Could a drug stop uncontrolled body growth? small study tests sirolimus
NCT ID NCT02443818
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This pilot study tested the drug sirolimus in 16 people aged 3 to 65 who have excessive growth in parts of their body due to a change in the PIK3CA gene. The goal was to see if sirolimus can stop or slow that overgrowth. Researchers used MRI scans and other measures to track changes, and also looked at quality of life. This small study helps prepare for a larger international trial.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- Sirolimus (a drug that slows cell growth)
- What this could lead to
- If it works, this could point toward a treatment that stops or slows excessive growth in people with PIK3CA-related overgrowth disorders.
- What could go wrong
- This is a very small pilot study with only 16 people, so results may not apply to everyone. The drug may not stop growth or could cause side effects.
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.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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CHRU de Montpellier - Hôpital Saint-Eloi
Montpellier, 34295, France
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CHRU de Tours
Tours, 37044, France
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CHU Paris - Necker
Paris, 75015, 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 DIJON
Dijon, 21079, France
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HCL Lyon - CH Lyon Sud
Pierre-Bénite, 69495, France
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Hopitaux de Brabois Chu Nancy
Nancy, 54511, France
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Hôpital Jeanne de Flandre
Lille, 59037, France
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Hôpital mère-enfant de Nantes
Nantes, 44093, France
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Pôle Cardiovasculaire et Métabolique - Hôpital Larrey
Toulouse, 31059, France