Phase 2 N=20 Everolimus (RAD001) Therapy for Epilepsy in Patients With Tuberous Sclerosis Complex (TSC)
Epilepsy · Tuberous Sclerosis Complex
Primary: Reduction in Seizure Frequency — 60 percentage
Clinical Trial Search
Completed trials with posted results — search by concept, filter by the parameters that matter at the bedside.
Phase 2 N=47 Trial of RAD001 and Neurocognition in Tuberous Sclerosis Complex (TSC)
Tuberous Sclerosis Complex
Primary: Evaluation of the Safety of RAD001 on Neurocognition in Patients With TSC Compared With Placebo in Patients With TSC. — 325; 147 Adverse Events
Phase 2 N=84 Preventing Epilepsy Using Vigabatrin In Infants With Tuberous Sclerosis Complex
Tuberous Sclerosis Complex
Primary: Cognitive Assessment Scores and Developmental Impact — 83.9; 80.9; 97.3; 85.0 score on a scale — p=0.8681
Phase 2 N=28 Everolimus (RAD001) Therapy of Giant Cell Astrocytoma in Patients With Tuberous Sclerosis Complex
Tuberous Sclerosis · Subependymal Giant Cell Astrocytoma
Primary: Number With Observed Adverse Side Effects — 26; 25; 15; 14 participants
Phase 2 N=30 Everolimus for Cancer With TSC1 or TSC2 Mutation
TSC1 · TSC2 · Tuberous Sclerosis Complex
Primary: Objective Response Rate — 2 Participants
Phase 3 N=366 A Placebo-controlled Study of Efficacy & Safety of 2 Trough-ranges of Everolimus as Adjunctive Therapy in Patients With Tuberous Sclerosis Complex (TSC) & Refractory Partial-onset Seizures
Tuberous Sclerosis Complex-associated Refractory Seizures
Primary: Core Phase: European Medicine Agency (EMA): Seizure Frequency Response Rate — 28.2; 40.0; 15.1 Percentage of responders — p=0.008
Phase 2 N=5 Stopping TSC Onset and Progression 2: Epilepsy Prevention in TSC Infants
Tuberous Sclerosis Complex · Epilepsy
Primary: Safety - Adverse Events — 40 percentage of participants
Phase 2 N=179 Topical Rapamycin to Erase Angiofibromas in TSC
Angiofibromas · Tuberous Sclerosis
Primary: Change From Baseline in the Facial Angiofibroma Grading Scale (AGS) Score — -13.89; -12.71; -1.39 score on a scale
Phase 3 N=107 Dose-Ranging Efficacy and Safety Study of Topical Rapamycin Cream for Facial Angiofibroma Associated With Tuberous Sclerosis Complex
Facial Angiofibroma · Tuberous Sclerosis
Primary: Percentage of Participants Obtaining Successful Treatment — 4; 3; 2 Participants
Phase 3 N=117 Efficacy and Safety of Everolimus (RAD001) in Patients of All Ages With Subependymal Giant Cell Astrocytoma Associated With Tuberous Sclerosis Complex (TSC)(EXIST-1)
Tuberous Sclerosis · Subependymal Giant Cell Astrocytoma
Primary: Percentage of Participants With Best Overall Subependymal Giant Cell Astrocytomas (SEGA) Response — 34.6; 0.0; 57.7 Percentage of participants
Phase 3 N=118 Efficacy and Safety of RAD001 in Patients Aged 18 and Over With Angiomyolipoma Associated With Either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM)
Tuberous Sclerosis Complex (TSC) · Lymphangioleiomyomatosis (LAM)
Primary: Angiomyolipoma Response Rate as Per Central Radiology Review — 41.8; 0; 58.0 Percentage of Participants — p=<0.0001
Phase 2 N=3 Rapalogues for Autism Phenotype in TSC: A Feasibility Study
Tuberous Sclerosis Complex · Self-injury · Autism
Primary: Number of Participants With Compliance to the Treatment Protocol. — 100; 58; 58 percentage of completed visits/measures
Phase 4 N=40 Safety and Efficacy of Everolimus (Afinitor®) in Chinese Adult Patients With Angiomyolipoma Associated With Tuberous Sclerosis Complex.
Renal Angiomyolipoma
Primary: Percentage of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) — 40; 39; 6; 2 Participants
Phase 2 N=15 A Pilot Study To Evaluate The Effects of Everolimus on Brain mTOR Activity and Cortical Hyperexcitability in TSC and FCD
Epilepsy · Tuberous Sclerosis Complex · Focal Cortical Dysplasia
Primary: Number of Patients With Adverse Events — 0; 0 Participants
Phase 2 N=46 RAD001 and Neurocognition in PTEN Hamartoma Tumor Syndrome
PTEN Gene Mutation · PTEN Hamartoma Tumor Syndrome
Primary: Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability], Dropout — 3; 2; 5; 2 Participants
Phase 2 N=18 Sirolimus to Treat Cowden Syndrome and Other PTEN Hamartomatous Tumor Syndromes
Cowden's Disease · Hamartoma Syndrome, Multiple
Primary: Biochemical Changes in Benign and Malignant Tumor Tissues as Assessed by Immunohistochemistry.
Phase 2 N=36 RAPAMYCIN FOR KIDNEY ANGIOMYOLIPOMAS
Nonmalignant Neoplasm · Tuberous Sclerosis · Lymphangioleimyomatosis
Primary: Objective Response Rate — 44.4 percentage of participants
Phase 2 N=12 Everolimus in Patients With Advanced Solid Malignancies With TSC1, TSC2, NF1, NF2, or STK11 Mutations
Solid Malignancy · Solid Tumor
Primary: Response Rate (RR) — 1 Participants
Phase 3 N=224 A Randomized Controlled Trial of Cannabidiol (GWP42003-P, CBD) for Seizures in Tuberous Sclerosis Complex (GWPCARE6)
Tuberous Sclerosis Complex · Seizures
Primary: Percent Change From Baseline in the Number of Tuberous Sclerosis Complex (TSC)-Associated Seizures During the Treatment Period (Maintenance and Titration) — -43.36…
Phase 2 N=23 Adjunctive Ganaxolone Treatment (Part A) in TSC Followed by Long-term Treatment (Part B)
Tuberous Sclerosis
Primary: Percent Change From Baseline in 28-day Seizure Frequency Through the End of 12-Week Treatment Period — -16.61 Percent change
Phase 3 N=129 Adjunctive GNX Treatment Compared With Placebo in Children and Adults With TSC-related Epilepsy
Tuberous Sclerosis Complex
Primary: Percent Change From Baseline in 28-day Seizure Frequency for Primary Seizure Type During Double Blind Period — -7.50; 13.57 Percent change — p=0.0904
Phase 2 N=62 A Phase II Study of the mTOR Inhibitor Sirolimus in Neurofibromatosis Type 1 Related Plexiform Neurofibromas
Neurofibromatosis Type 1
Primary: Time to Disease Progression Based on Volumetric MRI — 15.4 Months
Phase 3 N=89 Efficacy and Safety of Sirolimus in LAM
Lymphangioleiomyomatosis
Primary: Changes in Forced Expiratory Volume in One-second (FEV1) Slope in Milliliters Per Month — 1; -12 milliliters per month — p=<0.001
Phase 4 N=15 Long-term Monitoring of Growth and Development of Pediatric Patients Previously Treated With Everolimus
Growth and Development
Primary: Number of Participants Who Achieved Tanner Stage V at or Before Age 16 (Females) or 17 (Males) — 4; 6 Participants
N/A N=10 Tourette Syndrome Deep Brain Stimulation
Tourette Syndrome
Primary: Number of Participants With a 40% Reduction in Total Tics on the Yale Global Tic Severity Scale (YGTSS) With CDBS (Period 1) — 4 Participants — p=0.05
Phase 3 N=199 An Open-label Extension Trial of Cannabidiol (GWP42003-P, CBD) for Seizures in Tuberous Sclerosis Complex (GWPCARE6)
Tuberous Sclerosis Complex · Seizures
Primary: Number of Participants With Any Treatment-emergent Adverse Events, Discontinuations Due to AEs, Serious AEs, and Treatment-related AEs (TEAE) — 117; 75; 80; 60…
Phase 2 N=20 Transcranial Magnetic Stimulation (TMS) for Individuals With Tourette's Syndrome
Tourette Syndrome
Primary: Yale Global Tic Severity Scale (Y-GTSS) — 29.5; 31.5 units on a scale
Phase 3 N=10 Trial of Sirolimus for Cognitive Impairment in Sturge-Weber Syndrome
Sturge-Weber Syndrome
Primary: Change in Cognitive Function as Assessed by the National Institute of Health (NIH) Toolbox Cognitive Battery — 44.2; 42.3; 34.1; 35.2 T score
Phase 2 N=61 Safety and Efficacy Study of Sirolimus in Complicated Vascular Anomalies
Kaposiform Hemangioendotheliomas · Tufted Angioma · Capillary Venous Lymphatic Malformation
Primary: Overall Response by Radiologic Evaluation, Quality of Life Assessment, and Functional Impairment Score — 47; 7; 3 Participants
N/A N=10 Response Inhibition in Tourette Syndrome
Tourette Syndrome
Primary: Stop Signal Reaction Time (SSRT) — 249; 245 milliseconds — p=0.49