Alpelisib for pediatric and adult PROS
Treatments studied
Part of Genetic & congenital clinical trials.
This trial tests a drug called alpelisib for children and adults with a rare overgrowth condition called PROS that is caused by a PIK3CA gene change. It aims to see if the drug can safely shrink overgrowth and improve symptoms.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must be at least 2 years old.
- You must have a confirmed diagnosis of PROS (a rare condition causing abnormal growth of body parts) that is getting worse.
- A genetic test must show a change in the PIK3CA gene in a sample from the overgrown area.
- You should have at least one overgrowth that is at least 2 cm wide and causes symptoms or limits daily activities.
- You cannot have only isolated features like an enlarged finger, a large head, or a skin mole without other PROS-related growths.
- You must not have taken other PI3K inhibitors (a class of medicines) for more than 2 weeks, or have had major surgery or radiation for PROS recently.
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.
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This study allows people who are already taking alpelisib in another Novartis trial to continue receiving it long-term. The goal is to keep giving the drug to those who are benefiting from it, and to track safety over a longer period.
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.
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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.
This study investigates Proteus syndrome, a rare condition that causes abnormal overgrowth of body parts, and related disorders. Researchers want to understand how these conditions develop, progress over time, and affect people of different ages—including how they may change into adulthood.
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