We are studying whether alpelisib can improve adaptive behavior and overall health in children and adults with MCAP. The trial will also assess the safety and effects of the treatment on brain and spinal cord health.
Health conditions and diseases that the clinical trial is designed to study and treat.
PIK3CA Related Overgrowth Spectrum is a group of rare genetic disorders caused by mutations in the PIK3CA gene, leading to abnormal growth of tissues and organs, which can result in various symptoms depending on the areas affected.
Megalencephaly-Capillary Malformation-Polymicrogyria Syndrome is a rare genetic condition characterized by an enlarged brain (megalencephaly), capillary malformations, and polymicrogyria, which can lead to developmental delays and neurological issues.
These questions help us understand your situation so we can let the trial team know whether you might be a potential match. This is not a medical evaluation and is not part of the official screening, the study doctor will make the final decision..
Experimental substances that are being investigated in this clinical trial. These are not yet approved for general use.
Locations and medical facilities where this clinical trial is currently recruiting or conducting research. Select the locations where you would like to participate.
Disclaimer: Parts of this content have been automatically extracted from the EU Clinical Trials registry. While we strive for accuracy, please always contact the trial site or sponsor directly for correct and official information before making any decisions about participation. View on EU Clinical Trials.