Clinical Trial for individuals diagnosed with Tuberous Sclerosis Complex (TSC) and cutaneous angiofibromas

(January, 2019)

We are currently conducting a clinical trial at Boston Children’s Hospital for individuals diagnosed with TSC with facial bumps called cutaneous angiofibromas. The goal of this study is to investigate if the study drug, sirolimus, improves cutaneous angiofibromas in individuals diagnosed with TSC. The trial will involve 8 centers and is sponsored by a pharmaceutical company named Aucta Pharmaceuticals, LLC.

We are looking to recruit individuals between the ages of 2 and 18 years who have TSC with cutaneous angiofibromas. If you/your child qualifies for the treatment phase of the trial after the initial screening visit, you/your child would be asked to take either the study ointment or a placebo (ointment with no medicine), which is determined by chance.

The study involves 4 visits to Boston Children’s Hospital and two telephone visits over a 4-month (16-week) timeframe. After completing the first phase of the study, you/your child will be offered the opportunity to participate in an optional 12-week open-label phase with sirolimus ointment. If you agree for you/your child to participate in the open-label phase, you/your child will make 2 additional clinic visits and 1 additional telephone visit bringing the total clinic visits to 6 and telephone visits to 3.

The study visits include blood draws, skin exams, vital signs, laboratory tests and other evaluations such as questionnaires and assessments. The study drug will be provided at no charge, and a voucher for parking will be provided to you at each study visit. In addition, there are travel funds to cover costs associated with participation in the study.

If you are interested in participating in this trial, please contact study coordinator Julia Costantini 617-355-5230 or Julia.Costantini@childrens.harvard.edu.