This trial will study a type of advanced breast cancer (ABC) defined as endocrine receptor (ER)-positive/human epidermal growth factor receptor 2(HER2)-negative and estrogen receptor 1 (ESR1)-mutated. Patients will be treated with elacestrant, a compound that acts as a selective estrogen receptor degrader, and everolimus (or placebo), a kinase inhibitor indicated for the treatment of postmenopausal women with advanced hormone receptor-positive, HER2-negative breast cancer. The main purpose of the study is to analyze the efficacy (to find out how effective a treatment is) of elacestrant plus everolimus therapy in patients who have ER-positive/HER2-negative, ESR1-mutated, ABC progressing to endocrine therapy and cyclin-dependent kinase 4/6 (CDK4/6) inhibitor. The efficacy of elacestrant plus everolimus combination will be determined by assessing the period from elacestrant plus everolimus (or placebo) treatment initiation until to the first occurrence of disease progression, unacceptable toxicity, death, or discontinuation from the study treatment for any other reason, whichever occurs first, defined as progression free survival. Rigorous eligibility criteria based on specific co-morbidities and clinicopathologic features of their disease have been designed to minimize the risk of patients participating in this study. The anticipated favorable clinical benefits of elacestrant combined with everolimus are projected to outweigh the risks of this treatment. This study will be performed in full compliance with International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) and all applicable local Good Clinical Practice (GCP) and regulations.
Upon meeting all selection criteria, a total of 240 patients will be enrolled. After signing the Informed Consent Form (ICF), patients will be randomized (ratio 1:1) as follows: * Interventional Arm (Arm A) (N=120): Patients will receive 345 mg of elacestrant and 7.5 mg of everolimus orally once daily. * Control Arm (Arm B) (N=120): Patients will receive elacestrant at 345 mg orally once daily plus everolimus placebo. Patients will be stratified by presence of visceral metastases (yes versus no) and duration of prior CDK4/6 inhibitor-based therapy (≥ 12 months versus \< 12 months). Patients progressing to CDK4/6 inhibitor-based therapy in the adjuvant setting will be stratified as patients with a duration of prior CDK4/6 inhibitor-based therapy \< 12 months. Patients will receive study treatment in 28-day cycles until documented disease progression, death, unacceptable toxicity, or discontinuation from the study treatment for any other reason, whichever occurs first. Patients discontinuing the study treatment period will enter a post-treatment follow-up period during which survival and new anti-cancer therapy information will be collected every 3 months (± 14 days) from the last dose of IMPs until the End of Study (EoS) defined as 12 months after last patient is randomized unless premature termination of the trial. For patients who discontinue the study treatment for reasons other than disease progression, tumor assessments will be conducted following the frequency of the study until the start of a new anti-cancer treatment, death, or disease progression.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
240
Patients will receive 7.5 mg of everolimus orally once daily.
Patients will receive elacestrant 345 mg orally once daily
Patients will receive placebo orally once daily
10 mL of alcohol-free dexamethasone 0.5 mg per 5 mL mouthwashes (swish for 2 min and spit, four times daily for 8 weeks). After 8 weeks, dexamethasone mouthwash could be continued for up to eight additional weeks at the discretion of the clinician and patient. Used for prevention of treatment-induced stomatitis.
According to clinical practice (for premenopausal/perimenopausal patients and male patients in both treatment arms). Used to suppress estrogen production.
Medizinische Universität Innsbruck
Innsbruck, Austria
Ordensklinikum Linz Barmherzige Schwestern
Linz, Austria
Ordination Priv.-Doz. Dr. Michael Hubalek
Schwaz, Austria
Masaryk Memorial Cancer Institute
Brno, Czechia
Multiscan Nemocnice Horovice
Hořovice, Czechia
Tomas Bata Regional Hospital in Zlin
To demonstrate superiority of elacestrant+everolimus vs. elacestrant+placebo in prolonging PFS based on a BIRC in patients with ER[+]/HER2[-], ESR1-mutated, ABC that have previously received ET+CDK4/6i (all patients).
Progression Free Survival (PFS), defined as the period from randomization to the first occurrence of disease progression or death from any cause, whichever occurs first, as assessed by a Blinded Imaging Review Committee (BIRC) through the use of RECIST v.1.1.
Time frame: Until disease progression, treatment discontinuation, the start of new anti-cancer treatment, withdrawal of consent, death, or EoS, whichever occurs first, assessed through study completion, an average of 12 months.
To compare overall survival (OS) between treatment groups, in all patients.
OS, defined as the period from randomization to death from any cause, as determined locally by the Investigator.
Time frame: Until disease progression, treatment discontinuation, the start of new anti-cancer treatment, withdrawal of consent, death, or EoS, whichever occurs first, assessed through study completion, an average of 12 months.
To compare investigator-assessed PFS based on local assessment between treatment groups, in all patients.
Investigator-assessed PFS, defined as the period from randomization to the first occurrence of disease progression or death from any cause, whichever occurs first, as determined locally using RECIST v.1.1.
Time frame: Until disease progression, treatment discontinuation, the start of new anti-cancer treatment, withdrawal of consent, death, or EoS, whichever occurs first, assessed through study completion, an average of 12 months.
To compare objective response rate (ORR) between treatment groups, in all patients.
ORR is defined as the rate of patients with a Best Overall Response (BOR) of complete response (CR) or partial response (PR) based on a BIRC and local Investigator assessment through the use of RECIST v.1.1.
Time frame: Until disease progression, treatment discontinuation, the start of new anti-cancer treatment, withdrawal of consent, death, or EoS, whichever occurs first, assessed through study completion, an average of 12 months.
To compare clinical benefit rate (CBR) between treatment groups, in all patients.
CBR is defined as the rate of patients with an objective response (CR or PR), or stable disease for at least 24 weeks based on a BIRC and local Investigator assessment through the use of RECIST v.1.1.
Time frame: Until disease progression, treatment discontinuation, the start of new anti-cancer treatment, withdrawal of consent, death, or EoS, whichever occurs first, assessed through study completion, an average of 12 months.
To compare overall time to response (TTR) between treatment groups, in all patients.
TTR is defined as the period from the randomization to time of the first objective tumor response (tumor shrinkage of ≥ 30%) observed for patients who achieved a BOR of CR or PR based on a BIRC and local Investigator assessment through the use of RECIST v.1.1.
Time frame: Until disease progression, treatment discontinuation, the start of new anti-cancer treatment, withdrawal of consent, death, or EoS, whichever occurs first, assessed through study completion, an average of 12 months.
To compare duration of response (DoR) between treatment groups, in all patients.
DoR is defined as the period from the first occurrence of a documented objective response to disease progression or death from any cause, whichever occurs first, based on a BIRC and local Investigator assessment through the use of RECIST v.1.1.
Time frame: Until disease progression, treatment discontinuation, the start of new anti-cancer treatment, withdrawal of consent, death, or EoS, whichever occurs first, assessed through study completion, an average of 12 months.
To compare best percentage of change in tumor burden between treatment groups, in all patients.
Best percentage of change from baseline in the size of target tumor lesions is defined as the biggest decrease, or smallest increase if no decrease will be observed, based on a BIRC and local Investigator assessment through the use of RECIST v.1.1.
Time frame: Until disease progression, treatment discontinuation, the start of new anti-cancer treatment, withdrawal of consent, death, or EoS, whichever occurs first, assessed through study completion, an average of 12 months.
To describe the changes in health-related quality-of-life (HRQoL) from baseline using the EuroQoL 5 Dimension 5 Level (EQ-5D-5L) scale.
Changes from baseline in the EQ-5D-5L scale. The descriptive system comprises five dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each dimension has 5 levels, indicating, from 1 to 5: no problems, slight problems, moderate problems, severe problems and extreme problems. The patient is asked to indicate their health state by ticking the box next to the most appropriate statement in each of the five dimensions. This decision results in a 1-digit number that expresses the level selected for that dimension. The digits for the five dimensions can be combined into a 5-digit number that describes the patient's health state.
Time frame: Until disease progression, treatment discontinuation, the start of new anti-cancer treatment, withdrawal of consent, death, or EoS, whichever occurs first, assessed through study completion, an average of 12 months.
To describe the changes in HRQoL from baseline using the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30).
Changes from baseline in the EORTC QLQ-C30 scale. All of the scales and single item measures range in score from 0 to 100. A high score for the functional scales and functional single items represents a high/healthy level of functioning, whereas a high score for the symptom scales and symptom item represents a high level of symptomatology or problems.
Time frame: Until disease progression, treatment discontinuation, the start of new anti-cancer treatment, withdrawal of consent, death, or EoS, whichever occurs first, assessed through study completion, an average of 12 months.
To describe the changes in HRQoL from baseline using the EORTC Quality of Life Questionnaire Breast Cancer module (EORTC QLQ-BR42).
Changes from baseline in the QLQ-BR42 scale. All of the scales and single item measures range in score from 0 to 100. A high score for the functional scales and functional single items represents a high/healthy level of functioning, whereas a high score for the symptom scales and symptom item represents a high level of symptomatology or problems.
Time frame: Until disease progression, treatment discontinuation, the start of new anti-cancer treatment, withdrawal of consent, death, or EoS, whichever occurs first, assessed through study completion, an average of 12 months.
To assess adverse events (AEs) between treatment groups in all patients.
Measures will include AEs and treatment-emergent AEs (TEAEs), including NCI CTCAE v5.0 severity grade and relationship to study drugs.
Time frame: Until disease progression, treatment discontinuation, the start of new anti-cancer treatment, withdrawal of consent, death, or EoS, whichever occurs first, assessed through study completion, an average of 12 months.
To assess adverse events leading to study treatment withdrawal.
Measures will include deaths, serious adverse events (SAEs), and AEs leading to study treatment withdrawal.
Time frame: Until disease progression, treatment discontinuation, the start of new anti-cancer treatment, withdrawal of consent, death, or EoS, whichever occurs first, assessed through study completion, an average of 12 months.
To assess adverse events leading to dose modification.
Measures will include dose interruption and reductions due to AEs.
Time frame: Until disease progression, treatment discontinuation, the start of new anti-cancer treatment, withdrawal of consent, death, or EoS, whichever occurs first, assessed through study completion, an average of 12 months.
To assess the number of participants with abnormal hematology and/or chemistry values that are related to treatment.
Number of participants with treatment-related AEs as measured by laboratory parameters (hematology and chemistry) and assessed by NCI CTCAE v5.0 grade.
Time frame: Until disease progression, treatment discontinuation, the start of new anti-cancer treatment, withdrawal of consent, death, or EoS, whichever occurs first, assessed through study completion, an average of 12 months.
To assess the number of participants with changes on vital signs and/or AEs that are related to treatment.
Number of participants with treatment-related changes on vital sign values as determined by respiratory rate, blood pressure (systolic and diastolic), pulse rate, and body temperature over time and assessed by NCI-CTCAE v.5.0.
Time frame: Until disease progression, treatment discontinuation, the start of new anti-cancer treatment, withdrawal of consent, death, or EoS, whichever occurs first, assessed through study completion, an average of 12 months.
To assess the number of participants with changes on cardiac function measured by ECG QT Interval Corrected by Fridericia formula (QTcF) that are related to treatment effect .
Measurement of treatment-related changes in cardiac function measured by ECG QTcF over time.
Time frame: Until disease progression, treatment discontinuation, the start of new anti-cancer treatment, withdrawal of consent, death, or EoS, whichever occurs first, assessed through study completion, an average of 12 months.
To assess the number of participants with changes on Eastern Cooperative Oncology Group (ECOG) Performance Status (PS) that are related to treatment.
Measurement of treatment-related changes on patient's level of functioning in terms of their ability to care for themself, daily activity, and physical ability (walking, working, etc.), as measured with the ECOG PS scale over time.
Time frame: Until disease progression, treatment discontinuation, the start of new anti-cancer treatment, withdrawal of consent, death, or EoS, whichever occurs first, assessed through study completion, an average of 12 months.
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