NCT04591431

Brief Summary

This is a randomized, prospective, multicenter, Proof of Concept, Phase II clinical trial Study. The main objective of the study is to evaluate the efficacy (meant as overall response rate ORR) of TT (targeted Therapy) vs SoC (standard of Care) in patients with progressive disease (recurrent and/or metastatic) of breast cancer, metastatic gastro-intestinal tumors, non small cell lung cancer (NSCLC) or others. Patients should have completed at least 1 line of treatment and no more than 2 as defined by the current version of the AIOM (Italian Association of Medical Oncology) guidelines. Patients are included if surgery is contraindicated.

Trial Health

43
At Risk

Trial Health Score

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

Trial has exceeded expected completion date
Enrollment
400

participants targeted

Target at P75+ for phase_2 breast-cancer

Timeline
Completed

Started Oct 2020

Typical duration for phase_2 breast-cancer

Geographic Reach
1 country

37 active sites

Status
unknown

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

September 18, 2020

Completed
19 days until next milestone

Study Start

First participant enrolled

October 7, 2020

Completed
12 days until next milestone

First Posted

Study publicly available on registry

October 19, 2020

Completed
4.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2024

Completed
6 months until next milestone

Study Completion

Last participant's last visit for all outcomes

June 1, 2025

Completed
Last Updated

October 3, 2023

Status Verified

October 1, 2023

Enrollment Period

4.2 years

First QC Date

September 18, 2020

Last Update Submit

October 2, 2023

Conditions

Outcome Measures

Primary Outcomes (1)

  • OVERALL RESPONSE RATE (ORR)

    Evaluation of the ORR of the Treatment at choice of physicians, according to Standard of Care (SoC) or of the Tailored Treatment (TT). The ORR will be constructed according to the specific design of the study, therefore including also the Rescue Therapy Phase data. This means that the ORR will take into account 3 evaluations: * on the original final population ( i.e 384 patients divided into the 4 groups of type of cancer) * on the TT patients, which will include the original randomized TT patients and the patients switched from the standard of care therapy (SoC therapy) to the TT Therapy, this latter within the Rescue Therapy Phase (patients switching upon the first documented progression) * on the population composed by the original TT patients, the original SoC patients and the switched TT patients. This means that the total population analyzed will include the original 384 population data (as per randomization) and the additional switched TT patients.

    42 months

Secondary Outcomes (7)

  • Progression Free Survival (PFS) of SoC vs TT

    42 months

  • Time to Treatment Failure (TTF) of SoC vs TT

    42 months

  • Time to Next Treatment (TTNT) of SoC vs TT

    42 months

  • Concordance between molecular profile on tumor tissue and ctDNA

    42 months

  • QoLs included in the two arms of the study of SoC vs TT

    42 months

  • +2 more secondary outcomes

Study Arms (2)

Tailored Therapy

EXPERIMENTAL

Experimental (TT) Patients will be treated with target therapy and/or immunotherapy according to their genomic profile evidenced by Foundation One test and independently from their type of cancer with one or more drugs of the following list (administered according to the SmPCs or IBs if under development): TARGET THERAPY: ERLOTINIB (EGFR mutation) TRASTUZUMAB, PERTUZUMAB, TDM1, LAPATINIB (ERBB2 amplifications/mut) EVEROLIMUS (mTOR mutations, AKT mut) VEMURAFENIB, COBIMETINIB (BRAFV600E mutations) ALECTINIB, BRIGATINIB (ALK, RET) PALBOCICLIB (CDK4/6, CDKN2A/p16) PONATINIB (Bcr-abl) VISMODEGIB (SMO/PTCH1) ITACITINIB (JAK mutation) INCB054828 (FGFR1/2/3) IPATASERTIB (PI3K, AKT, PTEN) ENTRECTINIB (NTRK1/2/3 -TRK fusion proteins-, ROS1) ALPELISIB (PI3K, AKT) TEPOTINIB (MET amplification/exon14 skipping mutations) PRALSETINIB (RET) TALAZOPARIB (BRCA1/2, ATM, other HRD status) SELPERCATINIB (RET) IMMUNOTHERAPY: ATEZOLIZUMAB, NIVOLUMAB, IPILIMUMAB (MSI, HIGH TUMOR MUTATIONAL BURDEN, OTHER)

Drug: ErlotinibDrug: TrastuzumabDrug: Trastuzumab emtansineDrug: PertuzumabDrug: LapatinibDrug: EverolimusDrug: VemurafenibDrug: CobimetinibDrug: AlectinibDrug: BrigatinibDrug: PalbociclibDrug: PonatinibDrug: VismogedibDrug: ItacitinibDrug: IpatasertibDrug: EntrectinibDrug: AtezolizumabDrug: NivolumabDrug: IpilimumabDrug: PemigatinibDrug: PralsetinibDrug: SelpercatinibDrug: TalazoparibDrug: TepotinibDrug: Alpelisib

Standard of Care

ACTIVE COMPARATOR

Patients will be treated according the current version of the AIOM (Italian Association of Medical Oncology) guidelines for their type of cancer. As an example, patients could be treated with standard chemotherapy and/or targeted therapy according to the histological results.

Drug: Oncology Drugs

Interventions

TT arm

Tailored Therapy

TT arm

Tailored Therapy

TT arm

Tailored Therapy

TT arm

Tailored Therapy

TT arm

Tailored Therapy

TT arm

Tailored Therapy

TT arm

Tailored Therapy

TT arm

Tailored Therapy

TT arm

Tailored Therapy

TT arm

Tailored Therapy

TT arm

Tailored Therapy

TT arm

Tailored Therapy

TT arm

Tailored Therapy

TT arm

Tailored Therapy

TT arm

Tailored Therapy

TT arm

Tailored Therapy

TT arm

Tailored Therapy

TT arm

Tailored Therapy

TT arm

Tailored Therapy

TT arm

Tailored Therapy

Standard of Care Arm

Standard of Care

TT arm

Tailored Therapy

TT arm

Tailored Therapy

TT arm

Tailored Therapy

TT arm

Tailored Therapy

TT arm

Tailored Therapy

Eligibility Criteria

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

You may qualify if:

  • Age ≥ 18 at time of signing Informed Consent Form
  • Patients able and willing to provide a written informed consent to participate to the study
  • Patients with recurrent/metastatic breast, gastrointestinal cancer,non small cell lung cancer or others
  • Patients not treatable with potentially curative surgery ot other loco-regional treatments.
  • Patients should have been completed at least or failed the first line of treatment for breast cancer, gastro-intestinal, non small cell lung cancer or other cancer
  • ECOG performance status from 0 to 1
  • Molecular target not actionable with approved drugs identified during screening by profiling with FoundationOne CDX on biopsy and FoundationOne Liquid CDx on blood
  • Biopsiable disease (tumor biopsy mandatory for tumor profiling). The biopsy must be performed during the screening period, when patients complete the conventional therapy for their recurrent/metastatic cancer. Historical samples will be considered for the study if collected within 3 months before the ICF signature of the patient. Samples older than 3 months, with a maximum timeframe of 6 months, and collected before progression of disease after the last treatment administered will be considered upon clinical judgement of the Investigator, after confirmation by the coordinating site or MTB. Samples obtained from a biospy of a metastatic lesion in progression after the last treatment administered represent the optimal tissue sample for genomic testing. Patients with glioblastomas and high grade malignant gliomas can be enrolled with the historical tissue samples.
  • Measurable disease, eligible to standard treatment. Patients must have measurable or evaluable disease defined, per RECIST 1.1 or irCS (immune related Response Criteria), as at least one lesion that can be accurately measured in at least one dimension (longest diameter to be recorded for non-nodal lesions and short axis for nodal lesions) as ≥20 mm with conventional techniques or as ≥10 mm with spiral computed tomography (CT) scan, Magnetic Resonance Imaging (MRI), or a subcutaneous or superficial lesion that can be measured with calipers by clinical exam. For lymph nodes, the short axis must be ≥15 mm. Patients who have assessable disease by physical or radiographic examination but do not fully meet the above definitions of measurable disease (but still remains measurable) are eligible and will be considered to have evaluable disease. Patient's whose disease cannot be objectively measured by physical or radiographic examination (e.g., elevated serum tumor marker only) are NOT eligible. PET scan could be performed, if clinically indicated. For PET response evaluation PERCIST criteria will be applied.
  • Adequate renal function defined by a serum creatinine \<1.5xUNL (upper normal limit).
  • Adequate liver function test defined by SGOT \& SGPT \<3xUNL (5xUNL in case of liver metastases), and bilirubin level \<1.5xUNL
  • Adequate bone marrow function defined by platelets \>100,000/mm3, hemoglobin \>10 g/dL, and neutrophils \>1,000/mm3
  • For female of child-bearing potential and for all women \< 1 years after the onset of menopause: a negative pregnancy test \<72 hours before starting study treatment is required. If sexually active, female of childbearing potential must use "highly effective" methods of contraception for the study duration. Contraception should continue after the last treatment for 3 months or for longer periods according to what reported in the Appendix 1 of the Protocol
  • For male of reproductive potential: any sexually active male patient must use a condom while on study treatment. Contraception should continue after the last treatment for 3 months or for longer periods according to what reported in the Appendix 1 of the Protocol.

You may not qualify if:

  • Patients who have only bone and/or brain metastases
  • Patients treated with more than 2 lines for breast cancer, gastro-intestinal, non small cell lung cancer and other cancer
  • Patients with uncontrolled disease (untreated and/or sintomatic) and patients whose brain metastases have not been monitored for \>2 months
  • Patients with well-established actionable targets for which approved and marketed targeted drugs are available (i.e. lung cancer with EGFR mutation, or ALK translocation, B-RAF mutant melanoma, GIST with KIT mutations or breast cancer with HER2 amplification)
  • Patient participating in another clinical trial with an experimental drug
  • Anticoagulation with anti-vitamin K (Low Molecular Weight Heparin \[LMWH\] is allowed)
  • Patients with other concurrent severe and/or uncontrolled medical disease which could compromise participation in the study, including uncontrolled diabetes, cardiac disease, uncontrolled hypertension, congestive cardiac failure, ventricular arrhythmias, active ischemic heart disease, myocardial infarction within one year, chronic liver or renal disease, active gastrointestinal tract ulceration, severely impaired lung function
  • Pregnant and/or breastfeeding women
  • Patients with any psychological, familial, sociological or geographical condition potentially hampering compliance with the study protocol and follow-up schedule
  • HIV, HBV, or HCV infection as per specific test performed at the screening visit or known as per Medical History
  • Patients with documented contraindication to any of the IMPs that will be used for the study, as reported in the respective SmPcs/IBs and in Appendix 2
  • Patients treated with the following drugs, because of the risk of immunosuppression: Chronic or high-dose oral corticosteroid therapy, TNF-inhibitors and Anti-T cell antibodies

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (37)

OSPEDALI RIUNITI di ANCONA

Ancona, Italy

Location

Centro Riferimento Oncologico

Aviano, Italy

Location

Irccs Oncologico Istituto Tumori Giovanni Paolo Ii - Bari

Bari, Italy

Location

Asst Papa Giovanni Xxiii

Bergamo, Italy

Location

Ospedale Bellaria

Bologna, Italy

Location

Ospedale di Carpi

Carpi, Italy

Location

Arnas Garibaldi- Nuovo Ospedale Garibaldi - Nesima

Catania, Italy

Location

A.O. Mater Domini Catanzaro

Catanzaro, Italy

Location

Azienda Ospedaliero-Universitaria Di Ferrara

Ferrara, Italy

Location

E.O. Ospedali Galliera

Genova, Italy

Location

Ospedale Policlinico San Martino

Genova, Italy

Location

Ospedale Della Misericordia

Grosseto, Italy

Location

I.R.S.T. Srl Irccs

Meldola, Italy

Location

Ao Papardo

Messina, Italy

Location

Istituto Europeo Di Oncologia

Milan, Italy

Location

Istituto Nazionale Tumori Di Napoli Irccs Pascale

Napoli, Italy

Location

Ospedale Classificato Sacro Cuore - Don Calabria

Negrar, Italy

Location

I.R.C.C.S. Istituto Oncologico Veneto

Padua, Italy

Location

Az.Osp.Univ.P.Giaccone

Palermo, Italy

Location

Azienda Ospedaliera Di Perugia

Perugia, Italy

Location

Casa Di Cura Privata Osp. P. Pederzoli

Peschiera del Garda, Italy

Location

Azienda Usl Di Piacenza

Piacenza, Italy

Location

Azienda Ospedaliero-Universitaria Pisana

Pisa, Italy

Location

Nuovo Ospedale Di Prato - S. Stefano

Prato, Italy

Location

Ospedale "Santa Maria Delle Croci"

Ravenna, Italy

Location

Arcispedale Santa Maria Nuova Di Reggio Emilia

Reggio Emilia, Italy

Location

Az. Osp. Uni. Policlinico Umberto I

Roma, Italy

Location

Azienda Ospedaliera Sant'Andrea

Roma, Italy

Location

Istituti Fisioterapici Ospitalieri- Ifo - Istituto Regina Elena

Roma, Italy

Location

Ospedale Fatebenefratelli

Roma, Italy

Location

Policl. Univ. Campus Bio Medico

Roma, Italy

Location

Casa Sollievo della Sofferenza - Opera Padre Pio

San Giovanni Rotondo, Italy

Location

Azienda Ospedaliera 'S. Maria' - Terni

Terni, Italy

Location

AO Ordine Mauriziano

Torino, Italy

Location

Humanitas Gradenigo

Torino, Italy

Location

IRCCS Candiolo

Torino, Italy

Location

Complesso Ospedaliero Di Belcolle- Ospedale Di Belcolle

Viterbo, Italy

Location

Related Publications (1)

  • Marchetti P, Curigliano G, Biffoni M, Lonardi S, Scagnoli S, Fornaro L, Guarneri V, De Giorgi U, Ascierto PA, Blandino G, D'Amati G, Aglietta M, Cremolini C, Conte P, Crimini E, Ceracchi M, Pisegna S, Verkhovskaia S, Bordonaro R, Bracarda S, Butturini G, Del Mastro L, DeCensi A, Fabbri A, Fenocchio E, Gori S, Metro G, Pessino A, Pozzessere D, Puglisi F, Tamberi S, Zambelli A, Marino D, Capoluongo E, Cappuzzo F, Cerbelli B, Giannini G, Malapelle U, Mazzuca F, Nuti M, Pruneri G, Simmaco M, Strigari L, Tonini G, Martini N, Botticelli A; ROME trial investigators consortia. Genomically matched therapy in advanced solid tumors: the randomized phase 2 ROME trial. Nat Med. 2025 Oct;31(10):3514-3523. doi: 10.1038/s41591-025-03918-x. Epub 2025 Sep 29.

MeSH Terms

Conditions

Breast NeoplasmsGastrointestinal NeoplasmsCarcinoma, Non-Small-Cell Lung

Interventions

Erlotinib HydrochlorideTrastuzumabAdo-Trastuzumab EmtansinepertuzumabLapatinibEverolimusVemurafenibcobimetinibalectinibbrigatinibpalbociclibponatinibitacitinibipatasertibentrectinibatezolizumabNivolumabIpilimumabpemigatinibpralsetinibselpercatinibtalazoparibtepotinibAlpelisib

Condition Hierarchy (Ancestors)

Neoplasms by SiteNeoplasmsBreast DiseasesSkin DiseasesSkin and Connective Tissue DiseasesDigestive System NeoplasmsDigestive System DiseasesGastrointestinal DiseasesCarcinoma, BronchogenicBronchial NeoplasmsLung NeoplasmsRespiratory Tract NeoplasmsThoracic NeoplasmsLung DiseasesRespiratory Tract Diseases

Intervention Hierarchy (Ancestors)

QuinazolinesHeterocyclic Compounds, 2-RingHeterocyclic Compounds, Fused-RingHeterocyclic CompoundsAntibodies, Monoclonal, HumanizedAntibodies, MonoclonalAntibodiesImmunoglobulinsImmunoproteinsBlood ProteinsProteinsAmino Acids, Peptides, and ProteinsSerum GlobulinsGlobulinsMaytansineMacrolidesLactonesOrganic ChemicalsLactams, MacrocyclicMacrocyclic CompoundsPolycyclic CompoundsSirolimusSulfonamidesAmidesSulfonesSulfur CompoundsIndoles

Study Officials

  • Paolo Marchetti

    Fondazione per la Medicina Personalizzata

    STUDY CHAIR
  • Andrea Botticelli

    Università degli Studi di Roma Sapienza

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Model Details: Randomized, prospective, multicenter, Proof of Concept, Phase II clinical trial
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

September 18, 2020

First Posted

October 19, 2020

Study Start

October 7, 2020

Primary Completion

December 1, 2024

Study Completion

June 1, 2025

Last Updated

October 3, 2023

Record last verified: 2023-10

Data Sharing

IPD Sharing
Will not share

Locations