administered intravenously (IV) on Day 1 of each 3-week treatment cycle together with an investigator-chosen chemotherapy doublet until unacceptable toxicity, disease progression or withdrawal for other reasons. Chemotherapy options were a platinum agent (cisplatin 60-80 mg/m² intravenously on Day 1 or oxaliplatin 130 mg/m² intravenously on Day 1) combined with a fluoropyrimidine (fluorouracil \[750-800 mg/m² intravenously on Days 1-5\] or capecitabine \[1000 mg/m² orally twice daily on Days 1-14\]) or paclitaxel (175 mg/m² intravenously on Day 1).

Intervention: Paclitaxel

Tislelizumab + Chemotherapy

Participants received tislelizumab 200 milligrams (mg) administered intravenously (IV) on Day 1 of each 3-week treatment cycle together with an investigator-chosen chemotherapy doublet until unacceptable toxicity, disease progression or withdrawal for other reasons. Chemotherapy options were a platinum agent (cisplatin 60-80 mg/m² intravenously on Day 1 or oxaliplatin 130 mg/m² intravenously on Day 1) combined with a fluoropyrimidine (fluorouracil \[750-800 mg/m² intravenously on Days 1-5\] or capecitabine \[1000 mg/m² orally twice daily on Days 1-14\]) or paclitaxel (175 mg/m² intravenously on Day 1).

Intervention: Tislelizumab

Placebo + Chemotherapy

Participants received placebo administered IV on Day 1 of each 3-week treatment cycle together with an investigator-chosen chemotherapy doublet until unacceptable toxicity, disease progression or withdrawal for other reasons. Chemotherapy options were a platinum agent (cisplatin 60-80 mg/m² intravenously on Day 1 or oxaliplatin 130 mg/m² intravenously on Day 1) combined with a fluoropyrimidine (fluorouracil \[750-800 mg/m² intravenously on Days 1-5\] or capecitabine \[1000 mg/m² orally twice daily on Days 1-14\]) or paclitaxel (175 mg/m² intravenously on Day 1).

Intervention: Cisplatin

Placebo + Chemotherapy

Participants received placebo administered IV on Day 1 of each 3-week treatment cycle together with an investigator-chosen chemotherapy doublet until unacceptable toxicity, disease progression or withdrawal for other reasons. Chemotherapy options were a platinum agent (cisplatin 60-80 mg/m² intravenously on Day 1 or oxaliplatin 130 mg/m² intravenously on Day 1) combined with a fluoropyrimidine (fluorouracil \[750-800 mg/m² intravenously on Days 1-5\] or capecitabine \[1000 mg/m² orally twice daily on Days 1-14\]) or paclitaxel (175 mg/m² intravenously on Day 1).

Intervention: Oxaliplatin

Placebo + Chemotherapy

Participants received placebo administered IV on Day 1 of each 3-week treatment cycle together with an investigator-chosen chemotherapy doublet until unacceptable toxicity, disease progression or withdrawal for other reasons. Chemotherapy options were a platinum agent (cisplatin 60-80 mg/m² intravenously on Day 1 or oxaliplatin 130 mg/m² intravenously on Day 1) combined with a fluoropyrimidine (fluorouracil \[750-800 mg/m² intravenously on Days 1-5\] or capecitabine \[1000 mg/m² orally twice daily on Days 1-14\]) or paclitaxel (175 mg/m² intravenously on Day 1).

Intervention: Fluorouracil (5-FU)

Placebo + Chemotherapy

Participants received placebo administered IV on Day 1 of each 3-week treatment cycle together with an investigator-chosen chemotherapy doublet until unacceptable toxicity, disease progression or withdrawal for other reasons. Chemotherapy options were a platinum agent (cisplatin 60-80 mg/m² intravenously on Day 1 or oxaliplatin 130 mg/m² intravenously on Day 1) combined with a fluoropyrimidine (fluorouracil \[750-800 mg/m² intravenously on Days 1-5\] or capecitabine \[1000 mg/m² orally twice daily on Days 1-14\]) or paclitaxel (175 mg/m² intravenously on Day 1).

Intervention: Capecitabine

Placebo + Chemotherapy

Participants received placebo administered IV on Day 1 of each 3-week treatment cycle together with an investigator-chosen chemotherapy doublet until unacceptable toxicity, disease progression or withdrawal for other reasons. Chemotherapy options were a platinum agent (cisplatin 60-80 mg/m² intravenously on Day 1 or oxaliplatin 130 mg/m² intravenously on Day 1) combined with a fluoropyrimidine (fluorouracil \[750-800 mg/m² intravenously on Days 1-5\] or capecitabine \[1000 mg/m² orally twice daily on Days 1-14\]) or paclitaxel (175 mg/m² intravenously on Day 1).

Intervention: Paclitaxel

Placebo + Chemotherapy

Participants received placebo administered IV on Day 1 of each 3-week treatment cycle together with an investigator-chosen chemotherapy doublet until unacceptable toxicity, disease progression or withdrawal for other reasons. Chemotherapy options were a platinum agent (cisplatin 60-80 mg/m² intravenously on Day 1 or oxaliplatin 130 mg/m² intravenously on Day 1) combined with a fluoropyrimidine (fluorouracil \[750-800 mg/m² intravenously on Days 1-5\] or capecitabine \[1000 mg/m² orally twice daily on Days 1-14\]) or paclitaxel (175 mg/m² intravenously on Day 1).

Intervention: Placebo

Outcomes

Primary Outcomes

Overall Survival (OS)

Time Frame: From randomization to the primary analysis cutoff date of 28 February 2022; maximum time on follow-up was 3 years and 2 months.

Overall survival is defined as the time from the date of randomization until the date of death due to any cause. Median OS was estimated using the Kaplan-Meier method. Primary analysis (data cut-off date of 28 February 2022) was the pre-defined analysis at which the primary endpoint was tested for superiority.

Secondary Outcomes

  • Progression-Free Survival (PFS)(From randomization to the primary analysis cutoff date of 28 February 2022; maximum time on follow-up was 3 years and 2 months.)
  • Objective Response Rate (ORR)(Response was assessed every 6 weeks for the first 48 weeks, then every 9 weeks thereafter; up to the primary analysis cutoff date of 28 February 2022; maximum time on follow-up was 3 years and 2 months.)
  • Overall Survival (OS) in Participants With a PD-L1 Score ≥ 10%(From randomization to the primary analysis cutoff date of 28 February 2022; maximum time on follow-up was 3 years and 2 months.)
  • Duration of Response (DOR)(From randomization to the primary analysis cutoff date of 28 February 2022; maximum time on follow-up was 3 years and 2 months.)
  • Change From Baseline in European Organization for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire - Oesophageal Cancer 18 Question Module (QLQ-OES18) Dysphagia, Eating, Reflux, Pain, and Index Scores(Baseline, Cycle 6 (Week 15))
  • Change From Baseline in European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire - Core 30 (QLQ-C30) Global Health Status/Quality of Life (GHS/QoL) and Physical Functioning Scales(Baseline, Cycle 6 (Week 15))
  • Change From Baseline in EORTC QLQ-C30 Fatigue Scale(Baseline, Cycle 6 (Week 15))
  • Change From Baseline in European Quality of Life 5-Dimension 5-Level (EQ-5D-5L) Visual Analog Scale (VAS)(Baseline, Cycle 6 (Week 15))
  • Number of Participants Experiencing Treatment-emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)(From first dose of study drug up to 30 days after last dose; maximum time on treatment was 63.5 months.)

Study Sites (124)

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FDA Approves Tevimbra-Chemotherapy Combination for First-Line Treatment of Advanced Esophageal Squamous Cell Carcinoma- The FDA has approved BeiGene's Tevimbra (tislelizumab-jsgr) in combination with platinum-containing chemotherapy for first-line treatment of adults with unresectable or metastatic esophageal squamous cell carcinoma whose tumors express PD-L1. - In the pivotal RATIONALE-306 trial, patients treated with Tevimbra plus chemotherapy demonstrated a median overall survival of 16.8 months compared to 9.6 months with chemotherapy alone, representing a 34% reduction in risk of death. - This marks BeiGene's third FDA approval in less than a year, following previous approvals for Tevimbra in second-line ESCC and first-line gastric/gastroesophageal junction cancers, highlighting the company's expanding oncology portfolio.European Commission Approves Tislelizumab Plus Chemotherapy for ESCC and GEJ Cancer- The European Commission has approved tislelizumab in combination with chemotherapy for first-line treatment of esophageal squamous cell carcinoma (ESCC) and gastroesophageal junction (GEJ) adenocarcinoma. - Approval was based on results from the RATIONALE-306 and RATIONALE-305 trials, which demonstrated statistically significant improvements in overall survival (OS). - In RATIONALE-306, median OS for ESCC patients treated with tislelizumab plus chemotherapy was 17.2 months compared to 10.6 months with chemotherapy alone. - RATIONALE-305 showed that tislelizumab plus chemotherapy improved median OS to 15.0 months in GEJ cancer, versus 12.9 months with chemotherapy alone.