Alpelisib in Pediatric and Adult Patients With Lymphatic Malformations Associated With a PIK3CA Mutation.
Condition(s) studied
Investigational drug(s) / intervention(s)
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
Study summary
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.
Eligibility
Primary outcome measure(s)
- Stage 2:Radiological response rate at Week 24 of Stage 2 (adult and pediatric (6 - 17 years of age) participants) — Baseline, Week 24
Radiological response defined by achieving at least 20% reduction in the sum of target lesion volumes (1 to 3 lesions), assessed by MRI by a BIRC at Week 24, provided that none of the individual target lesions has at least 20% increase from baseline and in absence of progression of non-target lesions and without new lesions. The percentage of participants with a radiological response at Week 24 of Stage 2 in adult and pediatric (6-17 years of age) groups will be assessed
Trial sites (61)
| 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.
More Novartis Pharmaceuticals trials in Italy
Other trials for the same condition
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 Italy