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Compound · clinical trials

Alpelisib

Source: ClinicalTrials.gov, U.S. National Library of Medicine — the 12 trials in our USA, UK, China, Ireland and India registers that name Alpelisib as an intervention. Synced 24 Sep 2026
12Trials tracked
4Recruiting now
7Lead sponsors
5Country registers
Maintained by Sreepriya Prasannan
MSc Digital Transformation of Life Sciences (Innopharma Education / Griffith College); MSc & BSc Botany HSE Spark Ignite 2026 โ€” Top 14 finalist
Compiled from ClinicalTrials.gov registry records ยท Last verified ยท Editorial standards

Alpelisib is named as a drug or biological intervention in 12 clinical trials in our USA, UK, China, Ireland and India trial registers, 4 of which are recruiting. Lead sponsors include Novartis Pharmaceuticals (6), Hoffmann-La Roche (1) and Stemline Therapeutics, Inc. (1). Registered phases: Phase 2, Phase 3, Phase 1, Phase 1/2 and Phase 2/3. Conditions studied include Breast Cancer; PIK3CA-related Overgrowth Spectrum (PROS); Metastatic Breast Cancer.

01

Clinical trials of Alpelisib

12 trials
NCT IDStudyPhaseStatusLead sponsorRegister
NCT05646862 A Study Evaluating the Efficacy and Safety of Inavolisib Plus Fulvestrant Compared With Alpelisib Plus Fulvestrant in Participants With HR-Positive, HER2-Negative, PIK3CA Mutated, Locally Advanced or Metastatic Breast Cancer Post CDK4/6i and Endocrine Combination Therapy
Breast Cancer
Phase 3 Recruiting Hoffmann-La Roche UK, USA, China
NCT05948943 Alpelisib in Pediatric and Adult Patients With Lymphatic Malformations Associated With a PIK3CA Mutation. (ClinicalTrials.gov ↗)
Lymphatic Malformations
Phase 2/3 Recruiting Novartis Pharmaceuticals UK, USA
NCT06145308 Precision Treatment of Recurrent/Metastatic Salivary Gland Carcinoma Guided by Molecular Typing (ClinicalTrials.gov ↗)
Salivary Gland Carcinoma, Precision Therapy
Phase 2 Recruiting Peking Union Medical College China
NCT06997588 EPIK-P4: A Phase II Single-arm Study to Assess the Efficacy, Safety and Pharmacokinetics of Alpelisib (BYL719) in Pediatric and Adult Patients With PIK3CA-related Overgrowth Spectrum (PROS) (ClinicalTrials.gov ↗)
PIK3CA-related Overgrowth Spectrum (PROS)
Phase 2 Recruiting Novartis Pharmaceuticals UK, USA
NCT04208178 Study of Alpelisib (BYL719) in Combination With Trastuzumab and Pertuzumab as Maintenance Therapy in Patients With HER2-positive Advanced Breast Cancer With a PIK3CA Mutation (ClinicalTrials.gov ↗)
Advanced HER2+Breast Cancer
Phase 3 Active, closed Novartis Pharmaceuticals USA, China
NCT05038735 Study to Assess the Efficacy and Safety of Alpelisib Plus Fulvestrant in Participants With HR-positive (HR+), HER2-negative, Advanced Breast Cancer After Treatment With a CDK4/6 Inhibitor and an Aromatase Inhibitor. (ClinicalTrials.gov ↗)
Breast Cancer
Phase 3 Active, closed Novartis Pharmaceuticals Ireland
NCT05501886 Gedatolisib Plus Fulvestrant With or Without Palbociclib vs Standard-of-Care for the Treatment of Patients With Advanced or Metastatic HR+/HER2- Breast Cancer (VIKTORIA-1) (ClinicalTrials.gov ↗)
Breast Cancer
Phase 3 Active, closed Celcuity Inc UK, USA, India, China
NCT04589650 Study Assessing the Efficacy, Safety and PK of Alpelisib (BYL719) in Pediatric and Adult Patients With PIK3CA-related Overgrowth Spectrum (ClinicalTrials.gov ↗)
PIK3CA-related Overgrowth Spectrum (PROS)
Phase 2 Active, closed Novartis Pharmaceuticals UK, USA, China
NCT04980833 Study Assessing Long-term Safety and Efficacy of Alpelisib in Patients With PIK3CA-Related Overgrowth Spectrum (PROS) Who Previously Participated in Study CBYL719F12002 (EPIK-P1) (ClinicalTrials.gov ↗)
PIK3CA-related Overgrowth Spectrum (PROS)
Phase 2 Active, closed Novartis Pharmaceuticals Ireland, USA
NCT05563220 Open-Label Umbrella Study To Evaluate Safety And Efficacy Of Elacestrant In Various Combination In Participants With Metastatic Breast Cancer
Breast Cancer, Metastatic Breast Cancer
Phase 1/2 Active, closed Stemline Therapeutics, Inc. UK, USA
NCT04188548 A Study of LY3484356 in Participants With Advanced or Metastatic Breast Cancer or Endometrial Cancer
Breast Cancer, Advanced Breast Cancer, Metastatic Breast Cancer
Phase 1 Active, closed Eli Lilly and Company USA, China
NCT05508906 Phase 1b Study of OP-1250 (Palazestrant) in Combination With Ribociclib, Alpelisib, Everolimus, or Atirmociclib in ER+, HER2- Breast Cancer (ClinicalTrials.gov ↗)
Metastatic Breast Cancer, ER-positive Breast Cancer, HER2-negative Breast Cancer
Phase 1 Active, closed Olema Pharmaceuticals, Inc. USA

Registers list studies that are recruiting, about to open, active but closed to enrolment, or enrolling by invitation. The UK and USA registers cover industry-sponsored studies only. Trial titles and statuses are copied from the registry as published.

02

Conditions studied

11 conditions
Condition (as registered)Trials
Breast Cancer6
PIK3CA-related Overgrowth Spectrum (PROS)3
Metastatic Breast Cancer3
Lymphatic Malformations1
Advanced Breast Cancer1
Endometrial Cancer1
Advanced HER2+Breast Cancer1
ER-positive Breast Cancer1
HER2-negative Breast Cancer1
Salivary Gland Carcinoma1
Precision Therapy1
03

Sponsors

lead sponsor per trial
Lead sponsorTrials
Novartis Pharmaceuticals6
Hoffmann-La Roche1
Stemline Therapeutics, Inc.1
Celcuity Inc1
Eli Lilly and Company1
Olema Pharmaceuticals, Inc.1
Peking Union Medical College1
04

Trials by country register

A trial with sites in more than one country appears in each of those registers.

How this page is built

Everything on this page is copied from public ClinicalTrials.gov registry records โ€” it is not written from memory, and nothing here describes how Alpelisib works or whether it is effective. Each trial links to its own record so you can check it yourself.

This page is built automatically: a daily sync job reads the registry records behind our country trial registers, collects every trial that lists Alpelisib (or a registered synonym) as a drug or biological intervention, and tabulates the registry fields directly. Placebos and generic comparators are excluded. There is no AI drafting and no per-compound editorial pass, and the page has not been clinically reviewed. The “last verified” date above is the timestamp of that sync, not a review date. Our general editorial approach is described in our editorial standards and corrections policy. The ClinicalTrials.gov data on this page was last retrieved on 24 Sep 2026.

Please verify before you rely on this. This page is general information for life-science and pharmaceutical professionals. It is not medical advice, and it has not been reviewed by a clinician โ€” our editorial team holds life-science qualifications, not clinical ones. It is not exhaustive and may not reflect the most recent label change. Always check the official prescribing information (US Prescribing Information or EU SmPC) and speak to your doctor or pharmacist before acting on anything here. Drugs in the same class are not automatically interchangeable, and approvals, brand names and indications differ between the US, the EU/Ireland (EMA/HPRA) and other regions. Spotted an error? Tell us โ€” we correct promptly and log it.