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Recruiting Phase 2/3

Alpelisib in Pediatric and Adult Patients With Lymphatic Malformations Associated With a PIK3CA Mutation.

NCT05948943 · tracked via the Priya Life Science Italy tracker
Phase
Phase 2/3
Started
2023-11-24
Last updated
2026-09-18

Condition(s) studied

Lymphatic Malformations

Investigational drug(s) / intervention(s)

Alpelisib →Placebo

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

Sex
ALL
Min age
0 Years
Max age
100 Years
Healthy volunteers
No
Key inclusion criteria: 1. Signed informed consent and assent (when applicable) from the participant, parent, legal authorized representative or guardian. 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. Key exclusion criteria: 1. Participant has a physician-confirmed and documented diagnosis of PROS at the time of informed consent. 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. Other inclusion/exclusion criteria may apply

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)

FacilityCityRegionStatus
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.

Official registry record

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