New drug trial targets root cause of rare 'MCAP' syndrome in kids and adults

NCT ID NCT05577754

Recruiting now Disease control Sponsor: Centre Hospitalier Universitaire Dijon Source: ClinicalTrials.gov ↗

Summary

This study is testing whether a drug called alpelisib can help patients with MCAP syndrome, a rare genetic disorder that causes brain overgrowth, intellectual disability, and other health problems. For the first 6 months, some participants will receive the drug and others a placebo, followed by an open period where all can receive the treatment. The main goal is to see if the drug improves patients' daily living skills, communication, and socialization.

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

Contact

Phone: •••-•••-•••• Email: •••••@•••••

Locations

AP-HP Hôpital Necker-Enfants Malades
RECRUITING

Paris, 75015, France

Contact Phone: •••-•••-•••• Email: •••••@•••••
AP-HP Hôpital Necker-Enfants Malades - CIC
ACTIVE_NOT_RECRUITING

Paris, 75015, France
CHRU Brest
NOT_YET_RECRUITING

Brest, 29200, France

Contact Phone: •••-•••-•••• Email: •••••@•••••
CHRU Nîmes
RECRUITING

Nîmes, 30029, France

Contact Phone: •••-•••-•••• Email: •••••@•••••
CHRU Tours
RECRUITING

Tours, 37044, France

Contact Phone: •••-•••-•••• Email: •••••@•••••
CHU Amiens
RECRUITING

Amiens, 80054 Amiens, France

Contact Phone: •••-•••-•••• Email: •••••@•••••
CHU Dijon Bourgogne - CIC-P
ACTIVE_NOT_RECRUITING

Dijon, 21079, France
CHU Rennes
RECRUITING

Rennes, 35023, France

Contact Phone: •••-•••-••••
CHU d'Angers
RECRUITING

Angers, 49933, France

Contact Phone: •••-•••-•••• Email: •••••@•••••
CHU de Lille
RECRUITING

Lille, 59037, France

Contact Phone: •••-•••-•••• Email: •••••@•••••
Chu Dijon Bourgogne
RECRUITING

Dijon, 21000, France

Contact Phone: •••-•••-•••• Email: •••••@•••••
Chu Estaing
RECRUITING

Clermont-Ferrand, 63003, France

Contact Phone: •••-•••-•••• Email: •••••@•••••
HCL - Groupement Hospitalier Est Hôpital Femme-Mère-Enfant
RECRUITING

Bron, 69677, France

Contact Phone: •••-•••-•••• Email: •••••@•••••

Conditions

Explore the condition pages connected to this study.

MEGALENCEPHALY-CAPILLARY MALFORMATION POLYMICROGYRIA SYNDROME (MCAP)