Roll-Over Study of Alpelisib (BYL719) for Continued Access and Long-Term Safety. (EPIK-RO)

June 25, 2026 updated by: Novartis Pharmaceuticals

An Open Label, Multi-center Alpelisib Roll-over Study to Assess Long-term Safety in Participants Who Have Completed a Novartis Sponsored Alpelisib (BYL719) Study and Are Judged by the Investigator to Benefit From Continued Treatment With Alpelisib.

The purpose of this study is to provide post-trial access to alpelisib and to assess its long-term safety when administered as a single agent or in combination with other drugs. This study is intended for participants who are currently receiving alpelisib in a Novartis-sponsored clinical trial (parent study) and, in the Investigator's judgment, would benefit from continued treatment with alpelisib.

Study Overview

Status

Not yet recruiting

Conditions

Intervention / Treatment

Detailed Description

Eligible participants are those who are receiving alpelisib, either as a single agent or in combination with other drugs, at the end of one of the following parent studies: CBYL719F12401 (NCT04980833), CBYL719G12301 (NCT04208178), CBYL719C2303 (NCT05038735), CBYL719C1201 (NCT04524000), CBYL719C2201 (NCT04544189), or CLEE011X2107 (NCT01872260).

Participants transition directly from the parent study into this roll-over study without a separate screening period. At enrollment, participants continue treatment with the same combination and the same dose that were administered at the end of the parent study. Study visits, treatment administration, and safety assessments generally follow the schedules used in the parent study.

Participants continue receiving alpelisib until clinical benefit is no longer observed, study treatment is discontinued for safety or other protocol-defined reasons, or another discontinuation criterion is met. Clinical benefit is evaluated by the investigator at scheduled visits.

Safety is monitored throughout the study through the collection of adverse events and serious adverse events. Safety assessments are conducted at regular intervals during treatment in accordance with the study protocol and local clinical practice.

At discontinuation of study treatment, participants complete an end-of-treatment visit. All participants are followed for safety for 30 days after the last dose of alpelisib.

Study Type

Interventional

Enrollment (Estimated)

51

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Key Inclusion Criteria:

  • Written informed consent/assent, according to local guidelines, signed by the participants and/or by the parents or legal guardian prior to enrolling in the roll-over study.
  • Participant currently enrolled in a Novartis-sponsored study, is currently receiving alpelisib as a single agent or in combination with other drugs, and has fulfilled all on-treatment requirements in the parent study.
  • Participant is currently benefiting from the treatment with alpelisib as determined by the Investigator in the parent study.
  • Participant demonstrated compliance with the visit schedule in the parent study, and in the opinion of the Investigator has shown willingness and ability to comply with future visit schedules, treatment plans, and any other study procedures in this protocol.

Key Exclusion Criteria:

  • Participant had permanently discontinued from alpelisib in the parent study for any reason including withdrawal of consent.
  • Participant currently has ongoing/unresolved treatment related Grade 3 or higher AEs, and/or any ongoing/unresolved AE or toxicities for which alpelisib dosing has been interrupted in the parent study. Participants meeting all other eligibility criteria may be enrolled once toxicities have improved to allow alpelisib dosing to resume as stated in the parent protocol.
  • Participant's ongoing treatment is currently approved and reimbursed for their indication at their country level. In exceptional cases where the treatment is reimbursed at the country level, but not individual level, please contact the Novartis Study Team.
  • Concurrent participation in any other investigational clinical trial other than the parent study.
  • Pregnant or nursing (breastfeeding) women.
  • Female participants of childbearing potential who do not consent to use a highly effective method of contraception, and male participants who do not consent to use a condom and/or a highly effective method of contraception, including refraining from sperm donation and complying with measures to prevent exposure of a partner to alpelisib via seminal fluid, for the duration of the study and for one week following discontinuation of alpelisib (or longer if required per parent protocol).

Other inclusion/exclusion criteria may apply.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Other: Alpelisib (BYL719) or in Combination Therapy
Eligible participants will continue treatment with the same combination and dose as in the parent study until end of treatment (EOT), followed by a 30-day safety follow-up.
Drug: Alpelisib
Administered as oral tablets at dose levels as per the parent study, taken once daily.
Other Names:
  • BYL719
Drug: Fulvestrant
Administered as an intramuscular injection at dose levels and dosing schedule as per standard of care, in accordance with the parent study.
Drug: Trastuzumab
Administered as an intravenous infusion of a reconstituted lyophilized powder at dose levels as per the parent study, given every 21 days.
Drug: Pertuzumab
Administered as an intravenous infusion of a solution concentrate at dose levels as per the parent study, given every 21 days.
Drug: Letrozole
Administered as oral tablets at a dose of 2.5 mg, taken once daily, as per the parent study.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time Frame: From start of treatment up to 30 days after last dose of study treatment, assessed up to approximately 53 months
Incidence of adverse events by type, frequency, and severity, as graded by the NCI CTCAE version 4.03.
From start of treatment up to 30 days after last dose of study treatment, assessed up to approximately 53 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Percentage of participants with Clinical Benefit Rate (CBR)
Time Frame: Up to approximately 54 months
Clinical benefit is defined as the investigator's assessment that the participant continues to derive benefit from alpelisib treatment, based on overall clinical evaluation at scheduled visits, and that continued treatment is appropriate.
Up to approximately 54 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Novartis Pharmaceuticals

Investigators

  • Study Director: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

January 1, 2027

Primary Completion (Estimated)

June 27, 2031

Study Completion (Estimated)

July 25, 2031

Study Registration Dates

First Submitted

June 25, 2026

First Submitted That Met QC Criteria

June 25, 2026

First Posted (Actual)

July 1, 2026

Study Record Updates

Last Update Posted (Actual)

July 1, 2026

Last Update Submitted That Met QC Criteria

June 25, 2026

Last Verified

June 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CBYL719F12003B
  • 2026-525890-38-00 (Registry Identifier: EU CT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD 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.

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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