Actively Recruiting

Phase 2
Age: 2Years +
All Genders
NCT04861064

Weekly Sirolimus Therapy

Led by Medical University of South Carolina · Updated on 2026-03-05

24

Participants Needed

1

Research Sites

306 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

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

Official Title

Weekly Sirolimus Therapy

Who Can Participate

Age: 2Years +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Patient 2 years of age and older
  • Diagnosis of venous, lymphatic, or venolymphatic malformations
Not Eligible

You will not qualify if you...

  • 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 who are pregnant or may become pregnant; women of childbearing potential must use effective contraception before, during, and for 12 weeks after therapy

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 1 location

1

Medical University of South Carolina

Charleston, South Carolina, United States, 29403

Actively Recruiting

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Research Team

S

Stephnie Munie, BS

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NA

Model

SINGLE_GROUP

Primary Purpose

TREATMENT

Number of Arms

1

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Weekly Sirolimus Therapy | DecenTrialz