Tuberous Sclerosis
41
11
13
19
Key Insights
Highlights
Success Rate
90% trial completion (above average)
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 50/100
4.9%
2 terminated out of 41 trials
90.5%
+4.0% vs benchmark
10%
4 trials in Phase 3/4
37%
7 of 19 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 19 completed trials
Clinical Trials (41)
Study of Skin Tumors in Tuberous Sclerosis
Role of Genetic Factors in the Development of Lung Disease
Familial Investigations of Childhood Cancer Predisposition
Study of the Disease Process of Lymphangioleiomyomatosis
Remote Assessment and Intervention for Behavior Problems in Kids With TSC
Web Intervention for Parents of Youth With Genetic Syndromes (WINGS)
Topical Sirolimus Ointment for Cutaneous Angiofibromas in Subjects With Tuberous Sclerosis Complex
Gait in Rare Diseases
Parent and Infant Inter(X)Action Intervention (PIXI)
Sertraline vs. Placebo in the Treatment of Anxiety in Children and AdoLescents With NeurodevelopMental Disorders
TSC Biosample Repository and Natural History Database
Qualitative Study in Patients With Genodermatoses and Healthcare Professionals on Reproductive Counselling
Reproductive Options in Inherited Skin Diseases
Clinical Trial Data Set Re-use With Statistical Methodologies Tailored for Clinical Trials in Rare Diseases
KidneYou - Innovative Digital Therapy
Autism Spectrum Disorder (ASD) and Intellectual Disability (ID) Determinants in Tuberous Sclerosis Complex (TSC)
Genetic Bases of Neuroendocrine Neoplasms in Mexican Patients
Use of a Tonometer to Identify Epileptogenic Lesions During Pediatric Epilepsy Surgery
National Registry of Rare Kidney Diseases
Dose-Ranging Efficacy and Safety Study of Topical Rapamycin Cream for Facial Angiofibroma Associated With Tuberous Sclerosis Complex