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NCT Number: NCT04861064

Weekly Sirolimus Therapy

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.

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Key information

Conditions

Venous Malformation Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Hemic and Lymphatic Diseases Lymphatic Abnormalities Lymphatic Diseases Lymphatic Malformation

Age range

2 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Medical University of South Carolina

Charleston, South Carolina, 29403, United States

Location status: Recruiting

Location contact

Stephanie Munie, BS

CONTACT

[email protected]

843-792-9784

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

sirolimus

Drug

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

Primary outcomes

  1. Change in size of lesion

    Time frame: Baseline and 6 months

    Will be measuring the size of the lesions (mm) at each patient visit

  2. Change in size of lesion through photograph

    Time frame: Baseline and 6 months

    Will be evaluating clinical photographs of lesions at each patient visit

Secondary outcomes

  1. Number of side effects experienced

    Time frame: Month One

    Patient will complete side effect questionnaires at each visit

  2. Number of side effects experienced

    Time frame: Month Two

    Patient will complete side effect questionnaires at each visit

  3. Number of side effects experienced

    Time frame: Month Three

    Patient will complete side effect questionnaires at each visit

  4. Number of side effects experienced

    Time frame: Month Four

    Patient will complete side effect questionnaires at each visit

  5. Number of side effects experienced

    Time frame: Month Five

    Patient will complete side effect questionnaires at each visit

  6. Number of side effects experienced

    Time frame: Month Six

    Patient will complete side effect questionnaires at each visit

  7. Change in quality of life as assessed by questionnaire

    Time frame: Baseline and 6 months

    Patient will complete quality of life questionnaire at each visit

  8. Number of participants with laboratory abnormalities

    Time frame: From baseline visit to 2 month visit

    Standard of care laboratory results (CBC, CMP, triglycerides) will be monitored

Study contacts

Contact information is provided by the study sponsor or research team.

Stephnie Munie, BS

CONTACT

[email protected]

843-566-2453

Sponsors and collaborators

Lead sponsor

Medical University of South Carolina

Other

Registry information

Official study title

Weekly Sirolimus Therapy for the Treatment of Venous and Lymphatic Malformations

Important dates

Study start
2022
Primary completion
2027
Study completion
2027
First posted
Apr 27, 2021
Registry last updated
Mar 5, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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