Could a cancer drug tame rare overgrowth disease?

NCT ID NCT03290092

First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 2 times

Summary

This early-phase trial tested taselisib, a drug that blocks a faulty protein causing excessive growth in people with PIK3CA-related overgrowth. Nineteen participants took the drug daily for four weeks to see if it was safe and tolerable. The study was stopped early, so we have limited information, but it may help design future trials.

What this could mean

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

Active substance

Taselisib (a drug that blocks a protein called PI3K, which is overactive in this condition)

What this could lead to

If it works, this could point toward a drug treatment for PIK3CA-related overgrowth, potentially reducing the need for repeated surgeries.

What could go wrong

This trial was terminated early with only 19 participants, so results are very limited. The drug may cause side effects like high blood sugar or diarrhea, and it is not yet clear if it helps symptoms.

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.

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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.

Contacts and locations

Locations

  • CHU Hôtel Dieu

    Nantes, 44093, France

  • CHU La Réunion - Site GHSR

    Saint-Pierre, 97448, France

  • CHU d'Angers

    Angers, 49933, France

  • CHU de Bordeaux - GH Pellegrin

    Bordeaux, 33076, France

  • CHU de Nancy

    Vandœuvre-lès-Nancy, 54511, France

  • CHU de Nancy - Hôpital de Brabois

    Vandœuvre-lès-Nancy, 54511, France

  • CHU de Nice - Hôpital l'Archet 2

    Nice, 06202, France

  • CHU de Saint-Etienne Hôpital Nord

    Saint-Priest-en-Jarez, 42270, France

  • Chu Dijon Bourgogne

    Dijon, 21000, France

  • HCL - Hôpital femme-mère-enfant

    Lyon, France

  • Hôpital Arnaud de Villeneuve

    Montpellier, 34090, France

  • Hôpital Jeanne de Flandre

    Lille, 59037, France

  • Hôpital Larrey

    Toulouse, 31059, France

  • Hôpital Necker-Enfants malades

    Paris, 75743, France

  • Hôpital Raymond Poincaré (AP-HP)

    Garches, 92380, France

  • Hôpital Saint Eloi

    Montpellier, 34295, France

  • Hôpital Sud

    Rennes, 35203, France

  • Hôpital Trousseau

    Tours, 37044, France