Actively Recruiting
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)
Led by Novartis Pharmaceuticals · Updated on 2026-04-30
104
Participants Needed
30
Research Sites
259 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
This study is designed to demonstrate the efficacy and assess safety and tolerability of oral daily alpelisib in participants with PIK3CA-related overgrowth spectrum (PROS).
CONDITIONS
Official Title
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)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male or female participants aged 652 years at the time of informed consent or assent
- Diagnosis of PROS with symptomatic and progressive overgrowth, including syndromic disease or isolated features except isolated macrodactyly, macrocephaly or epidermal nevus
- Documented somatic mutation in the PIK3CA gene confirmed by DNA-based test and available archival tissue or fresh biopsy if needed
- Karnofsky performance status 6550 for participants older than 16 years or Lansky performance status 6550 for those 16 years or younger
- PGI-S score of mild, moderate, severe, or very severe at screening
- Adequate bone marrow and organ function
- At least one measurable PROS-related lesion (longest diameter 652 cm) confirmed by BIRC and causing complaints, symptoms, or functional limitations affecting daily life
You will not qualify if you...
- Participants with only isolated macrodactyly, epidermal nevus/nevi, and macroencephaly without other PROS-related lesions
- Previous treatment with alpelisib or other PI3K inhibitors except short treatment attempts less than 2 weeks stopped at least 4 weeks before study
- Major surgery within 3 months prior to consent or assent
- Radiation treatment for PROS within 12 months prior to consent or assent
- Significant PROS-related thrombotic event (Grade 2 or higher) within 30 days before consent or assent
- Sclerotherapy or embolization for vascular complications within 6 weeks before consent or assent
- Clinically significant bleeding from a PROS lesion (Grade 2 or higher) within 30 days before treatment start
- Worsening PROS-related lab abnormalities or symptoms indicating uncontrolled condition during screening
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 30 locations
1
Washington University
St Louis, Missouri, United States, 63110
Actively Recruiting
2
Cinn Children Hosp Medical Center
Cincinnati, Ohio, United States, 45206
Actively Recruiting
3
Novartis Investigative Site
Sydney, New South Wales, Australia, 2010
Actively Recruiting
4
Novartis Investigative Site
Sydney, New South Wales, Australia, 2031
Actively Recruiting
5
Novartis Investigative Site
North Adelaide, South Australia, Australia, 5066
Actively Recruiting
6
Novartis Investigative Site
Salzburg, Austria, 5020
Actively Recruiting
7
Novartis Investigative Site
Vienna, Austria, A 1090
Actively Recruiting
8
Novartis Investigative Site
Ghent, Belgium, 9000
Actively Recruiting
9
Novartis Investigative Site
Angers, France, 49933
Actively Recruiting
10
Novartis Investigative Site
Bron, France, 69677
Actively Recruiting
11
Novartis Investigative Site
Dijon, France, 21000
Actively Recruiting
12
Novartis Investigative Site
Montpellier, France, 34295
Actively Recruiting
13
Novartis Investigative Site
Paris, France, 75015
Actively Recruiting
14
Novartis Investigative Site
Toulouse, France, 31400
Actively Recruiting
15
Novartis Investigative Site
Tours, France, 37044
Actively Recruiting
16
Novartis Investigative Site
Freiburg im Breisgau, Baden-Wurttemberg, Germany, 79106
Actively Recruiting
17
Novartis Investigative Site
Düsseldorf, North Rhine-Westphalia, Germany, 40225
Actively Recruiting
18
Novartis Investigative Site
Leipzig, Saxony, Germany, 04103
Actively Recruiting
19
Novartis Investigative Site
Halle, Saxony-Anhalt, Germany, 06120
Actively Recruiting
20
Novartis Investigative Site
Berlin, Germany, 13353
Actively Recruiting
21
Novartis Investigative Site
Stuttgart, Germany, 70374
Actively Recruiting
22
Novartis Investigative Site
Roma, RM, Italy, 00168
Actively Recruiting
23
Novartis Investigative Site
Torino, TO, Italy, 10126
Actively Recruiting
24
Novartis Investigative Site
Trieste, TS, Italy, 34137
Actively Recruiting
25
Novartis Investigative Site
Esplugues, Barcelona, Spain, 08950
Actively Recruiting
26
Novartis Investigative Site
A Coruña, Spain, 15006
Actively Recruiting
27
Novartis Investigative Site
Barcelona, Spain, 08035
Actively Recruiting
28
Novartis Investigative Site
Madrid, Spain, 28046
Actively Recruiting
29
Novartis Investigative Site
Lausanne, Switzerland, 1011
Actively Recruiting
30
Novartis Investigative Site
Liverpool, United Kingdom, L12 2AP
Actively Recruiting
Research Team
N
Novartis Pharmaceuticals
CONTACT
N
Novartis Pharmaceuticals
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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