Alpelisib: In Stage 1: adult participants (≥18 years of age) will receive dose 1 or dose 2 of alpelisib; pediatric participants (6-17 years of age) will receive dose 2 or dose 3 of alpelisib. In Stage 2: Adult participants will receive alpelisib at the dose selected for confirmatory phase in adult participants; pediatric participants (6-17 years of age) will will receive alpelisib at the dose selected for confirmatory phase in pediatric participants; and pediatric participants of 0-5 years of age will receive dose 3 of alpelisib
Placebo: In Stage 2, participants will receive matching placebo for 24 weeks of the study
The main purpose of this study in participants with PIK3CA-mutated LyM is to assess the change in radiological response and symptom severity upon treatment with alpelisib film-coated tablets (FCT) as compared to placebo.
| Facility | City | Region | Status |
|---|---|---|---|
| UCSF Benioff Children s Hospital | Oakland | California | Recruiting |
| Childrens Hospital of Orange County | Orange | California | Recruiting |
| Lucile Packard Childrens Hosp | Palo Alto | California | Recruiting |
| Childrens National Medical Center | Washington D.C. | District of Columbia | Recruiting |
| Nemours Childrens Clinic | Jacksonville | Florida | Recruiting |
| Childrens Hosp Boston Dept of Heme | Boston | Massachusetts | Recruiting |
| WA Uni School Of Med | St Louis | Missouri | Recruiting |
| University of North Carolina at Chapel Hill | Chapel Hill | North Carolina | Recruiting |
| Cinn Children Hosp Medical Center | Cincinnati | Ohio | Recruiting |
| Univ Hospital Of Cleveland | Cleveland | Ohio | Recruiting |
| Cleveland Clinic Foundation | Cleveland | Ohio | Recruiting |
| Nationwide Children s Hospital | Columbus | Ohio | Recruiting |
| Oregon Health Science University | Portland | Oregon | Recruiting |
| CHOP Abramson Pediatric Resch Ctr | Philadelphia | Pennsylvania | Recruiting |
| Childrens Hosp Pittsburgh UPMC | Pittsburgh | Pennsylvania | Recruiting |
| Baylor College Of Medicine | Houston | Texas | Recruiting |
| U of TX Health Science Ct | Houston | Texas | Recruiting |
| Childrens Hospital and Regional Medical Center | Seattle | Washington | Recruiting |
| Novartis Investigative Site | CABA | Buenos Aires | Recruiting |
| Novartis Investigative Site | CABA | Buenos Aires | Recruiting |
| Novartis Investigative Site | Capital Federal | Argentina | Recruiting |
| Novartis Investigative Site | Sydney | New South Wales | Recruiting |
| Novartis Investigative Site | Sydney | New South Wales | Recruiting |
| Novartis Investigative Site | Brisbane | Queensland | Recruiting |
| Novartis Investigative Site | Brussels | Belgium | Recruiting |
| Novartis Investigative Site | Brno | Czechia | Recruiting |
| Novartis Investigative Site | Angers | France | Recruiting |
| Novartis Investigative Site | Bordeaux | France | Recruiting |
| Novartis Investigative Site | Bron | France | Recruiting |
| Novartis Investigative Site | Caen | France | Recruiting |
| Novartis Investigative Site | Dijon | France | Recruiting |
| Novartis Investigative Site | Lille | France | Recruiting |
| Novartis Investigative Site | Marseille | France | Recruiting |
| Novartis Investigative Site | Montpellier | France | Recruiting |
| Novartis Investigative Site | Paris | France | Recruiting |
| Novartis Investigative Site | Paris | France | Recruiting |
| Novartis Investigative Site | Toulouse | France | Recruiting |
| Novartis Investigative Site | Tours | France | Recruiting |
| Novartis Investigative Site | Freiburg im Breisgau | Baden-Wurttemberg | Recruiting |
| Novartis Investigative Site | Mannheim | Baden-Wurttemberg | Withdrawn |
+ 21 more sites — see the full list on the official registry below.
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT05948943 on ClinicalTrials.gov ↗ ← All trials in the UK