Weekly Sirolimus Therapy

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE2

Total Enrollment

24 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-01-18

Study Completion Date

2026-06-30

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.

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Detailed Description

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Conditions

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Venous Malformation Lymphatic Malformation

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Treatment Group

Group Type EXPERIMENTAL

Sirolimus

Intervention Type DRUG

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

Interventions

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Sirolimus

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

Intervention Type DRUG

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.

Minimum Eligible Age

2 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No