Clinical Trial: A Study of Everolimus in the Treatment of Neurocognitive Problems in Tuberous Sclerosis

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

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

Brief 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.


Detailed Summary:
Sponsor: Cardiff University

Current Primary Outcome:

  • List Learning test (from the BIRT Memory and Information Processing Battery) [ Time Frame: 6 months ]
  • Complex Figure test (from the BIRT Memory and Information Processing Battery) [ Time Frame: 6 months ]
  • CANTAB - Stockings of Cambridge (SOC) [ Time Frame: 6 months ]
  • CANTAB - Spatial Working Memory (SWM) [ Time Frame: 6 months ]
  • Telephone search dual task (from the Test of Everyday Attention) [ Time Frame: 6 months ]


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • CANTAB - Rapid Visual Information Processing Battery (RVIP) [ Time Frame: 6 months ]
  • CANTAB - Spatial Span (SSP) [ Time Frame: 6 months ]
  • CANTAB - Attentional Set-shifting (IDED) [ Time Frame: 6 month ]
  • Verbal Fluency /Controlled Oral Word Association Test (COWAT) [ Time Frame: 6 months ]
  • Cancellation task [ Time Frame: 6 months ]
  • Symptom Checklist 90R (SCL-90R) [ Time Frame: 6 months ]
  • Quality of Life in Epilepsy (QOLIE) [ Time Frame: 6 months ]
  • Liverpool Seizure Severity Scale (LSSS) [ Time Frame: 6 months ]
  • Vineland Adaptive Behavior Scales-II (VABS-II) (survey form) [ Time Frame: 6 months ]
  • Social Responsiveness Scale - Adult version (SRS-A) [ Time Frame: 6 months ]
  • Social communication questionnaire (SCQ) [ Time Frame: 6 months ]
  • National Adult Reading Test (NART) [ Time Frame: 6 months ]


Original Secondary Outcome: Same as current

Information By: Cardiff University

Dates:
Date Received: September 5, 2013
Date Started: June 2012
Date Completion: July 2018
Last Updated: January 30, 2017
Last Verified: January 2017