New hope for rare brain disorder: drug trial seeks to improve lives
NCT ID NCT05577754
Summary
This study is testing whether the drug alpelisib can help children and adults with MCAP syndrome, a rare genetic disorder that affects brain growth and development. For the first six months, some participants will receive the drug while others get a placebo, followed by an open period where everyone can receive the treatment. The main goal is to see if the drug improves patients' daily living skills, communication, and socialization.
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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.