E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
HR-positive/HER2-negative advanced breast cancer with a PIK3CA mutation |
|
E.1.1.1 | Medical condition in easily understood language |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Cancer [C04] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 21.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10072737 |
E.1.2 | Term | Advanced breast cancer |
E.1.2 | System Organ Class | 100000004864 |
|
E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To assess participant satisfaction with the DCT experience |
|
E.2.2 | Secondary objectives of the trial |
• To explore patient retention on DCT approach
• To explore if the DCT approach ensures safe and suitable remote management of patients
• Patient compliance to treatment
• To assess adverse events (AEs) of special interest (AESIs) and AEs leading to in-clinic visits
• To evaluate participant-reported global health status, quality of life (QOL) and pain
• To assess the effectiveness of alpelisib plus fulvestrant
|
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Participant is an adult ≥18 years old at the time of consent
2. Participant with ABC (loco regionally recurrent or metastatic) not amenable to curative therapy.
3. Participant with a histologically and/or cytologically confirmed diagnosis of ER-positive and/or PR-positive breast cancer by local laboratory.
4. Participant with a confirmed HER2-negative ABC.
5. Participant with a pathology report confirming PIK3CA mutant status by a certified laboratory using a validated PIK3CA mutation assay (from either tissue or blood).
6. Participant is a man or a pre- or post-menopausal woman.
7. Participant is willing to operate a smartphone compatible with the software of the medical device and willing to manage applications
8. Participant is willing to use the telemedicine platform and to follow the remote participant monitoring procedure.
9. Participant has signed an informed consent form before any trial related activities and according to local guidelines.
|
|
E.4 | Principal exclusion criteria |
1. Participant has received prior treatment with any PI3K, mTOR or AKT inhibitor.
2. Participant with known hypersensitivity to alpelisib or fulvestrant, or to any of the excipients of alpelisib or fulvestrant.
3. 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.
|
|
E.5 End points |
E.5.1 | Primary end point(s) |
Participant satisfaction, assessed at the start of the trial, every 12 weeks, and at the end of trial through the Trial Feedback Questionnaire (TFQ) |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
at the start of the trial, every 12 weeks, and at the end of trial |
|
E.5.2 | Secondary end point(s) |
Endpoints for secondary objectives
• Proportion of participants on remote monitoring at 3, 6, 12 months for participants still on treatment.
Statistical assumptions (descriptive): targeted proportion of patients remaining on remaining on remote monitoring after 6 months, 75%
Note: unscheduled in-clinic visit does not exclude for DCT monitoring
• Total number of unscheduled in-clinic visits because of safety reasons
• Total number of unscheduled in-clinic visits and the reason
• Number of unscheduled in-clinic visits per participant in the study
• Discontinuation rate related to adverse events
Note: Visits initially planned to be performed remotely but finally performed on site and unscheduled visits on site or at the local oncologist practice will be considered as unscheduled in-clinic visits. Visits that require treating oncologists or district nurses assessments are considered unscheduled visits.
• Overall compliance
• Type, frequency and severity of AESIs (hyperglycemia, rash, and diarrhea) per Common Terminology Criteria for Adverse events (CTCAE) v4.03 (incidence proportion)
• Number and proportion of AEs leading to in-clinic visits
• Change in scores of participant reported outcome (PRO) questionnaire (within same participant) from baseline to each time point of questionnaire administration (every 12 weeks)
• PFS according to RECIST 1.1 |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
• Proportion of participants on remote monitoring at 3, 6, 12 months for participants still on treatment.
The following will be monitored on an ongoing basis:
• Total number of unscheduled in-clinic visits because of safety reasons
• Total number of unscheduled in-clinic visits and the reason
• Number of unscheduled in-clinic visits per participant in the study
• Discontinuation rate related to adverse events
• Overall compliance
• Type, frequency and severity of AESIs
• Number and proportion of AEs leading to in-clinic visits
• Change in scores of participant reported outcome (PRO) questionnaire (within same participant) from baseline to each time point of questionnaire administration (every 12 weeks)
|
|
E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | No |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | Yes |
E.6.13.1 | Other scope of the trial description |
The purpose of this pilot trial is to demonstrate that the hybrid DCT approach is safe and suitable for ABC patients treated with alpelisib, which has a balanced safety profile. |
|
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | 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.8.4.1 | Number of sites anticipated in Member State concerned | 2 |
E.8.5 | The trial involves multiple Member States | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.7 | Trial 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
|
End of trial will be the LVLS. Participants will be followed remotely up to 12 cycles of treatment or until treatment discontinuation, death, withdrawal of consent, or loss to follow-up, whichever occurs first. Discontinuation of remote participation is not a reason for trial discontinuation. Participants who do not wish to continue with remote participation will have the option to attend on-site visits. For each participant, the trial will conclude with an on-site end of trial (EOT) visit. |
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | |
E.8.9.1 | In the Member State concerned months | 24 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial months | 24 |