NCT01289912.

Methods	Phase II randomised, double‐blinded, placebo‐controlled study.
Participants	52 participants diagnosed with TSC between the ages of 6 and 21 years of age and had an IQ of greater than or equal to 60.
Interventions	Drug: RAD001 (other names: everolimus; afinitor). RAD001 is formulated as tablets of 5.0 mg strength, blister‐packed under aluminium foil in units of 10 tablets and dosed on a regular basis. Matching placebo will be provided as a matching tablet and will also be blister packed under aluminium foil in units of 10. RAD001 or matching placebo tablets should be opened only at the time of administration as drug is both hygroscopic and light‐sensitive. Participants will be instructed to take 4.5 mg/m2 of RAD001 or matching placebo orally with a glass of water at regular intervals at the same time (delete: each day) in the morning after a light, non‐fat breakfast.
Outcomes	Primary outcomes: neurocognition and safety. Secondary outcomes: frequency of epileptiform events; sleep disturbances; autism spectrum disorders features; academic skills; behavioral problems.
Notes	Sponsored by Mustafa Sahin (Children's Hospital, Boston) in collaboration with Tuberous Sclerosis Alliance, Autism Speaks, Novartis Pharmaceuticals and Seizure Tracker LLC.