Tuberous Sclerosis Clinical Trial
Official title:
TRON: A Randomised, Double Blind, Placebo-controlled Study of RAD001 (Everolimus) in the Treatment of Neurocognitive Problems in Tuberous Sclerosis
This is a single centre, two-arm, individually randomised, Phase II, double- blind,
placebo-controlled trial of RAD001 (Everolimus) versus placebo in the treatment of
neurocognitive problems in patients with tuberous sclerosis (TSC). The IMP is a licensed
medicine in this patient group but for a different target of effect. The current trial is a
proof of principle study for memory and executive function outcomes.
Following an eligibility visit, patients will be scheduled for baseline visit and
randomization. They will then be followed up for 6 months undergoing both safety and
neurocognitive assessments whilst taking either the placebo or study drug.
48 patients aged 16 to 60 years with tuberous sclerosis (TSC) who have IQ > 60 and a
significant deficit in one or more primary outcome measures will be randomly allocated in a
ratio of 2:1 to either RAD001 (Everolimus) or Placebo.
n/a
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