9 completed, 3 active not recruiting, 2 unknown, 1 not yet recruiting. 16 Phase 2, 5 Phase 1, 2 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 16 | Completed 9, Active not recruiting 3, Unknown 2, Not yet recruiting 1, Recruiting 1 |
| Phase 1 | 5 | Completed 3, Active not recruiting 2 |
| Phase 3 | 2 | Recruiting 1, Unknown 1 |
Follow Tuberous Sclerosis Complex: new trials by email Literature Review of Tuberous Sclerosis Complex trials Research Report on Tuberous Sclerosis Complex
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Safety and Efficacy of AV078 in Participants With Tuberous Sclerosis Complex (TSC) Refractory Epilepsy (RESTOR-1) AV078, Placebo - Tuberous Sclerosis Complex, Epilepsy | Phase 2 | Not yet recruiting | Aeovian Pharmaceuticals, Inc. | 42 | 2026-09-01 | 2026-09-29 |
| Astroscape: A Study of Radiprodil on Safety, Tolerability, Pharmacokinetics, and Effect on Seizures and Behavioral Symptoms in Patients With TSC or FCD Type II Radiprodil - Tuberous Sclerosis Complex, Focal Cortical Dysplasia | Phase 1 / Phase 2 | Active not recruiting | GRIN Therapeutics, Inc. | 30 | 2024-07-10 | 2026-04-01 |
| Basimglurant (NOE-101) in Children, Adolescents, and Young Adults With TSC Basimglurant with crossover to Placebo, Placebo with crossover to Basimglurant - Tuberous Sclerosis Complex | Phase 2 | Completed | Noema Pharma AG | 61 | 2022-03-03 | 2025-06-04 |
| Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study Sirolimus, Placebo - Tuberous Sclerosis Complex, Epilepsy | Phase 2 | Active not recruiting | Darcy Krueger | 64 | 2021-10-13 | 2026-10-29 |
| Feasibility of [11C]Acetate-PET in LAM and TSC [11C]acetate - Lymphangioleiomyomatosis, Tuberous Sclerosis Complex | Phase 1 / Phase 2 | Active not recruiting | Brigham and Women's Hospital | 7 | 2021-08-30 | 2026-01-21 |
| Efficacy and Safety of Rapamycin Versus Vigabatrin in the Prevention of Tuberous Sclerosis Complex Symptoms in Infants Vigabatrin, Rapamycin, Placebo - Tuberous Sclerosis Complex | Phase 2 / Phase 3 | Recruiting | Katarzyna Kotulska | 60 | 2021-05-07 | 2026-10-22 |
| Stopping TSC Onset and Progression 2: Epilepsy Prevention in TSC Infants TAVT-18 (sirolimus) - Tuberous Sclerosis Complex, Epilepsy | Phase 1 / Phase 2 | Completed | Children's Hospital Medical Center, Cincinnati | 5 | 2020-09-08 | 2024-05-16 |
| Aspirin as an add-on Treatment of Refractory Epilepsy in Tuberous Sclerosis Complex Aspirin, AED, Placebo - Tuberous Sclerosis Complex, Aspirin | Phase 2 | Unknown | Peking Union Medical College Hospital | 98 | 2017-11-20 | 2020-06-09 |
| Preventing Epilepsy Using Vigabatrin In Infants With Tuberous Sclerosis Complex Early Vigabatrin, Delayed Vigabatrin (Placebo) - Tuberous Sclerosis Complex | Phase 2 | Completed | Martina Bebin | 84 | 2016-12-01 | 2024-08-19 |
| Everolimus for Cancer With TSC1 or TSC2 Mutation Everolimus - TSC1, TSC2 | Phase 2 | Completed | Dana-Farber Cancer Institute | 30 | 2014-09-01 | 2020-09-16 |
| Safety of Simvastatin in LAM and TSC Simvastatin, Sirolimus Oral Product, Everolimus Oral Product - Lymphangioleiomyomatosis, Tuberous Sclerosis Complex | Phase 1 / Phase 2 | Completed | University of Pennsylvania | 10 | 2014-03-01 | 2020-08-25 |
| A Pilot Study To Evaluate The Effects of Everolimus on Brain mTOR Activity and Cortical Hyperexcitability in TSC and FCD Everolimus - Epilepsy, Tuberous Sclerosis Complex | Phase 2 | Completed | NYU Langone Health | 15 | 2014-01-01 | 2021-09-01 |
| Rapalogues for Autism Phenotype in TSC: A Feasibility Study Sirolimus, Everolimus - Tuberous Sclerosis Complex, Self-injury | Phase 2 | Completed | Hugo W. Moser Research Institute at Kennedy Krieger, Inc. | 3 | 2013-07-01 | 2021-03-29 |
| Efficacy of RAD001/Everolimus in Autism and NeuroPsychological Deficits in Children With Tuberous Sclerosis Complex Everolimus, Placebo - Tuberous Sclerosis Complex, TSC Related Cognitive Disability | Phase 2 / Phase 3 | Unknown | Erasmus Medical Center | 60 | 2012-11-01 | 2015-05-05 |
| Trial of RAD001 and Neurocognition in Tuberous Sclerosis Complex (TSC) RAD001, Placebo - Tuberous Sclerosis Complex | Phase 2 | Completed | Mustafa Sahin | 52 | 2011-01-01 | 2018-01-25 |
| Everolimus (RAD001) Therapy for Epilepsy in Patients With Tuberous Sclerosis Complex (TSC) Everolimus - Epilepsy, Tuberous Sclerosis Complex | Phase 1 / Phase 2 | Completed | Children's Hospital Medical Center, Cincinnati | 20 | 2010-01-01 | 2017-03-13 |
Tuberous Sclerosis (16) · Epilepsy (7) · Autistic Disorder (3) · Seizures (3) · Focal Cortical Dysplasia (2) · Lymphangioleiomyomatosis (2) · Cognitive Dysfunction (1) · Drug Resistant Epilepsy (1) · Hereditary Sensory and Autonomic Neuropathies (1) · Intellectual Disability (1) · Neoplasms (1) · Self-Injurious Behavior (1)
Congenital Abnormalities (16) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (16) · Genetic Diseases, Inborn (16) · Hamartoma (16) · Heredodegenerative Disorders, Nervous System (16) · Malformations of Cortical Development (16) · Malformations of Cortical Development, Group I (16) · Neoplasms, Multiple Primary (16) · Neoplastic Syndromes, Hereditary (16) · Nervous System Diseases (16) · Nervous System Malformations (16) · Neurocutaneous Syndromes (16) · Neurodegenerative Diseases (16) · Neoplasms (15) · Brain Diseases (7)
Everolimus (7) · Sirolimus (5) · Vigabatrin (2) · 2-chloro-4-(1-(4-fluorophenyl)-2,5-dimethyl-1H-imidazol-4-ylethynyl)pyridine (1) · Anticonvulsants (1) · Aspirin (1) · Fluid Therapy (1) · Simvastatin (1) · carbon-11 acetate (1) · radiprodil (1)
Children's Hospital Medical Center, Cincinnati (2) · Aeovian Pharmaceuticals, Inc. (1) · Brigham and Women's Hospital (1) · Dana-Farber Cancer Institute (1) · Darcy Krueger (1) · Erasmus Medical Center (1) · GRIN Therapeutics, Inc. (1) · Hugo W. Moser Research Institute at Kennedy Krieger, Inc. (1) · Katarzyna Kotulska (1) · Martina Bebin (1) · Mustafa Sahin (1) · NYU Langone Health (1) · Noema Pharma AG (1) · Peking Union Medical College Hospital (1) · University of Pennsylvania (1)
United States (11) · Poland (3) · Canada (2) · Italy (2) · Netherlands (2) · Spain (2) · United Kingdom (2) · Australia (1) · Belgium (1) · China (1) · India (1) · Israel (1) · Turkey (Türkiye) (1)
Martina Bebin, MD, MPA (2) · Carmen P Priolo, MD PhD (1) · Darcy A Krueger, MD, PhD (1) · Darcy Krueger, M.D. Ph.D (1) · Darcy Krueger, MD, PhD (1) · David Kwiatkowski, MD, PhD (1) · Frank McCormack, MD (1) · Katarzyna Kotulska-Jozwiak (1) · Maryl Kreider, MD, MSCE (1) · Mustafa Sahin, MD, PhD (1) · Orrin Devinsky, MD (1) · Qing Liu, MD PhD (1) · Tanjala Gipson, MD (1)
PMID 21482669, PMID 23798472, PMID 31217257, PMID 31912454, PMID 35587487, PMID 37638552, PMID 39331837
2023 (1), 2025 (1), U01NS092595 (1)