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 (TELEPIK)

• ClinicalTrials.gov Identifier: NCT04862143
• Recruitment Status: Recruiting
• First Posted: April 27, 2021
• Last Update Posted: February 9, 2022
• Sponsor: Novartis Pharmaceuticals
• Information provided by (Responsible Party): Novartis ( Novartis Pharmaceuticals )

Study Description

Brief Summary:

The study is 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.

Condition or disease	Intervention/treatment	Phase
Advanced Breast Cancer	Drug: Alpelisib; Drug: fulvestrant	Phase 2

Detailed Description:

Open-label, single arm, multi-center, Phase II interventional pilot trial. Primary objective will be to assess participant satisfaction with the decentralized clinical trial (DCT) experience.

The trial will enroll men and pre- and post-menopausal women with a HR-positive/HER2-negative ABC (loco regionally recurrent not amenable to curative therapy or metastatic) with a PIK3CA mutation, which progressed on or after endocrine-based treatment.

Participants will receive alpelisib 300 mg daily and fulvestrant 500 mg administered intramuscularly on Cycle 1, Day 1 and Cycle 1, Day 15, and on Day 1 of each cycle thereafter until Cycle 12.

Pre-menopausal women will receive goserelin 3.6 mg on Day 1 of each cycle.

Study Design

• Study Type: Interventional (Clinical Trial)
• Estimated Enrollment: 20 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• 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
• Estimated Study Start Date: February 28, 2022
• Estimated Primary Completion Date: August 22, 2023
• Estimated Study Completion Date: August 22, 2023

Arms and Interventions

Arm	Intervention/treatment
Experimental: single arm; alpelisib 300 mg daily and fulvestrant 500 mg administered intramuscularly on Cycle 1, Day 1 and Cycle 1, Day 15, and on Day 1 of each cycle thereafter until Cycle 12.	Drug: Alpelisib; Alpelisib 300 mg daily up to 12 cycles of 28 days each; Drug: fulvestrant; fulvestrant 500 mg administered intramuscularly on Cycle 1, Day 1 and Cycle 1, Day 15, and on Day 1 of each cycle thereafter until Cycle 12.

Outcome Measures

Primary Outcome Measures :

1. Participant satisfaction with the decentralized clinical trial (DCT) experience [ Time Frame: up to 12 months ]
   change in the Trial Feedback Questionnaire (TFQ) assessed at the start of the trial, every 12 weeks, and at the end of trial through the Trial Feedback Questionnaire (TFQ)

Secondary Outcome Measures :

1. Patient retention on DCT [ Time Frame: 3, 6, 12 months ]
   Proportion of participants on remote monitoring at 3, 6, 12 months for participants still on treatment.
2. Compliance to treatment [ Time Frame: up to 12 months ]
   Overall compliance
3. Proportion of participants with AESIs (hyperglicemia, rash and diarrhea) [ Time Frame: up to 12 months ]
   Adverse events of special interest (AESIs) per Common Terminology Criteria for Adverse Events (CTCAE) v4.03
4. Proportion of participants with AEs leading to in-clinic visits [ Time Frame: up to 12 months ]
   Adverse events (AEs) that lead to in-clinic visits.
5. Change from baseline in the EORTC QLQ C30 score. [ Time Frame: baseline, 3,6,9,12 months ]
   The European Organization for Research and Treatment of Cancer's core quality of life questionnaire (EORTC QLQ-C30) contains 30 items and is composed of both multi-item scales and single item measures. These include 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/QoL scale.
6. Progression-free survival (PFS) according to RECIST 1.1 [ Time Frame: 3,6,9,12 months ]
   Effectiveness of alpelisib plus fulvestrant
7. Total number of unscheduled in-clinic visits because of safety reasons [ Time Frame: up to 12 months ]
   safe and suitable remote management of patients
8. Total number of unscheduled in-clinic visits and the reason [ Time Frame: up to 12 months ]
   safe and suitable remote management of patients
9. Number of unscheduled in-clinic visits per participant in the study [ Time Frame: up to 12 months ]
   safe and suitable remote management of patients
10. Discontinuation rate related to adverse events [ Time Frame: up to 12 months ]
    safe and suitable remote management of patients
11. Number and proportion of AEs leading to in-clinic visits [ Time Frame: up to 12 months ]
    AEs of special interest (AESIs) and AEs leading to in-clinic visits
12. Change from baseline in the EQ-5D-5L score [ Time Frame: baseline, 3,6,9,12 months ]
    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. Each dimension has 5 levels: no problems, slight problems, moderate problems, severe problems and extreme problems.
13. Change from baseline in the BPI-SF score [ Time Frame: baseline, 3,6,9,12 months ]
    The Brief Pain Inventory Short Form (BPI-SF) is a self-administered assessment tool validated to assess pain The basic aim of the BPI is to provide the information on the intensity of pain, along with the degree to which the pain interferes with the everyday functioning of life. The BPI uses simple numeric rating scales from 0 (no pain) to 10 (severe pain).

Eligibility Criteria

• Ages Eligible for Study: 18 Years to 99 Years (Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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 is a man or a pre- or post-menopausal woman.
7. Participant is willing to operate a smartphone compatible with the software of the medical device and willing to manage applications
8. Participant is willing to use the telemedicine platform and to follow the remote participant monitoring procedure.
9. Participant has signed an informed consent form before any trial

Exclusion Criteria:

1. Participant has 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 is longer.

Other protocol-defined inclusion/exclusion criteria may apply.

Contacts and Locations

Contacts

Contact: Novartis Pharmaceuticals; +41613241111; novartis.email@novartis.com

Contact: Novartis Pharmaceuticals

Locations

Sweden

Novartis Investigative Site; Recruiting

Orebro, Sweden, 701 85

Sponsors and Collaborators

Novartis Pharmaceuticals

Investigators

• Study Director: Novartis Pharmaceuticals; Novartis Pharmaceuticals

More Information

• Responsible Party: Novartis Pharmaceuticals
• ClinicalTrials.gov Identifier: NCT04862143
• Other Study ID Numbers: CBYL719A03201; 2020-005882-15 ( EudraCT Number )
• First Posted: April 27, 2021
• Last Update Posted: February 9, 2022
• Last Verified: February 2022

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: Yes
• Plan Description: Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com.

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Novartis ( Novartis Pharmaceuticals ):

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

Additional relevant MeSH terms:

Breast Neoplasms; Neoplasms by Site; Neoplasms; Breast Diseases; Skin Diseases; Fulvestrant; Antineoplastic Agents, Hormonal; Antineoplastic Agents; Estrogen Receptor Antagonists; Estrogen Antagonists; Hormone Antagonists; Hormones, Hormone Substitutes, and Hormone Antagonists; Physiological Effects of Drugs