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Rare overgrowth disorder drug tested for long-term safety

NCT ID NCT04980833

Ongoing Disease control Sponsor: Novartis Pharmaceuticals Source: ClinicalTrials.gov ↗

First seen Sep 30, 2025 · Last updated May 10, 2026 · Updated 32 times

Summary

This study looks at the long-term safety and effectiveness of the drug alpelisib in children and adults with PIK3CA-related overgrowth spectrum (PROS), a rare condition that causes abnormal growth. All 41 participants had already taken alpelisib in a previous study. Researchers will track side effects and how well the drug controls the condition over time.

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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.

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Contacts and locations

Locations

  • Boston Childrens Hospital

    Boston, Massachusetts, 02215, United States

  • Novartis Investigative Site

    Dijon, 21000, France

  • Novartis Investigative Site

    Montpellier, 34295, France

  • Novartis Investigative Site

    Paris, 75015, France

  • Novartis Investigative Site

    Dublin, 12, Ireland

  • Novartis Investigative Site

    Madrid, 28046, Spain

Conditions

Explore the condition pages connected to this study.

PIK3CA-RELATED OVERGROWTH SPECTRUM (PROS)