Weekly Sirolimus Therapy

Description

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

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Study Overview

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Study Details

Study overview

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.

Weekly Sirolimus Therapy for the Treatment of Venous and Lymphatic Malformations

Weekly Sirolimus Therapy

Condition
Venous Malformation
Intervention / Treatment

-

Contacts and Locations

Charleston

Medical University of South Carolina, Charleston, South Carolina, United States, 29403

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

  • * Patient 2 years of age and older
  • * Venous, lymphatic, or venolymphatic malformations
  • * 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.

Ages Eligible for Study

2 Years to

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Medical University of South Carolina,

Chelsea Shope, MSCR, PRINCIPAL_INVESTIGATOR, Medical University of South Carolina

Study Record Dates

2025-06