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
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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.
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.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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CHU Hôtel Dieu
Nantes, 44093, France
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CHU La Réunion - Site GHSR
Saint-Pierre, 97448, France
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CHU d'Angers
Angers, 49933, France
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CHU de Bordeaux - GH Pellegrin
Bordeaux, 33076, France
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CHU de Nancy
Vandœuvre-lès-Nancy, 54511, France
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CHU de Nancy - Hôpital de Brabois
Vandœuvre-lès-Nancy, 54511, France
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CHU de Nice - Hôpital l'Archet 2
Nice, 06202, France
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CHU de Saint-Etienne Hôpital Nord
Saint-Priest-en-Jarez, 42270, France
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Chu Dijon Bourgogne
Dijon, 21000, France
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HCL - Hôpital femme-mère-enfant
Lyon, France
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Hôpital Arnaud de Villeneuve
Montpellier, 34090, France
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Hôpital Jeanne de Flandre
Lille, 59037, France
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Hôpital Larrey
Toulouse, 31059, France
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Hôpital Necker-Enfants malades
Paris, 75743, France
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Hôpital Raymond Poincaré (AP-HP)
Garches, 92380, France
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Hôpital Saint Eloi
Montpellier, 34295, France
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Hôpital Sud
Rennes, 35203, France
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Hôpital Trousseau
Tours, 37044, France