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

What Was Tested

The trial (phase 2) is built around Goserelin.

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.

It had 1 arm (groups): Alpelisib + fulvestrant (experimental).

The registry classes the interventions as drug.

More on the Plan

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.

Who Took Part

2 actual participants took part.

It was open to people of any sex aged from 18 years up to 99 years.

Healthy volunteers were not accepted; participants needed the condition being studied.

Examples of who could join: 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).

The full record lists 7 inclusion and 3 exclusion criteria; among the exclusions: participant had 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.

How the Study Was Run

Design: single-group and open-label.

Being open-label, the Goserelin study did not mask who received what.

Everyone received the same intervention, so there was no separate comparison group inside the trial.

It started in 2022-03-08 with completion listed for 2022-09-19.

It ran at 1 site in Sweden.

Its stated primary purpose is treatment.

What Was Measured

Researchers chose this as the main measure: Participant Satisfaction Assessed Through the Trial Feedback Questionnaire (TFQ) (time frame: Baseline, and on Day 1 of Cycle 4 and 7. Cycle= 28 days).

It also tracked 12 secondary outcomes, for example Patient Retention on the Decentralized Clinical Trial (DCT) Approach, Number of Unscheduled In-clinic Visits, Number of Unscheduled In-clinic Visits Because of Safety Reasons, Number of Unscheduled In-clinic Visits Per Participant in the Study, Number of Participants Who Discontinue Treatment Due to Adverse Events (AEs) and Number of Participants With Adverse Events of Special Interest (AESIs)- Hyperglycemia, Rash and Diarrhea.

What This Means in Plain Language

Put simply, this is a phase 2 trial of Goserelin in 2 people.

Its size is small, so on its own it can only give an early look.

It ended early (terminated); results are posted on the registry, and this page does not restate them.

More context: the Goserelin compound page, all human trials.