Lymphatic malformations (LM) are rare masses where fluid has built up causing swelling. This study will compare an oral drug called sildenafil to a placebo to see if it safe and effective for the treatment of LM. The study has a main phase and an open-label phase. The main phase is randomized and double-blinded. This means your child will be randomly put into a group that receives either the sildenafil or a placebo and neither you or the staff will know what treatment your child is receiving (unless this information is needed in an emergency). Any participants who receive the placebo will have the option to participate in the open-label phase during which all participants will take the sildenafil. This study involves 3 visits to a clinic at Stanford University, 3 visits to Lurie Children’s clinic; and 3 phone calls. If your child has lymphatic malformations; weights at least 17.5 lbs; and is 6 months to 10 years old, he or she may be eligible for this research study. All children participating will have a medical evaluation including an MRI, physical, eye, and hearing exams. Financial compensation for completing all study visits and travel to Stanford University.
Thy Huynh, MD