Recruiting
Phase 2

Sirolimus

Sponsor:

Medical University of South Carolina

Code:

NCT04861064

Conditions

Venous Malformation

Lymphatic Malformation

Eligibility Criteria

Sex: All

Age: 2+

Healthy Volunteers: Not accepted

Interventions

Sirolimus

Study Details

Brief 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.

Conditions

Venous Malformation

Lymphatic Malformation

Study ID

NCT04861064

Start date

Jan 18, 2022

Status verified date

Mar, 2026

Completion date

Dec, 2027

Anticipated

Primary completion date

Dec, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 2+

Healthy Volunteers: Not accepted

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.

Study Design

Enrollment

24 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Treatment Group

Interventions

Sirolimus

Participants will get Sirolimus (1.5-2 2mg/m2) weekly for 6 months.

Primary outcome measure

  • Change in size of lesion [ Time Frame: Baseline and 6 months ]
  • Change in size of lesion through photograph [ Time Frame: Baseline and 6 months ]

Central Contacts and Locations

Central contacts

Stephnie Munie, BS

843-566-2453munie@musc.edu

Locations

Medical University of South Carolina

Recruiting

Charleston, South Carolina, United States, 29403

Contacts

Stephanie Munie, BS

843-792-9784munie@musc.edu

More Information

Sponsor

Medical University of South Carolina

Last update posted

Mar 5, 2026

Last verified

Mar, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Medical University of South Carolina on 2026-03-05.