Neoadjuvant Trastuzumab, Pertuzumab and Zongertinib Without Chemotherapy in HER2-positive Breast Cancer: the TRAIN-5 Study (TRAIN-5)
This is a single-center, phase 1b study evaluating the safety, feasibility and initial efficacy of a neoadjuvant treatment with zongertinib, trastuzumab and pertuzumab in stage II-IIIA HER2-positive breast cancer.
Study Overview
Status
Status
Conditions
Conditions
Intervention / Treatment
Intervention / Treatment
Detailed Description
Neoadjuvant chemotherapy combined with dual HER2-targeted therapy results in high rates of pathological complete response (pCR) and excellent long-term survival outcomes in patients with HER2-positive breast cancer. However, this intensive treatment carries significant toxicity, leading to both acute and long-term side effects. Long-term and potentially irreversible toxicities such as neuropathy, persistent fatigue, and cognitive impairment frequently occur and may significantly impair long-term quality of life. Additionally, brain recurrence remains a well-documented, devastating complication in HER2-positive disease.
Previous studies have demonstrated that neoadjuvant treatment with dual HER2-targeted therapy consisting of trastuzumab and pertuzumab without chemotherapy can induce pCR in 11% to 47% of patients, while event-free survival appeared non-inferior. These findings suggest that omission of chemotherapy may be feasible in selected patients. Strategies to improve patient selection and to enhance HER2-directed therapy using novel highly selective agents may further increase pCR rates and expand the proportion of patients eligible for chemotherapy-free treatment. The use of agents with adequate central nervous system penetration may additionally reduce the risk of brain recurrence.
Zongertinib is an orally administered, highly selective, and irreversible HER2 tyrosine kinase inhibitor with minimal off-target toxicity. It has demonstrated robust preclinical and clinical activity against HER2-altered solid tumours, including efficacy in patients with brain metastases. Given its favourable pharmacologic profile and promising therapeutic potential, zongertinib is a strong candidate for incorporation into a chemotherapy-free neoadjuvant regimen for HER2-positive breast cancer.
Study Type
Study Type
Enrollment (Estimated)
Enrollment
Phase
Phase
- Phase 1
Contacts and Locations
Study Locations
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Amsterdam, Netherlands
- Netherlands Cancer Institute
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Contact:
- Gabe Sonke, MD PhD
- Phone Number: 0205129111
- Email: trainstudies@nki.nl
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Principal Investigator:
- Gabe Sonke, MD PhD
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Participation Criteria
Eligibility Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
Inclusion Criteria:
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Signed written informed consent
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Pathologically confirmed primary invasive breast cancer
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Stage II - IIIA primary breast cancer according to TNM-staging (8th edition, AJCC); (largest tumour diameter DCE-MRI > 2cm (cT2-3) and/or cN1-2 confirmed with FNA or histology)
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Homogeneous HER2 overexpression (IHC 3+) on pre-treatment biopsy, with confirmatory HER2 amplification by in situ hybridization
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Known oestrogen- and progesterone-receptor expression of the invasive tumour
a. Hormone receptor negativity is defined as expression of both oestrogen and progesterone receptors being less than 10%
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WHO performance status of 0 or 1
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18 years of age or older
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LVEF ≥50% measured by echocardiography or MUGA
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Eligible for neoadjuvant treatment
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Laboratory requirements within 4 weeks prior to the start of study treatment:
- Adequate bone marrow function (ANC ≥1.5 x 109/l, platelets ≥100 x 109/l, haemoglobin ≥5.6 mmol/L (≥90 g/L); without the use of hematopoietic growth factors within 4 weeks prior to start of trial medication.
- Adequate hepatic function (ALT and AST ≤3 times upper limit of normal (ULN), Alkaline phosphatase <5 times ULN, Total bilirubin ≤1.5 times the ULN, except for patients with Gilbert's syndrome: total bilirubin ≤3 times ULN or direct bilirubin ≤1.5 times ULN);
- Adequate renal function: Estimated Glomerular Filtration Rate (eGFR) ≥30 mL/min/1.73m2 calculated using Chronic Kidney Disease Epidemiology (CKD-EPI) formula.
- Either International Normalized Ratio (INR) or Prothrombin Time (PT) ≤1.5 times ULN unless on a stable dose of an anticoagulant with no unexplained elevation of INR
- Activated Partial Thromboplastin Time (aPTT) ≤ 1.5 times ULN unless on a stable dose of an anticoagulant with no unexplained elevation of INR
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Women of childbearing potential (WOCBP) must be ready and able to use dual highly effective methods of birth control per ICH M3 (R2) that result in a low failure rate of less than 1% per year when used consistently and correctly during study treatment and 7 months after last dose of trastuzumab
Exclusion Criteria:
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Inflammatory breast cancer, cT4 and/or cN3 tumours
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Occult breast cancer (cT0)
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Bilateral breast cancer
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Current pregnancy or breastfeeding. Women who are breastfeeding can be enrolled if they stop breastfeeding.
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Current or previous other malignancy unless treated without systemic therapy and more than five years ago
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HER2 heterogeneity on pre-treatment biopsy
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Psychological, familial, sociological, or geographical condition potentially hampering compliance with the study protocol and follow-up schedule
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History or presence of cardiovascular abnormalities such as uncontrolled hypertension, congestive heart failure NYHA classification of III or IV, unstable angina or poorly controlled arrhythmia which are considered as clinically relevant by the investigator. Myocardial infarction, stroke, or pulmonary embolism within 6 months prior to registration.
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Any clinically important abnormalities (as assessed by the investigator) in rhythm, conduction, or morphology of resting electrocardiograms
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Mean resting corrected QT interval (QTcF) >470 msec (females) or >450 msec (males) based on screening 12-lead ECG
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Patients with chronic HBV infection with active disease who meet the criteria for anti-HBV therapy (according to local / institutional standard) who have not been treated with suppressive antiviral therapy prior to initiation of study treatment
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Patients with a history of HCV infection who meet one or both of the following criteria:
- Currently receiving curative antiviral treatment
- HCV viral load is above the limit of quantification (HCV RNA positive)
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Patients with history of HIV infection who meet one or more of the following criteria;
- CD4+ count <350 cells/μL
- Viral load >400 copies/μL (local lab assessment)
- Not receiving antiretroviral therapy
- Receiving established antiretroviral therapy for less than 4 weeks prior to the start of trial treatment
- History of AIDS-defining opportunistic infections within 12 months prior to start of trial treatment
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Active or known or history of non-infectious interstitial lung disease/pneumonitis
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Any history or presence of uncontrolled gastrointestinal disorders that could affect the intake and/or absorption of the trial drug (e.g., nausea, vomiting, Crohn's disease, ulcerative colitis, chronic diarrhoea, malabsorption) in the opinion of the investigator
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Active infectious disease requiring systemic therapy, which puts the patient at increased risk in the opinion of the investigator.
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Patients with severe hepatic impairment (classified as Child-Pugh C or score 10-15)
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Known history of allergy to the trial drug(s) or any excipients of the trial drug(s) or contraindication to any of the trial drugs
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Contraindications for MRI or or non-evaluable MRI
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Number of Arms
Arms and Interventions
Participant Group / ArmParticipant Group / Arm |
Intervention / TreatmentIntervention / Treatment |
|---|---|
|
Experimental: This is a single-arm, non-comparative, non-randomized, single-center phase 1b study
Eligible patients are adults (≥18 years) with pathologically confirmed primary stage II-IIIA HER2-positive breast cancer, defined as homogenous HER2 3+ on immunohistochemistry. Additional eligibility criteria include WHO performance status 0-1, left ventricular ejection fraction ≥50%, adequate hepatic, renal and bone marrow function, and absence of current or previous other malignancy unless treated without systemic therapy and disease-free for more than five years. |
All patients will receive neoadjuvant treatment with trastuzumab, pertuzumab and zongertinib. Patients with hormone receptor-positive tumours will additionally receive concurrent endocrine therapy with an aromatase inhibitor; premenopausal patients will also receive an LHRH antagonist. Breast MRI will be performed at baseline and after three cycles to assess functional tumour volume (FTV). Patients demonstrating ≥65% FTV reduction will continue the chemotherapy-free regimen for an additional three cycles. Patients with <65% FTV will switch to standard neoadjuvant chemotherapy for three cycles. Following completion of neoadjuvant therapy, patients will undergo surgery within six weeks. Those achieving pCR will continue adjuvant trastuzumab according to ESMO guidelines. Patients without pCR will receive adjuvant chemotherapy according to standard care. |
What is the study measuring?
Primary Outcome Measures
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
|---|---|---|
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Safety
Time Frame: Incidence and severity of adverse events (all grades) occuring during treatment and up to 30 days after last administration of study treatment.
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The primary objective is to evaluate the safety of neoadjuvant chemotherapy-free regimen consisting of trastuzumab, pertuzumab and zongertinib in patients with stage II-IIIA HER2-positive breast cancer. |
Incidence and severity of adverse events (all grades) occuring during treatment and up to 30 days after last administration of study treatment.
|
Secondary Outcome Measures
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
|---|---|---|
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Progressive disease during neoadjuvant treatment
Time Frame: Patients will participate in the study for approximately 24 weeks (including surgery), with a follow-up period of up to 10 years.
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Progressive disease during neoadjuvant treatment |
Patients will participate in the study for approximately 24 weeks (including surgery), with a follow-up period of up to 10 years.
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Pathological complete response
Time Frame: Patients will participate in the study for approximately 24 weeks (including surgery), with a follow-up period of up to 10 years.
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Pathological complete response |
Patients will participate in the study for approximately 24 weeks (including surgery), with a follow-up period of up to 10 years.
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Residual cancer burden
Time Frame: Patients will participate in the study for approximately 24 weeks (including surgery), with a follow-up period of up to 10 years.
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Residual cancer burden |
Patients will participate in the study for approximately 24 weeks (including surgery), with a follow-up period of up to 10 years.
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|
Radiological response
Time Frame: Patients will participate in the study for approximately 24 weeks (including surgery), with a follow-up period of up to 10 years.
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Radiological response |
Patients will participate in the study for approximately 24 weeks (including surgery), with a follow-up period of up to 10 years.
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Event-free survival
Time Frame: Patients will participate in the study for approximately 24 weeks (including surgery), with a follow-up period of up to 10 years.
|
Event-free survival |
Patients will participate in the study for approximately 24 weeks (including surgery), with a follow-up period of up to 10 years.
|
|
Overall survival
Time Frame: Patients will participate in the study for approximately 24 weeks (including surgery), with a follow-up period of up to 10 years.
|
Overall survival |
Patients will participate in the study for approximately 24 weeks (including surgery), with a follow-up period of up to 10 years.
|
Collaborators and Investigators
Sponsor
Sponsor
Collaborators
Collaborators
Study record dates
Study Major Dates
Study Start (Estimated)
Study Start
Primary Completion (Estimated)
Primary Completion
Study Completion (Estimated)
Study Completion
Study Registration Dates
First Submitted
First Submitted
First Submitted That Met QC Criteria
First Submitted That Met QC Criteria
First Posted (Actual)
First Posted
Study Record Updates
Last Update Posted (Actual)
Last Update Posted
Last Update Submitted That Met QC Criteria
Last Update Submitted That Met QC Criteria
Last Verified
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
Other Study ID Numbers
- N25TRN
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
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