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Weekly Sirolimus Therapy
Sponsor: Medical University of South Carolina
Summary
In current practice, options for venous and lymphatic malformations remain limited. Recently an oral medication, sirolimus, has been found to benefit patients when taken once or twice a day for several months. Unfortunately there are many side effects associated with this medication, some of which can be severe including, neutropenia, oral ulcerations, and lab abnormalities. The purpose of this study is to determine if once weekly dosed sirolimus will be effective for the treatment of venous and lymphatic malformations. Additionally, the study will evaluate patient satisfaction and identify adverse effects. Participants will be on the medication for 6 months with an option to continue after this time period.
Official title: Weekly Sirolimus Therapy for the Treatment of Venous and Lymphatic Malformations
Key Details
Gender
All
Age Range
2 Years - Any
Study Type
INTERVENTIONAL
Enrollment
24
Start Date
2022-01-18
Completion Date
2027-12
Last Updated
2026-03-05
Healthy Volunteers
No
Interventions
Sirolimus
Participants will get Sirolimus (1.5-2 2mg/m2) weekly for 6 months.
Locations (1)
Medical University of South Carolina
Charleston, South Carolina, United States