Actively Recruiting
Alpelisib in Pediatric and Adult Patients With Lymphatic Malformations Associated With a PIK3CA Mutation.
Led by Novartis Pharmaceuticals · Updated on 2026-05-12
232
Participants Needed
56
Research Sites
492 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Alpelisib in Pediatric and Adult Patients With Lymphatic Malformations Associated With a PIK3CA Mutation.
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Signed informed consent and assent when applicable
- Willingness to stay at the clinical site and follow study rules
- Physician-confirmed symptomatic lymphatic malformation not classified under PROS
- Not a candidate or unwilling to receive non-drug therapies like sclerotherapy, embolization, or surgery until Week 24 of Stage 1 and 2
- Evidence of a somatic PIK3CA gene mutation before randomization
- At least one measurable lymphatic malformation lesion confirmed by BIRC before randomization
- Ability to take study drug in tablet, suspension, granules, or oral suspension form, including via feeding tubes
You will not qualify if you...
- Diagnosis of PROS at the time of consent
- Diagnosis of Central Conducting Lymphatic Anomaly, General Lymphatic Anomaly, Gorham-Stout disease, or Kaposiform lymphangiomatosis
- History of Stevens-Johnson syndrome, erythema multiforme, or toxic epidermal necrolysis
- Diagnosis of type I diabetes or uncontrolled type II diabetes at consent
- Previous treatment with alpelisib or other PI3K inhibitors for more than 2 weeks
- Other criteria may apply
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 56 locations
1
UCSF Benioff Children s Hospital
Oakland, California, United States, 94609
Actively Recruiting
2
Lucile Packard Childrens Hosp
Palo Alto, California, United States, 94304
Actively Recruiting
3
Childrens National Medical Center
Washington D.C., District of Columbia, United States, 20010-2970
Actively Recruiting
4
Nemours Childrens Clinic
Jacksonville, Florida, United States, 32207
Actively Recruiting
5
Childrens Hosp Boston Dept of Heme
Boston, Massachusetts, United States, 02115
Actively Recruiting
6
WA Uni School Of Med
St Louis, Missouri, United States, 63110
Actively Recruiting
7
Cinn Children Hosp Medical Center
Cincinnati, Ohio, United States, 45206
Actively Recruiting
8
Univ Hospital Of Cleveland
Cleveland, Ohio, United States, 44106
Actively Recruiting
9
Cleveland Clinic Foundation
Cleveland, Ohio, United States, 44195
Actively Recruiting
10
Nationwide Children s Hospital
Columbus, Ohio, United States, 43205
Actively Recruiting
11
Oregon Health Science University
Portland, Oregon, United States, 97239
Actively Recruiting
12
CHOP Abramson Pediatric Resch Ctr
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
13
Childrens Hosp Pittsburgh UPMC
Pittsburgh, Pennsylvania, United States, 15224
Actively Recruiting
14
Baylor College Of Medicine
Houston, Texas, United States, 77030
Actively Recruiting
15
U of TX Health Science Ct
Houston, Texas, United States, 77030
Actively Recruiting
16
Childrens Hospital and Regional Medical Center
Seattle, Washington, United States, 98105
Actively Recruiting
17
Novartis Investigative Site
CABA, Buenos Aires, Argentina, C1181ACH
Actively Recruiting
18
Novartis Investigative Site
CABA, Buenos Aires, Argentina, C1425BEA
Actively Recruiting
19
Novartis Investigative Site
Capital Federal, Argentina, C1023AAB
Actively Recruiting
20
Novartis Investigative Site
Sydney, New South Wales, Australia, 2010
Actively Recruiting
21
Novartis Investigative Site
Sydney, New South Wales, Australia, 2031
Actively Recruiting
22
Novartis Investigative Site
Brisbane, Queensland, Australia, 4101
Actively Recruiting
23
Novartis Investigative Site
Brussels, Belgium, 1200
Actively Recruiting
24
Novartis Investigative Site
Angers, France, 49933
Actively Recruiting
25
Novartis Investigative Site
Bordeaux, France, 33076
Actively Recruiting
26
Novartis Investigative Site
Bron, France, 69677
Actively Recruiting
27
Novartis Investigative Site
Caen, France, 14033
Actively Recruiting
28
Novartis Investigative Site
Dijon, France, 21000
Actively Recruiting
29
Novartis Investigative Site
Lille, France, 59000
Actively Recruiting
30
Novartis Investigative Site
Marseille, France, 13885
Actively Recruiting
31
Novartis Investigative Site
Montpellier, France, 34295
Actively Recruiting
32
Novartis Investigative Site
Paris, France, 75010
Actively Recruiting
33
Novartis Investigative Site
Paris, France, 75015
Actively Recruiting
34
Novartis Investigative Site
Toulouse, France, 31054
Actively Recruiting
35
Novartis Investigative Site
Tours, France, 37044
Actively Recruiting
36
Novartis Investigative Site
Freiburg im Breisgau, Baden-Wurttemberg, Germany, 79106
Actively Recruiting
37
Novartis Investigative Site
Mannheim, Baden-Wurttemberg, Germany, 68305
Withdrawn
38
Novartis Investigative Site
Cologne, North Rhine-Westphalia, Germany, 50937
Actively Recruiting
39
Novartis Investigative Site
Leipzig, Saxony, Germany, 04103
Actively Recruiting
40
Novartis Investigative Site
Berlin, Germany, 13353
Actively Recruiting
41
Novartis Investigative Site
Bologna, BO, Italy, 40138
Actively Recruiting
42
Novartis Investigative Site
Milan, MI, Italy, 20122
Actively Recruiting
43
Novartis Investigative Site
Roma, RM, Italy, 00165
Actively Recruiting
44
Novartis Investigative Site
Roma, RM, Italy, 00168
Actively Recruiting
45
Novartis Investigative Site
Torino, TO, Italy, 10126
Actively Recruiting
46
Novartis Investigative Site
Naples, Italy, 80122
Actively Recruiting
47
Novartis Investigative Site
Nijmegen, Gelderland, Netherlands, 6500HB
Actively Recruiting
48
Novartis Investigative Site
Rotterdam, South Holland, Netherlands, 3015 GD
Actively Recruiting
49
Novartis Investigative Site
Palma, Balearic Islands, Spain, 07120
Actively Recruiting
50
Novartis Investigative Site
Esplugues, Barcelona, Spain, 08950
Actively Recruiting
51
Novartis Investigative Site
L'Hospitalet de Llobregat, Barcelona, Spain, 08907
Actively Recruiting
52
Novartis Investigative Site
A Coruña, Spain, 15006
Actively Recruiting
53
Novartis Investigative Site
Barcelona, Spain, 08035
Actively Recruiting
54
Novartis Investigative Site
Madrid, Spain, 28009
Actively Recruiting
55
Novartis Investigative Site
Madrid, Spain, 28046
Actively Recruiting
56
Novartis Investigative Site
Lausanne, Switzerland, 1011
Actively Recruiting
Research Team
N
Novartis Pharmaceuticals
CONTACT
N
Novartis Pharmaceuticals
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
9
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