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
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Enrolling by invitation |
NCT06139172 -
Promoting Prosocial Behavior in Syndromic Intellectual and Developmental Disabilities
|
N/A | |
| Recruiting |
NCT00001532 -
Role of Genetic Factors in the Development of Lung Disease
|
||
| Completed |
NCT00552955 -
Effect of Fasting on the Size of Abdominal Lymphatic Tumors in Women
|
||
| Recruiting |
NCT03422367 -
JASPER Early Intervention for Tuberous Sclerosis
|
N/A | |
| Not yet recruiting |
NCT06081348 -
Sertraline vs. Placebo in the Treatment of Anxiety in Children and AdoLescents With NeurodevelopMental Disorders
|
Phase 2 | |
| Unknown status |
NCT00490789 -
Trial of Efficacy and Safety of Sirolimus in Tuberous Sclerosis and LAM
|
Phase 2 | |
| Recruiting |
NCT04463316 -
GROWing Up With Rare GENEtic Syndromes
|
||
| Recruiting |
NCT06065852 -
National Registry of Rare Kidney Diseases
|
||
| Enrolling by invitation |
NCT03836300 -
Parent and Infant Inter(X)Action Intervention (PIXI)
|
N/A | |
| Completed |
NCT02104011 -
Treatment of Renal Angiomyolipomas in Tuberous Sclerosis by Beta-blockers
|
Phase 2 | |
| Recruiting |
NCT02461459 -
Autism Spectrum Disorder (ASD) and Intellectual Disability (ID) Determinants in Tuberous Sclerosis Complex (TSC)
|
||
| Completed |
NCT01526356 -
Topical Rapamycin to Erase Angiofibromas in TSC
|
Phase 2 | |
| Withdrawn |
NCT03254680 -
Turmeric as Treatment in Epilepsy
|
N/A | |
| Terminated |
NCT01092208 -
Studies of Autistic Patients: Gene Networks and Clinical Subtypes
|
||
| Completed |
NCT03826628 -
Dose-Ranging Efficacy and Safety Study of Topical Rapamycin Cream for Facial Angiofibroma Associated With Tuberous Sclerosis Complex
|
Phase 2/Phase 3 | |
| Completed |
NCT00989742 -
Doxycycline In Lymphangioleiomyomatosis (LAM)
|
Phase 4 | |
| Recruiting |
NCT05286632 -
KidneYou - Innovative Digital Therapy
|
N/A | |
| Withdrawn |
NCT04344626 -
Use of a Tonometer to Identify Epileptogenic Lesions During Pediatric Epilepsy Surgery
|
N/A | |
| Recruiting |
NCT05199402 -
Clinical Trial Data Set Re-use With Statistical Methodologies Tailored for Clinical Trials in Rare Diseases
|
||
| Recruiting |
NCT00001975 -
Study of Skin Tumors in Tuberous Sclerosis
|