NCT04341181

Brief Summary

The ProTarget study is a phase II, prospective, non-randomized clinical trial with the primary purpose of investigating the safety and efficacy of commercially available cancer drugs that target specific changes in cancer cell DNA to treat patients with advanced cancer. The primary endpoint is anti-tumor activity or stable disease documented after 16 weeks of experimental drug treatment. The drugs used in the trial have been approved by EMA/FDA for the treatment of certain cancers. Choice of drug is based on whether the patient's cancer cells contain precisely the DNA change (i) targeted by the EMA/FDA-approved drug or (ii) related to sensitivity to the EMA/FDA-approved drug. The trial drug is thus not approved by the EMA/FDA or in Denmark for the treatment of the patient's cancer - it is so-called "off-label use". The secondary purposes are:

  • To detect side effects in patients treated with commercially available targeted cancer drugs.
  • Performing biomarker analyzes, including (but not limited to) whole-genome analysis (WGS) on a fresh tumor tissue sample (biopsy) at baseline and progression.
  • To investigate mechanisms of resistance using recurrent / serial fresh tumor biopsies for WGS and so-called liquid biopsies, which are blood samples in which the cancer cell DNA is analyzed. The secondary endpoints include response duration, progression-free survival, and overall survival.

Trial Health

77
On Track

Trial Health Score

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

Enrollment
300

participants targeted

Target at P75+ for phase_2 cancer

Timeline
49mo left

Started Aug 2020

Longer than P75 for phase_2 cancer

Geographic Reach
1 country

7 active sites

Status
recruiting

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

Study Progress59%
Aug 2020Apr 2030

First Submitted

Initial submission to the registry

April 8, 2020

Completed
2 days until next milestone

First Posted

Study publicly available on registry

April 10, 2020

Completed
5 months until next milestone

Study Start

First participant enrolled

August 24, 2020

Completed
8.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

April 30, 2029

Expected
1 year until next milestone

Study Completion

Last participant's last visit for all outcomes

April 30, 2030

Last Updated

July 25, 2025

Status Verified

July 1, 2025

Enrollment Period

8.7 years

First QC Date

April 8, 2020

Last Update Submit

July 22, 2025

Conditions

Outcome Measures

Primary Outcomes (1)

  • Response rate

    Response according to RECIST

    16 weeks

Study Arms (10)

Alectinib

EXPERIMENTAL

Alectinib for patients with a molecular tumor profile that can potentially be targeted by Alectinib.

Drug: Alectinib

Atezolizumab

EXPERIMENTAL

Atezolizumab for patients with a molecular tumor profile that can potentially be targeted by Atezolizumab.

Drug: Atezolizumab

Avelumab

EXPERIMENTAL

Avelumab for patients with a molecular tumor profile that can potentially be targeted by Avelumab.

Drug: Avelumab

Axitinib

EXPERIMENTAL

Axitinib for patients with a molecular tumor profile that can potentially be targeted by Axitinib.

Drug: Axitinib

Erlotinib

EXPERIMENTAL

Erlotinib for patients with a molecular tumor profile that can potentially be targeted by Erlotinib.

Drug: Erlotinib

Vemurafenib plus Cobimetinib (combination)

EXPERIMENTAL

Vemurafenib plus Cobimetinib (combination treatment) for patients with a molecular tumor profile that can potentially be targeted by Vemurafenib plus Cobimetinib.

Drug: Vemurafenib plus Cobimetinib (combination)

Trastuzumab plus Pertuzumab (combination)

EXPERIMENTAL

Trastuzumab plus Pertuzumab (combination treatment) for patients with a molecular tumor profile that can potentially be targeted by Trastuzumab plus Pertuzumab.

Drug: Trastuzumab plus Pertuzumab (combination)

Trastuzumab emtansin

EXPERIMENTAL

Trastuzumab emtansin for patients with a molecular tumor profile that can potentially be targeted by Trastuzumab emtansin.

Drug: Trastuzumab emtansine

Vismodegib

EXPERIMENTAL

Vismodegib for patients with a molecular tumor profile that can potentially be targeted by Vismodegib.

Drug: Vismodegib

Niraparib

EXPERIMENTAL

Niraparib for patients with a molecular tumor profile that can potentially be targeted by Niraparib.

Drug: Niraparib

Interventions

Alectinib treatment for patients with an advanced solid tumor, multiple myeloma or B cell non-Hodgkin lymphoma, who are not eligible for on-label treatment but for whom anti-tumor activity of Alectinib might be expected based on their molecular tumor profile.

Also known as: Alecensa
Alectinib

Atezolizumab treatment for patients with an advanced solid tumor, multiple myeloma or B cell non-Hodgkin lymphoma, who are not eligible for on-label treatment but for whom anti-tumor activity of Atezolizumab might be expected based on their molecular tumor profile.

Also known as: Tecentriq
Atezolizumab

Avelumab treatment for patients with an advanced solid tumor, multiple myeloma or B cell non-Hodgkin lymphoma, who are not eligible for on-label treatment but for whom anti-tumor activity of Avelumab might be expected based on their molecular tumor profile.

Also known as: Bavencio
Avelumab

Axitinib treatment for patients with an advanced solid tumor, multiple myeloma or B cell non-Hodgkin lymphoma, who are not eligible for on-label treatment but for whom anti-tumor activity of Axitinib might be expected based on their molecular tumor profile.

Also known as: Inlyta
Axitinib

Erlotinib treatment for patients with an advanced solid tumor, multiple myeloma or B cell non-Hodgkin lymphoma, who are not eligible for on-label treatment but for whom anti-tumor activity of Erlotinib might be expected based on their molecular tumor profile.

Also known as: Tarceva
Erlotinib

Vemurafenib plus Cobimetinib treatment for patients with an advanced solid tumor, multiple myeloma or B cell non-Hodgkin lymphoma, who are not eligible for on-label treatment but for whom anti-tumor activity of Vemurafenib plus Cobimetinib might be expected based on their molecular tumor profile.

Also known as: Zelboraf plus Cotellic (combination)
Vemurafenib plus Cobimetinib (combination)

Trastuzumab plus Pertuzumab treatment for patients with an advanced solid tumor, multiple myeloma or B cell non-Hodgkin lymphoma, who are not eligible for on-label treatment but for whom anti-tumor activity of Trastuzumab plus Pertuzumab might be expected based on their molecular tumor profile.

Also known as: Herceptin plus Perjeta (combination)
Trastuzumab plus Pertuzumab (combination)

Trastuzumab emtansine treatment for patients with an advanced solid tumor, multiple myeloma or B cell non-Hodgkin lymphoma, who are not eligible for on-label treatment but for whom anti-tumor activity of Trastuzumab emtansine might be expected based on their molecular tumor profile.

Also known as: Kadcyla
Trastuzumab emtansin

Vismodegib treatment for patients with an advanced solid tumor, multiple myeloma or B cell non-Hodgkin lymphoma, who are not eligible for on-label treatment but for whom anti-tumor activity of Vismodegib might be expected based on their molecular tumor profile.

Also known as: Erivedge
Vismodegib

Niraparib treatment for patients with an advanced solid tumor, multiple myeloma or B cell non-Hodgkin lymphoma, who are not eligible for on-label treatment but for whom anti-tumor activity of Niraparib might be expected based on their molecular tumor profile.

Also known as: Zejula
Niraparib

Eligibility Criteria

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

You may qualify if:

  • Patient (age≥ 18 years) with a histologically-proven locally advanced or metastatic malignant disease who is no longer benefitting from standard anti-cancer treatment or for whom, in the opinion of the investigator, no such treatment is available or indicated.
  • ECOG performance status 0-2

You may not qualify if:

  • Absolute neutrophil count ≥ 1500 µl
  • Hemoglobin \> 5.6 mmol/l
  • Platelets \> 75,000/µl
  • Total bilirubin \< 1.5 x institutional upper limit of normal (ULN)
  • AST (SGOT) and ALT(SGPT) \< 2.5 x institutional upper limit of normal (ULN) (or \< 5 x ULN in patients with known hepatic metastases)
  • Calculated or measured creatinine clearance ≥ 50 mL/min/1.73 m2.
  • Patients must have measurable or evaluable disease (per RECIST v1.1 for solid tumor), defined 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 CT scan, 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 meet these definitions of measurable disease 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, with the exception of CA-125 for ovarian cancer and PSA for prostate cancer.
  • Results must be available from a genomic test or immunohistochemistry (IHC) test for protein expression performed in a laboratory accredited by the competent local regulatory authority. The genomic or IHC test used to qualify a patient for participation in ProTarget may have been performed on any specimen of the patient's tumor obtained at any point during the patient's care at the discretion of the patient's treating physician. Genomic assays performed on cell-free DNA in plasma ("liquid biopsies") will also be acceptable if the genomic analysis is performed in a laboratory accredited by the competent local regulatory authority.
  • A new biopsy must be performed if possible, for central confirmation by WGS (the result may be awaited and is not required before first dosing).
  • Note: Eligible genomic tests may include any of the following technologies: fluorescence in situ hybridization (FISH), polymerase chain reaction (PCR), comparative genomic hybridization (CGH), next generation sequencing (NGS), whole exome sequencing (WES). The test may have been performed on a fresh (frozen or in RNA-later) or paraffin-embedded specimen of the primary tumor or a metastatic deposit or on cell free DNA derived from plasma, as determined by the treating physician, and must reveal a potentially actionable genomic variant as defined in Section 5.0, or protein overexpression by IHC.
  • Ability to understand and the willingness to sign a written informed consent/assent document
  • Have a tumor genomic profile for which treatment with one of the approved targeted anti-cancer therapies included in this study has potential clinical benefit based on the criteria described in Section 7.0.
  • For orally administered drugs, the patient must be able to swallow and tolerate oral medication and must have no known malabsorption syndrome.
  • Because of the risks of drug treatment to the developing fetus, women of child-bearing potential and men must agree to use highly effective contraception (hormonal or barrier method of birth control; abstinence) in combination with inhibition of ovulation (intrauterine device (IUD), intrauterine hormone-releasing system ( IUS), bilateral tubal occlusion, vasectomized partner or sexual abstinence) for the duration of study participation, and for four to 24 months following completion of study therapy (depending on SPC from individual drugs). Should a woman become pregnant or suspect she is pregnant while participating in this study or if she is the partner of a male participant in this study and becomes pregnant while he is participating in this study, she should inform her or her partner's treating physician immediately as well as her obstetrician. Female study patients who become pregnant must immediately discontinue treatment with any study therapy. Male patients should avoid impregnating a female partner. Male study patients, even if surgically sterilized, (i.e. post-vasectomy) must agree to one of the following: practice effective barrier contraception during the entire study treatment period and through 4 months after the last dose of study drug, or completely abstain from sexual intercourse.
  • Ongoing toxicity \> CTCAE grade 2, other than peripheral neuropathy, related to anti-tumor treatment that was completed within 4 weeks prior to registration. Patients with ongoing peripheral neuropathy of ≥ CTCAE grade 3 will be excluded.
  • +14 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (7)

Aalborg University Hospital

Aalborg, Aalborg, 9000, Denmark

NOT YET RECRUITING

Aarhus University Hospital

Aarhus, Aarhus, 8200, Denmark

NOT YET RECRUITING

Rigshospitalet

Copenhagen, Copenhagen, 2100, Denmark

RECRUITING

Herlev Hospital

Herlev, Herlev, 2730, Denmark

NOT YET RECRUITING

Odense University Hospital

Odense, Odense, 5000, Denmark

NOT YET RECRUITING

Zealand University Hospital

Roskilde, Roskilde, 4000, Denmark

NOT YET RECRUITING

Vejle Sygehus

Vejle, Vejle, 7100, Denmark

NOT YET RECRUITING

Related Publications (1)

  • Kringelbach T, Hojgaard M, Rohrberg K, Spanggaard I, Laursen BE, Ladekarl M, Haslund CA, Harslof L, Belcaid L, Gehl J, Sondergaard L, Eefsen RL, Hansen KH, Kodahl AR, Jensen LH, Holt MI, Oellegaard TH, Yde CW, Ahlborn LB, Lassen U. ProTarget: a Danish Nationwide Clinical Trial on Targeted Cancer Treatment based on genomic profiling - a national, phase 2, prospective, multi-drug, non-randomized, open-label basket trial. BMC Cancer. 2023 Feb 22;23(1):182. doi: 10.1186/s12885-023-10632-9.

MeSH Terms

Conditions

Neoplasms

Interventions

alectinibatezolizumabavelumabAxitinibErlotinib HydrochlorideVemurafenibcobimetinibTrastuzumabpertuzumabAdo-Trastuzumab EmtansineHhAntag691niraparib

Intervention Hierarchy (Ancestors)

BenzamidesAmidesOrganic ChemicalsBenzoatesAcids, CarbocyclicCarboxylic AcidsBenzene DerivativesHydrocarbons, AromaticHydrocarbons, CyclicHydrocarbonsIndazolesPyrazolesAzolesHeterocyclic Compounds, 1-RingHeterocyclic CompoundsHeterocyclic Compounds, 2-RingHeterocyclic Compounds, Fused-RingQuinazolinesSulfonamidesSulfonesSulfur CompoundsIndolesAntibodies, Monoclonal, HumanizedAntibodies, MonoclonalAntibodiesImmunoglobulinsImmunoproteinsBlood ProteinsProteinsAmino Acids, Peptides, and ProteinsSerum GlobulinsGlobulinsMaytansineMacrolidesLactonesLactams, MacrocyclicMacrocyclic CompoundsPolycyclic Compounds

Central Study Contacts

Ulrik Lassen, Prof.

CONTACT

Kristoffer S Rohrberg, MD, PhD

CONTACT

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
SPONSOR INVESTIGATOR
PI Title
Professor, MD, PhD

Study Record Dates

First Submitted

April 8, 2020

First Posted

April 10, 2020

Study Start

August 24, 2020

Primary Completion (Estimated)

April 30, 2029

Study Completion (Estimated)

April 30, 2030

Last Updated

July 25, 2025

Record last verified: 2025-07

Locations