Actively Recruiting
A Two-stage Double-blind, Randomized, Placebo-controlled Study to Assess the Efficacy, Safety and Pharmacokinetics of Alpelisib in Pediatric and Adult Patients With Lymphatic Malformations Associated With a PIK3CA Mutation
Led by Novartis Pharmaceuticals · Updated on 2026-06-02
232
Participants Needed
57
Research Sites
256 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating alpelisib film-coated tablets (FCT) in participants with lymphatic malformations (LyM) associated with a PIK3CA mutation. The study aims to assess how alpelisib affects radiological response and symptom severity compared to placebo. This phase II/III, multi-center trial includes both pediatric and adult participants with this genetic mutation. The study has two stages. Stage 1 is a 24-week open-label phase to select doses of alpelisib for adults (18 years and older) and children aged 6 to 17 years, followed by an extension. Participants are randomized to different doses based on age. Stage 2 is a 24-week randomized, double-blind, placebo-controlled phase to confirm efficacy and safety of the selected dose in adults and children 6 to 17 years old, followed by an open-label extension. Additionally, children aged 0 to 5 years participate in a 24-week open-label phase with dose 3 of alpelisib, with an extension if they enroll. Participants will undergo regular assessments including radiological evaluations, symptom severity scales, and quality of life questionnaires up to approximately 8 years. Safety and response are monitored throughout both stages. The primary outcome is the radiological response rate at Week 24 of Stage 2. Other measures include patient and investigator impressions of change, symptom and lesion assessments, and alpelisib blood levels. Participants are expected to take study medication orally and attend scheduled visits during the study period.
CONDITIONS
Brief 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 remain at the clinical site and follow study schedules and restrictions
- Physician-confirmed diagnosis of symptomatic lymphatic malformations not included under PROS criteria
- Not a candidate for or unwilling to receive non-drug therapies like surgery, sclerotherapy, or embolization until after Week 24
- Evidence of a somatic mutation in the PIK3CA gene before randomization
- At least one measurable lymphatic malformation lesion confirmed before randomization
- Ability to ingest study medication orally or via feeding tube as assessed within 7 days before treatment start
You will not qualify if you...
- Physician-confirmed diagnosis of PROS at the time of informed 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
- Established type I diabetes or uncontrolled type II diabetes
- Previous treatment with alpelisib or other PI3K inhibitors for more than 2 weeks
- Other exclusion criteria may apply
AI-Screening
AI-Powered Screening
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Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 24 weeks
Participants aged 6 years and older receive alpelisib at assigned doses in an open-label fashion to determine optimal dosing over 24 weeks.
Visits during 24 weeks of treatment
Duration - 24 weeks
Participants aged 6 years and older are randomized to receive alpelisib at the selected confirmatory dose or placebo for 24 weeks in a double-blind manner. Pediatric participants aged 0-5 years receive open-label alpelisib dosing for 24 weeks.
Visits during 24 weeks of treatment
Duration - Duration varies based on participant continuation
Participants who complete Stage 1 or Stage 2 may continue receiving alpelisib in an open-label extension phase to further assess safety and efficacy.
Visits as scheduled during extension
Trial Site Locations
Total: 57 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, 45229-3039
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
Ulm, Germany, 89081
Actively Recruiting
42
Novartis Investigative Site
Bologna, BO, Italy, 40138
Actively Recruiting
43
Novartis Investigative Site
Milan, MI, Italy, 20122
Actively Recruiting
44
Novartis Investigative Site
Roma, RM, Italy, 00165
Actively Recruiting
45
Novartis Investigative Site
Roma, RM, Italy, 00168
Actively Recruiting
46
Novartis Investigative Site
Torino, TO, Italy, 10126
Actively Recruiting
47
Novartis Investigative Site
Naples, Italy, 80122
Actively Recruiting
48
Novartis Investigative Site
Nijmegen, Gelderland, Netherlands, 6500HB
Actively Recruiting
49
Novartis Investigative Site
Rotterdam, South Holland, Netherlands, 3015 GD
Actively Recruiting
50
Novartis Investigative Site
Palma, Balearic Islands, Spain, 07120
Actively Recruiting
51
Novartis Investigative Site
Esplugues, Barcelona, Spain, 08950
Actively Recruiting
52
Novartis Investigative Site
L'Hospitalet de Llobregat, Barcelona, Spain, 08907
Actively Recruiting
53
Novartis Investigative Site
A Coruña, Spain, 15006
Actively Recruiting
54
Novartis Investigative Site
Barcelona, Spain, 08035
Actively Recruiting
55
Novartis Investigative Site
Madrid, Spain, 28009
Actively Recruiting
56
Novartis Investigative Site
Madrid, Spain, 28046
Actively Recruiting
57
Novartis Investigative Site
Lausanne, Switzerland, 1011
Actively Recruiting
Research Team
N
Novartis Pharmaceuticals
N
Novartis Pharmaceuticals
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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