NCT03430843

Brief Summary

The purpose of this study was to evaluate the efficacy and safety of tislelizumab as second line treatment in participants with advanced unresectable/metastatic ESCC that had progressed during or after first line therapy.

Trial Health

98
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Strong global presence with extensive site network
Enrollment
512

participants targeted

Target at P75+ for phase_3

Timeline
Completed

Started Jan 2018

Longer than P75 for phase_3

Geographic Reach
11 countries

104 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

January 26, 2018

Completed
3 days until next milestone

First Submitted

Initial submission to the registry

January 29, 2018

Completed
15 days until next milestone

First Posted

Study publicly available on registry

February 13, 2018

Completed
2.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2020

Completed
2.1 years until next milestone

Study Completion

Last participant's last visit for all outcomes

December 28, 2022

Completed
1 year until next milestone

Results Posted

Study results publicly available

December 29, 2023

Completed
Last Updated

October 26, 2024

Status Verified

October 1, 2024

Enrollment Period

2.8 years

First QC Date

January 29, 2018

Results QC Date

November 10, 2023

Last Update Submit

October 23, 2024

Conditions

Keywords

Unresectablemetastaticsecond-linesquamousesophaguschemotherapypaclitaxeldocetaxelirinotecan

Outcome Measures

Primary Outcomes (1)

  • Overall Survival (OS) in the Intent-to-Treat (ITT) Analysis Set

    OS is defined as the length of time from the date of randomization until the date of death due to any cause in all randomized participants

    Approximately 2 years and 10 months from date of first randomization

Secondary Outcomes (14)

  • Overall Survival (OS) in the PDL-1 Positive Analysis Set

    Through End-of-Trial Analysis data cutoff date of 28-Dec-2022 (up to approximately 5 years)

  • Objective Response Rate (ORR) in the ITT Analysis Set

    Through End-of-Trial Analysis data cutoff date of 28-Dec-2022 (up to approximately 5 years)

  • Overall Response Rate (ORR) in the PD-L1 Positive Analysis Sets

    Through End-of-Trial Analysis data cutoff date of 28-Dec-2022 (up to approximately 5 years)

  • Progression-free Survival (PFS) in the ITT Analysis Set

    Through End-of-Trial Analysis data cutoff date of 28-Dec-2022 (up to approximately 5 years)

  • Progression-free Survival (PFS) in the PDL-1 Positive Analysis Set

    Through End-of-Trial Analysis data cutoff date of 28-Dec-2022 (up to approximately 5 years)

  • +9 more secondary outcomes

Study Arms (2)

Tislelizumab

EXPERIMENTAL

Tislelizumab on Day 1, given every 21 days

Drug: Tislelizumab

Investigator chosen chemotherapy (ICC)

ACTIVE COMPARATOR

Paclitaxel on Day 1, given every 21 days or on a weekly schedule; OR Docetaxel on Day 1, given every 21 days; OR Irinotecan on Days 1 and 8, given every 21 days

Drug: PaclitaxelDrug: DocetaxelDrug: Irinotecan

Interventions

200 mg administered intravenously (IV)

Also known as: BGB-A317
Tislelizumab

135-175 mg /m² administered IV , or 80-100 mg/m\^2 administered IV according to local guidelines for standard of care

Investigator chosen chemotherapy (ICC)

75 mg/m\^2 administered IV or 70 mg/m\^2 IV in Japan

Investigator chosen chemotherapy (ICC)

125 mg/m\^2 administered IV

Investigator chosen chemotherapy (ICC)

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Histologically confirmed diagnosis of esophageal squamous cell carcinoma (ESCC)
  • Tumor progression during or after first-line treatment for advanced unresectable / metastatic ESCC
  • At least one measurable/evaluable lesion by Response Evaluation Criteria in Solid Tumors (RECIST) v1.1
  • Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1 prior to randomization

You may not qualify if:

  • Receipt of 2 or more prior systemic treatments for advanced/metastatic unresectable ESCC
  • History of gastrointestinal perforation and /or fistula or aorto-esophageal fistula within 6 months prior to randomization
  • Tumor invasion into organs located adjacent to the esophageal disease site (eg, aorta or respiratory tract) at an increased risk of fistula in the study treatment assessed by investigator
  • Uncontrollable pleural effusion, pericardial effusion, or ascites requiring frequent drainage
  • Received prior therapies targeting programmed death 1 (PD-1) or programmed death ligand 1 (PD-L1)
  • Prior malignancy active within the previous 2 years (exceptions include the tumor under investigation in this trial, and locally recurring cancers that have undergone curative treatment, such as resected basal or squamous cell skin cancer, superficial bladder cancer or carcinoma in situ of the prostate, cervix or breast)
  • Active brain or leptomeningeal metastasis.
  • Has active autoimmune disease or history of autoimmune diseases at high risk for relapse
  • Known history of, or any evidence of interstitial lung disease, non-infectious pneumonitis, pulmonary fibrosis diagnosed based on imaging or clinical findings, or uncontrolled systemic diseases, including diabetes, hypertension, acute lung diseases, etc
  • Known history of Human Immunodeficiency Virus (HIV)
  • Has cardiovascular risk factors
  • Pregnant or breastfeeding woman.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (104)

Henry Ford Hospital

Detroit, Michigan, 48202, United States

Location

Toledo Clinic Cancer Center

Toledo, Ohio, 43623, United States

Location

Imelda Ziekenhuis Bonheiden

Bonheiden, 2820, Belgium

Location

Cliniques Universitaires Saint Luc

Brussels, 1200, Belgium

Location

Uz Antwerpen

Edegem, 2650, Belgium

Location

University Hospitals Leuven

Leuven, 3000, Belgium

Location

The Second Hospital of Anhui Medical University

Hefei, Anhui, 230601, China

Location

Beijing Friendship Hospital, Capital Medical University

Beijing, Beijing Municipality, 100050, China

Location

Beijing Cancer Hospital

Beijing, Beijing Municipality, 100142, China

Location

Peking Union Medical College Hospital

Beijing, Beijing Municipality, 100730, China

Location

Fujian Medical University Union Hospital

Fuzhou, Fujian, 350001, China

Location

The First Affiliated Hospital of Fujian Medical University

Fuzhou, Fujian, 350005, China

Location

Fujian Cancer Hospital

Fuzhou, Fujian, 350014, China

Location

The First Affiliated Hospital of Xiamen University

Xiamen, Fujian, 361003, China

Location

The First Affiliated Hospital, Sun Yat Sen University

Guangzhou, Guangdong, 510080, China

Location

The Sixth Affiliated Hospital, Sun Yat Sen University

Guangzhou, Guangdong, 510655, China

Location

Cancer Hospital of Shantou University Medical College

Shantou, Guangdong, 515031, China

Location

Peking University Shenzhen Hospital

Shenzhen, Guangdong, 518036, China

Location

Affiliated Hospital of Hebei University

Baoding, Hebei, 071000, China

Location

Harbin Medical University Cancer Hospital

Harbin, Heilongjiang, 150000, China

Location

The First Affiliated Hospital of Xinxiang Medical University

Xinxiang, Henan, 453100, China

Location

Henan Cancer Hospital

Zhengzhou, Henan, 450000, China

Location

The First Affiliated Hospital of Zhengzhou University

Zhengzhou, Henan, 450052, China

Location

Hubei Cancer Hospital

Wuhan, Hubei, 430079, China

Location

Xiangyang Central Hospital

Xiangyang, Hubei, 441021, China

Location

Hunan Cancer Hospital

Changsha, Hunan, 410013, China

Location

The First Peoples Hospital of Changzhou

Changzhou, Jiangsu, 213000, China

Location

Huaian Second Peoples Hospital

Huaian, Jiangsu, 223002, China

Location

Nanjing Drum Tower Hospital,the Affiliated Hospital of Nanjing University Medical School

Nanjing, Jiangsu, 210008, China

Location

Jiangsu Province Hospital

Nanjing, Jiangsu, 210029, China

Location

Nantong Tumor Hospital Branch South

Nantong, Jiangsu, 226000, China

Location

The Affiliated Hospital of Xuzhou Medical University

Xuzhou, Jiangsu, 221000, China

Location

Jiangxi Province Cancer Hospital

Nanchang, Jiangxi, 330029, China

Location

The First Hospital of Jilin University

Changchun, Jilin, 130021, China

Location

The Second Affiliated Hospital of Xian Jiaotong University

Xi'an, Shaanxi, 710004, China

Location

Shandong Cancer Hospital

Jinan, Shandong, 250117, China

Location

Linyi Cancer Hospital

Linyi, Shandong, 276001, China

Location

Weifang Peoples Hospital

Weifang, Shandong, 261000, China

Location

Fudan University Shanghai Cancer Center

Shanghai, Shanghai Municipality, 200000, China

Location

Shanxi Provincial Peoples Hospital

Taiyuan, Shanxi, 030012, China

Location

Sichuan Academy of Medical Sciences and Sichuan Provincial Peoples Hospital

Chengdu, Sichuan, 610071, China

Location

Yunnan Cancer Hospital

Kunming, Yunnan, 650100, China

Location

The First Affiliated Hospital, Zhejiang University School of Medicine

Hangzhou, Zhejiang, 310003, China

Location

Zhejiang Cancer Hospital

Hangzhou, Zhejiang, 310022, China

Location

Chru de Brest Hospital Morvan

Brest, 29200, France

Location

Centre Georges Francois Leclerc

Dijon, 21079, France

Location

Chu Besancon Hopital Jean Minjoz

Doubs, 25030, France

Location

Centre Oscar Lambret

Lille, 59000, France

Location

Hopital Prive Jean Mermoz

Lyon, 69008, France

Location

Icm Val Daurelle

Montpellier, 34090, France

Location

Chu Bordeaux Hopital Haut Leveque

Pessac, 33600, France

Location

Chu de Poitiers Site de La Mileterie

Poitiers, 86000, France

Location

Ico Site Rene Gauducheau

SaintHerblain, 44805, France

Location

Institut Gustave Roussy

Villejuif, 94805, France

Location

Charite Universitatsmedizin Berlin

Berlin, 13353, Germany

Location

Universitares Krebszentrum Leipzig

Leipzig, 04103, Germany

Location

Klinikum Johannes Gutenberg Universitaet Mainz

Mainz, 55131, Germany

Location

Ospedale Di Faenza

Faenza, 48018, Italy

Location

Istituto Romagnolo Per Lo Studio Dei Tumori Dino Amadori Irst

Meldola, 47014, Italy

Location

Azienda Ospedaliero Universitaria San Luigi Gonzaga

Orbassano, 10043, Italy

Location

Aou Pisana, Stabilimento Di Santa Chiara

Pisa, 56126, Italy

Location

Ao Citta Della Salute E Della Scienza Di Torino Presidio O

Torino, 10126, Italy

Location

Akita University Hospital

Akitashi, Akita, 010-8543, Japan

Location

National Cancer Center Hospital East

Kashiwa, Chiba, 277-8577, Japan

Location

Nho Shikoku Cancer Center

Matsuyama, Ehime, 791-0280, Japan

Location

Nho Kyushu Cancer Center

Fukuoka, Fukuoka, 811-1395, Japan

Location

Kobe City Medical Center General Hospital

Kobe, Hyōgo, 650-0047, Japan

Location

St Marianna University School of Medicine Hospital

Yokohama, Kanagawa, 216-8511, Japan

Location

Osaka International Cancer Institute

Osakashi, Osaka, 541-8567, Japan

Location

Kindai University Hospital

Sayama, Osaka, 589-8511, Japan

Location

Osaka University Hospital

Suitashi, Osaka, 565-0871, Japan

Location

Osaka Medical and Pharmaceutical University Hospital

Takatsukishi, Osaka, 569-8686, Japan

Location

Saitama Cancer Center

Kitaadachigun, Saitama, 362-0806, Japan

Location

Shizuoka Cancer Center

Suntogun, Shizuoka, 411-8777, Japan

Location

National Cancer Center Hospital

Chuoku, Tokyo, 104-0045, Japan

Location

Cancer Institute Hospital of Jfcr

Kotoku, Tokyo, 135-8550, Japan

Location

Hiroshima University Hospital

Hiroshima, 734-8551, Japan

Location

Kochi Health Sciences Center

Kochi, 781-8555, Japan

Location

Kyoto University Hospital

Kyoto, 6068507, Japan

Location

Seoul National University Bundang Hospital

Seongnam-si, Gyeonggi-do, 13620, South Korea

Location

Gachon University Gil Medical Center

Incheon, Incheon Gwang'yeogsi, 21565, South Korea

Location

Chonnam National University Hwasun Hospital

HwasunGun, Jeollanam-do, 58128, South Korea

Location

Asan Medical Center

Seoul, Seoul Teugbyeolsi, 05505, South Korea

Location

Samsung Medical Center

Seoul, Seoul Teugbyeolsi, 06351, South Korea

Location

Korea University Guro Hospital

Seoul, Seoul Teugbyeolsi, 08308, South Korea

Location

Hospital Del Mar

Barcelona, 08003, Spain

Location

Hospital Universitario Vall Dhebron

Barcelona, 08035, Spain

Location

Institut Catala Doncologia

Barcelona, 08908, Spain

Location

Hospital Clinic de Barcelona

Barcelona, 8036, Spain

Location

Hospital Universitario Ramon Y Cajal

Madrid, 28034, Spain

Location

Hospital Universitario de Octubre

Madrid, 28041, Spain

Location

Hospital Universitario Hm Madrid Sanchinarro

Madrid, 28050, Spain

Location

Hospital Universitario Miguel Servet

Zaragoza, 50009, Spain

Location

Kaohsiung Chang Gung Memorial Hospital

Kaohsiung City, 83301, Taiwan

Location

China Medical University Hospital

Taichung, 40447, Taiwan

Location

Chi Mei Hospital Liouying

Tainan, 736, Taiwan

Location

National Taiwan University Hospital East Campus

Taipei, 100225, Taiwan

Location

Taipei Veterans General Hospital

Taipei, 11217, Taiwan

Location

The Christie Hospital

Greater Manchester, M20 4BX, United Kingdom

Location

Guys and St Thomas Hospital Nhs Foundation Trust

London, SE1 9RT, United Kingdom

Location

Sarah Cannon Research Institute Uk

London, W1G 6AD, United Kingdom

Location

St Marys Hospital Imperial College Healthcare Nhs Trust

London, W2 1NY, United Kingdom

Location

Mount Vernon Hospital (Mount Vernon Cancer Center)

Northwood, HA6 2RN, United Kingdom

Location

Royal Marsden Nhs Foundation Royal Marsden Hospital

Sutton, SM2 5PT, United Kingdom

Location

Related Publications (7)

  • Shen L, Kato K, Kim SB, Ajani JA, Zhao K, He Z, Yu X, Shu Y, Luo Q, Wang J, Chen Z, Niu Z, Zhang L, Yi T, Sun JM, Chen J, Yu G, Lin CY, Hara H, Bi Q, Satoh T, Pazo-Cid R, Arkenau HT, Borg C, Lordick F, Li L, Ding N, Tao A, Shi J, Van Cutsem E; RATIONALE-302 Investigators. Tislelizumab Versus Chemotherapy as Second-Line Treatment for Advanced or Metastatic Esophageal Squamous Cell Carcinoma (RATIONALE-302): A Randomized Phase III Study. J Clin Oncol. 2022 Sep 10;40(26):3065-3076. doi: 10.1200/JCO.21.01926. Epub 2022 Apr 20.

  • Ajani JA, Wang K, Nikolaidis GF, Coaquira Castro J, Augusto Novis de Figueiredo M, Sun JM, Soulanis K, Tasoulas A, Zhan L. Comparative Efficacy and Safety of Tislelizumab in Second-Line Esophageal Squamous Cell Carcinoma: Systematic Literature Review and Simulated Treatment Comparisons. Adv Ther. 2025 Dec 3. doi: 10.1007/s12325-025-03410-5. Online ahead of print.

  • Lu Z, Du W, Jiao X, Wang Y, Shi J, Shi Y, Shu Y, Niu Z, Hara H, Wu J, Hsu CH, Van Cutsem E, Brock MV, Zhang Z, Ding N, Zhang Y, Shen Z, Shen L. NOTCH1 Mutation and Survival Analysis of Tislelizumab in Advanced or Metastatic Esophageal Squamous Cell Carcinoma: A Biomarker Analysis From the Randomized, Phase III, RATIONALE-302 Trial. J Clin Oncol. 2025 Jun;43(16):1898-1909. doi: 10.1200/JCO-24-01818. Epub 2025 Apr 3.

  • He Q, Shi X, Yan J, Wu M, Gu C, Yu X. Circulating tumor DNA serial monitoring of relapse and responses to tislelizumab immunotherapy as second-line monotherapy for metastatic esophageal squamous cell carcinoma: A prospective study. Mol Clin Oncol. 2024 Feb 9;20(4):29. doi: 10.3892/mco.2024.2727. eCollection 2024 Apr.

  • Hara H, Satoh T, Kojima T, Tsushima T, Sunakawa Y, Okada M, Ding N, Wu H, Li L, Yu T, Barnes G, Kato K. Second-line tislelizumab versus chemotherapy in Japanese patients with advanced or metastatic esophageal squamous cell carcinoma: subgroup analysis from RATIONALE-302. Esophagus. 2024 Apr;21(2):102-110. doi: 10.1007/s10388-023-01040-w. Epub 2024 Jan 19.

  • Kim SB, Van Cutsem E, Ajani J, Shen L, Barnes G, Ding N, Tao A, Xia T, Zhan L, Kato K. Tislelizumab in advanced/metastatic esophageal squamous cell carcinoma: health-related quality of life in Asian patients. Curr Med Res Opin. 2024 Jan;40(1):69-75. doi: 10.1080/03007995.2023.2270894. Epub 2024 Jan 3.

  • Van Cutsem E, Kato K, Ajani J, Shen L, Xia T, Ding N, Zhan L, Barnes G, Kim SB. Tislelizumab versus chemotherapy as second-line treatment of advanced or metastatic esophageal squamous cell carcinoma (RATIONALE 302): impact on health-related quality of life. ESMO Open. 2022 Aug;7(4):100517. doi: 10.1016/j.esmoop.2022.100517. Epub 2022 Jul 1.

MeSH Terms

Conditions

Esophageal Squamous Cell CarcinomaNeoplasm Metastasis

Interventions

tislelizumabPaclitaxelDocetaxelIrinotecan

Condition Hierarchy (Ancestors)

Carcinoma, Squamous CellCarcinomaNeoplasms, Glandular and EpithelialNeoplasms by Histologic TypeNeoplasmsNeoplasms, Squamous CellEsophageal NeoplasmsGastrointestinal NeoplasmsDigestive System NeoplasmsNeoplasms by SiteHead and Neck NeoplasmsDigestive System DiseasesEsophageal DiseasesGastrointestinal DiseasesNeoplastic ProcessesPathologic ProcessesPathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

TaxoidsCyclodecanesCycloparaffinsHydrocarbons, AlicyclicHydrocarbons, CyclicHydrocarbonsOrganic ChemicalsDiterpenesTerpenesCamptothecinAlkaloidsHeterocyclic Compounds

Limitations and Caveats

The impact on treatment was considered very limited due to the small number of patients remaining on treatment, the short COVID-19 restriction period (in Asia), and limited restrictions for healthcare visits (in Europe/North America).

Results Point of Contact

Title
Study Director
Organization
BeiGene

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 3
Allocation
RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

January 29, 2018

First Posted

February 13, 2018

Study Start

January 26, 2018

Primary Completion

December 1, 2020

Study Completion

December 28, 2022

Last Updated

October 26, 2024

Results First Posted

December 29, 2023

Record last verified: 2024-10

Data Sharing

IPD Sharing
Will share

Locations