NCT01954693

A Study of Everolimus in the Treatment of Neurocognitive Problems in Tuberous Sclerosis

TRON: A Randomised, Double Blind, Placebo-controlled Study of RAD001 (Everolimus) in the Treatment of Neurocognitive Problems in Tuberous Sclerosis

• Status: Unknown
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 48 (estimated)
• Sponsor: Cardiff University · Academic / Other
• Sex: All
• Age: 16 Years – 60 Years
• Healthy volunteers: Not accepted

Summary

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.

Conditions

• Tuberous Sclerosis

Interventions

Type	Name	Description
DRUG	Placebo	5mg daily administered for 6 months as two oral 2.5 mg tablets once daily.
DRUG	Everolimus (RAD001)	5mg daily administered for 6 months as two oral 2.5 mg tablets once daily

Timeline

• Start date: 2012-06-01
• Primary completion: 2018-08-06
• Completion: 2018-08-06
• First posted: 2013-10-07
• Last updated: 2018-01-30

Locations

1 site across 1 country: United Kingdom