Lymphatic Malformation, Venous Malformation
Conditions
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.
Interventions
DRUGSirolimus
Participants will get Sirolimus (1.5-2 2mg/m2) weekly for 6 months.
Sponsors
Medical University of South Carolina
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
2 Years to No maximum
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change in size of lesion | Baseline and 6 months | Will be measuring the size of the lesions (mm) at each patient visit |
| Change in size of lesion through photograph | Baseline and 6 months | Will be evaluating clinical photographs of lesions at each patient visit |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of side effects experienced | Month One | Patient will complete side effect questionnaires at each visit |
| Change in quality of life as assessed by questionnaire | Baseline and 6 months | Patient will complete quality of life questionnaire at each visit |
| Number of participants with laboratory abnormalities | From baseline visit to 2 month visit | Standard of care laboratory results (CBC, CMP, triglycerides) will be monitored |
Countries
United States
Contacts
CONTACTStephnie Munie, BS
PRINCIPAL_INVESTIGATORChelsea Shope, MSCR
Medical University of South Carolina
Outcome results
None listed