A Phase 2 interventional study of Alpelisib and Fulvestrant in PIK3CA-Related Overgrowth Spectrum (PROS) and Breast Neoplasms (Including Advanced or Metastatic Breast Cancer With PIK3CA Mutation), sponsored by Novartis Pharmaceuticals. Not yet recruiting. Per ClinicalTrials.gov, last updated 2026-09-18.
Sponsored by Novartis Pharmaceuticals · Phase 2, Interventional, and Treatment
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.
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.
12,543 studies on the registry are indexed under Breast Neoplasms; 2,892 are open to participants now.
This study's planned enrollment of 51 is below the median of 72 across 9,302 interventional studies indexed under Breast Neoplasms.
Browse Breast Neoplasms studies →Novartis Pharmaceuticals is the lead sponsor of 2,673 studies on the registry; 228 are open to participants now.
Of its 576 completed or terminated interventional studies of FDA-regulated products, 431 (75%) have results posted.
Counted across the registry records on this site, refreshed daily.
Key Inclusion Criteria:
Key Exclusion Criteria:
Other inclusion/exclusion criteria may apply.
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
Administered as oral tablets at dose levels as per the parent study, taken once daily.
Also known as: BYL719
Administered as an intramuscular injection at dose levels and dosing schedule as per standard of care, in accordance with the parent study.
Administered as an intravenous infusion of a reconstituted lyophilized powder at dose levels as per the parent study, given every 21 days.
Administered as an intravenous infusion of a solution concentrate at dose levels as per the parent study, given every 21 days.
Administered as oral tablets at a dose of 2.5 mg, taken once daily, as per the parent study.
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.
Time frame: From start of treatment up to 30 days after last dose of study treatment, assessed up to approximately 53 months
Percentage of participants with Clinical Benefit Rate (CBR)
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.
Time frame: Up to approximately 54 months
No study locations are listed for this record.
Plan to share: Yes — 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.
No publications or documents are linked to this record.
This study is not yet recruiting, as verified in Sep 2026. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Novartis Pharmaceuticals