Weekly Sirolimus Therapy

Launched by MEDICAL UNIVERSITY OF SOUTH CAROLINA · Apr 23, 2021

Keywords

ClinConnect Summary

This clinical trial is studying the use of a medication called sirolimus, taken once a week, to treat venous and lymphatic malformations, which are abnormal growths in the blood and lymph vessels. Currently, treatment options for these conditions are limited and daily doses of sirolimus have shown benefits but can come with significant side effects. Researchers want to see if a weekly dose can still be effective while possibly reducing these side effects. The trial will also look at how satisfied patients are with the treatment and what side effects they experience.

To participate in this study, you need to be at least 2 years old and have a diagnosis of venous or lymphatic malformations. Unfortunately, some children cannot participate, such as those with certain health conditions or those who have had transplants. If you join the trial, you will take the medication for 6 months, with an option to continue afterwards. It’s important to know that women who could become pregnant must use effective birth control during and for 12 weeks after treatment, as sirolimus may not be safe during pregnancy. The trial is currently recruiting participants, so there’s an opportunity to help advance treatment options for these conditions.

Gender

ALL

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.