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Drug shows promise for rare overgrowth disorder in long-term study

NCT ID NCT04980833

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

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study looks at the long-term safety and effectiveness of the drug alpelisib in people with PIK3CA-Related Overgrowth Spectrum (PROS), a rare condition causing abnormal tissue growth. It includes 41 children and adults who previously took alpelisib in an earlier trial. Researchers will track side effects and how well the drug controls overgrowth over time.

What this could mean

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

Active substance
alpelisib
What this could lead to
If successful, this could confirm alpelisib as a long-term treatment option to control overgrowth in PROS, improving quality of life.
What could go wrong
This is a small, phase 2 study with no placebo group, so results may not be definitive. Side effects like high blood sugar or rash are possible.

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.

PIK3CA-related overgrowth spectrum

As listed by the trial registrant

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

PIK3CA-RELATED OVERGROWTH SPECTRUM (PROS)

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

More trials for these conditions

Other studies related to the condition(s) this trial covers.