At a glance
ClinicalIndex Comparison Record- ✓Age 2-40 years at informed consent
- ✓Diagnosis of MCAP with neurodevelopmental disorder presentation
- ✓Documented postzygotic or constitutional PIK3CA mutation
- ✓Able to swallow study drug in appropriate formulation for age
- ✕Prior treatment with alpelisib
- ✕GI impairment significantly affecting drug absorption
- ✕Uncontrolled diabetes mellitus (Type I or II)
- ✕Hypersensitivity to PI3K inhibitors or alpelisib excipients
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
A Phase II Double-blind Multi-center, Placebo-controlled Trial, to Assess the Efficacy and Safety of Alpelisib (BYL719) in Pediatric and Adult Patients With Megalencephaly-CApillary Malformation Polymicrogyria Syndrome (MCAP)
In Brief
A Phase 2 clinical trial evaluating Alpelisib (BYL719), Matching placebo, and 1 other intervention for Megalencephaly-capillary Malformation Polymicrogyria Syndrome (MCAP). Currently recruiting, targeting 20 participants across 13 sites.
Detailed 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, to assess the efficacy and safety of alpelisib (BYL719) in pediatric and adult patients with Megalencephaly-CApillary malformation Polymicrogyria syndrome (MCAP)
Study Details
Timeline
Interventions
Administration during open label and double-blind period of group B: alpelisib will be taken once a day each day over 24 mois During open label period of group A: alpelisib will be taken once a day each day over 24 months
During double-blind period of group A: matching placebo will be taken once a day each day over 6 months
Between 6 and 24 months of treatment with Alpelisib