Advanced Breast Cancer
Conditions
Keywords
Decentralized clinical trial, advanced breast cancer, PIK3CA mutation, alpelisib
Brief summary
The study was 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
The purpose of this open-label, single arm, multi-center, Phase II interventional pilot trial was to evaluate if a decentralized clinical trial (DCT) using a telemedicine platform offers a satisfactory, safe and suitable management for HR-positive/HER2-negative participants with advanced breast cancer harboring a PIK3CA mutation and treated with alpelisib plus fulvestrant. The trial utilized a hybrid DCT approach to reduce participant burden by bringing visits, services, and supplies closer to them. The planned duration of treatment was 12 cycles of 28 days. Participants could discontinue treatment earlier due to unacceptable toxicity, disease progression and/or decision made at the discretion of the investigator or the participant. On-site visits occurred during screening, at Cycle 1 Day 1 (baseline), and at end-of-trial. Visits at the local oncologist practice were planned on Day 1 of Cycle 2, Cycle 4, Cycle 7, and Cycle 10. Other visits were performed by a district nurse, either at home or at the local oncologist's practice, depending on the participant's preference. During the on-site visit on Cycle 1, Day 1, participants were trained on using the telemedicine platform, and other monitoring devices used during remote participation: a glucometer and a smartphone with the telemedicine application installed. Study treatment was also initiated during this visit. The participants were then transitioned to remote participation enabled by the telemedicine platform with support of local healthcare providers (local oncologist, district nurse, or other qualified healthcare professional) under the investigator's oversight. Discontinuation of remote participation was not a reason for trial termination. Participants who did not wish to continue with remote participation had the option to attend on-site visits. The study planned to enroll approximately 20 participants, however the study was terminated prematurely with only 2 participants enrolled. The decision to terminate the study was due to delays during the start-up period and due to low enrollment. The decision to terminate was not related to any potential safety concern with alpelisib.
Interventions
DRUGAlpelisib
Participants received a daily oral dose of 300 mg of alpelisib film-coated tablets for a total of 12 cycles, with each cycle lasting 28 days.
DRUGFulvestrant
Participants were administered fulvestrant at a dose of 500 mg via intramuscular injection on Cycle 1 Day 1 and Cycle 1 Day 15, and on Day 1 of each 28-day cycle thereafter until Cycle 12.
DRUGGoserelin
Pre-menopausal women were administered a dose of 3.6 mg of goserelin injection via intramuscular route, beginning on Cycle 1 Day 1. Subsequently, the same dose was administered on Day 1 of each 28-day cycle throughout the study.
Sponsors
Novartis Pharmaceuticals
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 99 Years
Healthy volunteers
No
Inclusion criteria
Key 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 was willing to operate a smartphone compatible with the software of the medical device and willing to manage applications 7. Participant was willing to use the telemedicine platform and to follow the remote participant monitoring procedure. Key
Exclusion criteria
1. Participant had 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 was longer.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Participant Satisfaction Assessed Through the Trial Feedback Questionnaire (TFQ) | Baseline, and on Day 1 of Cycle 4 and 7. Cycle= 28 days | The TFQ was designed to capture the patient's experience during a clinical trial. The questionnaire consisted of 23 questions that assessed various aspects of the trial experience. Each question in the TFQ scored on a scale ranging from 1 (representing the worst response) to 5 (representing the best response). To calculate the total score, the scores obtained from each of the 23 questions were summed up. The resulting sum represented the participant's total score, which could ranged from 23 (indicating the lowest possible score) to 115 (indicating the highest possible score). |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of Unscheduled In-clinic Visits | From the date of the first study treatment up to the end of study, assessed up to 6 months | Unscheduled in-clinic visits were defined as visits that were originally intended to be conducted remotely but were ultimately carried out on-site, or visits that were not originally scheduled but took place on-site or at the local oncologist's (regional hospital). The total number of unscheduled in-clinic visits was evaluated |
| Number of Unscheduled In-clinic Visits Because of Safety Reasons | From the date of the first study treatment up to the end of study, assessed up to 6 months | Unscheduled in-clinic visits were defined as visits that were originally intended to be conducted remotely but were ultimately carried out on-site, or visits that were not originally scheduled but took place on-site or at the local oncologist's (regional hospital). The total number of unscheduled in-clinic visits that were prompted by safety reasons was evaluated |
| Number of Unscheduled In-clinic Visits Per Participant in the Study | From the date of the first study treatment up to the end of study, assessed up to 6 months | Unscheduled in-clinic visits were defined as visits that were originally intended to be conducted remotely but were ultimately carried out on-site, or visits that were not originally scheduled but took place on-site or at the local oncologist's (regional hospital). The total number of unscheduled in-clinic visits per participants was evaluated |
| Number of Participants Who Discontinue Treatment Due to Adverse Events (AEs) | From the date of the first study treatment up to the end of treatment, assessed up to 6 months | An AE refers to any untoward medical occurrence, such as an unfavorable and unintended sign (including abnormal laboratory findings), symptom, or disease. The number of participants who discontinued the treatment due to adverse events was evaluated |
| Number of Participants With Dose Reductions/Interruptions for Alpelisib | From the date of the first study treatment up to the end of treatment, assessed up to 6 months | Number of participants with dose reductions and interruptions for alpelisib |
| Patient Retention on the Decentralized Clinical Trial (DCT) Approach | At 3 and 6 months | Patient retention on the DCT approach was calculated as the percentage of participants on remote monitoring for participants still on treatment |
| Number of Participants With Adverse Events (AEs) Leading to In-clinic Visits | From the date of the first study treatment up to the end of study, assessed up to 6 months | An AE refers to any untoward medical occurrence, such as an unfavorable and unintended sign (including abnormal laboratory findings), symptom, or disease. The number of participants with AEs per the Common Terminology Criteria for Adverse Events (CTCAE) v4.03 leading to in-clinic visits was assessed. |
| European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) | Baseline, and on Day 1 of Cycle 4, and 7 and end of treatment, assessed up to 6 months. Cycle= 28 days | The EORTC QLQ-C30 questionnaire contained 30 items and was composed of both multi-item scales and single item measures. These included 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/quality of life (QoL) scale. All of the scales and single items ranged from 0 to 100. A high scale score represented a higher response level. Thus, a high score for a functional scale indicated a high/healthy level of functioning, a high score for the QoL indicated high QoL, but a high score for a symptom scale/single item indicated a high level of symptomatology/problems. The EORTC QLQ-C30 scores for all functional and symptom scales were evaluated |
| EuroQol 5-Dimension 5-Level (EQ-5D-5L)- Visual Analog Scale (VAS) Score | Baseline, and on Day 1 of Cycle 4, and 7 and end of treatment, assessed up to 6 months. Cycle= 28 days | 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. Along with the five dimensions of health, the EQ-5D-5L includes a VAS where respondents rate their overall health status on a scale from 0 to 100, where 0 represents the worst possible health state and 100 represents the best possible health state. The EQ-5D-5L VAS scores were evaluated |
| Brief Pain Inventory Short Form (BPI-SF) Scores | Baseline, and on Day 1 of Cycle 4, and 7 and end of treatment, assessed up to 6 months. Cycle= 28 days | The BPI-SF was a questionnaire used to assess pain intensity and interference with daily activities. The Pain Severity Subscale included four questions asking individuals to rate their pain intensity on a scale from 0 to 10, with higher scores indicating more severe pain. The Pain Interference Subscale consisted of seven items that assessed how pain had interfered with activities, rated on the same scale. Both subscales had a total score range of 0 to 10, with higher scores indicating more significant pain or interference. |
| Number of Participants With Progression-free Survival (PFS) According to RECIST 1.1 | Up to 6 months | The number of participants with PFS was defined as the count of participants who did not experience disease progression or death due to any cause during the study. Progression was assessed by the Response Evaluation Criteria in Solid Tumors (RECIST) v1.1 based on local radiology review. |
| Number of Participants With Adverse Events of Special Interest (AESIs)- Hyperglycemia, Rash and Diarrhea | From the date of the first study treatment up to the end of study, assessed up to 6 months | AESIs (Adverse Events of Special Interest) are defined as events, whether serious or non-serious, that are of scientific and medical concern specific to the sponsor's product or program. These events may require ongoing monitoring and communication by the investigator to the sponsor. For this study, the following AESIs were defined: hyperglycemia, rash, and diarrhea. The number of participants experiencing these events per the Common Terminology Criteria for Adverse Events (CTCAE) v4.03 was evaluated. |
Countries
Sweden
Participant flow
Recruitment details
The study was conducted in 1 center from Sweden
Participants by arm
| Arm | Count |
|---|---|
| Alpelisib + Fulvestrant Participants were administered alpelisib at a daily dose of 300 mg for 12 cycles of 28 days and fulvestrant at a dose of 500 mg via intramuscular injection on Cycle 1 Day 1 and Cycle 1 Day 15, and Day 1 of each subsequent cycle up to Cycle 12. Pre-menopausal women also received goserelin at a dose of 3.6 mg on Day 1 of each cycle. | 2 |
| Total | 2 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Adverse Event | 1 |
| Overall Study | Study terminated by sponsor | 1 |
Baseline characteristics
| Characteristic | Alpelisib + Fulvestrant | — |
|---|---|---|
| Age, Continuous | 54 Years STANDARD_DEVIATION 0 | — |
| Race and Ethnicity Not Collected | — | — Participants |
| Sex/Gender, Customized Unknown | 2 Participants | — |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 2 |
| other Total, other adverse events | 2 / 2 |
| serious Total, serious adverse events | 0 / 2 |
Outcome results
Primary
Participant Satisfaction Assessed Through the Trial Feedback Questionnaire (TFQ)
The TFQ was designed to capture the patient's experience during a clinical trial. The questionnaire consisted of 23 questions that assessed various aspects of the trial experience. Each question in the TFQ scored on a scale ranging from 1 (representing the worst response) to 5 (representing the best response). To calculate the total score, the scores obtained from each of the 23 questions were summed up. The resulting sum represented the participant's total score, which could ranged from 23 (indicating the lowest possible score) to 115 (indicating the highest possible score).
Time frame: Baseline, and on Day 1 of Cycle 4 and 7. Cycle= 28 days
Population: All enrolled participants who received at least one dose of any component of study treatment
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| Alpelisib + Fulvestrant | Participant Satisfaction Assessed Through the Trial Feedback Questionnaire (TFQ) | Baseline | 99.5 Score on a Scale |
| Alpelisib + Fulvestrant | Participant Satisfaction Assessed Through the Trial Feedback Questionnaire (TFQ) | Cycle 4 Day 1 | 89 Score on a Scale |
| Alpelisib + Fulvestrant | Participant Satisfaction Assessed Through the Trial Feedback Questionnaire (TFQ) | Cycle 7 Day 1 | 98 Score on a Scale |
Secondary
Brief Pain Inventory Short Form (BPI-SF) Scores
The BPI-SF was a questionnaire used to assess pain intensity and interference with daily activities. The Pain Severity Subscale included four questions asking individuals to rate their pain intensity on a scale from 0 to 10, with higher scores indicating more severe pain. The Pain Interference Subscale consisted of seven items that assessed how pain had interfered with activities, rated on the same scale. Both subscales had a total score range of 0 to 10, with higher scores indicating more significant pain or interference.
Time frame: Baseline, and on Day 1 of Cycle 4, and 7 and end of treatment, assessed up to 6 months. Cycle= 28 days
Population: All enrolled participants who received at least one dose of any study treatment. Number analyzed indicates the number of participants with data available at the designated time point.
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| Alpelisib + Fulvestrant | Brief Pain Inventory Short Form (BPI-SF) Scores | Pain severity- Baseline | 1.63 Score on a scale |
| Alpelisib + Fulvestrant | Brief Pain Inventory Short Form (BPI-SF) Scores | Pain severity- Cycle 4 Day 1 | 0 Score on a scale |
| Alpelisib + Fulvestrant | Brief Pain Inventory Short Form (BPI-SF) Scores | Pain severity- Cycle 7 Day 1 | 0.75 Score on a scale |
| Alpelisib + Fulvestrant | Brief Pain Inventory Short Form (BPI-SF) Scores | Pain severity- End of treatment | 1.13 Score on a scale |
| Alpelisib + Fulvestrant | Brief Pain Inventory Short Form (BPI-SF) Scores | Pain interference- Baseline | 0.79 Score on a scale |
| Alpelisib + Fulvestrant | Brief Pain Inventory Short Form (BPI-SF) Scores | Pain interference- Cycle 4 Day 1 | 0 Score on a scale |
| Alpelisib + Fulvestrant | Brief Pain Inventory Short Form (BPI-SF) Scores | Pain interference- Cycle 7 Day 1 | 0 Score on a scale |
| Alpelisib + Fulvestrant | Brief Pain Inventory Short Form (BPI-SF) Scores | Pain interference- End of treatment | 1.07 Score on a scale |
Secondary
European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30)
The EORTC QLQ-C30 questionnaire contained 30 items and was composed of both multi-item scales and single item measures. These included 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/quality of life (QoL) scale. All of the scales and single items ranged from 0 to 100. A high scale score represented a higher response level. Thus, a high score for a functional scale indicated a high/healthy level of functioning, a high score for the QoL indicated high QoL, but a high score for a symptom scale/single item indicated a high level of symptomatology/problems. The EORTC QLQ-C30 scores for all functional and symptom scales were evaluated
Time frame: Baseline, and on Day 1 of Cycle 4, and 7 and end of treatment, assessed up to 6 months. Cycle= 28 days
Population: All enrolled participants who received at least one dose of any study treatment. Number analyzed indicates the number of participants with data available at the designated time point.
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| Alpelisib + Fulvestrant | European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) | QoL- Baseline | 75 Score on a Scale |
| Alpelisib + Fulvestrant | European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) | QoL- Cycle 4 Day 1 | 83 Score on a Scale |
| Alpelisib + Fulvestrant | European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) | QoL- Cycle 7 Day 1 | 92 Score on a Scale |
| Alpelisib + Fulvestrant | European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) | QoL- End of Treatment | 75 Score on a Scale |
| Alpelisib + Fulvestrant | European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) | Physical Functioning- Baseline | 90 Score on a Scale |
| Alpelisib + Fulvestrant | European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) | Physical Functioning- Cycle 4 Day 1 | 93 Score on a Scale |
| Alpelisib + Fulvestrant | European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) | Physical Functioning- Cycle 7 Day 1 | 100 Score on a Scale |
| Alpelisib + Fulvestrant | European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) | Physical Functioning-End of Treatment | 86.5 Score on a Scale |
| Alpelisib + Fulvestrant | European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) | Role Functioning- Baseline | 91.5 Score on a Scale |
| Alpelisib + Fulvestrant | European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) | Role Functioning- Cycle 4 Day 1 | 100 Score on a Scale |
| Alpelisib + Fulvestrant | European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) | Role Functioning- Cycle 7 Day 1 | 100 Score on a Scale |
| Alpelisib + Fulvestrant | European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) | Role Functioning-End of Treatment | 83.5 Score on a Scale |
| Alpelisib + Fulvestrant | European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) | Emotional Functioning- Baseline | 71 Score on a Scale |
| Alpelisib + Fulvestrant | European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) | Emotional Functioning- Cycle 4 Day 1 | 83 Score on a Scale |
| Alpelisib + Fulvestrant | European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) | Emotional Functioning- Cycle 7 Day 1 | 92 Score on a Scale |
| Alpelisib + Fulvestrant | European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) | Emotional Functioning- End of treatment | 83 Score on a Scale |
| Alpelisib + Fulvestrant | European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) | Cognitive Functioning- Baseline | 91.5 Score on a Scale |
| Alpelisib + Fulvestrant | European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) | Cognitive Functioning- Cycle 4 Day 1 | 100 Score on a Scale |
| Alpelisib + Fulvestrant | European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) | Cognitive Functioning- Cycle 7 Day 1 | 100 Score on a Scale |
| Alpelisib + Fulvestrant | European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) | Cognitive Functioning- End of treatment | 75 Score on a Scale |
| Alpelisib + Fulvestrant | European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) | Social Functioning- Baseline | 91.5 Score on a Scale |
| Alpelisib + Fulvestrant | European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) | Social Functioning- Cycle 4 Day 1 | 100 Score on a Scale |
| Alpelisib + Fulvestrant | European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) | Social Functioning- Cycle 7 Day 1 | 100 Score on a Scale |
| Alpelisib + Fulvestrant | European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) | Social Functioning- End of treatment | 91.5 Score on a Scale |
| Alpelisib + Fulvestrant | European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) | Fatigue- Baseline | 22 Score on a Scale |
| Alpelisib + Fulvestrant | European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) | Fatigue- Cycle 4 Day 1 | 22 Score on a Scale |
| Alpelisib + Fulvestrant | European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) | Fatigue- Cycle 7 Day 1 | 0 Score on a Scale |
| Alpelisib + Fulvestrant | European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) | Fatigue- End of treatment | 33 Score on a Scale |
| Alpelisib + Fulvestrant | European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) | Nausea/vomiting- Baseline | 0 Score on a Scale |
| Alpelisib + Fulvestrant | European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) | Nausea/vomiting- Cycle 4 Day 1 | 17 Score on a Scale |
| Alpelisib + Fulvestrant | European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) | Nausea/vomiting- Cycle 7 Day 1 | 0 Score on a Scale |
| Alpelisib + Fulvestrant | European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) | Nausea/vomiting- End of treatment | 0 Score on a Scale |
| Alpelisib + Fulvestrant | European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) | Pain- Baseline | 8.5 Score on a Scale |
| Alpelisib + Fulvestrant | European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) | Pain- Cycle 4 Day 1 | 0 Score on a Scale |
| Alpelisib + Fulvestrant | European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) | Pain- Cycle 7 Day 1 | 0 Score on a Scale |
| Alpelisib + Fulvestrant | European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) | Pain- End of treatment | 16.5 Score on a Scale |
Secondary
EuroQol 5-Dimension 5-Level (EQ-5D-5L)- Visual Analog Scale (VAS) 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. Along with the five dimensions of health, the EQ-5D-5L includes a VAS where respondents rate their overall health status on a scale from 0 to 100, where 0 represents the worst possible health state and 100 represents the best possible health state. The EQ-5D-5L VAS scores were evaluated
Time frame: Baseline, and on Day 1 of Cycle 4, and 7 and end of treatment, assessed up to 6 months. Cycle= 28 days
Population: All enrolled participants who received at least one dose of any study treatment. Number analyzed indicates the number of participants with data available at the designated time point.
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| Alpelisib + Fulvestrant | EuroQol 5-Dimension 5-Level (EQ-5D-5L)- Visual Analog Scale (VAS) Score | Baseline | 80 Score on a scale |
| Alpelisib + Fulvestrant | EuroQol 5-Dimension 5-Level (EQ-5D-5L)- Visual Analog Scale (VAS) Score | Cycle 4 Day 1 | 93 Score on a scale |
| Alpelisib + Fulvestrant | EuroQol 5-Dimension 5-Level (EQ-5D-5L)- Visual Analog Scale (VAS) Score | Cycle 7 Day 1 | 95 Score on a scale |
| Alpelisib + Fulvestrant | EuroQol 5-Dimension 5-Level (EQ-5D-5L)- Visual Analog Scale (VAS) Score | End of treatment | 82.5 Score on a scale |
Secondary
Number of Participants Who Discontinue Treatment Due to Adverse Events (AEs)
An AE refers to any untoward medical occurrence, such as an unfavorable and unintended sign (including abnormal laboratory findings), symptom, or disease. The number of participants who discontinued the treatment due to adverse events was evaluated
Time frame: From the date of the first study treatment up to the end of treatment, assessed up to 6 months
Population: All enrolled participants who received at least one dose of any component of study treatment
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Alpelisib + Fulvestrant | Number of Participants Who Discontinue Treatment Due to Adverse Events (AEs) | 1 Participants |
Secondary
Number of Participants With Adverse Events (AEs) Leading to In-clinic Visits
An AE refers to any untoward medical occurrence, such as an unfavorable and unintended sign (including abnormal laboratory findings), symptom, or disease. The number of participants with AEs per the Common Terminology Criteria for Adverse Events (CTCAE) v4.03 leading to in-clinic visits was assessed.
Time frame: From the date of the first study treatment up to the end of study, assessed up to 6 months
Population: All enrolled participants who received at least one dose of any study treatment
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Alpelisib + Fulvestrant | Number of Participants With Adverse Events (AEs) Leading to In-clinic Visits | 0 Participants |
Secondary
Number of Participants With Adverse Events of Special Interest (AESIs)- Hyperglycemia, Rash and Diarrhea
AESIs (Adverse Events of Special Interest) are defined as events, whether serious or non-serious, that are of scientific and medical concern specific to the sponsor's product or program. These events may require ongoing monitoring and communication by the investigator to the sponsor. For this study, the following AESIs were defined: hyperglycemia, rash, and diarrhea. The number of participants experiencing these events per the Common Terminology Criteria for Adverse Events (CTCAE) v4.03 was evaluated.
Time frame: From the date of the first study treatment up to the end of study, assessed up to 6 months
Population: All enrolled participants who received at least one dose of any study treatment
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Alpelisib + Fulvestrant | Number of Participants With Adverse Events of Special Interest (AESIs)- Hyperglycemia, Rash and Diarrhea | Diarrhea | 1 Participants |
| Alpelisib + Fulvestrant | Number of Participants With Adverse Events of Special Interest (AESIs)- Hyperglycemia, Rash and Diarrhea | Hyperglycaemia | 2 Participants |
| Alpelisib + Fulvestrant | Number of Participants With Adverse Events of Special Interest (AESIs)- Hyperglycemia, Rash and Diarrhea | Rash | 0 Participants |
Secondary
Number of Participants With Dose Reductions/Interruptions for Alpelisib
Number of participants with dose reductions and interruptions for alpelisib
Time frame: From the date of the first study treatment up to the end of treatment, assessed up to 6 months
Population: All enrolled participants who received at least one dose of any study treatment
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Alpelisib + Fulvestrant | Number of Participants With Dose Reductions/Interruptions for Alpelisib | Dose interruptions | 1 Participants |
| Alpelisib + Fulvestrant | Number of Participants With Dose Reductions/Interruptions for Alpelisib | Dose reductions | 1 Participants |
Secondary
Number of Participants With Progression-free Survival (PFS) According to RECIST 1.1
The number of participants with PFS was defined as the count of participants who did not experience disease progression or death due to any cause during the study. Progression was assessed by the Response Evaluation Criteria in Solid Tumors (RECIST) v1.1 based on local radiology review.
Time frame: Up to 6 months
Population: All enrolled participants who received at least one dose of any component of study treatment
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Alpelisib + Fulvestrant | Number of Participants With Progression-free Survival (PFS) According to RECIST 1.1 | 2 Participants |
Secondary
Number of Unscheduled In-clinic Visits
Unscheduled in-clinic visits were defined as visits that were originally intended to be conducted remotely but were ultimately carried out on-site, or visits that were not originally scheduled but took place on-site or at the local oncologist's (regional hospital). The total number of unscheduled in-clinic visits was evaluated
Time frame: From the date of the first study treatment up to the end of study, assessed up to 6 months
Population: All enrolled participants who received at least one dose of any component of study treatment
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Alpelisib + Fulvestrant | Number of Unscheduled In-clinic Visits | 0 Visits |
Secondary
Number of Unscheduled In-clinic Visits Because of Safety Reasons
Unscheduled in-clinic visits were defined as visits that were originally intended to be conducted remotely but were ultimately carried out on-site, or visits that were not originally scheduled but took place on-site or at the local oncologist's (regional hospital). The total number of unscheduled in-clinic visits that were prompted by safety reasons was evaluated
Time frame: From the date of the first study treatment up to the end of study, assessed up to 6 months
Population: All enrolled participants who received at least one dose of any component of study treatment
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Alpelisib + Fulvestrant | Number of Unscheduled In-clinic Visits Because of Safety Reasons | 0 Visits |
Secondary
Number of Unscheduled In-clinic Visits Per Participant in the Study
Unscheduled in-clinic visits were defined as visits that were originally intended to be conducted remotely but were ultimately carried out on-site, or visits that were not originally scheduled but took place on-site or at the local oncologist's (regional hospital). The total number of unscheduled in-clinic visits per participants was evaluated
Time frame: From the date of the first study treatment up to the end of study, assessed up to 6 months
Population: All enrolled participants who received at least one dose of any component of study treatment
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Alpelisib + Fulvestrant | Number of Unscheduled In-clinic Visits Per Participant in the Study | 0 Visits per participant |
Secondary
Patient Retention on the Decentralized Clinical Trial (DCT) Approach
Patient retention on the DCT approach was calculated as the percentage of participants on remote monitoring for participants still on treatment
Time frame: At 3 and 6 months
Population: Participants receiving treatment at the specified time points
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Alpelisib + Fulvestrant | Patient Retention on the Decentralized Clinical Trial (DCT) Approach | 3 months | 1 Participants |
| Alpelisib + Fulvestrant | Patient Retention on the Decentralized Clinical Trial (DCT) Approach | 6 months | 1 Participants |