A clinical trial, sometimes called a “study,” is the investigation of a medicine in a new way. Sometimes the medicine has been studied and approved for other diseases. Clinical trials in the United States are closely regulated by the Food & Drug Administration (FDA). Study participants and their doctors are provided with information specific to the trial to help them make a decision about whether the study is right for them.  

All patients in the TOIVA Trial will treat themselves at home with a new gel formulation of an approved drug (rapamycin, also known as sirolimus).  The gel will be rubbed onto the skin once a day by the participant, or for very young children, by their caregiver. 

Venous Malformation (VM) is a type of vascular anomaly where a person’s veins grow in an abnormal way, sometimes called VM disease.  A cutaneous Venous Malformation (or cVM) is typically purplish or blue in color and is visible just beneath the skin, sometimes protruding or bulging out of the skin.  A cutaneous VM can be delicate and painful, and can sometimes cause problems, especially if it is located near joints or other organs.  In some patients the skin is also tender and fragile, and friction from clothes or bedding can lead to chafing and bleeding.  

Compensation is offered to offset patient burden and should not be considered the reason for participation.  If you or your child is a good match for the study, the details related to the study stipend will be provided to you.

The study sponsor will pay travel expenses to a study location that is convenient for both the participant and one caregiver.