A Randomized Phase II Study to Evaluate the Incidence of Discontinuations due to Diarrhoea at 3 Cycles in patients with Early-stage HER2-positive (HER2+), Hormone Receptor-positive (HR+) Breast Cancer treated with Neratinib plus Loperamide prophylaxis versus Neratinib with Initial Dose Escalation plus PRN Loperamide prophylaxis versus Neratinib plus Loperamide plus Colesevelam prophylaxis.
This is an international, multicenter, prospective, controlled, randomized, adaptative, phase II study to evaluate the incidence of discontinuations due to diarrhoea within the first 3 cycles in patients with early-stage HER2+ and HR+ breast cancer treated with neratinib plus loperamide prophylaxis for the first 2 cycles versus neratinib with initial 2-week dose escalation plus PRN loperamide versus neratinib and loperamide plus colesevelam prophylaxis for 28 days. After the preplanned therapy, prophylaxis or treatment for diarrhoea will be given as clinically indicated following the standard of care by the treating physician. Approximately 315 patients will be enrolled in the study. All enrolled patients will receive neratinib orally once daily for 13 cycles, continuously. Eligible patients will be randomly assigned in a 1:1:1 ratio to one of the diarrhoea prophylaxis arms, using an interactive response technology (IRT) module within the electronic data capture (EDC) system. Patients will be stratified according to menopausal status (premenopausal versus postmenopausal) and prior anti-HER2 therapy (trastuzumab only versus trastuzumab plus pertuzumab). Baseline assessments will be performed prior to C1D1 dosing. During the first 3 cycles of treatment, safety data will be collected during the cycle visits. After the first 3 cycles, all patients will enter in a follow-up period to complete approximately 1 year of neratinib treatment. During this follow-up period, safety data will be collected every 3 months. An End-of-Treatment (EOT) Visit is planned on cycle 13 day 28 for all treatment arms (unless patient discontinues earlier), followed by a Safety Follow-up Visit (30 +/-5 days after the last dose of neratinib). This will be the core phase of the study. After this safety follow-up visit, long term outcome data will be collected for 5 years to address the exploratory objectives. This will be the extended phase of the study. Archived primary tumor tissue (at baseline) and whole blood samples (at baseline, during the treatment and follow-up period, and at disease relapse) will be collected for the exploratory analyses. Patients are anticipated to participate in the core phase of the study for approximately 1 year to address primary and secondary objectives (28 days for screening, approximately 12 months to complete neratinib treatment, and 30 days for a safety follow-up visit after the last dose of neratinib). Later on patients will continue in the extended phase of the study and will be followed-up for at least 5 years to collect long term outcome data to conduct the exploratory analyses. The approximate duration of the full study is 8 years. The objectives of the study are indicated below: Primary objectives: To evaluate the incidence of neratinib discontinuations due to diarrhoea within the first 3 cycles (1 cycle = 28 days) in patients with early-stage HER2 overexpressed/amplified (HER2+), hormone receptor-positive (HR+) breast cancer who have completed adjuvant trastuzumab-based therapy. Primary End-point: Incidence of neratinib discontinuations due to diarrhoea at the end of 3 cycles of neratinib treatment. Secondary Objectives: * Incidence and time of neratinib discontinuations due to any treatment-emergent adverse event (TEAE). * Diarrhoea due to neratinib: incidence, duration, severity, and treatment interventions. * Incidence of neratinib discontinuation due to any reason. * Incidence of hospitalisations (overall and for diarrhoea). * Incidence of TEAEs and serious adverse events (SAEs) and adverse events of special interest (AESIs, ie, hepatic, cardiac, pulmonary, reproductive and developmental). * Neratinib exposure assessment. * Determine the effect of study treatment on quality of life, as measured by patient reported outcomes, in all treatment arms. Secondary End-points: * Incidence and time to neratinib discontinuations due to any TEAE. * Incidence, cumulative duration and time to first episode of any diarrhoea and grade 3 or higher diarrhoea. * Incidence and time to neratinib discontinuation due to any reason. * Incidence of hospitalisations due to any reason and diarrhoea. * Incidence of TEAEs and SAEs that included AESIs (i.e. hepatic, cardiac, pulmonary, reproductive and developmental). * Incidence of Neratinib dose modifications (reductions and dose holds), and dose intensity. * Systemic therapy-induced diarrhea Assessment Tool (STIDAT), Functional Assessment of Cancer Therapy Questionnaire for Breast Cancer (FACT B) and EuroQol 5 Dimensions 5 Levels (EQ5D-5L) questionnaires. Exploratory Objectives: Evaluate minimal residual disease (MRD) and molecular alterations associated with patient outcome, and/or the development of diarrhoea with neratinib. Exploratory End-points: Correlation of biomarkers data with patient outcome and safety data.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
PREVENTION
Masking
NONE
Enrollment
177
Neratinib orally once daily for 13 cycles, unless patient discontinues earlier.
Loperamide orally.
Colesevelam capsules orally, 2 times a day for the first month (28 days).
Hospital General Universitario de Elche
Elche, Alicante, Spain
Hospital Costa del Sol
Málaga, Andalusia, Spain
Hospital Clínico Universitario Lozano Blesa
Zaragoza, Aragon, Spain
Institut Català d'Oncología (ICO) L'Hospitalet
L'Hospitalet de Llobregat, Barcelona, Spain
Althaia Xarxa Asistencial de Manresa
Manresa, Barcelona, Spain
The incidence of neratinib discontinuations due to diarrhoea at the end of 3 cycles (1 cycle= 28 days) of neratinib treatment.
The proportion of patients who discontinue the treatment with neratinib due to diarrhoea within this time period.
Time frame: Up to 3 months
Incidence of neratinib discontinuations due to any TEAE (treatment-emergent adverse event).
The proportion of patients who discontinue the treatment of neratinib at any time due to any TEAE. TEAE are undesirable events not present prior to medical treatment, or an already present event that worsens either in intensity or frequency following the treatment.
Time frame: Up to 13 months
Time to neratinib discontinuations due to any TEAE (treatment-emergent adverse event).
The time from the start of neratinib therapy to the discontinuation due to any TEAE.
Time frame: Up to 13 months
Incidence of diarrhoea of any grade/grade 3 or higher.
The proportion of patients with at least one TEAE of diarrhoea of any grade/grade 3 or higher coded and graded by the investigator according to the NCI-CTCAE version 5.0.
Time frame: Up to 13 months
Cumulative duration of diarrhoea (Grade 2/3/4).
The time from the diagnosis of each of the different diarrhoea Grades 2 or 3 or 4 coded and graded by the investigator according to the NCI-CTCAE version 5.0 to the time of change to a different grade of this adverse event.
Time frame: Up to 13 months
Time to first episode of diarrhoea.
The time from the start of neratinib therapy to the first episode of diarrhoea of any grade, whichever occurs first.
Time frame: Up to 13 months
Incidence of neratinib discontinuation (for any reason).
The proportion of patients who discontinued neratinib early (before 1 year of therapy).
Time frame: Up to 13 months
Time to neratinib discontinuation/neratinib treatment duration.
The time from the start of neratinib to the last dose of neratinib.
Time frame: Up to 13 months
Incidence of hospitalisations due to any reason and diarrhoea.
The proportion of patients who have a hospitalisation during the treatment with neratinib or 30 days after the last dose.
Time frame: Up to 13 months
Incidence of TEAEs and SAEs that include AESIs (ie, hepatic, cardiac, pulmonary, reproductive and developmental).
The proportion of patients in which those events are observed.
Time frame: Up to 13 months
Cumulative dose of neratinib.
The total dose of neratinib administered during the study.
Time frame: Up to 13 months
Neratinib dose intensity.
The cumulative dose of neratinib divided by the neratinib treatment duration.
Time frame: Up to 13 months
Neratinib relative dose intensity.
The dose intensity divided by 240 mg.
Time frame: Up to 13 months
Patient reported outcomes (PRO) of health related quality of life measured by FACT B.
PRO of health related quality of life will be assessed using the FACT-B questionnaire. The FACT-B is a 36-item questionnaire composed of five multi-item functional subscales: physical well-being, social/family well-being, emotional well-being, functional well-being and a subscale related with the breast cancer and its treatment. The questionnaire employs 5 points Likert scales with responses from "not at all" to "very much". The total score is obtained from the sum of the score on each subscale. Patients will complete a questionnaire at cycle 1 day 1 (before the first dose of neratinib and anti-diarrhoeal therapy), on day 1 of cycles 2, 3, 4, 7 and 10 and at the End of Treatment Visit.
Time frame: Up to 13 months
Patient reported outcomes (PRO) of health related quality of life measured by EQ5D-5L
To be assessed using the EQ5D-5L questionnaire, a standardized instrument for measuring generic health status. It has 2 components: * health state description: measured in terms of 5 dimensions; mobility (person's walking ability), self-care (ability to wash or dress by oneself), usual activities (work, study, housework, family or leisure activities), pain/discomfort (how much pain or discomfort they have), and anxiety/depression (how anxious or depressed they are). The number of levels of severity is 5; having no problems, slight problems, moderate problems, severe problems and being unable to do/extreme problems. The respondents self-rate their level of severity for each dimension. * Evaluation: It also includes a visual analogue scale, EQ VAS, which records patient's self-rated health on a scale from 0 (worst imaginable) to 100 (best imaginable). To be completed at day 1 of cycles 1, 2, 3, 4, 7 and 10 and at the End of Treatment Visit.
Time frame: Up to 13 months
Patient reported outcomes (PRO) of health related quality of life measured by Systemic Therapy-Induced Diarrhea Assessment Test (STIDAT)
PRO of health related quality of life will be assessed using the STIDAT questionnaire. The STIDAT is a questionnaire that was developed using the FDA iterative process for patient-reported outcomes in which patients define diarrhoea based on presence of watery stool. The STIDAT assess patient's perception of having diarrhoea, daily number of bowel movements, daily number of diarrhoea episodes, antidiarrheal medication use, the presence of urgency, abdominal pain, abdominal spasms or fecal incontinence, patient's perception of diarrhoea severity, and QoL. Patients will complete the instrument at cycle 1 day 1 (before the first dose of neratinib and anti-diarrhoeal therapy), on day 1 of cycles 2, 3, 4, 7, 10 and at the End of Treatment Visit.
Time frame: Up to 13 months
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Hospital de Mataró
Mataró, Barcelona, Spain
Consorcio Hospitalario Provincial de Castellón
Castellon, Castellón, Spain
Hospital Clínico Universitario de Valladolid
Valladolid, Castille and León, Spain
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