New hope for rare brain disorder: drug trial targets MCAP symptoms
NCT ID NCT05577754
First seen Jan 07, 2026 · Last updated May 09, 2026 · Updated 18 times
Summary
This study tests a drug called alpelisib in 20 people aged 2 to 40 with MCAP syndrome, a rare condition causing an enlarged brain and developmental delays. For the first 6 months, some get the drug and some get a placebo to see if it improves daily living skills like communication and socialization. After that, everyone can receive the drug. The goal is to manage symptoms, not cure the condition.
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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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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: •••••@•••••
Conditions
Explore the condition pages connected to this study.