Alpelisib in Pediatric and Adult Patients With Lymphatic Malformations Associated With a PIK3CA Mutation.
NCT ID: NCT05948943
Last Updated: 2025-11-12
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
RECRUITING
PHASE2/PHASE3
232 participants
INTERVENTIONAL
2023-11-24
2033-05-02
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
* Stage 1 is designed to select the dose(s) for the confirmatory phase (DSCP) for alpelisib in Stage 2 and will comprise a 24-week open-label core phase in adult (≥18 years of age) and pediatric participants (6-17 years of age) with PIK3CA-mutated LyM, followed by an extension. After eligibility has been confirmed at screening, participants will be randomized in a 1:1 ratio to the different alpelisib doses according to their age. Depending on the results at the end of Stage 1 core phase, the Stage 2 will be opened to adult and/or pediatric participants or the study may be stopped.
* Stage 2 is designed to confirm the efficacy and assess safety of alpelisib at the DSCP in participants with PIK3CA-mutated LyM and will comprise a 24-week randomized, double blind, placebo-controlled confirmatory phase in adult (≥18 years of age) and pediatric participants 6-17 years of age followed by an open-label extension. After eligibility has been confirmed at screening participants will be randomized in a 2:1 ratio to alpelisib or placebo.
Additionally, in parallel, Stage 2 will include a 24-week open-label core phase in pediatric participants 0-5 years of age followed by an extension, if pediatric participants will be enrolling in Stage 2.
Based on the results of the 24-week open-label core phase of Stage 1, the dose(s) for Stage 2 will be selected by Novartis in consultation with the Steering Committee (SC). During the 24-week randomized, double blind, placebo-controlled core phase of Stage 2, an Independent Data Monitoring Committee (DMC) will conduct periodic safety and efficacy reviews to assess the risk benefit profile of the treatment.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Keywords
Explore important study keywords that can help with search, categorization, and topic discovery.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
RANDOMIZED
SEQUENTIAL
TREATMENT
QUADRUPLE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
Pediatric participants (6-17 years of age), alpelisib dose 2 (Stage 1)
Pediatric participants 6-17 years of age who will receive dose 2 of alpelisib in an open-label fashion for at least 24 weeks unless they discontinue earlier (Stage 1)
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
Pediatric participants (6-17 years of age), alpelisib dose 3 (Stage 1)
Pediatric participants 6-17 years of age who will receive dose 3 of alpelisib in an open-label fashion for at least 24 weeks unless they discontinue earlier (Stage 1).
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
Adult participants, alpelisib (Stage 2)
Adult participants (≥18 years of age) who will receive alpelisib at the dose selected for confirmatory phase in adult participants (Stage 2)
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
Adult participants, placebo (Stage 2)
Adult participants (≥18 years of age) who will receive matching placebo
Placebo
In Stage 2, participants will receive matching placebo for 24 weeks of the study
Pediatric participants (6-17 years of age), alpelisib (Stage 2)
Pediatric participants (6-17 years of age) who will receive alpelisib at the dose selected for confirmatory phase in pediatric participants (Stage 2)
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
Pediatric participants (6-17 years of age), placebo (Stage 2)
Pediatric participants (6-17 years of age) who will receive matching placebo
Placebo
In Stage 2, participants will receive matching placebo for 24 weeks of the study
Pediatric participants (0-5 years of age), alpelisib (Stage 2)
Pediatric participants of 0-5 years who will dose 3 of alpelisib in an open-label fashion for at least 24 weeks unless they discontinue earlier
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
Adult participants, alpelisib dose 1 (Stage 1)
Adult participants (≥18 years of age) who will receive dose 1 of alpelisib an open-label fashion for at least 24 weeks unless they discontinue earlier (Stage 1)
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
Adult participants, alpelisib dose 2 (Stage 1)
Adult participants (≥18 years of age) who will receive dose 2 of alpelisib in an open-label fashion for at least 24 weeks unless they discontinue earlier (Stage 1).
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
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
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
Other Intervention Names
Discover alternative or legacy names that may be used to describe the listed interventions across different sources.
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
2. Participant must be willing to remain at the clinical site as required by the protocol and be willing to adhere to study restrictions and examination schedules.
3. Participant has a physician confirmed and documented diagnosis of a symptomatic LyM at the time of informed consent (Note: the physician must confirm that the LyM cannot be included under the PROS diagnostic criteria).
4. Participant is not considered as a candidate for or is not willing to receive non-drug therapies including but not limited to sclerotherapy, embolization, and surgery until the completion of Week 24 in Stage 1 and 2.
5. Participant has evidence of a somatic mutation(s) in the PIK3CA gene prior to randomization.
6. Participant has at least one measurable LyM lesion confirmed by BIRC assessment prior to randomization.
7. Participants must be able to ingest study drug (either in tablet form or as a drinkable suspension \[Groups 1 to 4\] or granules or as an oral suspension \[Group 5\]) as assessed within 7 days before study treatment start. Drug administration via feeding tubes is allowed.
Exclusion Criteria
2. Participant has a physician-confirmed and documented diagnosis of a Central Conducting Lymphatic Anomaly, General Lymphatic Anomaly, Gorham-Stout disease, Kaposiform lymphangiomatosis at the time of informed consent.
3. Participant has a known history of Stevens-Johnson syndrome, erythema multiforme, or toxic epidermal necrolysis at the time of informed consent.
4. Participant has an established diagnosis of type I diabetes mellitus or uncontrolled type II diabetes mellitus at the time of informed consent.
5. Participant had previous treatment with alpelisib and/or any other PI3K inhibitors with treatment duration longer than 2 weeks at the time of informed consent.
0 Years
100 Years
ALL
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
Novartis Pharmaceuticals
INDUSTRY
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Principal Investigators
Learn about the lead researchers overseeing the trial and their institutional affiliations.
Novartis Pharmaceuticals
Role: STUDY_DIRECTOR
Novartis Pharmaceuticals
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
UCSF Benioff Children s Hospital
Oakland, California, United States
Lucile Packard Childrens Hosp
Palo Alto, California, United States
Childrens National Medical Center
Washington D.C., District of Columbia, United States
Childrens Hosp Boston Dept of Heme
Boston, Massachusetts, United States
WA Uni School Of Med
St Louis, Missouri, United States
Cinn Children Hosp Medical Center
Cincinnati, Ohio, United States
Univ Hospital Of Cleveland
Cleveland, Ohio, United States
Cleveland Clinic Foundation
Cleveland, Ohio, United States
Oregon Health Science University
Portland, Oregon, United States
CHOP Abramson Pediatric Resch Ctr
Philadelphia, Pennsylvania, United States
Baylor College Of Medicine
Houston, Texas, United States
U of TX Health Science Ct
Houston, Texas, United States
Childrens Hospital and Regional Medical Center
Seattle, Washington, United States
Novartis Investigative Site
Sydney, New South Wales, Australia
Novartis Investigative Site
Sydney, New South Wales, Australia
Novartis Investigative Site
Brisbane, Queensland, Australia
Novartis Investigative Site
Brussels, , Belgium
Novartis Investigative Site
Bordeaux, , France
Novartis Investigative Site
Bron, , France
Novartis Investigative Site
Caen, , France
Novartis Investigative Site
Dijon, , France
Novartis Investigative Site
Lille, , France
Novartis Investigative Site
Marseille, , France
Novartis Investigative Site
Montpellier, , France
Novartis Investigative Site
Paris, , France
Novartis Investigative Site
Paris, , France
Novartis Investigative Site
Tours, , France
Novartis Investigative Site
Freiburg im Breisgau, Baden-Wurttemberg, Germany
Novartis Investigative Site
Mannheim, Baden-Wurttemberg, Germany
Novartis Investigative Site
Cologne, North Rhine-Westphalia, Germany
Novartis Investigative Site
Leipzig, Saxony, Germany
Novartis Investigative Site
Berlin, , Germany
Novartis Investigative Site
Bologna, BO, Italy
Novartis Investigative Site
Milan, MI, Italy
Novartis Investigative Site
Roma, RM, Italy
Novartis Investigative Site
Roma, RM, Italy
Novartis Investigative Site
Napoli, , Italy
Novartis Investigative Site
Esplugues, Barcelona, Spain
Novartis Investigative Site
A Coruña, , Spain
Novartis Investigative Site
Madrid, , Spain
Novartis Investigative Site
Madrid, , Spain
Countries
Review the countries where the study has at least one active or historical site.
Central Contacts
Reach out to these primary contacts for questions about participation or study logistics.
Facility Contacts
Find local site contact details for specific facilities participating in the trial.
Audrey Hernando
Role: primary
Ramrada Lekwuttikarn
Role: primary
Taylor Brice
Role: primary
Kelsey Deemer
Role: primary
Sally Jones
Role: primary
Kimberly Whittaker
Role: primary
Hayley Sluka
Role: primary
Bea Ferreira Alves
Role: primary
Denae Clohessy
Role: primary
Samantha Santos
Role: primary
Derek Cole
Role: primary
Janaei Phillips
Role: primary
Maya Gopalan
Role: primary
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
2023-504146-60-00
Identifier Type: REGISTRY
Identifier Source: secondary_id
CBYL719P12201
Identifier Type: -
Identifier Source: org_study_id