Dr Kate Riney and the team at Queensland Children’s Hospital are currently recruiting patients for a formal phase II/III trial of topical rapamycin (sirolimus) for facial angiofibromas.
Patients, aged 6 to 65 years, need to be treatment naïve (ie have not received rapamycin/sirolimus, everolimus, or temsirolimus within 3 months of enrolment) or willing to take a break from their usual treatment for a while and then go into the trial. Patients who have received laser therapy or surgical therapy within 6 months prior to trial enrolment are also ineligible.
There are six sites involved in the trial, three in the US, one in Spain, one in Australia and also one in New Zealand.
The study aims to compare the safety and efficacy of two different strengths of rapamycin cream, topical and placebo over 26 weeks in the treatment of facial angiofibroma (FA) associated with TSC. The goal of the trial is to obtain the data for FDA approval which might possibly lead eventually to PBS listing, meaning topical rapamycin (sirolimus) might be more easily and widely available.
More information about the trial can be found at https://clinicaltrials.gov/ct2/show/NCT03826628