Weekly Sirolimus Treatment for Venous and Lymphatic Malformations in Patients Aged 2 Years and Older

What this Trial Is About

Researchers are evaluating the use of sirolimus, an oral medication, for treating venous and lymphatic malformations. Current treatment options are limited, and while sirolimus taken once or twice daily has shown benefit, it can cause serious side effects such as neutropenia, oral ulcers, and lab abnormalities. This study aims to determine if giving sirolimus once weekly will be effective and better tolerated for these conditions. It is a Phase 2 clinical trial sponsored by the Medical University of South Carolina. Participants will receive weekly doses of sirolimus 1.5-2 mgm2 for six months. After this initial period, there is an option to continue the medication. The study focuses on monitoring the size of the malformations through photographs and clinical assessments to evaluate treatment effect. It also tracks side effects monthly and measures quality of life changes using questionnaires. Throughout the study, participants will be regularly assessed for lesion size, side effects, and laboratory abnormalities. Quality of life will be evaluated at the start and six months later. Safety monitoring includes checking for adverse effects each month and ensuring proper use of contraception for women who could become pregnant. Total participation lasts at least six months, with ongoing options for those wishing to continue therapy.

Weekly Sirolimus Therapy