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Weekly Sirolimus Therapy
Weekly Sirolimus Therapy for the Treatment of Venous and Lymphatic Malformations
Who can join
Ages 2 and older · All sexes
Full eligibility criteria
Inclusion Criteria: * Patient 2 years of age and older * Venous, lymphatic, or venolymphatic malformations Exclusion Criteria: * Children with contraindication to use of sirolimus * Children with history of transplant * Children with a history of natural immunodeficiency * Children with a history of artificially induced immunodeficiency * Children with a history of a serious or life-threatening infection * Children taking CYP3A4 inhibiting medications * Children taking strong CYP3A4 inducers to avoid subtherapeutic dosing/exposure. * Inability or unwillingness of subject or legal guardian/representative to give informed consent * Women that are or may become pregnant o Sirolimus is a Pregnancy Category C drug. No randomized controlled studies have been done on pregnant women. Women of childbearing potential must be on effective contraception prior to, during, and for 12 weeks following sirolimus therapy.
About the study
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.
What is being tested
- Sirolimus (drug)
Sponsor: Medical University of South Carolina · Participants: 24 · Started: Jan 18, 2022
Contact the study team
- Stephnie Munie, BS · Phone: 843-566-2453
Official record on ClinicalTrials.gov — NCT04861064
Locations in the U.S.
| South Carolina | Medical University of South Carolina, Charleston |
Conditions
From ClinicalTrials.gov, data retrieved Oct 2, 2026. Each study sets its own eligibility; the study team decides who can join.