Study Comparing Low and High Doses of Sirolimus for Treating Cystic Lymphatic Malformations in Children and Adolescents

What this Trial Is About

This research aims to compare how well different concentration levels of sirolimus work and how safe they are for treating cystic lymphatic malformation, a condition caused by abnormal growth and structure of lymphatic vessels. This disease can cause fluid buildup, swelling, pain, and organ problems in areas like the skin, soft tissues, and internal organs. The study explores whether adjusting the sirolimus dose to maintain lower blood levels might improve patient outcomes and reduce side effects such as oral mucositis. Participants will be randomly assigned to two groups one group will receive sirolimus adjusted to keep a blood concentration between 5-8 ngml, and the other group will have a higher target range of 10-15 ngml. Both groups will receive treatment for one year. The study is single-blind and phase 23, meaning patients will not know which dose they receive, and the research will evaluate both doses over this period. During the study, patients will be monitored for treatment response, quality of life, and any side effects. Researchers will measure how many patients show an objective response at 6 and 12 months, track lesion changes, and assess disease-related aftereffects. Safety and adverse events will also be recorded throughout the 12-month treatment. The trial includes children aged 1 to 18 years and involves regular clinical assessments and laboratory tests to evaluate the impact and safety of different sirolimus doses.

Different Doses of Sirolimus for the Treatment of Cystic Lymphatic Malformations