MedPath

Comparing NUC-1031 Plus Cisplatin to Gemcitabine Plus Cisplatin in Patients With Advanced Biliary Tract Cancer

Phase 3
Terminated
Conditions
Biliary Tract Cancer
Interventions
Registration Number
NCT04163900
Lead Sponsor
NuCana plc
Brief Summary

NuTide:121 compares NUC-1031 with gemcitabine, both in combination with cisplatin, in patients with previously untreated advanced biliary tract cancer.

The primary hypotheses are:

* The combination of NUC-1031 plus cisplatin prolongs overall survival compared to the gemcitabine plus cisplatin standard of care

* The combination of NUC-1031 plus cisplatin increases overall response rate compared to the gemcitabine plus cisplatin standard of care

Detailed Description

Not available

Recruitment & Eligibility

Status
TERMINATED
Sex
All
Target Recruitment
773
Inclusion Criteria
  1. Written informed consent and authorization to use and disclose health information.

  2. Ability to comprehend and willingness to comply with the requirements of this protocol, including the QoL questionnaires.

  3. Female or male patients aged ≥18 years.

  4. Histologically- or cytologically-confirmed adenocarcinoma of the biliary tract (including gallbladder, intra and extra-hepatic biliary ducts and ampullary cancers) that is locally advanced, unresectable or metastatic (AJCC edition 8, 2018). Patients with measurable (as per RECIST v1.1 criteria) or non-measurable disease are permitted.

  5. Life expectancy ≥16 weeks.

  6. Eastern Cooperative Oncology Group (ECOG) performance status 0 or 1.

  7. Adequate biliary drainage with no evidence of ongoing infection. If applicable, treatable and clinically-relevant biliary duct obstruction has been relieved by internal endoscopic drainage/stenting at least 2 weeks previously or by palliative bypass surgery or percutaneous drainage prior to study treatment, and the patient has no active or suspected uncontrolled infection. Patients fitted with a biliary stent should be clinically stable and free of signs of infection for ≥2 weeks prior to study treatment. Patients with improving biliary function who meet all other inclusion criteria may be re-tested during the screening window.

  8. Adequate bone marrow, hepatic, and renal function, as evidenced by:

    • Absolute neutrophil count (ANC) ≥1,500/μL without colony-stimulating factor support
    • Platelet count ≥100,000/μL
    • Haemoglobin ≥9 g/dL without need for haematopoietic growth factor or transfusion support in prior 2 weeks
    • Total bilirubin <2 × upper limit of normal (ULN); does not apply to patients with Gilbert's syndrome. Consistent with inclusion criterion 7, patients whose whole bilirubin and biliary function is recovering may be re-tested during the screening period.
    • Alanine aminotransferase (ALT) and/or aspartate aminotransferase (AST) <5 × ULN
    • Creatinine clearance ≥45 mL/min actual or calculated by the Cockcroft-Gault method
    • International normalized ratio (INR) <1.5 and activated partial thromboplastin time (aPTT) <1.5 × ULN; does not apply to patients on an anti-coagulant with stable dose 28 days prior to first dose.
  9. QTc interval <450 msec (males) or <470 msec (females), in the absence of bundle branch block. In the presence of bundle branch block with consequent QTc prolongation, patients may be enrolled based on a careful risk-benefit assessment.

  10. Human Immunodeficiency Virus-infected patients who are healthy and have a low risk of Acquired Immunodeficiency Syndrome-related outcomes may be included in this study.

  11. Female patients of child-bearing potential (i.e., all women except those who are post-menopausal for ≥1 year or who have a history of hysterectomy or surgical sterilization) must have a negative pregnancy test within 3 days prior to the first study drug administration. All patients of child-bearing potential must agree to practice true abstinence or to use two highly effective forms of contraception, one of which must be a barrier method of contraception, from the time of screening until 6 months after the last dose of study medication.

  12. Male patients with a female partner must either have had a successful vasectomy or they and their female partner meet the criteria above (not of childbearing potential or practicing highly effective contraceptive methods).

Exclusion Criteria
  1. Combined or mixed hepatocellular/cholangiocarcinoma.

  2. Prior systemic therapy for advanced or metastatic biliary tract cancer. However, prior chemotherapy in the adjuvant setting or low-dose chemotherapy given in conjunction with radiotherapy in the adjuvant setting and completed at least 6 months prior to enrolment is permitted. The following prior interventions are allowed provided the patient has fully recovered:

    • Surgery: non-curative resection with macroscopic residual disease or palliative bypass surgery. Patients who have previously undergone curative surgery must now have evidence of non-resectable disease requiring systemic chemotherapy.
    • Radiotherapy: prior radiotherapy (with or without radio-sensitizing low-dose chemotherapy) for localized disease and there is now clear evidence of disease progression requiring systemic chemotherapy.
    • Photodynamic therapy: prior photodynamic therapy for localized disease with no evidence of metastatic disease or for localized disease to relieve biliary obstruction in the presence of metastatic disease provided there is now clear evidence of disease progression requiring systemic chemotherapy.
    • Palliative radiotherapy: palliative radiotherapy provided that all adverse events have resolved and the patient has measurable disease outside the field of radiation.
  3. Prior treatment with or known hypersensitivity to NUC-1031, gemcitabine, cisplatin or other platinum-based agents or history of allergic reactions attributed to any parenteral excipients (e.g. dimethylacetamide [DMA], Cremophor EL, Polysorbate 80, Solutol HS 15).

  4. Symptomatic central nervous system or leptomeningeal metastases.

  5. History of other malignancies, except adequately treated non-melanoma skin cancer, curatively treated in situ cancer of the cervix, surgically excised or potentially curatively treated ductal carcinoma in situ of the breast, or low grade prostate cancer or patients after prostatectomy not requiring treatment. Patients with previous invasive cancers are eligible if treatment was completed more than 3 years prior to initiating the current study treatment, and the patient has had no evidence of recurrence since then.

  6. Concurrent serious (as deemed by the Investigator) medical conditions, including, but not limited to, New York Heart Association class III or IV congestive heart failure, history of congenital prolonged QT syndrome, uncontrolled infection, active hepatitis B or C, or other co-morbid conditions that in the opinion of the Investigator would impair study participation or cooperation.

  7. Congenital or acquired immunodeficiency (e.g., serious active infection with HIV). As per inclusion criterion 10, patients with HIV who are healthy and have a low risk of AIDS related outcomes are eligible.

  8. Other acute or chronic medical, neurological, or psychiatric condition or laboratory abnormality that may increase the risk associated with study participation or investigational product administration or may interfere with the interpretation of study results and, in the judgment of the Investigator, would make the patient inappropriate for entry into this study.

  9. Prior exposure to another investigational agent within 28 days prior to randomization.

  10. Major surgery within 28 days prior to randomization; patient must have completely recovered from any prior surgical or other procedures.

  11. Pregnant or breastfeeding.

  12. Residual toxicities from prior treatments or procedures which have not regressed to Grade ≤1 severity (CTCAE v5.0), except for alopecia or ≤ Grade 2 peripheral neuropathy.

  13. Concomitant use of drugs at doses known to cause clinically relevant prolongation of QT/QTc interval.

  14. Administration of a live vaccination within 28 days prior to randomization.

  15. Ongoing or recent (≤6 months) hepatorenal syndrome.

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
A - NUC-1031 and cisplatinNUC-1031725 mg/m\^2 NUC-1031 administered in combination with 25 mg/m\^2 cisplatin on Days 1 and 8 of a 21-day cycle
B - gemcitabine and cisplatinCisplatin1000 mg/m\^2 gemcitabine administered in combination with 25 mg/m\^2 cisplatin on Days 1 and 8 of a 21-day cycle
A - NUC-1031 and cisplatinCisplatin725 mg/m\^2 NUC-1031 administered in combination with 25 mg/m\^2 cisplatin on Days 1 and 8 of a 21-day cycle
B - gemcitabine and cisplatinGemcitabine1000 mg/m\^2 gemcitabine administered in combination with 25 mg/m\^2 cisplatin on Days 1 and 8 of a 21-day cycle
Primary Outcome Measures
NameTimeMethod
Overall Survival (OS)From the date of randomization until the date of death from any cause, assessed up to 12 months on average

The median time, in months, from the date of randomization to the date of death from any cause. For patients who were alive at the time of a data cut-off or were permanently lost to follow up, duration of OS was censored at the date at which they were last known to be alive.

Objective Response Rate (ORR)Evaluated at Screening (baseline) then every 9 weeks from treatment start (C1D1), or every 12 weeks if treatment is stopped with no evidence of progression, until disease progression or death from any cause up to the end of study, an average of 6 months.

Percentage of patients achieving a confirmed complete response (CR) or partial response (PR) to treatment as assessed by blinded independent review according to RECIST v1.1 criteria. ORR = patients with CR + patients with PR, where" CR = disappearance of all target lesions PR = at least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum of diameters This outcome was assessed in the population of patients with measurable disease at baseline who were also randomized ≥28 weeks before the data cut-off (ITTMD28). Patients were to receive a confirmatory scan 28-42 days after response is first observed.

Secondary Outcome Measures
NameTimeMethod

Trial Locations

Locations (125)

University of Kansas Medical Center Research Institute, Inc.

🇺🇸

Westwood, Kansas, United States

The Research Foundation for The State University of New York

🇺🇸

Stony Brook, New York, United States

The Ohio State University James Cancer Hospital and Solove Research Institute

🇺🇸

Columbus, Ohio, United States

Virginia Mason Medical Center

🇺🇸

Seattle, Washington, United States

The Medical College of Wisconsin, Inc.

🇺🇸

Milwaukee, Wisconsin, United States

IEO Istituto Europeo di Oncologia

🇮🇹

Milano, Italy

IOV - Istituto Oncologico Veneto IRCCS

🇮🇹

Padova, Italy

Azienda Ospedaliero Universitaria Pisana

🇮🇹

Pisa, Italy

Baptist Health Medical Group Oncology, LLC

🇺🇸

Miami, Florida, United States

University of Arizona Cancer Center

🇺🇸

Tucson, Arizona, United States

Rocky Mountain Cancer Centers, LLP- Aurora

🇺🇸

Aurora, Colorado, United States

The Oncology Institute of Hope and Innovation

🇺🇸

Whittier, California, United States

Orlando Health, Inc.

🇺🇸

Orlando, Florida, United States

Wake Forest University Health Sciences

🇺🇸

Winston-Salem, North Carolina, United States

Royal Victoria Regional Health Centre

🇨🇦

Barrie, Ontario, Canada

Fakultní nemocnice Olomouc

🇨🇿

Olomouc, Czechia

Fiona Stanley Hospital

🇦🇺

Murdoch, Western Australia, Australia

Texas Oncology, P.A. - Tyler

🇺🇸

Tyler, Texas, United States

St George Hospital

🇦🇺

Kogarah, New South Wales, Australia

Tom Baker Cancer Centre

🇨🇦

Calgary, Alberta, Canada

ICO - Site René Gauducheau

🇫🇷

Saint Herblain, France

Thomayerova nemocnice

🇨🇿

Prague, Czechia

Medizinische Hochschule Hannover

🇩🇪

Hanover, Germany

Fakultní nemocnice Brno

🇨🇿

Brno, Czechia

Fakultní nemocnice Hradec Králové

🇨🇿

Hradec Králové, Czechia

Nemocnice Na Homolce

🇨🇿

Prague, Czechia

Centre Georges François Leclerc

🇫🇷

Dijon, France

Western General Hospital

🇬🇧

Edinburgh, United Kingdom

Hospital de la Santa Creu i Sant Pau

🇪🇸

Barcelona, Spain

Universitaetsklinikum Heidelberg

🇩🇪

Heidelberg, Baden Wuerttemberg, Germany

Korea University Anam Hospital

🇰🇷

Seoul, Korea, Republic of

Severance Hospital, Yonsei University

🇰🇷

Seoul, Korea, Republic of

FSBSI "Russian Oncological Scientific Center n.a. N.N. Blokhin"

🇷🇺

Moscow, Russian Federation

Centro Ricerche Cliniche di Verona S.r.l

🇮🇹

Verona, Italy

CHU de Grenoble - Hôpital Nord

🇫🇷

Grenoble, France

Institut Hospitalier Franco-Britannique

🇫🇷

Levallois-Perret, France

Trakya University Medical Faculty

🇹🇷

Edirne, Turkey

Hôpital Cochin

🇫🇷

Paris, France

ICO l'Hospitalet - Hospital Duran i Reynals

🇪🇸

L'Hospitalet De Llobregat, Spain

Vivantes Klinikum Neukoelln

🇩🇪

Berlin, Germany

Istituto Nazionale Tumori Fondazione G. Pascale

🇮🇹

Napoli, Italy

Jasz-Nagykun-Szolnok Megyei Hetenyi Geza Korhaz-Rendelointezet

🇭🇺

Szolnok, Hungary

Del-pesti Centrumkorhaz - Orszagos Hematologiai es Infektologiai Intezet

🇭🇺

Budapest, Hungary

Universitaetsklinikum Freiburg

🇩🇪

Freiburg, Germany

Borsod-Abauj-Zemplen Megyei Kozponti Korhaz es Egyetemi Oktatokorhaz

🇭🇺

Miskolc, Hungary

Semmelweis Egyetem

🇭🇺

Budapest, Hungary

Ospedale Policlinico San Martino

🇮🇹

Genova, Italy

FSBSI Russian Oncological Scientific Center n.a. N.N. Blokhin

🇷🇺

Moscow, Russian Federation

State Budget Institution of Healthcare "Leningrad Regional Clinical Oncology Dispensary"

🇷🇺

Saint Petersburg, Russian Federation

Korea University Guro Hospital

🇰🇷

Seoul, Korea, Republic of

Seoul National University Bundang Hospital

🇰🇷

Seongnam-si, Korea, Republic of

Dong-A University Hospital

🇰🇷

Pusan, Korea, Republic of

Chonnam National University Hwasun Hospital

🇰🇷

Hwasun, Jeollanam-do, Korea, Republic of

CHA Bundang Medical Center, CHA University

🇰🇷

Seongnam-si, Gyeonggi-do, Korea, Republic of

Samsung Medical Center

🇰🇷

Seoul, Korea, Republic of

Hospital Universitario HM Madrid Sanchinarro

🇪🇸

Madrid, Spain

SPb SBIH "City Clinical Oncological Dispensary"

🇷🇺

Saint Petersburg, Russian Federation

Medicinskiy gorod

🇷🇺

Tyumen, Russian Federation

"VitaMed" LLC

🇷🇺

Moscow, Russian Federation

Pavlov First Saint Petersburg State Medical University

🇷🇺

Saint Petersburg, Russian Federation

Baskent University Adana Application and Research Center

🇹🇷

Adana, Turkey

Asan Medical Center

🇰🇷

Seoul, Korea, Republic of

The Christie

🇬🇧

Manchester, Greater Manchester, United Kingdom

Beatson West of Scotland Cancer Centre

🇬🇧

Glasgow, United Kingdom

Hospital Universitario Virgen del Rocio

🇪🇸

Sevilla, Spain

Changhua Christian Medical Foundation Changhua Christian Hospital

🇨🇳

Changhua, Taiwan

Acibadem Adana Hospital

🇹🇷

Adana, Turkey

Akdeniz University Medical Faculty

🇹🇷

Antalya, Turkey

Dr. Abdurrahman Yurtaslan Oncology Teaching and Research Hospital

🇹🇷

Malatya, Turkey

Inonu University Medical Facility

🇹🇷

Malatya, Turkey

Kyiv City Clinical Oncological Center

🇺🇦

Kyiv, Ukraine

Treatment-Prevention Institution Volyn Regional Oncological Dispensary

🇺🇦

Luts'k, Ukraine

Hospital Universitari Vall d'Hebron

🇪🇸

Barcelona, Spain

Hospital General Universitario Gregorio Marañon

🇪🇸

Madrid, Spain

CI Chernivtsi RC Oncological Dispensary

🇺🇦

Chernivtsi, Ukraine

Communal Non-profit Enterprise Regional Center of Oncology, Kharkiv NMU

🇺🇦

Kharkiv, Ukraine

CI of Healthcare Regional Clinical Specialized Dispensary of the Radiation Protection

🇺🇦

Kharkiv, Ukraine

The Clatterbridge Cancer Centre

🇬🇧

Wirral, United Kingdom

Universitaetsklinikum Tuebingen

🇩🇪

Tuebingen, Germany

Hospital Clinic de Barcelona

🇪🇸

Barcelona, Spain

Hospital Universitario Virgen Macarena

🇪🇸

Sevilla, Spain

Arizona Oncology Associates , PC - HOPE

🇺🇸

Tucson, Arizona, United States

Massachusetts General Hospital

🇺🇸

Boston, Massachusetts, United States

Hospital Universitario Clinico San Carlos

🇪🇸

Madrid, Spain

IACT Health

🇺🇸

Columbus, Georgia, United States

Affiliated Oncologists LLC

🇺🇸

Chicago Ridge, Illinois, United States

Norton Cancer Institute

🇺🇸

Louisville, Kentucky, United States

Beth Israel Deaconess Medical Center

🇺🇸

Boston, Massachusetts, United States

Minnesota Oncology Hemtology

🇺🇸

Minneapolis, Minnesota, United States

University of Rochester Medical Center - Strong Memorial Hospital

🇺🇸

Rochester, New York, United States

Corporal Michael J. Crescenz VA Medical Center

🇺🇸

Philadelphia, Pennsylvania, United States

Regents of the University of Minnesota

🇺🇸

Minneapolis, Minnesota, United States

Texas Oncology, P.A. - Austin

🇺🇸

Austin, Texas, United States

Baylor Charles A. Sammons Cancer Center

🇺🇸

Dallas, Texas, United States

Joe Arrington Cancer Research and Treatment Center

🇺🇸

Lubbock, Texas, United States

Wenatchee Valley Hospital and Clinics

🇺🇸

Wenatchee, Washington, United States

Northwest Cancer Specialists, P.C.-Vancouver

🇺🇸

Vancouver, Washington, United States

Newcastle Private Hospital

🇦🇺

Newcastle, New South Wales, Australia

Chris O'Brien Lifehouse

🇦🇺

Camperdown, New South Wales, Australia

Westmead Hospital

🇦🇺

Westmead, New South Wales, Australia

Sir Charles Gairdner Hospital

🇦🇺

Nedlands, Western Australia, Australia

Sunnybrook Research Institute

🇨🇦

Toronto, Ontario, Canada

The Ottawa Hospital Cancer Centre

🇨🇦

Ottawa, Ontario, Canada

Princess Margaret Cancer Centre

🇨🇦

Toronto, Ontario, Canada

Debreceni Egyetem

🇭🇺

Debrecen, Hungary

Torbay Hospital

🇬🇧

Torquay, United Kingdom

Seoul National University Hospital

🇰🇷

Seoul, Korea, Republic of

CHUM Centre de Recherche

🇨🇦

Montréal, Quebec, Canada

SMBD Jewish General Hospital

🇨🇦

Montréal, Quebec, Canada

China Medical University Hospital

🇨🇳

Taichung, Taiwan

Communal Institution Odesa Regional Clinical Hospital

🇺🇦

Odesa, Ukraine

Nova Scotia Health Authority

🇨🇦

Halifax, Nova Scotia, Canada

Prisma Health Upstate

🇺🇸

Greenville, South Carolina, United States

Istanbul University Cerrahpasa - Cerrahpasa Medical Faculty

🇹🇷

Istanbul, Turkey

SI "Shalimov's National Institute of Surgery and Transplantation" of NAMSU

🇺🇦

Kyiv, Ukraine

RCI Sumy Regional Clinical Oncological Dispensary

🇺🇦

Sumy, Ukraine

Henry Ford Medical Group

🇺🇸

Detroit, Michigan, United States

MacKay Medical Foundation The Presbyterian Church in Taiwan MacKay Memorial Hospital

🇨🇳

Taipei, Taiwan

Townsville Cancer Centre

🇦🇺

Townsville, Queensland, Australia

Warringal Medical Centre

🇦🇺

Heidelberg, Victoria, Australia

The Alfred Hospital

🇦🇺

Melbourne, Victoria, Australia

Orszagos Onkologiai Intezet

🇭🇺

Budapest, Hungary

National Taiwan University Hospital

🇨🇳

Taipei, Taiwan

Taipei Veterans General Hospital

🇨🇳

Taipei, Taiwan

Guy's Hospital

🇬🇧

London, Greater London, United Kingdom

© Copyright 2025. All Rights Reserved by MedPath