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

NCT ID: NCT04862143

Last Updated: 2024-06-20

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE2
• Total Enrollment: 2 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2022-03-08
• Study Completion Date: 2022-09-19

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.

Conditions

Advanced Breast Cancer

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

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.

Group Type: EXPERIMENTAL

Alpelisib

Intervention Type: DRUG

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.

Fulvestrant

Intervention Type: DRUG

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.

Goserelin

Intervention Type: DRUG

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.

Interventions

Alpelisib

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.

Intervention Type: DRUG

Fulvestrant

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.

Intervention Type: DRUG

Goserelin

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.

Intervention Type: DRUG

Other Intervention Names

BYL719

Eligibility Criteria

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.

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.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 99 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Novartis Pharmaceuticals

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Novartis Pharmaceuticals

Role: STUDY_DIRECTOR

Novartis Pharmaceuticals

Locations

Novartis Investigative Site

Örebro, , Sweden

Countries

Sweden

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Related Links

https://www.novctrd.com/ctrdweb/patientsummary/patientsummaries?patientSummaryId=1606

Patient Lay Trial Summary

Other Identifiers

2020-005882-15

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

CBYL719A03201

Identifier Type: -

Identifier Source: org_study_id