PIK3CA-Related Overgrowth Spectrum (PROS)Phase 2EPIK-RONCT07679269
Roll-Over Study of Alpelisib (BYL719) for Continued Access and Long-Term Safety.
A Phase 2 study testing Alpelisib, Fulvestrant and Trastuzumab in people with PIK3CA-Related Overgrowth Spectrum (PROS) and Breast Neoplasms (Including Advanced or Metastatic Breast Cancer With PIK3CA Mutation), run by Novartis Pharmaceuticals.
Trial 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… Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- PIK3CA-Related Overgrowth Spectrum (PROS), Breast Neoplasms (Including Advanced or Metastatic Breast Cancer With PIK3CA Mutation)
- What is being tested?
- Alpelisib, Fulvestrant and Trastuzumab
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 51 participants
- Where
- No sites listed yet
- Study dates
- 15 Dec 2026to 20 Apr 2032
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: 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.
Read the 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.
- Conditions
- PIK3CA-Related Overgrowth Spectrum (PROS), Breast Neoplasms (Including Advanced or Metastatic Breast Cancer With PIK3CA Mutation)
- Being tested
- Alpelisib, Fulvestrant, Trastuzumab, Pertuzumab, Letrozole
- 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
- Medical subject headings
- Breast Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AlpelisibDrug
Also called BYL719
Administered as oral tablets at dose levels as per the parent study, taken once daily.
FulvestrantDrug
Administered as an intramuscular injection at dose levels and dosing schedule as per standard of care, in accordance with the parent study.
TrastuzumabDrug
Administered as an intravenous infusion of a reconstituted lyophilized powder at dose levels as per the parent study, given every 21 days.
PertuzumabDrug
Administered as an intravenous infusion of a solution concentrate at dose levels as per the parent study, given every 21 days.
LetrozoleDrug
Administered as oral tablets at a dose of 2.5 mg, taken once daily, as per the parent study.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: Alpelisib; Drug: Fulvestrant; Drug: Trastuzumab; Drug: Pertuzumab; Drug: Letrozole |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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)
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.
Measured: Up to approximately 54 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Jul 2026
- Study start15 Dec 2026 (estimated)
- Primary completion23 Mar 2032 (estimated)
- Study completion20 Apr 2032 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for PIK3CA-Related Overgrowth Spectrum (PROS)Studies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 51 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07679269
- Sponsor's protocol ID
- CBYL719F12003B
- Other IDs
- 2026-525890-38-00
- Acronym
- EPIK-RO
- Status last verified
- Sep 2026
- First submitted
- 25 Jun 2026
- Last updated
- 18 Sep 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- 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.
Study at a glance
From the registry record, last updated by the sponsor on 18 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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.
- ClinicalTrials.gov ID
- NCT07679269
- Acronym / sponsor ID
- EPIK-RO / CBYL719F12003B
- 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
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 51 participants (estimated)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 15 Dec 2026 (estimated)
- Primary completion
- 23 Mar 2032 (estimated)
- Study completion
- 20 Apr 2032 (estimated)
- First posted
- 1 Jul 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Alpelisib (BYL719) or in Combination Therapy | Other | Drug: Alpelisib, Drug: Fulvestrant, Drug: Trastuzumab, Drug: Pertuzumab, Drug: LetrozoleEligible 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. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- 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 (7 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AlpelisibAlso: BYL719Administered as oral tablets at dose levels as per the parent study, taken once daily. | Drug | Alpelisib (BYL719) or in Combination Therapy |
| FulvestrantAdministered as an intramuscular injection at dose levels and dosing schedule as per standard of care, in accordance with the parent study. | Drug | Alpelisib (BYL719) or in Combination Therapy |
| TrastuzumabAdministered as an intravenous infusion of a reconstituted lyophilized powder at dose levels as per the parent study, given every 21 days. | Drug | Alpelisib (BYL719) or in Combination Therapy |
| PertuzumabAdministered as an intravenous infusion of a solution concentrate at dose levels as per the parent study, given every 21 days. | Drug | Alpelisib (BYL719) or in Combination Therapy |
| LetrozoleAdministered as oral tablets at a dose of 2.5 mg, taken once daily, as per the parent study. | Drug | Alpelisib (BYL719) or in Combination Therapy |
Primary outcome
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.
1 secondary outcome
Percentage of participants with Clinical Benefit Rate (CBR)
Time frame: Up to approximately 54 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Sep 2026.
- Individual participant dataSharing planned: 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.
Related trials for PIK3CA-Related Overgrowth Spectrum (PROS)
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Novartis Pharmaceuticals study director
Novartis Pharmaceuticals
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Alpelisib, Fulvestrant and Trastuzumab in people with PIK3CA-Related Overgrowth Spectrum (PROS) and Breast Neoplasms (Including Advanced or Metastatic Breast Cancer With PIK3CA Mutation), run by Novartis Pharmaceuticals.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 18 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Novartis Pharmaceuticals (+41613241111, novartis.email@novartis.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Sep 2026.