What this Trial Is About

Researchers are evaluating the safety and effectiveness of different doses of sirolimus for treating cystic lymphatic malformations, which are caused by abnormal lymphatic vessel development leading to swelling, pain, and organ issues. This study focuses on children and adolescents up to 18 years old and aims to see if lower doses of sirolimus can improve patient outcomes while reducing side effects like oral mucositis, a common complication of high-dose treatment. Participants will receive varying doses of sirolimus (rapamycin), an immunosuppressant drug usually used to prevent organ transplant rejection but now being studied for lymphatic malformations. The study compares different concentration gradients of sirolimus to find the best balance between effectiveness and safety. Treatment details include monitoring drug plasma concentrations to optimize therapy and minimize complications. During the study, patients will be closely followed for 12 months to assess their response to treatment, with the main measure being the proportion of patients showing an objective response at the end of this period. Researchers will conduct clinical assessments, imaging, and laboratory tests to monitor disease status, drug effects, and safety. The study also includes screening for eligibility, ongoing safety checks, and follow-up visits to ensure comprehensive evaluation of sirolimus treatment in this population.

Different Doses of Sirolimus for the Treatment of Cystic Lymphatic Malformations