Weekly Sirolimus Therapy

Study Overview
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Brief Summary
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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.

Official Title
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Weekly Sirolimus Therapy for the Treatment of Venous and Lymphatic Malformations

Conditions
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Venous Malformation, Lymphatic Malformation

Intervention / Treatment
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* Drug: Sirolimus


Participation Criteria
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Eligibility Criteria
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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.

Ages Eligible for Study
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Minimum Age: 2 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Treatment Group<br> | Drug: Sirolimus<br>* Participants will get Sirolimus (1.5-2 2mg/m2) weekly for 6 months.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in size of lesion | Will be measuring the size of the lesions (mm) at each patient visit | Baseline and 6 months | 
| Change in size of lesion through photograph | Will be evaluating clinical photographs of lesions at each patient visit | Baseline and 6 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of side effects experienced | Patient will complete side effect questionnaires at each visit | Month One | 
| Number of side effects experienced | Patient will complete side effect questionnaires at each visit | Month Two | 
| Number of side effects experienced | Patient will complete side effect questionnaires at each visit | Month Three | 
| Number of side effects experienced | Patient will complete side effect questionnaires at each visit | Month Four | 
| Number of side effects experienced | Patient will complete side effect questionnaires at each visit | Month Five | 
| Number of side effects experienced | Patient will complete side effect questionnaires at each visit | Month Six | 
| Change in quality of life as assessed by questionnaire | Patient will complete quality of life questionnaire at each visit | Baseline and 6 months | 
| Number of participants with laboratory abnormalities | Standard of care laboratory results (CBC, CMP, triglycerides) will be monitored | From baseline visit to 2 month visit |