Clinical trial NCT04862143: 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
Official title: 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
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Advanced Breast Cancer
Keywords: Decentralized clinical trial
advanced breast cancer
PIK3CA mutation
alpelisib
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2021-04-27
Enrollment: 2
Sex: ALL
Minimum age (years): 18
Maximum age (years): 99
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

Eligibility 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.

Primary outcomes:
measure: Participant Satisfaction Assessed Through the Trial Feedback Questionnaire (TFQ)
timeFrame: Baseline, and on Day 1 of Cycle 4 and 7. Cycle= 28 days
Secondary outcomes:
measure: Patient Retention on the Decentralized Clinical Trial (DCT) Approach
timeFrame: At 3 and 6 months
measure: Number of Unscheduled In-clinic Visits
timeFrame: From the date of the first study treatment up to the end of study, assessed up to 6 months
measure: Number of Unscheduled In-clinic Visits Because of Safety Reasons
timeFrame: From the date of the first study treatment up to the end of study, assessed up to 6 months
measure: Number of Unscheduled In-clinic Visits Per Participant in the Study
timeFrame: From the date of the first study treatment up to the end of study, assessed up to 6 months
measure: Number of Participants Who Discontinue Treatment Due to Adverse Events (AEs)
timeFrame: From the date of the first study treatment up to the end of treatment, assessed up to 6 months
measure: Number of Participants With Dose Reductions/Interruptions for Alpelisib
timeFrame: From the date of the first study treatment up to the end of treatment, assessed up to 6 months
measure: Number of Participants With Adverse Events of Special Interest (AESIs)- Hyperglycemia, Rash and Diarrhea
timeFrame: From the date of the first study treatment up to the end of study, assessed up to 6 months
measure: Number of Participants With Adverse Events (AEs) Leading to In-clinic Visits
timeFrame: From the date of the first study treatment up to the end of study, assessed up to 6 months
measure: European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30)
timeFrame: Baseline, and on Day 1 of Cycle 4, and 7 and end of treatment, assessed up to 6 months. Cycle= 28 days
measure: EuroQol 5-Dimension 5-Level (EQ-5D-5L)- Visual Analog Scale (VAS) Score
timeFrame: Baseline, and on Day 1 of Cycle 4, and 7 and end of treatment, assessed up to 6 months. Cycle= 28 days
measure: Brief Pain Inventory Short Form (BPI-SF) Scores
timeFrame: Baseline, and on Day 1 of Cycle 4, and 7 and end of treatment, assessed up to 6 months. Cycle= 28 days
measure: Number of Participants With Progression-free Survival (PFS) According to RECIST 1.1
timeFrame: Up to 6 months
Other outcomes:
Locations:
city: Orebro
country: Sweden
facility: Novartis Investigative Site
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: