NCT03875235

Brief Summary

Durvalumab or Placebo in Combination With Gemcitabine/Cisplatin in Patients With 1st Line Advanced Biliary Tract Cancer (TOPAZ-1).

Trial Health

87
On Track

Trial Health Score

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

Strong global presence with extensive site network
Enrollment
810

participants targeted

Target at P75+ for phase_3

Timeline
12mo left

Started Apr 2019

Longer than P75 for phase_3

Geographic Reach
17 countries

126 active sites

Status
active not recruiting

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 Progress87%
Apr 2019May 2027

First Submitted

Initial submission to the registry

March 13, 2019

Completed
1 day until next milestone

First Posted

Study publicly available on registry

March 14, 2019

Completed
1 month until next milestone

Study Start

First participant enrolled

April 16, 2019

Completed
2.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 11, 2021

Completed
1.7 years until next milestone

Results Posted

Study results publicly available

April 13, 2023

Completed
4.1 years until next milestone

Study Completion

Last participant's last visit for all outcomes

May 16, 2027

Expected
Last Updated

March 19, 2026

Status Verified

February 1, 2026

Enrollment Period

2.3 years

First QC Date

March 13, 2019

Results QC Date

August 10, 2022

Last Update Submit

February 26, 2026

Conditions

Keywords

First-Line Advanced Biliary Tract Cancers (BTC)DurvalumabGemcitabine/CisplatinPlacebo

Outcome Measures

Primary Outcomes (3)

  • Overall Survival (OS)

    Overall Survival (OS) was defined as the time from the date of randomization until death due to any cause. Any patient not known to have died at the time of analysis was censored based on the last recorded date on which the patient was known to be alive. Median OS was calculated using the Kaplan-Meier technique. The second interim analysis was pre-specified after approximately 397 OS events occurred in both arms (59% maturity).

    From date of randomization until death due to any cause. Assessed up to maximum of approximately 27 months (from date of randomization to primary analysis data cut-off)

  • Overall Survival (OS) Rate at 18 Months

    Overall Survival (OS) was defined as the time from the date of randomization until death due to any cause. Any patient not known to have died at the time of analysis was censored based on the last recorded date on which the patient was known to be alive. Median OS was calculated using the Kaplan-Meier technique. The second interim analysis was pre-specified after approximately 397 OS events occurred in both arms (59% maturity).

    From date of randomization until death due to any cause. Calculated at 18 months using the Kaplan-Meier technique.

  • Overall Survival (OS) Rate at 24 Months

    Overall Survival (OS) was defined as the time from the date of randomization until death due to any cause. Any patient not known to have died at the time of analysis was censored based on the last recorded date on which the patient was known to be alive. Median OS was calculated using the Kaplan-Meier technique. The second interim analysis was pre-specified after approximately 397 OS events occurred in both arms (59% maturity).

    From date of randomization until death due to any cause. Calculated at 24 months using the Kaplan-Meier technique.

Secondary Outcomes (11)

  • Progression-free Survival (PFS)

    Tumor assessments every 6 weeks after randomization for the first 24 weeks and then every 8 weeks thereafter until date of RECIST 1.1 defined radiological progressive disease or death. Assessed up to maximum of approximately 27 months.

  • Progression-free Survival (PFS) Rate at 9 Months

    Tumor assessments every 6 weeks after randomization for the first 24 weeks and then every 8 weeks thereafter until date of RECIST 1.1 defined radiological progressive disease or death. Calculated at 9 months using the Kaplan-Meier technique.

  • Progression-free Survival (PFS) Rate at 12 Months

    Tumor assessments every 6 weeks after randomization for the first 24 weeks and then every 8 weeks thereafter until date of RECIST 1.1 defined radiological progressive disease or death. Calculated at 12 months using the Kaplan-Meier technique

  • Objective Response Rate (ORR)

    Tumor assessments (per RECIST 1.1) every 6 weeks for the first 24 weeks relative to the date of randomization and then every 8 weeks thereafter. Assessed up to maximum of approximately 27 months.

  • Duration of Response (DoR)

    Tumor assessments (per RECIST 1.1) every 6 weeks for first 24 weeks relative to the date of randomization and then every 8 weeks thereafter. Assessed up to maximum of approximately 27 months.

  • +6 more secondary outcomes

Study Arms (2)

Treatment Arm

EXPERIMENTAL

Durvalumab + Gemcitabine + Cisplatin

Drug: Durvalumab

Placebo Arm

PLACEBO COMPARATOR

Placebo + Gemcitabine + Cisplatin

Drug: Placebo

Interventions

IV infusion every 3 weeks with gemcitabine plus cisplatin up to 8 cycles followed by monotherapy every 4 weeks until disease progression or other discontinuation criteria.

Treatment Arm

IV infusion every 3 weeks with gemcitabine plus cisplatin up to 8 cycles followed by monotherapy every 4 weeks until disease progression or other discontinuation criteria.

Placebo Arm

Eligibility Criteria

Age18 Years - 130 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Histologically confirmed, unresectable advanced or metastatic biliary tract, including cholangiocarcinoma (intrahepatic or extrahepatic) and gallbladder carcinoma.
  • Patients with previously untreated disease if unresectable or metastatic at initial diagnosis will be eligible.
  • Patient with recurrent disease \>6 months after curative surgery or \>6 months after the completion of adjuvant therapy (chemotherapy and/or radiation) will be eligible.
  • WHO/ECOG PS of 0 or 1

You may not qualify if:

  • History of another primary malignancy
  • Brain metastases or spinal cord compression
  • Uncontrolled intercurrent illness
  • Major surgical procedure within 28 days prior to the first dose of IP.
  • Prior locoregional therapy such as radioembolization

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (126)

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Los Angeles, California, 90027, United States

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Orange, California, 92868, United States

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Washington D.C., District of Columbia, 20007, United States

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Fort Myers, Florida, 33905, United States

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St. Petersburg, Florida, 33705, United States

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Westwood, Kansas, 66205, United States

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Louisville, Kentucky, 40202, United States

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Burlington, Massachusetts, 01805, United States

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St Louis, Missouri, 63110, United States

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Chapel Hill, North Carolina, 27514, United States

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Portland, Oregon, 97213, United States

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Philadelphia, Pennsylvania, 19111, United States

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Chattanooga, Tennessee, 37404, United States

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Nashville, Tennessee, 37203, United States

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Seattle, Washington, 98109, United States

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Buenos Aires, C1118AAT, Argentina

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CABA, C1012AAR, Argentina

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CABA, C1019ABS, Argentina

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Ciudad de Buenos Aires, 1280, Argentina

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Ciudad de Buenos Aires, C1426ANZ, Argentina

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Rosario, 2000, Argentina

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San Salvador de Jujuy, 4600, Argentina

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Burgas, 8000, Bulgaria

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Sofia, 1330, Bulgaria

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Sofia, 1407, Bulgaria

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Sofia, 1606, Bulgaria

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Varna, 9010, Bulgaria

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Santiago, 7630370, Chile

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Temuco, 4810218, Chile

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Baoding, 071000, China

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Beijing, 100021, China

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Beijing, 100142, China

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Bengbu, 233004, China

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Chongqing, 400038, China

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Foshan, 528000, China

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Guangzhou, 510062, China

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Hangzhou, 310003, China

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Hangzhou, 310009, China

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Harbin, 150081, China

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Hefei, 230001, China

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Hefei, 230601, China

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Jinan, 250014, China

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Nanchang, 330006, China

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Nanjing, 210002, China

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Shandong, China

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Shanghai, 200032, China

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Shenyang, 100003, China

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Suzhou, 215004, China

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Xi'an, 710061, China

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Zhengzhou, 450008, China

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Zhuhai, 519000, China

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Clichy, 92210, France

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Dijon, 21079, France

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Montpellier, 34295, France

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Nice, 6189, France

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Paris, 75571, France

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Pessac, 33604, France

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Poitiers, 86021, France

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Hong Kong, 00000, Hong Kong

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Hong Kong, 150001, Hong Kong

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Hong Kong, Hong Kong

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Kowloon, Hong Kong

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Gūrgaon, 122001, India

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Kolkata, 700160, India

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Mumbai, 400012, India

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New Delhi, 110085, India

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Faenza, 48018, Italy

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Florence, 50134, Italy

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Milan, 20132, Italy

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Naples, 80131, Italy

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Roma, 00168, Italy

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Verona, 37134, Italy

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Chūōku, 104-0045, Japan

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Kashiwa, 227-8577, Japan

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Kitaadachi-gun, 362-0806, Japan

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Mitaka-shi, 181-8611, Japan

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Osaka, 541-8567, Japan

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Suita-shi, 565-0871, Japan

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Wakayama, 641-8510, Japan

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Yokohama, 241-8515, Japan

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Gdansk, 80-952, Poland

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Lodz, 93-513, Poland

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Olsztyn, 10-228, Poland

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Poznan, 60-780, Poland

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Warsaw, 02-106, Poland

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Wroclaw, 50-556, Poland

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Barnaul, 656049, Russia

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Kostroma, 156005, Russia

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Moscow, 115478, Russia

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Moscow, 121467, Russia

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Moscow, 125284, Russia

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Omsk, 644033, Russia

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Saint Petersburg, 197022, Russia

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Saint Petersburg, 197758, Russia

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Seongnam-si, 13620, South Korea

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Seoul, 03080, South Korea

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Seoul, 03722, South Korea

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Seoul, 06351, South Korea

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Seoul, 06591, South Korea

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Seoul, 152-703, South Korea

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Chiayi City, 613, Taiwan

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Kaohsiung City, 82445, Taiwan

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Kaohsiung City, 83301, Taiwan

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Taichung, 40705, Taiwan

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Tainan, 704, Taiwan

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Taipei, 10050, Taiwan

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Taipei, 11217, Taiwan

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Taoyuan District, 333, Taiwan

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Bangkok, 10330, Thailand

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Bangkok, 10400, Thailand

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Hat Yai, 90110, Thailand

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Khon Kaen, 40002, Thailand

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Muang, 50200, Thailand

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Si Sa Ket, 33000, Thailand

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Ankara, 06100, Turkey (Türkiye)

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Istanbul, 34030, Turkey (Türkiye)

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Izmir, 35100, Turkey (Türkiye)

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Mersin, 33110, Turkey (Türkiye)

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Bristol, BS2 8ED, United Kingdom

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Cambridge, CB2 0QQ, United Kingdom

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London, NW3 2QG, United Kingdom

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London, W12 0NN, United Kingdom

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Manchester, M20 4BX, United Kingdom

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Oxford, OX3 7LE, United Kingdom

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Romford, RM7 0AG, United Kingdom

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Sheffield, S10 2SJ, United Kingdom

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Related Publications (6)

  • Antonuzzo L, Takahashi H, Park JO, Sookprasert A, Gillmore R, Yang SS, Cundom J, Petrova M, Vaccaro G, Holmblad M, Zotkiewicz M, Wang J, Rokutanda N, Oh DY. Immune-mediated adverse events in the randomized phase 3 TOPAZ-1 study of durvalumab plus gemcitabine and cisplatin in advanced biliary tract cancer. Oncologist. 2025 Jul 4;30(7):oyaf148. doi: 10.1093/oncolo/oyaf148.

  • Oh DY, He AR, Qin S, Chen LT, Okusaka T, Kim JW, Suksombooncharoen T, Lee MA, Kitano M, Burris HA, Bouattour M, Tanasanvimon S, Zaucha R, Avallone A, Cundom J, Kuzko A, Wang J, Xynos I, Vogel A, Valle JW. Durvalumab plus chemotherapy in advanced biliary tract cancer: 3-year overall survival update from the phase III TOPAZ-1 study. J Hepatol. 2025 Nov;83(5):1092-1101. doi: 10.1016/j.jhep.2025.05.003. Epub 2025 May 15.

  • Oh DY, He AR, Bouattour M, Okusaka T, Qin S, Chen LT, Kitano M, Lee CK, Kim JW, Chen MH, Suksombooncharoen T, Ikeda M, Lee MA, Chen JS, Potemski P, Burris HA 3rd, Ostwal V, Tanasanvimon S, Morizane C, Zaucha RE, McNamara MG, Avallone A, Cundom JE, Breder V, Tan B, Shimizu S, Tougeron D, Evesque L, Petrova M, Zhen DB, Gillmore R, Gupta VG, Dayyani F, Park JO, Buchschacher GL Jr, Rey F, Kim H, Wang J, Morgan C, Rokutanda N, Zotkiewicz M, Vogel A, Valle JW. Durvalumab or placebo plus gemcitabine and cisplatin in participants with advanced biliary tract cancer (TOPAZ-1): updated overall survival from a randomised phase 3 study. Lancet Gastroenterol Hepatol. 2024 Aug;9(8):694-704. doi: 10.1016/S2468-1253(24)00095-5. Epub 2024 May 29.

  • Burris HA 3rd, Okusaka T, Vogel A, Lee MA, Takahashi H, Breder V, Blanc JF, Li J, Bachini M, Zotkiewicz M, Abraham J, Patel N, Wang J, Ali M, Rokutanda N, Cohen G, Oh DY. Durvalumab plus gemcitabine and cisplatin in advanced biliary tract cancer (TOPAZ-1): patient-reported outcomes from a randomised, double-blind, placebo-controlled, phase 3 trial. Lancet Oncol. 2024 May;25(5):626-635. doi: 10.1016/S1470-2045(24)00082-2.

  • Keller HR, Fluke L, Forrester JA, Wolf RF. Identifying Indications for Neoadjuvant Therapy in Cholangiocarcinoma. Oncology (Williston Park). 2024 Apr 11;38(4):160-162. doi: 10.46883/2024.25921017.

  • Oh DY, Ruth He A, Qin S, Chen LT, Okusaka T, Vogel A, Kim JW, Suksombooncharoen T, Ah Lee M, Kitano M, Burris H, Bouattour M, Tanasanvimon S, McNamara MG, Zaucha R, Avallone A, Tan B, Cundom J, Lee CK, Takahashi H, Ikeda M, Chen JS, Wang J, Makowsky M, Rokutanda N, He P, Kurland JF, Cohen G, Valle JW. Durvalumab plus Gemcitabine and Cisplatin in Advanced Biliary Tract Cancer. NEJM Evid. 2022 Aug;1(8):EVIDoa2200015. doi: 10.1056/EVIDoa2200015. Epub 2022 Jun 1.

Related Links

MeSH Terms

Conditions

Biliary Tract Neoplasms

Interventions

durvalumab

Condition Hierarchy (Ancestors)

Digestive System NeoplasmsNeoplasms by SiteNeoplasmsBiliary Tract DiseasesDigestive System Diseases

Results Point of Contact

Title
Global Clinical Lead
Organization
AstraZeneca Clinical Study Information Center

Study Officials

  • Gordon Cohen

    AstraZeneca

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restrictive Agreement
No

Study Design

Study Type
interventional
Phase
phase 3
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Model Details: Durvalumab in Combination with Gemcitabine plus Cisplatin Placebo in Combination with Gemcitabine plus Cisplatin
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

March 13, 2019

First Posted

March 14, 2019

Study Start

April 16, 2019

Primary Completion

August 11, 2021

Study Completion (Estimated)

May 16, 2027

Last Updated

March 19, 2026

Results First Posted

April 13, 2023

Record last verified: 2026-02

Data Sharing

IPD Sharing
Will share

Qualified researchers can request access to anonymized individual patient-level data from AstraZeneca group of companies sponsored clinical trials via the request portal. All request will be evaluated as per the AZ disclosure commitment: https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure.

Shared Documents
STUDY PROTOCOL, SAP
Time Frame
AstraZeneca will meet or exceed data availability as per the commitments made to the EFPIA Pharma Data Sharing Principles. For details of our timelines, please rerefer to our disclosure commitment at https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure.
Access Criteria
When a request has been approved AstraZeneca will provide access to the de-identified individual patient-level data in an approved sponsored tool. Signed Data Sharing Agreement (non-negotiable contract for data accessors) must be in place before accessing requested information. Additionally, all users will need to accept the terms and conditions of the SAS MSE to gain access. For additional details, please review the Disclosure Statements at https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure.
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