New drug targets rare growths in kids and adults

NCT ID NCT05948943

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

First seen Nov 01, 2025 · Last updated May 22, 2026 · Updated 29 times

Summary

This study tests whether alpelisib can shrink lymphatic malformations (abnormal fluid-filled growths) in people with a specific PIK3CA gene mutation. About 232 children and adults will receive either alpelisib or a placebo. The main goal is to see if the growths shrink by at least 20% on MRI scans and if symptoms improve.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

Get updates

Get notified about this study

Vår säkerhetsrekommendation!

Genom att skicka in godkänner du våra Användarvillkor

Contacts and locations

Show contact details

Study contacts

Contact

Phone: •••-•••-•••• Email: •••••@•••••
Contact

Phone: •••-•••-•••• Email: •••••@•••••

Locations

Baylor College Of Medicine
RECRUITING

Houston, Texas, 77030, United States

Contact Phone: •••-•••-•••• Email: •••••@•••••

Contact
CHOP Abramson Pediatric Resch Ctr
RECRUITING

Philadelphia, Pennsylvania, 19104, United States

Contact

Contact Email: •••••@•••••
Childrens Hosp Boston Dept of Heme
RECRUITING

Boston, Massachusetts, 02115, United States

Contact Phone: •••-•••-•••• Email: •••••@•••••

Contact
Childrens Hosp Pittsburgh UPMC
RECRUITING

Pittsburgh, Pennsylvania, 15224, United States

Contact Phone: •••-•••-•••• Email: •••••@•••••

Contact
Childrens Hospital and Regional Medical Center
RECRUITING

Seattle, Washington, 98105, United States

Contact Phone: •••-•••-••••

Contact
Childrens National Medical Center
RECRUITING

Washington D.C., District of Columbia, 20010-2970, United States

Contact Email: •••••@•••••

Contact
Cinn Children Hosp Medical Center
RECRUITING

Cincinnati, Ohio, 45206, United States

Contact

Contact Phone: •••-•••-•••• Email: •••••@•••••
Cleveland Clinic Foundation
RECRUITING

Cleveland, Ohio, 44195, United States

Contact Email: •••••@•••••

Contact
Lucile Packard Childrens Hosp
RECRUITING

Palo Alto, California, 94304, United States

Contact

Contact Email: •••••@•••••
Nationwide Children s Hospital
RECRUITING

Columbus, Ohio, 43205, United States

Contact

Contact Email: •••••@•••••
Nemours Childrens Clinic
RECRUITING

Jacksonville, Florida, 32207, United States

Contact

Contact Phone: •••-•••-•••• Email: •••••@•••••
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

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

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

Contact Email: •••••@•••••

Contact
U of TX Health Science Ct
RECRUITING

Houston, Texas, 77030, United States

Contact

Contact Phone: •••-•••-•••• Email: •••••@•••••
UCSF Benioff Children s Hospital
RECRUITING

Oakland, California, 94609, United States

Contact

Contact Phone: •••-•••-•••• Email: •••••@•••••
Univ Hospital Of Cleveland
RECRUITING

Cleveland, Ohio, 44106, United States

Contact Phone: •••-•••-•••• Email: •••••@•••••

Contact
WA Uni School Of Med
RECRUITING

St Louis, Missouri, 63110, United States

Contact Email: •••••@•••••

Contact

Conditions

Explore the condition pages connected to this study.

LYMPHATIC MALFORMATIONS LYMPHATIC MALFORMATIONS