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Efficacy of RAD001/Everolimus in Autism and NeuroPsychological Deficits in Children With Tuberous Sclerosis Complex

NCT01730209 · Status: UNKNOWN · Phase: PHASE2/PHASE3 · Type: INTERVENTIONAL · Enrollment: 60

Last updated 2015-05-05

No results posted yet for this study

Summary

Tuberous sclerosis complex (TSC) is a genetic disease that leads to mental retardation in over 50% of patients, and to learning problems, behavioral problems, autism and epilepsy in up to 90% of patients. The underlying deficit of TSC, loss of inhibition of the mammalian target of rapamycin (mTOR) protein due to dysfunction of the tuberin/hamartin protein complex, can be rescued by everolimus. Everolimus has been registered as treatment for renal cell carcinoma and giant cell astrocytoma (SEGA). Evidence in human and animal studies suggests that mTOR inhibitors improve learning and development in patients with TSC.

Conditions

  • Tuberous Sclerosis Complex
  • TSC Related Cognitive Disability
  • TSC Related Autism
  • TSC Related Learning Problems

Interventions

DRUG

Everolimus

Everolimus once daily titrated to trough levels of 5-10 ng/ml.

DRUG

Placebo

Sponsors & Collaborators

Principal Investigators

  • M.C.Y. de Wit, MD. PhD. · Erasmus Medical Center

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
4 Years
Max Age
15 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2012-11-30
Primary Completion
2015-11-30
Completion
2016-11-30

Countries

  • Netherlands

Study Locations

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Entities

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 NCT01730209 on ClinicalTrials.gov