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Cancer of Unknown Primary SitePhase 2CUPISCONCT03498521

A Phase II Randomized Study Comparing the Efficacy and Safety of Targeted Therapy or Cancer Immunotherapy Versus Platinum-Based Chemotherapy in Patients With Cancer of Unknown Primary Site

A Phase 2 study testing Alectinib, Vismodegib and Ipatasertib in people with Cancer of Unknown Primary Site, run by Hoffmann-La Roche at 125 sites in 33 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial summary

This study will compare the efficacy and safety of molecularly-guided therapy versus standard platinum-containing chemotherapy in participants with poor-prognosis cancer of unknown primary site (CUP; non-specific subset) who have achieved disease control after 3 cycles of first-line platinum based induction… Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Cancer of Unknown Primary Site
What is being tested?
Alectinib, Vismodegib and Ipatasertib
Could I receive a placebo?
No, everyone receives an active treatment
How many people
529 participants
Where
125 sitesin 33 countries
Study dates
10 Jul 2018to 7 Nov 2024
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Histologically-confirmed unresectable cancer of unknown primary site (CUP) diagnosed according to criteria defined in the 2015 European Society for Medical Oncology (ESMO) Clinical Practice Guidelines for CUP
  • No prior lines of systemic therapy for the treatment of CUP
  • Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1
  • Candidate for platinum-based chemotherapy (according to the reference information for the intended chemotherapy)
  • At least one measurable lesion according to Response Evaluation Criteria in Solid Tumors, version 1.1 (RECIST v1.1)
  • Formalin-Fixed Paraffin-Embedded (FFPE) tumor tissue sample </= 4 months old that is expected to be sufficient for generation of a comprehensive genomic profile at a central reference pathology laboratory

Who cannot join

  • Squamous cell CUP
  • Participants who can be assigned to a specific subset of CUP for which a specific treatment is recommended by the 2015 ESMO Clinical Practice Guidelines for CUP or with a clinical and IHC profile indicative of a specific primary tumor (favorable prognosis CUP subsets): Poorly differentiated carcinoma with midline distribution; women with papillary adenocarcinoma of the peritoneal cavity; women with adenocarcinoma involving only the axillary lymph nodes; squamous cell carcinoma of the cervical lymph nodes; poorly differentiated neuroendocrine tumors; men with blastic bone metastases and elevated prostate-specific antigen (PSA); participants with a single, small, potentially resectable tumor; colon cancer-type CUP, including participants with a CK7 negative, CK20 positive, CDX-2 positive immunohistochemistry profile; CK7-positive, CK20-negative and TTF-1 positive tumors in a context suggestive of lung adenocarcinoma or thyroid cancer; IHC profile definitely indicative of breast cancer OR an IHC profile indicative of breast cancer and either a history of breast cancer or lymph nodes in the drainage areas of the breast; high-grade serious carcinoma histology and elevated CA125 tumor marker and/or a mass in the gynecological tract or any tumor mass or lymph node in the abdominal cavity; IHC profile suggestive of renal cell carcinoma and renal lesions, with a Bosniak classification higher than IIF; IHC profile compatible with cholangiocarcinoma or pancreatobiliary (or upper gastrointestinal carcinoma) AND 1 or 2 liver lesions without extrahepatic disease or with only pulmonary metastases and/or lymph nodes in the drainage areas of the liver
  • Known presence of brain or spinal cord metastasis (including metastases that have been irradiated only)
  • Histology and immunohistology profiles (per 2015 ESMO guidelines) that are not adenocarcinoma or poorly differentiated carcinoma/adenocarcinoma
  • History or known presence of leptomeningeal disease
  • Known human immunodeficiency virus (HIV) infection
Show all 9Show fewer
  • Significant cardiovascular disease
  • Prior allogeneic stem cell or solid organ transplantation
  • Pregnancy or breastfeeding, or intention of becoming pregnant during study treatment or for up to 7 months after the final dose of treatment

Trial locations and contacts

125 sites in 33 countries.

125 sites in 33 countries

France15 sites

  • Ico - Paul Papin

    Angers, 49000

  • CHRU Besançon

    Besançon, 25030

  • Institut Bergonie

    Bordeaux, 33076

  • CRLCC-Francois Baclesse

    Caen, 14076

  • Centre Jean Perrin Centre Regional de Lutte Contre Le Cancer D auvergne

    Clermont-Ferrand, 63003

  • Centre Leon Berard

    Lyon, 69008

  • Institut Paoli-Calmettes

    Marseille, 13273

  • Institut régional du Cancer Montpellier

    Montpellier, 34298

  • Centre Antoine Lacassagne

    Nice, 06189

  • Institut Curie

    Paris, 75231

  • CHU Lyon - Centre Hospitalier Lyon Sud

    Pierre-Benite (Lyon), 69495

  • Centre Eugene Marquis

    Rennes, 35042

  • CHU Strasbourg Hpital Hautepierre

    Strasbourg, 67098

  • Hopital Foch

    Suresnes, 92151

  • Institut Gustave Roussy

    Villejuif, 94805

Other trials in France

Germany10 sites

  • Universitätsklinikum Augsburg

    Augsburg, 86156

  • Charité-Universitätsm. Berlin

    Berlin, 13353

  • Onkologisches Zentrum - Onkologie Dachau

    Dachau, 85221

  • Universitätsklinikum Frankfurt, UCT

    Frankfurt, 60590

  • SLK-Kliniken Heilbronn GmbH;Klinik für Innere Medizin III

    Heilbronn, 74078

  • Universitätsklinikum Jena, Klinik für Innere Medizin II

    Jena, 07740

  • Klinikum Mannheim III. Medizinische Klinik

    Mannheim, 68167

  • Klinikum der LMU München, Campus Großhadern, Krebszentrum München

    München, 81377

  • Universitätsklinikum Münster, Medizinische Klinik A, Translationale Onkologie

    Münster, 48149

  • RED-Oncology GmbH

    Oldenburg / Holstein, 23758

Other trials in Germany

Spain10 sites

  • Hospital Clínic i Provincial

    Barcelona, 08036

  • Institut Catala d Oncologia Hospital Duran i Reynals

    Barcelona, 08908

  • Hospital Ramon y Cajal

    Madrid, 28034

  • Hospital Clinico Universitario Virgen de la Victoria

    Málaga, 29010

  • Complejo Hospitalario de Navarra

    Pamplona, Navarre, 31008

  • Hospital Sant Joan Despi- Moises Broggi

    Sant Joan Despí, Barcelona, 08970

  • Hospital Universitario Virgen Macarena

    Seville, 41009

  • Hospital Universitari i Politecnic La Fe

    Valencia, 46026

  • Complexo Hospitalario de Vigo. Hospital Álvaro Cunqueiro

    Vigo, Pontevedra, 36312

  • Hospital Universitario Miguel Servet

    Zaragoza, 50009

Other trials in Spain

Turkey (Türkiye)8 sites

  • Baskent University Adana Dr. Turgut Noyan Practice and Research Hospital

    Adana, 01250

  • Ankara University Medical Faculty

    Ankara, 06100

  • Ankara Oncology Hospital

    Ankara, 06200

  • Akdeniz University Medical Faculty

    Antalya, 07070

  • Trakya Universitesi Tip Fakultesi, Medikal Onkoloji Bilim Dali, Balkan Yerleskesi

    Edirne, 22030

  • Istanbul University Cerrahpa?a-Cerrahpa?a Medical Faculty

    Istanbul, 34098

  • ?zmir Medical Point

    Kar?iyaka, 35575

  • Hacettepe Uni Medical Faculty Hospital

    S?hhiye, Ankara, 06100

Other trials in Turkey (Türkiye)

United Kingdom8 sites

  • Royal United Hospital

    Bath, BA1 3NG

  • Velindre Cancer Centre

    Cardiff, CF14 2TL

  • Western General Hospital

    Edinburgh, EH4 2XU

  • Hammersmith Hospital

    London, W12 0HS

  • Christie Hospital NHS Trust

    Manchester, M20 4BX

  • Freeman Hospital

    Newcastle upon Tyne, NE7 7DN

  • Southampton General Hospital

    Southampton, SO16 6YD

  • Torbay Hospital

    Torquay, TQ27AA

Other trials in United Kingdom

Italy7 sites

  • Asst Papa Giovanni XXIII

    Bergamo, Lombardy, 24128

  • Policlinico Univ. - A.O. Mater Domini

    Catanzaro, Calabria, 88100

  • Azienda Socio Sanitaria Territoriale Niguarda (Ospedale Niguarda Ca' Granda)

    Milan, Lombardy, 20162

  • Istituto Nazionale Tumori Fondazione G. Pascale

    Naples, Campania, 80131

  • IRCCS Istituto Oncologico Veneto (IOV)

    Padova, Veneto, 35128

  • U. O. Oncologia Medica, Ospedale Santa Chiara

    Pisa, Basilicate, 56100

  • Arcispedale Santa Maria Nuova

    Reggio Emilia, Emilia-Romagna, 42100

Other trials in Italy

Brazil5 sites

  • Hospital de Cancer de Barretos

    Barretos, São Paulo, 14784-400

  • Hospital Nossa Senhora da Conceicao

    Porto Alegre, Rio Grande do Sul, 91350-200

  • Instituto Nacional de Cancer - INCa

    Rio de Janeiro, 20560-120

  • Hospital Sao Rafael - HSR

    Salvador, Estado de Bahia, 41253-190

  • Instituto do Cancer do Estado de Sao Paulo - ICESP

    São Paulo, São Paulo, 01246-000

Other trials in Brazil

Greece5 sites

  • Anticancer Hospital Ag Savas

    Athens, 115 22

  • IASO General Hospital of Athens

    Athens, 155 62

  • Univ General Hosp Heraklion

    Heraklion, 711 10

  • Uni Hospital of Ioannina

    Ioannina, 455 00

  • Theagenio Anticancer Hospital

    Thessaloniki, 540 07

Other trials in Greece

Romania4 sites

  • Institutul Oncologic Prof. Dr. Ion Chiricuta Cluj Napoca

    Cluj-Napoca, 400015

  • Centrul de Oncologie Sfantul Nectarie

    Craiova, 200347

  • Institutul Regional de Oncologie Iasi

    Iași, 700483

  • Oncocenter Timisoara

    Timi?oara, 300166

Other trials in Romania

South Korea4 sites

  • Seoul National University Hospital

    Seoul, 03080

  • Asan Medical Center

    Seoul, 05505

  • Samsung Medical Center

    Seoul, 06351

  • Severance Hospital, Yonsei University Health System

    Seoul, 120-752

Other trials in South Korea

Australia3 sites

  • Blacktown Hospital

    Blacktown, New South Wales, NSW 2148

  • Icon Cancer Foundation

    South Brisbane, Queensland, 4101

  • GenesisCare North Shore

    St Leonards, New South Wales, 2065

Other trials in Australia

Austria3 sites

  • Lkh-Univ. Klinikum Graz

    Graz, 8036

  • Lkh Salzburg - Univ. Klinikum Salzburg

    Salzburg, 5020

  • Medizinische Universität Wien

    Vienna, 1090

Other trials in Austria

Colombia3 sites

  • Clinica del Country

    Bogotá, 11001

  • Inst. Nacional de Cancerologia

    Bogotá

  • Oncomedica S.A.

    Montería, 230002

Other trials in Colombia

Czechia3 sites

  • Masarykuv onkologicky ustav

    Brno, 656 53

  • Fakultni nemocnice Olomouc

    Olomouc, 779 00

  • Fakultni Poliklinika Vseobecne Fakultni Niemocnice

    Prague, 128 08

Other trials in Czechia

Hungary3 sites

  • Orszagos Onkologiai Intezet

    Budapest, 1122

  • Budapesti Uzsoki Utcai Kórház

    Budapest, 1145

  • Bács-Kiskun Vármegyei Oktatókórház

    Kecskemét, 6000

Other trials in Hungary

Israel3 sites

  • Rabin MC

    Petah Tikva, 4941492

  • Chaim Sheba medical center, Oncology division

    Ramat Gan, 5262000

  • Tel Aviv Sourasky Medical Ctr

    Tel Aviv, 6423906

Other trials in Israel

Japan3 sites

  • Aichi Cancer Center

    Aichi, 464-8681

  • National Cancer Center Hospital East

    Chiba, 277-8577

  • National Hospital Organization Kyushu Cancer Center

    Fukuoka, 811-1395

Other trials in Japan

Netherlands3 sites

  • Erasmus MC

    Rotterdam, 3015 GD

  • Universitair Medisch Centrum Utrecht

    Utrecht, 3584 CX

  • Ziekenhuis VieCuri Medisch Centrum

    Venlo, 5912 BL

Other trials in Netherlands

Norway3 sites

  • Sørlandet Sykehus Kristiansand

    Kristiansand, 4604

  • Akershus universitetssykehus HF

    Lørenskog, 1478

  • Oslo universitetssykehus HF, Ullevål, Kreftsenteret

    Oslo, 0450

Other trials in Norway

Bulgaria2 sites

  • MHAT Nadezhda

    Sofia, 1330

  • MBAL Serdika EOOD

    Sofia, 1632

Other trials in Bulgaria

Chile2 sites

  • Bradford Hill Centro de Investigaciones Clinicas

    Recoleta, 8420383

  • James Lind Centro de Investigación Del Cáncer

    Temuco, 4800827

Other trials in Chile

Finland2 sites

  • Helsinki University Central Hospital

    Helsinki, 00250

  • Tampere University Hospital

    Tampere, 33520

Other trials in Finland

Ireland2 sites

  • St Vincent'S Uni Hospital

    Dublin, D04 T6F4

  • Waterford Regional Hospital

    Waterford, X91 ER8E

Other trials in Ireland

Mexico2 sites

  • Health Pharma Professional Research

    CD Mexico, Mexico CITY (federal District), 03810

  • AVIX Investigación Clínica S.C

    Monterrey, Nuevo León, 64710

Other trials in Mexico

Peru2 sites

  • Instituto Nacional de Enfermedades Neoplasicas

    Lima, 15038

  • Oncosalud Sac

    Lima, 41

Other trials in Peru

Switzerland2 sites

  • Universitaetsspital Basel

    Basel, 4031

  • UniversitätsSpital Zürich

    Zurich, 8091

Other trials in Switzerland

Thailand2 sites

  • Ramathibodi Hospital;Medicine/Oncology

    Bangkok, 10400

  • Faculty of Med. Siriraj Hosp.

    Bangkok, 10700

Other trials in Thailand

Croatia1 site

  • Clinical Hospital Centre Zagreb

    Zagreb, 10000

Other trials in Croatia

Denmark1 site

  • Aarhus Universitetshospital

    Aarhus N, 8200

Other trials in Denmark

Estonia1 site

  • North Estonia Medical Centre, Oncology and hematology Clinic

    Tallinn, 13419

Other trials in Estonia

Latvia1 site

  • Riga East Clinical University Hospital Latvian Oncology Centre

    Riga, LV-1079

Other trials in Latvia

Poland1 site

  • Szpital Uniwersytecki w Krakowie, Oddzia? Kliniczny Kliniki Onkologii

    Krakow, 30-688

Other trials in Poland

Portugal1 site

  • IPO do Porto

    Porto, 4200-072

Other trials in Portugal

About this study

Official title: A Phase II, Randomized, Active-Controlled, Multi-Center Study Comparing the Efficacy and Safety of Targeted Therapy or Cancer Immunotherapy Guided by Genomic Profiling Versus Platinum-Based Chemotherapy in Patients With Cancer of Unknown Primary Site Who Have Received Three Cycles of Platinum Doublet Chemotherapy

This study will compare the efficacy and safety of molecularly-guided therapy versus standard platinum-containing chemotherapy in participants with poor-prognosis cancer of unknown primary site (CUP; non-specific subset) who have achieved disease control after 3 cycles of first-line platinum based induction chemotherapy.

Being tested
Alectinib, Vismodegib, Ipatasertib, Olaparib, Erlotinib
Medical subject headings
Neoplasms, Unknown Primary

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • AlectinibDrug

    Alectinib will be administered orally at the label-recommended dose (600 mg) twice daily until loss of clinical benefit or unacceptable toxicity, through the end of the study (approximately 70 months).

  • VismodegibDrug

    Vismodegib will be administered orally at the label-recommended dose (150 mg) once daily until loss of clinical benefit or unacceptable toxicity, through the end of the study (approximately 70 months).

  • IpatasertibDrug

    Ipatasertib will be administered orally at the label-recommended dose (400 mg) once daily on Days 1-21 of each 28-day Cycle in combination with paclitaxel, and as monotherapy after the final administration of paclitaxel, until loss of clinical benefit or unacceptable toxicity, through the end of the study (approximately 70 months).

  • OlaparibDrug

    Olaparib will be administered orally at the label-recommended dose (400 mg) twice daily until loss of clinical benefit or unacceptable toxicity, through the end of the study (approximately 70 months).

  • ErlotinibDrug

    Erlotinib will be administered orally in combination with Bevacizumab at the label recommended dose (150 mg) once daily until loss of clinical benefit or unacceptable toxicity, through the end of the study (approximately 70 months)

  • BevacizumabDrug

    Bevacizumab will be administered intravenously at 15mg/kg every 3 weeks in combination with Erlotinib until loss of clinical benefit or unacceptable toxicity, through the end of the study (approximately 70 months)

  • VemurafenibDrug

    Vemurafenib will be administered orally, 960 mg twice daily, in combination with Cobimetinib, until loss of clinical benefit or unacceptable toxicity, through the end of the study (approximately 70 months)

  • CobimetinibDrug

    Cobimetinib will be administered orally, 60mg once daily, in combination with Vemurafenib, on Days 1-21 of each 28-day Cycle, until loss of clinical benefit or unacceptable toxicity, through the end of the study (approximately 70 months)

  • Trastuzumab Subcutaneous (SC)Drug

    Trastuzumab will be administered subcutaneously, 600 mg every 3 weeks, in combination with Pertuzumab and chemotherapy, until loss of clinical benefit or unacceptable toxicity, through the end of the study (approximately 70 months)

  • PertuzumabDrug

    Pertuzumab will be initially be administered intravenously, 840 mg, followed by 420 mg every 3 weeks, in combination with Trastuzumab and chemotherapy, until loss of clinical benefit or unacceptable toxicity, through the end of the study (approximately 70 months)

  • AtezolizumabDrug

    Atezolizumab will be administered intravenously at the label-recommended dose (1200 mg), alone or in combination with chemotherapy, every 3 weeks until loss of clinical benefit or unacceptable toxicity, through the end of the study (approximately 70 months).

  • CarboplatinDrug

    Carboplatin will be administered intravenously at the area under the curve (AUC) dose once every 3 weeks for up to 9 Cycles (Cycle = 21 days) in some combination with the following: Paclitaxel, Gemcitabine, Atezolizumab, Pertuzumab, and Trastuzumab SC.

  • PaclitaxelDrug

    Paclitaxel will be administered intravenously, 175 mg/m^2, once every 3 weeks for up to 9 cycles (Cycle = 21 days) in some combination with the following: Carboplatin, Ipatasertib, Atezolizumab, Pertuzumab, and Trastuzumab SC

  • CisplatinDrug

    Cisplatin will be administered intravenously, 60-75 mg/m^2, once every three weeks, for up to 9 cycles (Cycle = 21 days) in some combination with the following: Gemcitabine, Paclitaxel, Atezolizumab, Pertuzumab, and Trastuzumab SC.

  • GemcitabineDrug

    Gemcitabine will be administered intravenously, 1000 mg/m^2, twice every three weeks for up to 9 cycles (Cycle = 21 days) in some combination with the following: Cisplatin, Carboplatin, Atezolizumab, Pertuzumab, and Trastuzumab SC.

  • EntrectinibDrug

    Entrectinib will be administered orally at the label-recommended dose (600 mg) once daily until loss of clinical benefit or unacceptable toxicity, through the end of the study (approximately 70 months).

  • IvosidenibDrug

    Ivosidenib will be administered orally at the label-recommended dose (500mg) once daily across a 28-day treatment cycle until loss of clinical benefit or unacceptable toxicity.

  • PemigatinibDrug

    Pemigatinib will be administered orally at the label-recommended dose (13.5mg) once daily across a 21-day treatment cycle until loss of clinical benefit or unacceptable toxicity.

Study groups in NCT03498521
Study groupRoleWhat they receive
Molecularly-Guided TherapyExperimentalParticipants will be assigned to molecularly-guided therapy based on genomic profile.
Receives: Drug: Alectinib; Drug: Vismodegib; Drug: Ipatasertib; Drug: Olaparib; Drug: Erlotinib; Drug: Bevacizumab; Drug: Vemurafenib; Drug: Cobimetinib; Drug: Trastuzumab Subcutaneous (SC); Drug: Pertuzumab; Drug: Atezolizumab; Drug: Paclitaxel; Drug: Entrectinib; Drug: Ivosidenib; Drug: Pemigatinib
Platinum-Based ChemotherapyActive comparatorParticipants will receive platinum-based chemotherapy (Carboplatin or Cisplatin in combination with Gemcitabine or Paclitaxel).
Receives: Drug: Trastuzumab Subcutaneous (SC); Drug: Pertuzumab; Drug: Carboplatin; Drug: Paclitaxel; Drug: Cisplatin; Drug: Gemcitabine

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Progression Free Survival (PFS) as Assessed by the Investigator According to Response Evaluation Criteria In Solid Tumors v1.1 (RECIST v1.1)

    This efficacy objective was to evaluate the efficacy of MGT vs platinum chemotherapy in term of PFS in participants with CUP whose best response to 3 cycles of platinum induction chemotherapy was assessed CR, PR, or SD.

    Measured: From randomization to the first occurrence of disease progression or death from any cause, until 330 PFS events were observed (approx. 4.3 years for MGT Cat 1 and 3.4 years for Chemotherapy Cat 1).

Secondary outcomes (4)

  1. Overall Survival (OS)

    Measured: From randomization to death from any cause (approx. 4 years)

  2. Objective Response Rate (ORR)

    Measured: Two consecutive occurrences of complete or partial response >/=4 weeks apart (up to approximately 4 months)

  3. Duration of Response (DOR)

    Measured: From the first documentation of a complete response (CR) or partial response (PR) to disease progression or death from any cause, whichever occurs first (up to approximately 4 years)

  4. Disease Control Rate (DCR)

    Measured: From randomization to death from any cause, through the end of study (approximately 4 years)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted13 Apr 2018
  2. Study start10 Jul 2018
  3. Primary completion14 Feb 2023
  4. Study completion7 Nov 2024

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
529 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03498521
Sponsor's protocol ID
MX39795
Other IDs
2017-003040-20, 2017
Acronym
CUPISCO
Status last verified
Nov 2025
First submitted
5 Apr 2018
Last updated
19 Nov 2025
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: For eligible studies, qualified researchers may request access to individual patient level clinical data. See Roche's commitment to transparency of clinical study information here: https://go.roche.com/data\_sharing

Publications

  • Kramer A, Bochtler T, Pauli C, Shiu KK, Cook N, Janoski de Menezes J, Pazo-Cid RA, Losa F, Robbrecht DGJ, Tomasek J, Arslan C, Ozguroglu M, Arni P, Bigot F, Kim SY, Naito Y, Italiano A, Chalabi N, Duran-Pacheco G, Ma N, Scarato J, Ross JS, Moch H, Mileshkin L. Molecularly Guided Therapy Versus Continued Chemotherapy in Unfavorable Cancer of Unknown Primary: Updated Efficacy and Safety From the Randomized, Phase II CUPISCO Study. J Clin Oncol. 2026 Sep 3:JCO2502974. doi: 10.1200/JCO-25-02974. Online ahead of print.

    PubMed 42691436

  • Kramer A, Bochtler T, Pauli C, Shiu KK, Cook N, de Menezes JJ, Pazo-Cid RA, Losa F, Robbrecht DG, Tomasek J, Arslan C, Ozguroglu M, Stahl M, Bigot F, Kim SY, Naito Y, Italiano A, Chalabi N, Duran-Pacheco G, Michaud C, Scarato J, Thomas M, Ross JS, Moch H, Mileshkin L. Molecularly guided therapy versus chemotherapy after disease control in unfavourable cancer of unknown primary (CUPISCO): an open-label, randomised, phase 2 study. Lancet. 2024 Aug 10;404(10452):527-539. doi: 10.1016/S0140-6736(24)00814-6. Epub 2024 Jul 31.

    PubMed 39096924

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Alectinib, Vismodegib and Ipatasertib in people with Cancer of Unknown Primary Site, run by Hoffmann-La Roche at 125 sites in 33 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 6 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 19 Nov 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 125 sites in France, Germany, Spain, Turkey (Türkiye), United Kingdom and 28 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 14 Jun 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Progression Free Survival (PFS) as Assessed by the Investigator According to Response Evaluation Criteria In Solid Tumors v1.1 (RECIST v1.1) (time frame: From randomization to the first occurrence of disease progression or death from any cause, until 330 PFS events were observed (approx. 4.3 years for MGT Cat 1 and 3.4 years for Chemotherapy Cat 1).). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03498521, which also lists 2 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Nov 2025.

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