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Alpelisib in Pediatric and Adult MCAP Syndrome (Phase II)
2022-500197-34-01Phase 2Authorised
- Sponsor
- Centre Hospitalier Universitaire Dijon Bourgogne
- Start
- Sep 2022
- Modality
- Small Molecules
- Countries
- 1
01
Overview
Phase 2, randomised, double-blind trial in Megalencephaly-Capillary Malformation Syndrome (MCAP) comparing Alpelisib with Placebo (2 arms). Primary endpoint: Primary efficacy: improvement in adaptive behavior at 24 months. Planned start Sep 2022; status: authorised.
02
Drugs
alpelisib
03
Conditions
Megalencephaly-Capillary Malformation Syndrome (MCAP)
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