NCT04163900

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

Trial Health

68
Monitor

Trial Health Score

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

Strong global presence with extensive site network
Enrollment
773

participants targeted

Target at P75+ for phase_3

Timeline
Completed

Started Dec 2019

Geographic Reach
15 countries

125 active sites

Status
terminated

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

First Submitted

Initial submission to the registry

November 1, 2019

Completed
14 days until next milestone

First Posted

Study publicly available on registry

November 15, 2019

Completed
1 month until next milestone

Study Start

First participant enrolled

December 24, 2019

Completed
2.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 2, 2022

Completed
1 month until next milestone

Study Completion

Last participant's last visit for all outcomes

April 5, 2022

Completed
1.1 years until next milestone

Results Posted

Study results publicly available

May 24, 2023

Completed
Last Updated

May 24, 2023

Status Verified

May 1, 2023

Enrollment Period

2.2 years

First QC Date

November 1, 2019

Results QC Date

March 2, 2023

Last Update Submit

May 22, 2023

Conditions

Keywords

AdenocarcinomaAmpullaryAntineoplastic AgentsBiliary Tract CancerChemotherapyCholangiocarcinomaCisplatinDigestive System NeoplasmsDistal Bile DuctExtrahepaticFirst-line ChemotherapyGallbladderGastrointestinalGemcitabineHepatobiliaryIntrahepaticLocally advancedMetastaticNeoplasmNUC-1031ProTidesUntreated

Outcome Measures

Primary Outcomes (2)

  • Objective Response Rate (ORR)

    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.

    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.

  • Overall Survival (OS)

    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.

    From the date of randomization until the date of death from any cause, assessed up to 12 months on average

Study Arms (2)

A - NUC-1031 and cisplatin

EXPERIMENTAL

725 mg/m\^2 NUC-1031 administered in combination with 25 mg/m\^2 cisplatin on Days 1 and 8 of a 21-day cycle

Drug: NUC-1031Drug: Cisplatin

B - gemcitabine and cisplatin

ACTIVE COMPARATOR

1000 mg/m\^2 gemcitabine administered in combination with 25 mg/m\^2 cisplatin on Days 1 and 8 of a 21-day cycle

Drug: GemcitabineDrug: Cisplatin

Interventions

IV infusion in 500 mL of 0.9% sterile saline for injection given over 30 minutes

Also known as: fosgemcitabine palabenamide
A - NUC-1031 and cisplatin

IV infusion in 250 mL of 0.9% sterile saline for injection given in accordance with the package insert

Also known as: Difluorodeoxycytidine, Gemzar
B - gemcitabine and cisplatin

IV in accordance with local institutional practice for biliary tract cancer, including the use of an appropriate hydration protocol

Also known as: CDDP
A - NUC-1031 and cisplatinB - gemcitabine and cisplatin

Eligibility Criteria

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

You may qualify if:

  • Written informed consent and authorization to use and disclose health information.
  • Ability to comprehend and willingness to comply with the requirements of this protocol, including the QoL questionnaires.
  • Female or male patients aged ≥18 years.
  • 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.
  • Life expectancy ≥16 weeks.
  • Eastern Cooperative Oncology Group (ECOG) performance status 0 or 1.
  • 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
  • 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.
  • 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.
  • 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.
  • +2 more criteria

You may not qualify if:

  • Combined or mixed hepatocellular/cholangiocarcinoma.
  • 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.
  • 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).
  • Symptomatic central nervous system or leptomeningeal metastases.
  • 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.
  • 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.
  • 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.
  • Prior exposure to another investigational agent within 28 days prior to randomization.
  • Major surgery within 28 days prior to randomization; patient must have completely recovered from any prior surgical or other procedures.
  • Pregnant or breastfeeding.
  • 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.
  • +3 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (125)

Arizona Oncology Associates , PC - HOPE

Tucson, Arizona, 85711, United States

Location

University of Arizona Cancer Center

Tucson, Arizona, 85719, United States

Location

The Oncology Institute of Hope and Innovation

Whittier, California, 90602-3171, United States

Location

Rocky Mountain Cancer Centers, LLP- Aurora

Aurora, Colorado, 80012, United States

Location

Baptist Health Medical Group Oncology, LLC

Miami, Florida, 33176, United States

Location

Orlando Health, Inc.

Orlando, Florida, 32806, United States

Location

IACT Health

Columbus, Georgia, 31904, United States

Location

Affiliated Oncologists LLC

Chicago Ridge, Illinois, 60415, United States

Location

University of Kansas Medical Center Research Institute, Inc.

Westwood, Kansas, 66205, United States

Location

Norton Cancer Institute

Louisville, Kentucky, 40217, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

Beth Israel Deaconess Medical Center

Boston, Massachusetts, 02215-5400, United States

Location

Henry Ford Medical Group

Detroit, Michigan, 48202, United States

Location

Minnesota Oncology Hemtology

Minneapolis, Minnesota, 55404, United States

Location

Regents of the University of Minnesota

Minneapolis, Minnesota, 55455, United States

Location

University of Rochester Medical Center - Strong Memorial Hospital

Rochester, New York, 14642, United States

Location

The Research Foundation for The State University of New York

Stony Brook, New York, 11794-3369, United States

Location

Wake Forest University Health Sciences

Winston-Salem, North Carolina, 27157, United States

Location

The Ohio State University James Cancer Hospital and Solove Research Institute

Columbus, Ohio, 43210, United States

Location

Corporal Michael J. Crescenz VA Medical Center

Philadelphia, Pennsylvania, 19104, United States

Location

Prisma Health Upstate

Greenville, South Carolina, 29605, United States

Location

Texas Oncology, P.A. - Austin

Austin, Texas, 78705, United States

Location

Baylor Charles A. Sammons Cancer Center

Dallas, Texas, 75246, United States

Location

Joe Arrington Cancer Research and Treatment Center

Lubbock, Texas, 79410, United States

Location

Texas Oncology, P.A. - Tyler

Tyler, Texas, 75702, United States

Location

Virginia Mason Medical Center

Seattle, Washington, 98101, United States

Location

Northwest Cancer Specialists, P.C.-Vancouver

Vancouver, Washington, 98684, United States

Location

Wenatchee Valley Hospital and Clinics

Wenatchee, Washington, 98801, United States

Location

The Medical College of Wisconsin, Inc.

Milwaukee, Wisconsin, 53226, United States

Location

Chris O'Brien Lifehouse

Camperdown, New South Wales, 2050, Australia

Location

St George Hospital

Kogarah, New South Wales, 2217, Australia

Location

Newcastle Private Hospital

Newcastle, New South Wales, 2305, Australia

Location

Westmead Hospital

Westmead, New South Wales, 2145, Australia

Location

Townsville Cancer Centre

Townsville, Queensland, 4814, Australia

Location

Warringal Medical Centre

Heidelberg, Victoria, 3084, Australia

Location

The Alfred Hospital

Melbourne, Victoria, 3004, Australia

Location

Fiona Stanley Hospital

Murdoch, Western Australia, 6150, Australia

Location

Sir Charles Gairdner Hospital

Nedlands, Western Australia, 6009, Australia

Location

Tom Baker Cancer Centre

Calgary, Alberta, T2N 4N2, Canada

Location

Nova Scotia Health Authority

Halifax, Nova Scotia, B3H1V7, Canada

Location

Royal Victoria Regional Health Centre

Barrie, Ontario, L4M 6M2, Canada

Location

The Ottawa Hospital Cancer Centre

Ottawa, Ontario, K1H 8L6, Canada

Location

Sunnybrook Research Institute

Toronto, Ontario, M4N 3M5, Canada

Location

Princess Margaret Cancer Centre

Toronto, Ontario, M5G 1X6, Canada

Location

CHUM Centre de Recherche

Montreal, Quebec, H2X 0A9, Canada

Location

SMBD Jewish General Hospital

Montreal, Quebec, H3T 1E2, Canada

Location

Fakultní nemocnice Brno

Brno, 625 00, Czechia

Location

Fakultní nemocnice Hradec Králové

Hradec Králové, 500 05, Czechia

Location

Fakultní nemocnice Olomouc

Olomouc, 775 20, Czechia

Location

Thomayerova nemocnice

Prague, 140 59, Czechia

Location

Nemocnice Na Homolce

Prague, 150 30, Czechia

Location

Centre Georges François Leclerc

Dijon, 21079, France

Location

CHU de Grenoble - Hôpital Nord

Grenoble, 38043, France

Location

Institut Hospitalier Franco-Britannique

Levallois-Perret, 92300, France

Location

Hôpital Cochin

Paris, 75679, France

Location

ICO - Site René Gauducheau

Saint-Herblain, 44805, France

Location

Universitaetsklinikum Heidelberg

Heidelberg, Baden-Wurttemberg, 69120, Germany

Location

Vivantes Klinikum Neukoelln

Berlin, 12351, Germany

Location

Universitaetsklinikum Freiburg

Freiburg im Breisgau, 79106, Germany

Location

Medizinische Hochschule Hannover

Hanover, 30625, Germany

Location

Universitaetsklinikum Tuebingen

Tübingen, 72076, Germany

Location

Semmelweis Egyetem

Budapest, 1083, Hungary

Location

Del-pesti Centrumkorhaz - Orszagos Hematologiai es Infektologiai Intezet

Budapest, 1097, Hungary

Location

Orszagos Onkologiai Intezet

Budapest, 1122, Hungary

Location

Debreceni Egyetem

Debrecen, 4032, Hungary

Location

Borsod-Abauj-Zemplen Megyei Kozponti Korhaz es Egyetemi Oktatokorhaz

Miskolc, 3526, Hungary

Location

Jasz-Nagykun-Szolnok Megyei Hetenyi Geza Korhaz-Rendelointezet

Szolnok, 5004, Hungary

Location

Ospedale Policlinico San Martino

Genova, 16132, Italy

Location

IEO Istituto Europeo di Oncologia

Milan, 20141, Italy

Location

Istituto Nazionale Tumori Fondazione G. Pascale

Napoli, 80131, Italy

Location

IOV - Istituto Oncologico Veneto IRCCS

Padua, 35128, Italy

Location

Azienda Ospedaliero Universitaria Pisana

Pisa, 56126, Italy

Location

Centro Ricerche Cliniche di Verona S.r.l

Verona, 37124, Italy

Location

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

Moscow, 115478, Russia

Location

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

Moscow, 115478, Russia

Location

"VitaMed" LLC

Moscow, 129515, Russia

Location

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

Saint Petersburg, 191104, Russia

Location

Pavlov First Saint Petersburg State Medical University

Saint Petersburg, 197022, Russia

Location

SPb SBIH "City Clinical Oncological Dispensary"

Saint Petersburg, 197022, Russia

Location

Medicinskiy gorod

Tyumen, 625041, Russia

Location

CHA Bundang Medical Center, CHA University

Seongnam-si, Gyeonggi-do, 13496, South Korea

Location

Chonnam National University Hwasun Hospital

Hwasun, Jeollanam-do, 58128, South Korea

Location

Dong-A University Hospital

Pusan, 602-715, South Korea

Location

Seoul National University Bundang Hospital

Seongnam-si, 13620, South Korea

Location

Korea University Anam Hospital

Seoul, 02841, South Korea

Location

Seoul National University Hospital

Seoul, 03080, South Korea

Location

Samsung Medical Center

Seoul, 06351, South Korea

Location

Korea University Guro Hospital

Seoul, 08308, South Korea

Location

Severance Hospital, Yonsei University

Seoul, 3722, South Korea

Location

Asan Medical Center

Seoul, 5505, South Korea

Location

Hospital Universitari Vall d'Hebron

Barcelona, 08035, Spain

Location

Hospital Clinic de Barcelona

Barcelona, 08036, Spain

Location

Hospital de la Santa Creu i Sant Pau

Barcelona, 08041, Spain

Location

ICO l'Hospitalet - Hospital Duran i Reynals

L'Hospitalet de Llobregat, 08908, Spain

Location

Hospital General Universitario Gregorio Marañon

Madrid, 28007, Spain

Location

Hospital Universitario Clinico San Carlos

Madrid, 28040, Spain

Location

Hospital Universitario HM Madrid Sanchinarro

Madrid, 28050, Spain

Location

Hospital Universitario Virgen Macarena

Seville, 41009, Spain

Location

Hospital Universitario Virgen del Rocio

Seville, 41013, Spain

Location

Changhua Christian Medical Foundation Changhua Christian Hospital

Changhua, 50004, Taiwan

Location

China Medical University Hospital

Taichung, 40447, Taiwan

Location

National Taiwan University Hospital

Taipei, 10016, Taiwan

Location

MacKay Medical Foundation The Presbyterian Church in Taiwan MacKay Memorial Hospital

Taipei, 10449, Taiwan

Location

Taipei Veterans General Hospital

Taipei, 11217, Taiwan

Location

Acibadem Adana Hospital

Adana, 01130, Turkey (Türkiye)

Location

Baskent University Adana Application and Research Center

Adana, 01220, Turkey (Türkiye)

Location

Akdeniz University Medical Faculty

Antalya, 07058, Turkey (Türkiye)

Location

Trakya University Medical Faculty

Edirne, 22030, Turkey (Türkiye)

Location

Istanbul University Cerrahpasa - Cerrahpasa Medical Faculty

Istanbul, 34098, Turkey (Türkiye)

Location

Dr. Abdurrahman Yurtaslan Oncology Teaching and Research Hospital

Malatya, 06105, Turkey (Türkiye)

Location

Inonu University Medical Facility

Malatya, 44280, Turkey (Türkiye)

Location

CI Chernivtsi RC Oncological Dispensary

Chernivtsi, 58013, Ukraine

Location

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

Kharkiv, 61070, Ukraine

Location

CI of Healthcare Regional Clinical Specialized Dispensary of the Radiation Protection

Kharkiv, 61166, Ukraine

Location

Kyiv City Clinical Oncological Center

Kyiv, 03115, Ukraine

Location

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

Kyiv, 03126, Ukraine

Location

Treatment-Prevention Institution Volyn Regional Oncological Dispensary

Lutsk, 43018, Ukraine

Location

Communal Institution Odesa Regional Clinical Hospital

Odesa, 65025, Ukraine

Location

RCI Sumy Regional Clinical Oncological Dispensary

Sumy, 40022, Ukraine

Location

Guy's Hospital

London, Greater London, SE1 9RY, United Kingdom

Location

The Christie

Manchester, Greater Manchester, M20 4BX, United Kingdom

Location

Western General Hospital

Edinburgh, EH4 2XU, United Kingdom

Location

Beatson West of Scotland Cancer Centre

Glasgow, G12 OYN, United Kingdom

Location

The Clatterbridge Cancer Centre

Metropolitan Borough of Wirral, CH63 4JY, United Kingdom

Location

Torbay Hospital

Torquay, TQ2 7AA, United Kingdom

Location

Related Publications (2)

  • Knox JJ, McNamara MG, Bazin IS, Oh DY, Zubkov O, Breder V, Bai LY, Christie A, Goyal L, Cosgrove DP, Springfeld C, Sjoquist KM, Park JO, Verdaguer H, Braconi C, Ross PJ, De Gramont A, Shroff RT, Zalcberg JR, Palmer DH, Smith JR, Oelmann E, Bruce T, Valle JW. A phase III randomized study of first-line NUC-1031/cisplatin vs. gemcitabine/cisplatin in advanced biliary tract cancer. J Hepatol. 2025 Aug;83(2):358-366. doi: 10.1016/j.jhep.2025.01.040. Epub 2025 Feb 18.

  • McNamara MG, Goyal L, Doherty M, Springfeld C, Cosgrove D, Sjoquist KM, Park JO, Verdaguer H, Braconi C, Ross PJ, Gramont A, Zalcberg JR, Palmer DH, Valle JW, Knox JJ. NUC-1031/cisplatin versus gemcitabine/cisplatin in untreated locally advanced/metastatic biliary tract cancer (NuTide:121). Future Oncol. 2020 Jun;16(16):1069-1081. doi: 10.2217/fon-2020-0247. Epub 2020 May 6.

MeSH Terms

Conditions

Biliary Tract NeoplasmsAdenocarcinomaCholangiocarcinomaDigestive System NeoplasmsNeoplasm MetastasisNeoplasms

Interventions

NUC-1031GemcitabineCisplatin

Condition Hierarchy (Ancestors)

Neoplasms by SiteBiliary Tract DiseasesDigestive System DiseasesCarcinomaNeoplasms, Glandular and EpithelialNeoplasms by Histologic TypeNeoplastic ProcessesPathologic ProcessesPathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Heterocyclic CompoundsDeoxycytidineCytidinePyrimidine NucleosidesPyrimidinesHeterocyclic Compounds, 1-RingChlorine CompoundsInorganic ChemicalsNitrogen CompoundsPlatinum Compounds

Limitations and Caveats

At Interim Analysis 1, the futility boundary for OS was crossed and the study was closed on 02 Mar 2022. Following study closure, data from the 05 April 2022 final data cut-off (30 day follow-up post the last patient last visit) were analysed. In general, the analyses performed on this final database were those that were scheduled to take place at Interim Analysis 2. However, any p-values or CIs obtained are only viewed as descriptive as the study was stopped for futility.

Results Point of Contact

Title
Medical and Scientific Affairs Department
Organization
NuCana plc

Study Officials

  • Jennifer Knox, MD

    Professor of Medicine, University of Toronto

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
No
Restrictive Agreement
No

Study Design

Study Type
interventional
Phase
phase 3
Allocation
RANDOMIZED
Masking
SINGLE
Who Masked
OUTCOMES ASSESSOR
Masking Details
Imaging scans will be assessed by blinded independent review according to RECIST v1.1
Purpose
TREATMENT
Intervention Model
PARALLEL
Model Details: 1:1 randomization model to either Arm A or Arm B
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

November 1, 2019

First Posted

November 15, 2019

Study Start

December 24, 2019

Primary Completion

March 2, 2022

Study Completion

April 5, 2022

Last Updated

May 24, 2023

Results First Posted

May 24, 2023

Record last verified: 2023-05

Data Sharing

IPD Sharing
Will not share

Locations