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

NCT01954693 · Status: UNKNOWN · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 48

Last updated 2018-01-30

No results posted yet for this study

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

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

Sponsors & Collaborators

  • Novartis

    collaborator INDUSTRY

  • Cardiff University

    lead OTHER

Principal Investigators

  • Julian Sampson, Prof · Cardiff University

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
16 Years
Max Age
60 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2012-06-30
Primary Completion
2018-08-06
Completion
2018-08-06

Countries

  • United Kingdom

Study Locations

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Drugs
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01954693 on ClinicalTrials.gov