Pilot Decentralized Clinical Trial in Men and Pre and Post-menopausal Women With Breast Cancer and a Specific Mutation (PIK3CA) Treated With Alpelisib in Combination With Fulvestrant

Study Overview
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Brief Summary
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The study is designed to identify and register practical observations and experiences in connection with planning and implementing decentralized, patient-centered clinical trials at a geographic distance with virtual elements.

Detailed Description
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Open-label, single arm, multi-center, Phase II interventional pilot trial. Primary objective will be to assess participant satisfaction with the decentralized clinical trial (DCT) experience. The trial will enroll men and pre- and post-menopausal women with a HR-positive/HER2-negative ABC (loco regionally recurrent not amenable to curative therapy or metastatic) with a PIK3CA mutation, which progressed on or after endocrine-based treatment. Participants will receive alpelisib 300 mg daily and fulvestrant 500 mg administered intramuscularly on Cycle 1, Day 1 and Cycle 1, Day 15, and on Day 1 of each cycle thereafter until Cycle 12. Pre-menopausal women will receive goserelin 3.6 mg on Day 1 of each cycle.

Official Title
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Open-label, Multicenter, Pilot-trial Evaluating the Safety and Utility of a Hybrid Decentralized Clinical Trial (DCT) Approach Using a TELEmedicine Platform in Patients With HR-positive/HER2-negative Advanced Breast Cancer With a PIK3CA Mutation Treated With Alpelisib - Fulvestrant TELEPIK Trial

Conditions
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Advanced Breast Cancer

Intervention / Treatment
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* Drug: Alpelisib
* Drug: fulvestrant


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Participant is an adult ≥18 years old at the time of consent Participant with ABC (loco regionally recurrent or metastatic) not amenable to curative therapy. Participant with a histologically and/or cytologically confirmed diagnosis of ER-positive and/or PR-positive breast cancer by local laboratory. Participant with a confirmed HER2-negative ABC. Participant with a pathology report confirming PIK3CA mutant status by a certified laboratory using a validated PIK3CA mutation assay (from either tissue or blood). Participant is a man or a pre- or post-menopausal woman. Participant is willing to operate a smartphone compatible with the software of the medical device and willing to manage applications Participant is willing to use the telemedicine platform and to follow the remote participant monitoring procedure. Participant has signed an informed consent form before any trial Exclusion Criteria: Participant has received prior treatment with any PI3K, mTOR or AKT inhibitor. Participant with known hypersensitivity to alpelisib or fulvestrant, or to any of the excipients of alpelisib or fulvestrant. Participant participated in a prior investigational study within 30 days prior to the start of trial treatment or within 5 half-lives of the trial treatment, whichever is longer. Other protocol-defined inclusion/exclusion criteria may apply.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 99 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: single arm<br>alpelisib 300 mg daily and fulvestrant 500 mg administered intramuscularly on Cycle 1, Day 1 and Cycle 1, Day 15, and on Day 1 of each cycle thereafter until Cycle 12. | Drug: Alpelisib<br>* Alpelisib 300 mg daily up to 12 cycles of 28 days each<br>Drug: fulvestrant<br>* fulvestrant 500 mg administered intramuscularly on Cycle 1, Day 1 and Cycle 1, Day 15, and on Day 1 of each cycle thereafter until Cycle 12.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Participant satisfaction with the decentralized clinical trial (DCT) experience | change in the Trial Feedback Questionnaire (TFQ) assessed at the start of the trial, every 12 weeks, and at the end of trial through the Trial Feedback Questionnaire (TFQ) | up to 12 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Patient retention on DCT | Proportion of participants on remote monitoring at 3, 6, 12 months for participants still on treatment. | 3, 6, 12 months | 
| Compliance to treatment | Overall compliance | up to 12 months | 
| Proportion of participants with AESIs (hyperglicemia, rash and diarrhea) | Adverse events of special interest (AESIs) per Common Terminology Criteria for Adverse Events (CTCAE) v4.03 | up to 12 months | 
| Proportion of participants with AEs leading to in-clinic visits | Adverse events (AEs) that lead to in-clinic visits. | up to 12 months | 
| Change from baseline in the EORTC QLQ C30 score. | The European Organization for Research and Treatment of Cancer's core quality of life questionnaire (EORTC QLQ-C30) contains 30 items and is composed of both multi-item scales and single item measures. These include five functional scales (physical, role, emotional, cognitive and social functioning), three symptom scales (fatigue, nausea/vomiting, and pain), six single items (dyspnea, insomnia, appetite loss, constipation, diarrhea and financial impact) and a global health status/QoL scale. | baseline, 3,6,9,12 months | 
| Progression-free survival (PFS) according to RECIST 1.1 | Effectiveness of alpelisib plus fulvestrant | 3,6,9,12 months | 
| Total number of unscheduled in-clinic visits because of safety reasons | safe and suitable remote management of patients | up to 12 months | 
| Total number of unscheduled in-clinic visits and the reason | safe and suitable remote management of patients | up to 12 months | 
| Number of unscheduled in-clinic visits per participant in the study | safe and suitable remote management of patients | up to 12 months | 
| Discontinuation rate related to adverse events | safe and suitable remote management of patients | up to 12 months | 
| Number and proportion of AEs leading to in-clinic visits | AEs of special interest (AESIs) and AEs leading to in-clinic visits | up to 12 months | 
| Change from baseline in the EQ-5D-5L score | The 5-level EQ-5D (EQ-5D-5L) questionnaire is a standardized measure of health status. The EQ-5D descriptive system comprises of the 5 following dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each dimension has 5 levels: no problems, slight problems, moderate problems, severe problems and extreme problems. | baseline, 3,6,9,12 months | 
| Change from baseline in the BPI-SF score | The Brief Pain Inventory Short Form (BPI-SF) is a self-administered assessment tool validated to assess pain The basic aim of the BPI is to provide the information on the intensity of pain, along with the degree to which the pain interferes with the everyday functioning of life. The BPI uses simple numeric rating scales from 0 (no pain) to 10 (severe pain). | baseline, 3,6,9,12 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Decentralized clinical trial, advanced breast cancer, PIK3CA mutation, alpelisib