New hope for rare overgrowth disorder: alpelisib trial targets root cause

NCT ID NCT06997588

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

First seen Nov 01, 2025 · Last updated May 06, 2026 · Updated 32 times

Summary

This study tests an oral drug, alpelisib, in children and adults with PIK3CA-related overgrowth spectrum (PROS), a rare condition causing abnormal tissue growth. The goal is to shrink overgrown tissue and improve symptoms. About 104 participants aged 2 and older will take the drug daily, and doctors will monitor changes using MRI scans. This is a Phase 2 trial, meaning it checks both effectiveness and safety.

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Contacts and locations

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Study contacts

Contact

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

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

Locations

Cinn Children Hosp Medical Center
RECRUITING

Cincinnati, Ohio, 45206, United States

Contact

Contact Phone: •••-•••-•••• Email: •••••@•••••
Novartis Investigative Site
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Sydney, New South Wales, 2010, Australia
Novartis Investigative Site
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Sydney, New South Wales, 2031, Australia
Novartis Investigative Site
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North Adelaide, South Australia, 5066, Australia
Novartis Investigative Site
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Salzburg, 5020, Austria
Novartis Investigative Site
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Vienna, A 1090, Austria
Novartis Investigative Site
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Ghent, 9000, Belgium
Novartis Investigative Site
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Angers, 49933, France
Novartis Investigative Site
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Bron, 69677, France
Novartis Investigative Site
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Dijon, 21000, France
Novartis Investigative Site
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Montpellier, 34295, France
Novartis Investigative Site
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Paris, 75015, France
Novartis Investigative Site
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Toulouse, 31400, France
Novartis Investigative Site
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Tours, 37044, France
Novartis Investigative Site
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Freiburg im Breisgau, Baden-Wurttemberg, 79106, Germany
Novartis Investigative Site
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Düsseldorf, North Rhine-Westphalia, 40225, Germany
Novartis Investigative Site
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Leipzig, Saxony, 04103, Germany
Novartis Investigative Site
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Halle, Saxony-Anhalt, 06120, Germany
Novartis Investigative Site
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Berlin, 13353, Germany
Novartis Investigative Site
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Stuttgart, 70374, Germany
Novartis Investigative Site
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Roma, RM, 00168, Italy
Novartis Investigative Site
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Torino, TO, 10126, Italy
Novartis Investigative Site
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Trieste, TS, 34137, Italy
Novartis Investigative Site
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Esplugues, Barcelona, 08950, Spain
Novartis Investigative Site
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A Coruña, 15006, Spain
Novartis Investigative Site
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Barcelona, 08035, Spain
Novartis Investigative Site
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Madrid, 28046, Spain
Novartis Investigative Site
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Lausanne, 1011, Switzerland
Novartis Investigative Site
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Liverpool, L12 2AP, United Kingdom
Washington University
RECRUITING

St Louis, Missouri, 63110, United States

Contact

Contact Email: •••••@•••••

Conditions

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