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    Summary
    EudraCT Number:2021-001246-36
    Sponsor's Protocol Code Number:ACE-Breast-03
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2021-12-20
    Trial results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedFrance - ANSM
    A.2EudraCT number2021-001246-36
    A.3Full title of the trial
    A Global, Phase 2 Study of ARX788 in HER2-positive Metastatic Breast Cancer Patients Whose Disease is Resistant or Refractory to T-DM1, and/or T-DXd, and/or Tucatinib-containing Regimens
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A clinical research study to test different doses of a study drug (ARX788), to see which dose is safest and its effectiveness(shrinks tumor size) in patients with advanced cancer.
    A.4.1Sponsor's protocol code numberACE-Breast-03
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT04829604
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorAmbrx, Inc.
    B.1.3.4CountryUnited States
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportAmbrx, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAmbrx, Inc.
    B.5.2Functional name of contact pointClinical Trial Information Desk
    B.5.3 Address:
    B.5.3.1Street Address10975 North Torrey Pines Road
    B.5.3.2Town/ cityLa Jolla
    B.5.3.3Post codeCA 92037
    B.5.3.4CountryUnited States
    B.5.6E-mailbreast03trialinquiry@ambrx.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameARX788
    D.3.2Product code ARX788
    D.3.4Pharmaceutical form Solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INN-
    D.3.9.1CAS number 2636710-07-9
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number20
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product Yes
    D.3.11.13.1Other medicinal product typeARX788 is an antibody-drug conjugate consisting of 2 intermediates: The anti-human epidermal growth factor receptor 2 (HER2) monoclonal antibody (mAb) The cytotoxic payload, AS269 trifluoroacetate
    D.8 Information on Placebo
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Metastatic Breast Cancer Resistant or Refractory
    E.1.1.1Medical condition in easily understood language
    Advanced Cancer that is metastasic
    E.1.1.2Therapeutic area Diseases [C] - Cancer [C04]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10027475
    E.1.2Term Metastatic breast cancer
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level PT
    E.1.2Classification code 10055113
    E.1.2Term Breast cancer metastatic
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The confirmed objective response rate (ORR) of ARX788 by blinded independent central review (BICR) based on RECIST v1.1 in subjects with metastatic HER2-positive breast cancer whose disease is resistant or refractory to T-DM1, and/or T-DXd, and/or tucatinib-containing regimens.
    E.2.2Secondary objectives of the trial
    - To evaluate duration of response (DOR), best overall response (BOR), disease control rate (DCR), progression-free survival (PFS), and overall survival (OS)
    - To evaluate the safety, tolerability, and immunogenicity profile of ARX788
    - To determine the pharmacokinetics (PK) of ARX788, total anti-HER2 antibody, and pAF-AS269
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Age ≥ 18 years
    2. Life expectancy > 3 months.
    3. ECOG Performance Status ≤ 1
    4. Metastatic breast cancer subjected previously treated with T-DM1, and/or T-DXd, and/or tucatinib-containing regimens. Subjects must have been previously treated with one (or more) of these regimens to be eligible. Subjects must have been treated with trastuzumab plus taxane.
    Subjects who received prior pertuzumab, lapatinib, neratinib, margetuximab, and/or other available and accessible HER2-directed therapies or investigational therapies are eligible.
    5. Presence of at least one measurable lesion per RECIST v 1.1 as determined by BICR.
    6. A tumor block or formalin-fixed paraffin-embedded (FFPE) tissue from tumor biopsies as 10 pre-cut unstained slides must be collected for the HER2 status evaluation and biomarker analysis based on most recent tumor tissue sample. If insufficient tissue, a fresh tumor biopsy must be collected. All tumor tissue (including the pathology report and methods of pathology laboratory's tissue preparation/testing) needs to be sourced for central laboratory for HER2 testing.
    7. Central laboratory-confirmed HER2-positive expression (estrogen receptor/progesterone receptor positive subjects may be enrolled if they are HER2-positive) according to 2018 American Society of Clinical Oncology – College of American Pathologists (ASCO-CAP) guidelines. See Laboratory Manual for details.
    8. Subjects whose brain metastases have been treated may participate provided they show radiographic stability (defined as 1 brain MR image, obtained at least four weeks after treatment to the brain metastases, shows no evidence of intracranial progression). In addition, any neurologic symptoms (including seizures) that developed either as a result of the brain metastases or their treatment must have returned to baseline or resolved. Any steroids administered as part of this therapy must be completed at least three days prior to study medication. After approximately 20 such subjects [20% of the first 100 subjects] have been enrolled, subsequent subjects with any current or past history of brain metastases will be excluded.
    9. Acute toxicities from any prior therapy, surgery, or radiotherapy must have resolved to Grade 0 or 1 as per the NCI-CTCAE v 5.0, except alopecia.
    10. Adequate bone marrow function defined by absolute neutrophil count of ≥ 1.5×109/L, platelet count of ≥ 100.0×109/L, and hemoglobin of ≥ 9.0 g/dL.
    11. Adequate hepatic function, as defined by serum total bilirubin ≤ 1.5 × ULN, serum aspartate aminotransferase and alanine aminotransferase ≤ 2.5 × ULN. Subjects with known hepatic metastases or primary biliary cancer may have serum total bilirubin ≤ 3.5 × ULN and transaminases ≤ 5 × ULN.
    12. Adequate renal function, defined as creatinine clearance ≥ 30 mL/min, as calculated using the Cockcroft Gault equation: ([{140 -age in years} × {actual weight in kg}] divided by [{72 × serum creatinine in mg/dL}
    multiplied by 0.85 if female]).
    13. Adequate cardiac function as assessed by left ventricular ejection fraction ≥ 50% or institutional lower limit of normal; cumulative anthracycline dose < 360 mg/m2 doxorubicin or equivalent.
    14. Willing and able to understand and sign an informed consent inform and to comply with all aspects of the protocol.
    15. Male and female subjects of reproductive/childbearing potential must agree to use a highly effective form of contraception or avoid intercourse during and upon completion of the study and for at least 6 months after the last dose of study drug.
    16. Male subjects must not freeze or donate sperm starting at Screening and throughout the study period, and at least 6 months after the final study drug administration.
    17. Female subjects must not donate, or retrieve for their own use, ova from the time of Screening and throughout the study treatment period, and for at least 6 months after the final study drug administration.
    E.4Principal exclusion criteria
    1. History of allergic reactions to any component of ARX788.
    2. Prior history of interstitial lung disease, pneumonitis, or other clinically significant lung disease within 12 months prior to screening, with the exception of that directly attributable to the presence of lung metastases from their underlying cancer. Subjects with significant treatment-related lung injury, defined as any of the following, will be excluded:
    a) Any prior history of drug-induced immune-mediated pneumonitis.
    b) Prior history of radiation therapy to the chest of > 18 Gy with residual sequelae considered clinically significant by Investigator assessment.
    c) Radiographic evidence of radiation fibrosis involving > 15% of the lung parenchyma associated with clinical symptoms.
    3. Any active ocular infections until resolved or any chronic corneal disorder unless approved by Medical Monitor. Keratopathy of grade ≤1 due to prior treatment with anti-HER2 ADC or chemotherapy are allowed.
    4. History of congestive heart failure, unstable angina pectoris, unstable atrial fibrillation, or cardiac arrhythmia within 12 months prior to enrollment. Medical history of myocardial infarction within 6 months prior to enrollment. Baseline QTc, averaged over 3 screening ECGs must be ≤ 470 msec (females) or ≤ 450 msec (males).
    5. Grade 3 to 4 peripheral neuropathy (NCI-CTCAE v 5.0). Patients with Grade 2 neuropathy can be enrolled at investigator’s discretion.
    6. Non-manageable electrolyte imbalances including hypokalemia, hypocalcemia, or hypomagnesemia (Grade 2 or greater based on NCI-CTCAE v 5.0).
    7. Any uncontrollable intercurrent illness, infection (including subjects with active, symptomatic Covid-19 infections), or other conditions that could limit study compliance or interfere with assessments.
    8. Exposure to any other investigational or commercial anticancer agents or therapies administered with the intention to treat malignancy within 2814 days before the first dose of ARX788. Anti-hormonal therapy may be administered up to 7 days prior to the first dose of ARX788.
    9. Major surgical intervention within 21 days of the first dose of ARX788 or with ongoing post-operative complications.
    10. Radiotherapy administered less than 21 days prior to the first dose of ARX788, or localized palliative radiotherapy administered less than 7 days prior to the first dose of ARX788, or radiotherapy-induced toxicity of Grade 2 or greater based on NCI-CTCAE v 5.0.
    11. Pregnant or breast feeding.
    12. Known active HCV, HBV, and/or HIV infection. HIV test is not required for the screening, unless required by the local Health Authorities.
    E.5 End points
    E.5.1Primary end point(s)
    Objective response rate (ORR) by blinded independent central review (BICR).
    E.5.1.1Timepoint(s) of evaluation of this end point
    Monitored throughout the study.
    E.5.2Secondary end point(s)
    - Efficacy: Anticancer activity of ARX788, including:
    o Duration of response (DOR)
    o Best overall response (BOR) (CR, PR, SD, or progressive disease)
    o Disease control rate o Progression-free survival (PFS)
    o Overall survival (OS)
    - Safety: The safety, tolerability, and immunogenicity profile assessed by AE, vital signs, ECG, LVEF, clinical laboratory tests, ADA assays, physical examination, medical history and prior and concomitant medication, and where applicable,
    changes from baseline .
    - Pharmacokinetics: Serum concentrations of ARX788, total antibody, and metabolite pAF-AS269 will. PK and PDx characteristics of ARX788 and its major metabolites
    - Immunogenicity: Presence of ADA and neutralizing ADA and their potential impact on the PK, efficacy, and safety of ARX788.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Monitored throughout the study
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned12
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA60
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Australia
    Hong Kong
    Belgium
    Brazil
    Canada
    Czechia
    Germany
    Italy
    Korea, Republic of
    Portugal
    Spain
    Switzerland
    Taiwan
    United States
    France
    E.8.7Trial has a data monitoring committee No
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LPLV
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years4
    E.8.9.1In the Member State concerned months2
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial months2
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 157
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 53
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state12
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 62
    F.4.2.2In the whole clinical trial 210
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    Standard of care. At investigator decision.
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2022-02-22
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-02-15
    P. End of Trial
    P.End of Trial StatusOngoing
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