Clinical trial NCT04861064: Weekly Sirolimus Therapy
Official title: Weekly Sirolimus Therapy for the Treatment of Venous and Lymphatic Malformations
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: Venous Malformation
Lymphatic Malformation
Lead sponsor: Medical University of South Carolina
Sponsor class: OTHER
First posted: 2021-04-27
Enrollment: 24
Sex: ALL
Minimum age (years): 2
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility criteria: 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.

Primary outcomes:
measure: Change in size of lesion
timeFrame: Baseline and 6 months
measure: Change in size of lesion through photograph
timeFrame: Baseline and 6 months
Secondary outcomes:
measure: Number of side effects experienced
timeFrame: Month One
measure: Number of side effects experienced
timeFrame: Month Two
measure: Number of side effects experienced
timeFrame: Month Three
measure: Number of side effects experienced
timeFrame: Month Four
measure: Number of side effects experienced
timeFrame: Month Five
measure: Number of side effects experienced
timeFrame: Month Six
measure: Change in quality of life as assessed by questionnaire
timeFrame: Baseline and 6 months
measure: Number of participants with laboratory abnormalities
timeFrame: From baseline visit to 2 month visit
Other outcomes:
Locations:
city: Charleston
country: United States
facility: Medical University of South Carolina
state: South Carolina
Study officials:
affiliation: Medical University of South Carolina
name: Chelsea Shope, MSCR
role: PRINCIPAL_INVESTIGATOR
References: