RecruitingPhase 2Phase 3NCT05786716

DETERMINE Trial Treatment Arm 04: Trastuzumab in Combination With Pertuzumab in Adult, Paediatric and Teenage/Young Adult Patients With Cancers With HER2 Amplification or Activating Mutations

DETERMINE (Determining Extended Therapeutic Indications for Existing Drugs in Rare Molecularly Defined Indications Using a National Evaluation Platform Trial): An Umbrella-Basket Platform Trial to Evaluate the Efficacy of Targeted Therapies in Rare Adult, Paediatric and Teenage/Young Adult (TYA) Cancers With Actionable Genomic Alterations, Including Common Cancers With Rare Actionable Alterations. Treatment Arm 04: Trastuzumab in Combination With Pertuzumab in Adult, Paediatric and Teenage/Young Adult Patients With Cancers With HER2 Amplification or Activating Mutations.


Sponsor

Cancer Research UK

Enrollment

30 participants

Start Date

Mar 7, 2023

Study Type

INTERVENTIONAL

Conditions

Summary

This clinical trial is looking at a combination of drugs called trastuzumab and pertuzumab. This combination of drugs is approved together as standard of care treatment for adult patients with breast cancer (often with other anti-cancer drugs). This means it has gone through clinical trials and been approved by the Medicines and Healthcare products Regulatory Agency (MHRA) in the UK. Trastuzumab and pertuzumab work in patients with these types of cancers which have a molecular alteration called HER2 amplification or HER2 activating mutation. Investigators now wish to find out if it will be useful in treating patients with other cancer types which are also HER2 amplified or HER2 mutated. If the results are positive, the study team will work with the NHS and the Cancer Drugs Fund to see if these drugs can be routinely accessed for patients in the future. This trial is part of a trial programme called DETERMINE. The programme will also look at other anti-cancer drugs in the same way, through matching the drug to rare cancer types or ones with specific mutations.


Eligibility

Min Age: 12 Years

Inclusion Criteria25

  • THE PATIENT MUST FULFIL THE ELIGIBILITY CRITERIA WITHIN THE DETERMINE MASTER PROTOCOL (NCT05722886) AND WITHIN THE TREATMENT ARM 04 (TRASTUZUMAB AND PERTUZUMAB) OUTLINED BELOW\*
  • A. Confirmed diagnosis of a malignancy harbouring HER2 amplification, or an appropriate activating mutation as defined by the MTB, using an analytically validated next-generation sequencing method.
  • • A HER2 amplification copy number between 5-9 will require an MTB discussion. A HER2 amplification copy number ≥10 will be fast-tracked for an MTB recommendation, unless there are any patient-specific individualities (such as multiple gene amplifications) that require MTB discussion.
  • B. Age 12 years or above.
  • C. Women of childbearing potential are eligible provided that they meet the following criteria:
  • Have a negative serum or urine pregnancy test before enrolment and;
  • Agree to use one form of effective birth control method such as:
  • I. combined (oestrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation (oral, intravaginal or transdermal):
  • II. progestogen-only hormonal contraception associated with or without inhibition of ovulation (oral, injectable or implantable)
  • III. intrauterine device (IUD)
  • IV. intrauterine hormone-releasing system (IUS)
  • V. bilateral tubal occlusion
  • VI. vasectomised partner
  • VII. sexual abstinence
  • VIII. male or female condom with or without spermicide
  • IX. cap, diaphragm or sponge with spermicide
  • Effective from the first administration of trastuzumab or pertuzumab (whichever is first), throughout the trial and for seven months after the last administration of trastuzumab or pertuzumab (whichever is later).
  • D. Male patients with partners who are women of childbearing potential are eligible provided that they agree to the following, from the first administration of trastuzumab or pertuzumab (whichever is first), throughout the trial and for seven months after the last administration of trastuzumab or pertuzumab (whichever is later):
  • Agree to take measures not to father children by using a barrier method of contraception (condom plus spermicide) or to sexual abstinence.
  • Non-vasectomised male patients with partners who are women of childbearing potential must also be willing to ensure that their partner uses an effective method of contraception as in C, above.
  • Male patients with pregnant or lactating partners must be advised to use barrier method contraception (e.g. condom) to prevent drug exposure of the foetus or neonate.
  • All male patients must refrain from donating sperm for the same period.
  • E. Patients must be able and willing to undergo a fresh tissue biopsy at baseline and blood samples for translational research. Note that for patients with haematological malignancies or neuroblastomas, blood, bone marrow aspiration and/or trephine or lymph node biopsy samples may be taken.
  • F. ADULT PATIENTS (≥18 years): Adequate organ function as per haematological and biochemical indices within the ranges defined in the protocol. These measurements should be performed to confirm the patient's eligibility.
  • G. PAEDIATRIC PATIENTS (\<18 years): Adequate organ function as per haematological and biochemical indices within the ranges defined in the protocol. These measurements should be performed to confirm the patient's eligibility.

Exclusion Criteria16

  • A. Diagnosis of HER2-positive early or metastatic breast cancer.
  • B. Female patients who are pregnant, breastfeeding or planning to become pregnant during the trial or within seven months following their last dose of trastuzumab or pertuzumab (whichever is later).
  • C. Severe dyspnoea at rest due to complications of advanced malignancy or requiring supplementary oxygen therapy.
  • D. Known hypersensitivity to trastuzumab or pertuzumab, murine proteins, or to any of the excipients.
  • E. Patients who were administered a live, attenuated vaccine within 28 days prior to enrolment, or anticipation of need for such a vaccine during trastuzumab and pertuzumab treatment or within six months after the final dose of trastuzumab and pertuzumab.
  • F. Patients with clinically significant pre-existing cardiac conditions, including uncontrolled or symptomatic angina, uncontrolled atrial or ventricular arrhythmias (within three months), NYHA class III or IV congestive heart failure.
  • Left Ventricular Ejection Fraction \<55%.
  • Patients with a cerebrovascular event (including stroke or transient ischaemic attack \[TIA\]) or cardiovascular event (including acute myocardial infarction \[MI\]) within three months before the first dose of trastuzumab and pertuzumab.
  • • Patients with primary CNS tumours may be considered unless intra-tumoural bleeding has occurred within 2 weeks of the first dose of trastuzumab and pertuzumab, and patients with punctate CNS haemorrhages \<3 mm may be considered.
  • G. Prior treatment with the same class of drug unless genetic profile demonstrates a mechanism of resistance known to be potentially sensitive to trastuzumab or pertuzumab.
  • H. Any clinically significant concomitant disease or condition (or its treatment) that could interfere with the conduct of the trial or absorption of oral medications or that would, in the opinion of the Investigator, pose an unacceptable risk to the patient in this trial.
  • I. Known active infections (bacterial, fungal or viral) that would interfere with the assessment of safety or efficacy of trastuzumab and pertuzumab, including human immunodeficiency virus (HIV) positivity. Patients with history of testing positive for HIV infection are eligible provided the each of the following conditions are met:
  • CD4 count ≥350/μL;
  • undetectable viral load;
  • receiving antiretroviral therapy (ART) that does not interact with IMP (patients should be on established ART for at least four weeks); and
  • no HIV/ acquired immune deficiency syndrome (AIDS)-associated opportunistic infection in the last 12 months.

Interventions

DRUGTrastuzumab

An initial loading dose of 8 mg/kg body weight, followed thereafter by a maintenance dose of 6 mg/kg body weight administered intravenously every 21 days. Patients may continue until disease progression without clinical benefit, unacceptable AEs or withdrawal of consent.

DRUGPertuzumab

An initial loading dose of 640 mg, followed thereafter by a maintenance dose of 420 mg administered intravenously every 21 days. Paediatric patients will receive an initial loading dose of 14 mg/kg (maximum 840 mg), followed thereafter by a maintenance dose of 7 mg/kg (maximum 420 mg) administered intravenously every 21 days. Patients may continue until disease progression without clinical benefit, unacceptable AEs or withdrawal of consent.


Locations(27)

Belfast City Hospital

Belfast, United Kingdom

University Hospital Birmingham

Birmingham, United Kingdom

Birmingham Children's Hospital

Birmingham, United Kingdom

Bristol Royal Hospital for Children

Bristol, United Kingdom

Bristol Haematology and Oncology Centre

Bristol, United Kingdom

Addenbrooke's Hospital

Cambridge, United Kingdom

Velindre Cancer Centre

Cardiff, United Kingdom

Cardiff Children's Hospital

Cardiff, United Kingdom

Western General Hospital

Edinburgh, United Kingdom

The Beatson Hospital

Glasgow, United Kingdom

Royal Hospital for Children Glasgow

Glasgow, United Kingdom

Leicester Royal Infirmary

Leicester, United Kingdom

Alder Hey Hospital

Liverpool, United Kingdom

University College London Hospital

London, United Kingdom

Guy's Hospital

London, United Kingdom

Great Ormond Street Hospital

London, United Kingdom

Royal Manchester Children's Hospital

Manchester, United Kingdom

The Christie Hospital

Manchester, United Kingdom

Clatterbridge Cancer Centre

Metropolitan Borough of Wirral, United Kingdom

Great North Children's Hospital

Newcastle, United Kingdom

Freeman Hospital

Newcastle, United Kingdom

Churchill Hospital

Oxford, United Kingdom

John Radcliffe Hospital

Oxford, United Kingdom

Weston Park Hospital

Sheffield, United Kingdom

Sheffield's Children's Hospital

Sheffield, United Kingdom

Southampton General Hospital

Southampton, United Kingdom

The Royal Marsden Hospital

Sutton, United Kingdom

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