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

NCT04862143 | Terminated Phase 2 Results

• 2 other identifiers: CBYL719A032012020-005882-15
• Study Type: interventional
• Target: 2
• Locations: 1 country
• Sites: 1

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.

Conditions

Advanced Breast Cancer

Keywords

Decentralized clinical trial; advanced breast cancer; PIK3CA mutation; alpelisib

Outcome Measures

Primary Outcomes (1)

• 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).

  Baseline, and on Day 1 of Cycle 4 and 7. Cycle= 28 days

Secondary Outcomes (12)

• Patient Retention on the Decentralized Clinical Trial (DCT) Approach

  At 3 and 6 months

• 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

• 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

• 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

• 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

Study Arms (1)

Alpelisib + fulvestrant

EXPERIMENTAL

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.

Drug: Alpelisib; Drug: Fulvestrant; Drug: Goserelin

Interventions

Alpelisib 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.

Also known as: BYL719

Alpelisib + fulvestrant

Fulvestrant 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.

Alpelisib + fulvestrant

Goserelin 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.

Alpelisib + fulvestrant

Eligibility Criteria

• Age: 18 Years - 99 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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).
• Participant was willing to operate a smartphone compatible with the software of the medical device and willing to manage applications
• Participant was willing to use the telemedicine platform and to follow the remote participant monitoring procedure.

You may not qualify if:

• 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.
• 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.

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (1)

Novartis Investigative Site

Örebro, 701 85, Sweden

Location

Related Links

• Patient Lay Trial Summary

MeSH Terms

Interventions

Alpelisib; Fulvestrant; Goserelin

Intervention Hierarchy (Ancestors)

Estradiol; Estrenes; Estranes; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Estradiol Congeners; Gonadal Steroid Hormones; Gonadal Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Gonadotropin-Releasing Hormone; Pituitary Hormone-Releasing Hormones; Hypothalamic Hormones; Peptide Hormones; Neuropeptides; Peptides; Amino Acids, Peptides, and Proteins; Oligopeptides; Nerve Tissue Proteins; Proteins

Results Point of Contact

• Title: Study director
• Organization: Novartis Pharmaceuticals

Novartis.email@novartis.com

862-778-8300

Study Officials

• Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 21, 2021
• First Posted: April 27, 2021
• Study Start: March 8, 2022
• Primary Completion: September 19, 2022
• Study Completion: September 19, 2022
• Last Updated: June 20, 2024
• Results First Posted: February 20, 2024

Record last verified: 2024-06

Data Sharing

• IPD Sharing: Will share

Locations

SE (1)