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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 | Clinical Trials | Clareo Health

COMPLETEDPHASE2

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 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

NCT03498521•Hoffmann-La Roche

View on ClinicalTrials.gov

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 chemotherapy.

Eligibility

Age

18 - No limit years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•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

Exclusion Criteria

•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
•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

Locations (125)

Blacktown Hospital

Blacktown, New South Wales, Australia

GenesisCare North Shore

St Leonards, New South Wales, Australia

Icon Cancer Foundation

South Brisbane, Queensland, Australia

Lkh-Univ. Klinikum Graz

Graz, Austria

Lkh Salzburg - Univ. Klinikum Salzburg

Salzburg, Austria

Medizinische Universität Wien

Vienna, Austria

Hospital Sao Rafael - HSR

Salvador, Estado de Bahia, Brazil

Hospital Nossa Senhora da Conceicao

Porto Alegre, Rio Grande do Sul, Brazil

Hospital de Cancer de Barretos

Barretos, São Paulo, Brazil

Instituto do Cancer do Estado de Sao Paulo - ICESP

São Paulo, São Paulo, Brazil

Key Dates

Start Date

July 10, 2018

Primary Completion Date

February 14, 2023

Completion Date

November 7, 2024

Last Updated

November 19, 2025

Enrollment

529

ACTUAL participants

Conditions

Cancer of Unknown Primary Site

Interventions

Alectinib

DRUG

Vismodegib

DRUG

Ipatasertib

DRUG

Olaparib

DRUG

Erlotinib

DRUG

Bevacizumab

DRUG

Vemurafenib

DRUG

Cobimetinib

DRUG

Trastuzumab Subcutaneous (SC)

DRUG

Pertuzumab

DRUG

Atezolizumab

DRUG

Carboplatin

DRUG

Paclitaxel

DRUG

Cisplatin

DRUG

Gemcitabine

DRUG

Entrectinib

DRUG

Ivosidenib

DRUG

Pemigatinib

DRUG

Sintilimab in Cancer of Unknown Primary

NCT05024968

Cancer of Unknown Primary Site

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UNKNOWNPHASE2

Nivolumab/Ipilimumab in Second Line CUP-syndrome

NCT04131621

Cancer of Unknown Primary Site

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: November 19, 2025Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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