Overgrowth syndrome

This study provides continued access to alpelisib for people who are already benefiting from it in a previous Novartis trial. It focuses on tracking long-term safety, including any side effects, in participants with PIK3CA-related overgrowth spectrum or certain breast cancers. Pa…

This study tests whether everolimus, a drug already approved for other conditions, can safely improve social abilities in people with PTEN Hamartoma Tumor Syndrome (PHTS). About 60 adults and children with PHTS and social difficulties will take either everolimus or a placebo for …

This early-stage trial tests a new treatment for children with a rare, aggressive brain tumor called ATRT that has come back or not responded to standard therapy. The treatment uses the patient's own immune cells, modified in a lab to better recognize and attack the tumor, plus a…

This Phase 3 trial tests an oral drug called KP-001 in 150 people aged 2 and older with venous malformations, lymphatic malformations, or KTS/CLOVES syndrome. Participants receive either KP-001 or a placebo for 24 weeks, then everyone gets the drug for another 28 weeks. The goal …

This study tests whether myofascial release therapy, a type of hands-on treatment that loosens tight connective tissue, can improve body posture and facial symmetry in healthy adults. Sixty participants will be randomly assigned to receive the therapy twice a week for six weeks o…

This study will look at data from 918 adults who had planned heart surgery to find out why some develop atrial fibrillation, a common and serious irregular heartbeat. The researchers will also examine whether newer anesthesia methods, like a nerve block, might help protect agains…

This study aims to better understand a condition called multilocus imprinting disorder (MLID), where multiple genes are affected by abnormal chemical marks. Researchers will test a new technique to detect these marks in 96 people, including those with known imprinting disorders a…