| Recruiting | Epidyolex® in Lennox Gastaut, Dravet Syndrome and Tuberous Sclerosis Complex: an Observational Study in ITALY NCT05485831 | Jazz Pharmaceuticals | — |
| Active Not Recruiting | Astroscape: A Study of Radiprodil on Safety, Tolerability, Pharmacokinetics, and Effect on Seizures and Behavi NCT06392009 | GRIN Therapeutics, Inc. | Phase 1 / Phase 2 |
| Recruiting | Tuberous Sclerosis Complex and Lymphangioleiomyomatosis Pregnancy Registry (TSC-LAM Registry) NCT06160310 | David M. Ritter | — |
| Recruiting | Placebo Controlled Study to Assess the Efficacy and Safety of Rapamycin in Drug Resistant Epilepsy Associated NCT05534672 | Katarzyna Kotulska | Phase 3 |
| Completed | Clinical Study of NPC-12Y Gel in Patients With Skin Lesions Associated With TSC NCT05495425 | Nobelpharma | Phase 3 |
| Completed | Lymphangioleiomyomatosis, a Study on Cathepsin K NCT05323370 | University Hospital, Tours | — |
| Terminated | Open-label Study of Adjunctive GNX Treatment in Children and Adults With TSC-related Epilepsy NCT05604170 | Marinus Pharmaceuticals | Phase 3 |
| Completed | Adjunctive GNX Treatment Compared With Placebo in Children and Adults With TSC-related Epilepsy NCT05323734 | Marinus Pharmaceuticals | Phase 3 |
| Completed | Basimglurant (NOE-101) in Children, Adolescents, and Young Adults With TSC NCT05059327 | Noema Pharma AG | Phase 2 |
| Recruiting | Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study NCT05104983 | Darcy Krueger | Phase 2 |
| Active Not Recruiting | Feasibility of [11C]Acetate-PET in LAM and TSC NCT05467397 | Brigham and Women's Hospital | Phase 1 / Phase 2 |
| Unknown | Acceptance and Commitment Therapy in Tuberous Sclerosis Complex NCT05867576 | University Hospitals Bristol and Weston NHS Foundation Trust | N/A |
| Active Not Recruiting | Assessment of Potential for Chronic Liver Injury in Participants Treated With Epidiolex (Cannabidiol) Oral Sol NCT05044819 | Jazz Pharmaceuticals | Phase 4 |
| Unknown | Efficacy and Safety of Rapamycin Versus Vigabatrin in the Prevention of Tuberous Sclerosis Complex Symptoms in NCT04987463 | Katarzyna Kotulska | Phase 2 / Phase 3 |
| Completed | Stopping TSC Onset and Progression 2: Epilepsy Prevention in TSC Infants NCT04595513 | Children's Hospital Medical Center, Cincinnati | Phase 1 / Phase 2 |
| Active Not Recruiting | The Effectiveness and Safety of Resective Epilepsy Surgery for TRE NCT04198181 | Beijing Children's Hospital | — |
| Completed | The Effectiveness and Safety of Vagus Nerve Stimulation for TRE NCT04198207 | Beijing Children's Hospital | — |
| Unknown | Dermatologic Patterns of Tuberous Sclerosis Patients and Somatic Mutation Relationship NCT04112537 | University Hospital, Montpellier | — |
| Unknown | Aspirin as an add-on Treatment of Refractory Epilepsy in Tuberous Sclerosis Complex NCT03356769 | Peking Union Medical College Hospital | Phase 2 |
| Active Not Recruiting | Roll-over Study to Collect and Assess Long-term Safety of Everolimus in Patients With TSC and Refractory Seizu NCT02962414 | Novartis Pharmaceuticals | Phase 3 |
| Completed | Preventing Epilepsy Using Vigabatrin In Infants With Tuberous Sclerosis Complex NCT02849457 | Martina Bebin | Phase 2 |
| Completed | An Open-label Extension Trial of Cannabidiol (GWP42003-P, CBD) for Seizures in Tuberous Sclerosis Complex (GWP NCT02544750 | Jazz Pharmaceuticals | Phase 3 |
| Completed | Predicting Epileptogenic Tubers in Patients with Tuberous Sclerosis Complex NCT06789419 | Beijing Children's Hospital | — |
| Completed | Studies in Patients With Tuberous Sclerosis Complex NCT03276195 | Translational Genomics Research Institute | — |
| Completed | A Randomized Controlled Trial of Cannabidiol (GWP42003-P, CBD) for Seizures in Tuberous Sclerosis Complex (GWP NCT02544763 | Jazz Pharmaceuticals | Phase 3 |
| Completed | Early Behavioral Intervention to Improve Social Communication Function in Infants With Tuberous Sclerosis Comp NCT02687633 | University of California, Los Angeles | N/A |
| Unknown | Clinical Presentation and Renal Outcome of Patients With Tuberous Sclerosis Complex and/or Renal Angiomyolipom NCT02887781 | University Hospital, Brest | — |
| Unknown | The Cognitive Variability in NF1 and TSC Monozygotic Twins NCT02436746 | Erasmus Medical Center | — |
| Completed | Everolimus for Cancer With TSC1 or TSC2 Mutation NCT02201212 | Dana-Farber Cancer Institute | Phase 2 |
| Completed | Safety of Simvastatin in LAM and TSC NCT02061397 | University of Pennsylvania | Phase 1 / Phase 2 |
| Completed | A Pilot Study To Evaluate The Effects of Everolimus on Brain mTOR Activity and Cortical Hyperexcitability in T NCT02451696 | NYU Langone Health | Phase 2 |
| Unknown | Long-term, Prospective Study Evaluating Clinical and Molecular Biomarkers of Epileptogenesis in a Genetic Mode NCT02098759 | Sergiusz Jozwiak | N/A |
| Completed | Rapalogues for Autism Phenotype in TSC: A Feasibility Study NCT01929642 | Hugo W. Moser Research Institute at Kennedy Krieger, Inc. | Phase 2 |
| Completed | Early Biomarkers of Autism in Infants With Tuberous Sclerosis Complex (TSC) NCT01780441 | Boston Children's Hospital | — |
| Unknown | Efficacy of RAD001/Everolimus in Autism and NeuroPsychological Deficits in Children With Tuberous Sclerosis Co NCT01730209 | Erasmus Medical Center | Phase 2 / Phase 3 |
| Completed | Potential EEG Biomarkers and Antiepileptogenic Strategies for Epilepsy in TSC NCT01767779 | University of Alabama at Birmingham | — |
| Completed | Trial of RAD001 and Neurocognition in Tuberous Sclerosis Complex (TSC) NCT01289912 | Mustafa Sahin | Phase 2 |
| Completed | Everolimus (RAD001) Therapy for Epilepsy in Patients With Tuberous Sclerosis Complex (TSC) NCT01070316 | Children's Hospital Medical Center, Cincinnati | Phase 1 / Phase 2 |
| Completed | Tuberous Sclerosis Complex Natural History Study: Renal Manifestations NCT00598455 | Children's Hospital Medical Center, Cincinnati | — |