Testing alpelisib for overgrowth syndrome in children and adults
Treatments studied
Part of Genetic & congenital clinical trials.
This trial is testing a drug called alpelisib for people with PROS (a rare condition that causes parts of the body to grow too much). The drug targets a specific gene change (PIK3CA mutation) that is often the cause. If you have this condition and it's causing serious symptoms, this study might help shrink or slow the overgrowth, improving your quality of life.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must be 2 years old or older.
- You must have a confirmed diagnosis of PROS (a condition causing overgrowth of body parts), with symptoms that are getting worse and affecting your daily life.
- A genetic test must have found a change (mutation) in your PIK3CA gene.
- You must have at least one overgrown area (lesion) that is at least 2 centimeters (about the size of a grape) across.
- You must not have had treatment with alpelisib or similar drugs for more than 2 weeks before the study.
- You must not have had major surgery to reduce overgrowth in the last 3 months.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
Similar studies
Other trials that look related to this one.
This trial tests a new medicine, RLY-2608, that targets the specific genetic change (PIK3CA mutation) causing overgrowth conditions. It aims to see if the drug can slow or stop the growth of affected tissues.
This study lets people who are already taking alpelisib in another Novartis trial keep getting it after that trial ends. It checks long-term safety and continued benefit.
This trial tests a new drug called CYH33 for people with conditions caused by a specific gene mutation (PIK3CA) that leads to abnormal tissue growth or blood vessel problems. It aims to see if the drug is safe and can help shrink or control these growths.
This program provides access to an experimental medicine called alpelisib (also known as BYL719) for people with serious conditions who have no other treatment options and cannot participate in clinical trials. If a licensed doctor requests it, and you meet the criteria, you may be able to receive this medicine through a managed access program.
This trial tests alpelisib (a targeted medicine) for people who have lymphatic malformations (LyM) that cause symptoms and have a PIK3CA mutation. It may help shrink or control these lesions when other procedures are delayed until after the first 24 weeks.
This Phase 2 study tests alpelisib in children and adults with MCAP, a condition caused by a specific PIK3CA gene mutation. The goal is to see if the treatment is effective and safe, while carefully monitoring blood sugar and organ function.
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