Translational medicine and Targeted therapies unit, Hôpital Necker Enfants Malades
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 0 Trials 
4 Trials

   

 
Trial + Data / EventsStatusPhNRegionInterventionsSponsorConditionsPrimary complStudy compl
Canaud, Guillaume
SESAM, NCT05577754: Assessment of the Efficacy and Safety of Alpelisib (BYL719) in Pediatric and Adult Patients with Megalencephaly-CApillary Malformation Polymicrogyria Syndrome (MCAP)

Recruiting
2
20
Europe
Alpelisib (BYL719), Matching placebo, Optional lumbar puncture + blood sample
Centre Hospitalier Universitaire Dijon, Novartis Pharmaceuticals
Megalencephaly-capillary Malformation Polymicrogyria Syndrome (MCAP)
03/27
03/27
COSY, NCT05563831: National Evaluation of Patients With PIK3CA-Related Overgrowth Spectrum (PROS)

Recruiting
N/A
2500
Europe
national registry
Institut National de la Santé Et de la Recherche Médicale, France
PIK3CA-related Overgrowth Spectrum, CLOVES Syndrome, Klippel Trenaunay Syndrome, Megalencephaly, MCAP, Macrodactyly, Vascular Malformations, Lymphatic Malformation, Venous Malformation
12/27
12/28
Bahi-Buisson, Nadia
CANDID, NCT05373719: Observational Study in Patients With Cyclin-dependent Kinase-like 5 Deficiency Disorder

Active, not recruiting
N/A
112
Europe, Canada, US, RoW
No intervention; observational
Loulou Foundation
Cyclin-Dependent Kinase-Like 5 Deficiency Disorder
01/27
01/27
COSY, NCT05563831: National Evaluation of Patients With PIK3CA-Related Overgrowth Spectrum (PROS)

Recruiting
N/A
2500
Europe
national registry
Institut National de la Santé Et de la Recherche Médicale, France
PIK3CA-related Overgrowth Spectrum, CLOVES Syndrome, Klippel Trenaunay Syndrome, Megalencephaly, MCAP, Macrodactyly, Vascular Malformations, Lymphatic Malformation, Venous Malformation
12/27
12/28

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