Overview

Assessment of the Efficacy and Safety of Alpelisib (BYL719) in Pediatric and Adult Patients With Megalencephaly-CApillary Malformation Polymicrogyria Syndrome (MCAP)

Status:
Not yet recruiting
Trial end date:
2026-08-01
Target enrollment:
Participant gender:
Summary
This study is a two periods multi-center Phase II trial, with a 6 months double-blind, placebo-controlled period followed by open label period. It will involve 16 participants with MCAP syndrome. Assessment will be performed at baseline and after 24 months of treatment and will include MRI, biological and clinical results, attention, language, and Intellectual Quotient (IQ) assessment. Appropriate neuropsychological tests will be used according to the age of patients and their level of cognitive impairment.
Phase:
Phase 2
Details
Lead Sponsor:
Centre Hospitalier Universitaire Dijon