Trial of Efficacy and Safety of Sirolimus in Tuberous Sclerosis and LAM

Tuberous sclerosis

TSC Biosample Repository and Natural History Database Open-label Study of Adjunctive GNX Treatment in Children and Adults With TSC-related Epilepsy (TrustTSC OLE) Placebo Controlled Study to Assess the Efficacy and Safety of Rapamycin in Drug Resistant Epilepsy Associated With Tuberous Sclerosis Complex Clinical Study of NPC-12Y Gel in Patients With Skin Lesions Associated With TSC Feasibility of [11C]Acetate-Positron Emission Tomography (PET) in Lymphangioleiomyomatosis and Tuberous Sclerosis Complex Lymphangioleiomyomatosis, a Study on Cathepsin K Adjunctive GNX Treatment Compared With Placebo in Children and Adults With TSC-related Epilepsy KidneYou – Innovative Digital Therapy Gait in Rare Diseases Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study Assessment of Potential for Chronic Liver Injury in Participants Treated With Epidiolex (Cannabidiol) Oral Solution Efficacy and Safety of Rapamycin Versus Vigabatrin in the Prevention of Tuberous Sclerosis Complex Symptoms in Infants Doxycycline In Lymphangioleiomyomatosis (LAM) Effect of Fasting on the Size of Abdominal Lymphatic Tumors in Women Turmeric as Treatment in Epilepsy Study of the Disease Process of Lymphangioleiomyomatosis Adjunctive Ganaxolone Treatment (Part A) in TSC Followed by Long-term Treatment (Part B) Safety of Simvastatin in LAM and TSC Studies of Autistic Patients: Gene Networks and Clinical Subtypes Topical Rapamycin to Erase Angiofibromas in TSC Rapalogues for Autism Phenotype in TSC: A Feasibility Study A Pilot Study To Evaluate The Effects of Everolimus on Brain mTOR Activity and Cortical Hyperexcitability in TSC and FCD Everolimus for Cancer With TSC1 or TSC2 Mutation RAD001 Therapy of Angiomyolipomata in Patients With TS Complex and Sporadic LAM The Effectiveness and Safety of Vagus Nerve Stimulation for TRE The Effectiveness and Safety of Resective Epilepsy Surgery for TRE Roll-over Study to Collect and Assess Long-term Safety of Everolimus in Patients With TSC and Refractory Seizures Who Have Completed the EXIST-3 Study [CRAD001M2304] and Who Are Benefitting From Continued Treatment Topical Sirolimus Ointment for Cutaneous Angiofibromas in Subjects With Tuberous Sclerosis Complex The Cognitive Variability in NF1 and TSC Monozygotic Twins Long-term Trial of Topical Sirolimus to Angiofibroma in Patient With Tuberous Sclerosis Complex Efficacy of RAD001/Everolimus in Autism and NeuroPsychological Deficits in Children With Tuberous Sclerosis Complex Phase III Trial of Topical Formulation of Sirolimus to Skin Lesions in Patients With Tuberous Sclerosis Complex (TSC) Topical Rapamycin Therapy to Alleviate Cutaneous Manifestations of Tuberous Sclerosis Complex (TSC) and Neurofibromatosis I (NF1) Trial of Efficacy and Safety of Sirolimus in Tuberous Sclerosis and LAM Long-term, Prospective Study Evaluating Clinical and Molecular Biomarkers of Epileptogenesis in a Genetic Model of Epilepsy – Tuberous Sclerosis Complex A Randomized Controlled Trial of Cannabidiol (GWP42003-P, CBD) for Seizures in Tuberous Sclerosis Complex (GWPCARE6) An Open-label Extension Trial of Cannabidiol (GWP42003-P, CBD) for Seizures in Tuberous Sclerosis Complex (GWPCARE6) Aspirin as an add-on Treatment of Refractory Epilepsy in Tuberous Sclerosis Complex Dose-Ranging Efficacy and Safety Study of Topical Rapamycin Cream for Facial Angiofibroma Associated With Tuberous Sclerosis Complex Autism Spectrum Disorder (ASD) and Intellectual Disability (ID) Determinants in Tuberous Sclerosis Complex (TSC) Clinical Presentation and Renal Outcome of Patients With Tuberous Sclerosis Complex and/or Renal Angiomyolipoma in the Great West Region of France Preventing Epilepsy Using Vigabatrin In Infants With Tuberous Sclerosis Complex Early Behavioral Intervention to Improve Social Communication Function in Infants With Tuberous Sclerosis Complex Characterization of Patients With Tuberous Sclerosis Complex, Lymphangioleiomyomatosis and Angiomyolipoma Everolimus (RAD001) Therapy for Epilepsy in Patients With Tuberous Sclerosis Complex (TSC) Everolimus (RAD001) Therapy of Giant Cell Astrocytoma in Patients With Tuberous Sclerosis Complex JASPER Early Intervention for Tuberous Sclerosis Studies in Patients With Tuberous Sclerosis Complex Treatment of Renal Angiomyolipomas in Tuberous Sclerosis by Beta-blockers Efficacy and Safety of RAD001 in Patients Aged 18 and Over With Angiomyolipoma Associated With Either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM) Efficacy and Safety of Everolimus (RAD001) in Patients of All Ages With Subependymal Giant Cell Astrocytoma Associated With Tuberous Sclerosis Complex (TSC)(EXIST-1) Long Term Follow Up for RAD001 Therapy of Angiomyolipomata in Patients With Tuberous Sclerosis (TSC) and Sporadic Lymphangioleiomyomatosis (LAM) A Study of Everolimus in the Treatment of Neurocognitive Problems in Tuberous Sclerosis Trial of RAD001 and Neurocognition in Tuberous Sclerosis Complex (TSC) Study of Skin Tumors in Tuberous Sclerosis Dermatologic Patterns of Tuberous Sclerosis Patients and Somatic Mutation Relationship Genetic Heterogeneity and Genotype-phenotype Correlation of Children and Adults With Tuberous Sclerosis Complex (TuScCom) Early Biomarkers of Autism in Infants With Tuberous Sclerosis Complex (TSC) Tuberous Sclerosis Complex Natural History Study: Renal Manifestations Rapamycin Therapy for Patients With Tuberous Sclerosis Complex and Sporadic LAM