New drug trial aims to shrink painful lymphatic growths in kids and adults

NCT ID NCT05948943

Recruiting now Disease control Sponsor: Novartis Pharmaceuticals Source: ClinicalTrials.gov ↗

First seen Jun 24, 2026 · Last updated Jul 15, 2026 · Updated 4 times

Summary

This study tests alpelisib, a drug that targets a specific gene mutation (PIK3CA), in people with lymphatic malformations—abnormal growths of lymph vessels. The trial includes children and adults and compares alpelisib to a placebo to see if it can shrink the growths and ease symptoms. Researchers are recruiting 232 participants across multiple sites.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: alpelisib (a targeted drug that blocks a specific protein called PI3K, which is overactive in these growths)
What this could lead to: If successful, this could provide a new treatment option to shrink lymphatic malformations and improve symptoms for people with PIK3CA mutations.
What could go wrong: This is an early-to-mid-stage trial, so results are not yet proven. The drug may cause side effects, and not all participants may respond.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

cystic hygroma lymphangioma Lymphatic Abnormalities lymphatic malformation PIK3CA-related overgrowth spectrum

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

LYMPHATIC MALFORMATIONS

Contacts and locations

Locations

Baylor College Of Medicine
RECRUITING

Houston, Texas, 77030, United States
CHOP Abramson Pediatric Resch Ctr
RECRUITING

Philadelphia, Pennsylvania, 19104, United States
Childrens Hosp Boston Dept of Heme
RECRUITING

Boston, Massachusetts, 02115, United States
Childrens Hosp Pittsburgh UPMC
RECRUITING

Pittsburgh, Pennsylvania, 15224, United States
Childrens Hospital and Regional Medical Center
RECRUITING

Seattle, Washington, 98105, United States
Childrens Hospital of Orange County
RECRUITING

Orange, California, 92868, United States
Childrens National Medical Center
RECRUITING

Washington D.C., District of Columbia, 20010-2970, United States
Cinn Children Hosp Medical Center
RECRUITING

Cincinnati, Ohio, 45229-3039, United States
Cleveland Clinic Foundation
RECRUITING

Cleveland, Ohio, 44195, United States
Lucile Packard Childrens Hosp
RECRUITING

Palo Alto, California, 94304, United States
Nationwide Children s Hospital
RECRUITING

Columbus, Ohio, 43205, United States
Nemours Childrens Clinic
RECRUITING

Jacksonville, Florida, 32207, United States
Novartis Investigative Site
RECRUITING

CABA, Buenos Aires, C1181ACH, Argentina
Novartis Investigative Site
RECRUITING

CABA, Buenos Aires, C1425BEA, Argentina
Novartis Investigative Site
RECRUITING

Capital Federal, C1023AAB, Argentina
Novartis Investigative Site
RECRUITING

Sydney, New South Wales, 2010, Australia
Novartis Investigative Site
RECRUITING

Sydney, New South Wales, 2031, Australia
Novartis Investigative Site
RECRUITING

Brisbane, Queensland, 4101, Australia
Novartis Investigative Site
RECRUITING

Brussels, 1200, Belgium
Novartis Investigative Site
RECRUITING

Brno, 625 00, Czechia
Novartis Investigative Site
RECRUITING

Angers, 49933, France
Novartis Investigative Site
RECRUITING

Bordeaux, 33076, France
Novartis Investigative Site
RECRUITING

Bron, 69677, France
Novartis Investigative Site
RECRUITING

Caen, 14033, France
Novartis Investigative Site
RECRUITING

Dijon, 21000, France
Novartis Investigative Site
RECRUITING

Lille, 59000, France
Novartis Investigative Site
RECRUITING

Marseille, 13885, France
Novartis Investigative Site
RECRUITING

Montpellier, 34295, France
Novartis Investigative Site
RECRUITING

Paris, 75010, France
Novartis Investigative Site
RECRUITING

Paris, 75015, France
Novartis Investigative Site
RECRUITING

Toulouse, 31054, France
Novartis Investigative Site
RECRUITING

Tours, 37044, France
Novartis Investigative Site
RECRUITING

Freiburg im Breisgau, Baden-Wurttemberg, 79106, Germany
Novartis Investigative Site
WITHDRAWN

Mannheim, Baden-Wurttemberg, 68305, Germany
Novartis Investigative Site
RECRUITING

Cologne, North Rhine-Westphalia, 50937, Germany
Novartis Investigative Site
RECRUITING

Leipzig, Saxony, 04103, Germany
Novartis Investigative Site
RECRUITING

Berlin, 13353, Germany
Novartis Investigative Site
RECRUITING

Ulm, 89081, Germany
Novartis Investigative Site
RECRUITING

Bologna, BO, 40138, Italy
Novartis Investigative Site
RECRUITING

Milan, MI, 20122, Italy
Novartis Investigative Site
RECRUITING

Roma, RM, 00165, Italy
Novartis Investigative Site
RECRUITING

Roma, RM, 00168, Italy
Novartis Investigative Site
RECRUITING

Torino, TO, 10126, Italy
Novartis Investigative Site
RECRUITING

Naples, 80122, Italy
Novartis Investigative Site
RECRUITING

Nijmegen, Gelderland, 6500HB, Netherlands
Novartis Investigative Site
RECRUITING

Rotterdam, South Holland, 3015 GD, Netherlands
Novartis Investigative Site
RECRUITING

Palma, Balearic Islands, 07120, Spain
Novartis Investigative Site
RECRUITING

Esplugues, Barcelona, 08950, Spain
Novartis Investigative Site
RECRUITING

L'Hospitalet de Llobregat, Barcelona, 08907, Spain
Novartis Investigative Site
RECRUITING

A Coruña, 15006, Spain
Novartis Investigative Site
RECRUITING

Barcelona, 08035, Spain
Novartis Investigative Site
RECRUITING

Madrid, 28009, Spain
Novartis Investigative Site
RECRUITING

Madrid, 28046, Spain
Novartis Investigative Site
RECRUITING

Lausanne, 1011, Switzerland
Oregon Health Science University
RECRUITING

Portland, Oregon, 97239, United States
U of TX Health Science Ct
RECRUITING

Houston, Texas, 77030, United States
UCSF Benioff Children s Hospital
RECRUITING

Oakland, California, 94609, United States
UNC Chapel Hill
RECRUITING

Chapel Hill, North Carolina, 27599, United States
Univ Hospital Of Cleveland
RECRUITING

Cleveland, Ohio, 44106, United States
WA Uni School Of Med
RECRUITING

St Louis, Missouri, 63110, United States

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