RAPIT TRIAL
Phase 2 and 3 clinical study of the effect of the drug Sirolimus in children with TSC.
In this study, designed and conducted by ENCORE, the effect of the drug Everolimus on autism and neuropsychological complaints in children with TSC was tested. 60 participants took part. The study has been completed and the results have been published in 2019.
Overwater IE, et al. (2019) A randomized controlled trial with everolimus for IQ and autism in tuberous sclerosis complex. Neurology. 93; E200–9. Pubmed