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

NCT07679269 | Not Yet Recruiting Phase 2

• 2 other identifiers: CBYL719F12003B2026-525890-38-00
• Study Type: interventional
• Target: 51
• Locations: 0 countries
• Sites: N/A

Brief Summary

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.

Conditions

PIK3CA-Related Overgrowth Spectrum (PROS); Breast Neoplasms (Including Advanced or Metastatic Breast Cancer With PIK3CA Mutation)

Keywords

Alpelisib (BYL719); PI3K inhibitor; PIK3CA mutation; Post-trial access; Roll-over study; Long-term safety; Open-label; Continued treatment access; Solid tumors; Breast cancer; PROS

Outcome Measures

Primary Outcomes (1)

• Number of participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)

  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 Outcomes (1)

• Percentage of participants with Clinical Benefit Rate (CBR)

  Up to approximately 54 months

Study Arms (1)

Alpelisib (BYL719) or in Combination Therapy

OTHER

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; Drug: Fulvestrant; Drug: Trastuzumab; Drug: Pertuzumab; Drug: Letrozole

Interventions

Alpelisib DRUG

Administered as oral tablets at dose levels as per the parent study, taken once daily.

Also known as: BYL719

Alpelisib (BYL719) or in Combination Therapy

Fulvestrant DRUG

Administered as an intramuscular injection at dose levels and dosing schedule as per standard of care, in accordance with the parent study.

Alpelisib (BYL719) or in Combination Therapy

Trastuzumab DRUG

Administered as an intravenous infusion of a reconstituted lyophilized powder at dose levels as per the parent study, given every 21 days.

Alpelisib (BYL719) or in Combination Therapy

Pertuzumab DRUG

Administered as an intravenous infusion of a solution concentrate at dose levels as per the parent study, given every 21 days.

Alpelisib (BYL719) or in Combination Therapy

Letrozole DRUG

Administered as oral tablets at a dose of 2.5 mg, taken once daily, as per the parent study.

Alpelisib (BYL719) or in Combination Therapy

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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).

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

MeSH Terms

Conditions

Breast Neoplasms

Interventions

Alpelisib; Fulvestrant; Trastuzumab; pertuzumab; Letrozole

Condition Hierarchy (Ancestors)

Neoplasms by Site; Neoplasms; Breast Diseases; Skin Diseases; Skin and Connective Tissue Diseases

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; Antibodies, Monoclonal, Humanized; Antibodies, Monoclonal; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins; Nitriles; Organic Chemicals; Triazoles; Azoles; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds

Study Officials

• Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Central Study Contacts

Novartis Pharmaceuticals

CONTACT

1-888-669-6682; novartis.email@novartis.com

Novartis Pharmaceuticals

CONTACT

+41613241111; novartis.email@novartis.com

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: June 25, 2026
• First Posted: July 1, 2026
• Study Start (Estimated): January 1, 2027
• Primary Completion (Estimated): June 27, 2031
• Study Completion (Estimated): July 25, 2031
• Last Updated: July 1, 2026

Record last verified: 2026-06

Data Sharing

• IPD Sharing: Will share