Could a cancer drug help kids with rare brain disorder?
NCT ID NCT05577754
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This phase 2 trial tests alpelisib, a drug originally developed for cancer, in 20 people aged 2 to 40 with MCAP syndrome—a rare condition causing an enlarged brain, blood vessel issues, and developmental delays. Participants receive either alpelisib or a placebo for 6 months, then all get the drug for up to 24 months. The goal is to see if it improves daily living skills like communication and socialization.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
alpelisib (BYL719)
What this could lead to
If it works, this could point toward a treatment that improves daily living skills and brain function in people with MCAP syndrome.
What could go wrong
This is a small, early-phase trial with only 20 participants, so results may not apply to everyone. The drug may cause side effects or fail to show benefit over placebo.
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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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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AP-HP Hôpital Necker-Enfants Malades
RECRUITINGParis, 75015, France
Contact Phone: •••-•••-•••• Email: •••••@•••••
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AP-HP Hôpital Necker-Enfants Malades - CIC
ACTIVE_NOT_RECRUITINGParis, 75015, France
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CHRU Brest
NOT_YET_RECRUITINGBrest, 29200, France
Contact Phone: •••-•••-•••• Email: •••••@•••••
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CHRU Nîmes
RECRUITINGNîmes, 30029, France
Contact Phone: •••-•••-•••• Email: •••••@•••••
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CHRU Tours
RECRUITINGTours, 37044, France
Contact Phone: •••-•••-•••• Email: •••••@•••••
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CHU Amiens
RECRUITINGAmiens, 80054 Amiens, France
Contact Phone: •••-•••-•••• Email: •••••@•••••
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CHU Dijon Bourgogne - CIC-P
ACTIVE_NOT_RECRUITINGDijon, 21079, France
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CHU Rennes
RECRUITINGRennes, 35023, France
Contact Phone: •••-•••-••••
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CHU d'Angers
RECRUITINGAngers, 49933, France
Contact Phone: •••-•••-•••• Email: •••••@•••••
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CHU de Lille
RECRUITINGLille, 59037, France
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Chu Dijon Bourgogne
RECRUITINGDijon, 21000, France
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Chu Estaing
RECRUITINGClermont-Ferrand, 63003, France
Contact Phone: •••-•••-•••• Email: •••••@•••••
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HCL - Groupement Hospitalier Est Hôpital Femme-Mère-Enfant
RECRUITINGBron, 69677, France
Contact Phone: •••-•••-•••• Email: •••••@•••••