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Weekly Sirolimus Therapy

NCT04861064 · Status: RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 24

Last updated 2026-03-05

No results posted yet for this study

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

Interventions

DRUG

Sirolimus

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

Sponsors & Collaborators

  • Medical University of South Carolina

    lead OTHER

Principal Investigators

  • Chelsea Shope, MSCR · Medical University of South Carolina

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
2 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-01-18
Primary Completion
2027-12-31
Completion
2027-12-31
FDA Drug
Yes

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04861064 on ClinicalTrials.gov