20 completed. 20 Phase 2, 7 Phase 1, 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 20 | Completed 20 |
| Phase 1 | 7 | Completed 7 |
| Phase 3 | 1 | Completed 1 |
Follow Tuberous Sclerosis: new trials by email Literature Review of Tuberous Sclerosis trials Research Report on Tuberous Sclerosis
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 |
|---|---|---|---|---|---|---|
| Open-label, Long-term Safety Study of LP352 in Subjects With Developmental and Epileptic Encephalopathy LP352, bexicaserin - Developmental and Epileptic Encephalopathy, Dravet Syndrome | Phase 2 | Completed | Longboard Pharmaceuticals | 41 | 2022-11-08 | 2025-12-03 |
| Study to Investigate LP352 in Subjects With Developmental and Epileptic Encephalopathies LP352, Placebo - Developmental and Epileptic Encephalopathy, Dravet Syndrome | Phase 1 / Phase 2 | Completed | Longboard Pharmaceuticals | 52 | 2022-03-03 | 2024-11-08 |
| 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 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 |
| Adjunctive Ganaxolone Treatment (Part A) in TSC Followed by Long-term Treatment (Part B) Ganaxolone - Tuberous Sclerosis | Phase 2 | Completed | Marinus Pharmaceuticals | 23 | 2020-04-08 | 2023-04-04 |
| Dose-Ranging Efficacy and Safety Study of Topical Rapamycin Cream for Facial Angiofibroma Associated With Tuberous Sclerosis Complex rapamycin, placebo - Facial Angiofibroma, Tuberous Sclerosis | Phase 2 / Phase 3 | Completed | Dermatology Specialties Limited Partnership | 107 | 2019-07-28 | 2023-09-08 |
| A Phase 2, Multicenter, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy, Safety, and Tolerability of TAK-935 (OV935) as an Adjunctive Therapy in Pediatric Participants With Developmental and/or Epileptic Encephalopathies TAK-935, Placebo - Epilepsy, Dravet Syndrome | Phase 2 | Completed | Takeda | 141 | 2018-08-08 | 2026-09-14 |
| 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 |
| Topical Rapamycin to Erase Angiofibromas in TSC Placebo, Rapamycin - Angiofibromas, Tuberous Sclerosis | Phase 2 | Completed | The University of Texas Health Science Center, Houston | 179 | 2012-05-01 | 2020-10-26 |
| 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 |
| Long Term Follow Up for RAD001 Therapy of Angiomyolipomata in Patients With Tuberous Sclerosis (TSC) and Sporadic Lymphangioleiomyomatosis (LAM) everolimus (RAD001) - Tuberous Sclerosis, Angiolipoma | Phase 1 / Phase 2 | Completed | Children's Hospital Medical Center, Cincinnati | 20 | 2008-12-01 | 2013-09-18 |
| Everolimus (RAD001) Therapy of Giant Cell Astrocytoma in Patients With Tuberous Sclerosis Complex Everolimus - Tuberous Sclerosis, Subependymal Giant Cell Astrocytoma | Phase 1 / Phase 2 | Completed | Children's Hospital Medical Center, Cincinnati | 28 | 2007-01-01 | 2014-10-20 |
| RAPAMYCIN FOR KIDNEY ANGIOMYOLIPOMAS Rapamycin - Nonmalignant Neoplasm, Tuberous Sclerosis | Phase 2 | Completed | Dana-Farber Cancer Institute | 36 | 2005-12-20 | 2022-04-25 |
| RAD001 Therapy of Angiomyolipomata in Patients With TS Complex and Sporadic LAM RAD001 - Tuberous Sclerosis, Lymphangioleiomyomatosis | Phase 1 / Phase 2 | Completed | Children's Hospital Medical Center, Cincinnati | 36 | 2005-08-01 | 2013-09-25 |
| Rapamycin Therapy for Patients With Tuberous Sclerosis Complex and Sporadic LAM Rapamycin, sirolimus - Tuberous Sclerosis, Lymphangioleiomyomatosis | Phase 2 | Completed | Children's Hospital Medical Center, Cincinnati | 25 | 2002-12-01 | 2007-04-09 |
Epilepsy (6) · CDKL5 deficiency disorder (3) · Epilepsies, Myoclonic (3) · Hereditary Sensory and Autonomic Neuropathies (3) · Lennox Gastaut Syndrome (3) · Lymphangioleiomyomatosis (3) · Autistic Disorder (2) · Seizures (2) · Angiofibroma (1) · Angiolipoma (1) · Angiomyolipoma (1) · Astrocytoma (1) · Brain Diseases (1) · Central Nervous System Diseases (1)
Congenital Abnormalities (20) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (20) · Genetic Diseases, Inborn (20) · Hamartoma (20) · Heredodegenerative Disorders, Nervous System (20) · Malformations of Cortical Development, Group I (20) · Neoplasms, Multiple Primary (20) · Neoplastic Syndromes, Hereditary (20) · Nervous System Malformations (20) · Neurocutaneous Syndromes (20) · Neurodegenerative Diseases (20) · Malformations of Cortical Development (19) · Neoplasms (19) · Nervous System Diseases (19) · Neoplasms by Histologic Type (7)
Everolimus (9) · Sirolimus (7) · 2-chloro-4-(1-(4-fluorophenyl)-2,5-dimethyl-1H-imidazol-4-ylethynyl)pyridine (1) · Simvastatin (1) · Vigabatrin (1) · ganaxolone (1)
Children's Hospital Medical Center, Cincinnati (6) · Dana-Farber Cancer Institute (2) · Longboard Pharmaceuticals (2) · Dermatology Specialties Limited Partnership (1) · Hugo W. Moser Research Institute at Kennedy Krieger, Inc. (1) · Marinus Pharmaceuticals (1) · Martina Bebin (1) · Mustafa Sahin (1) · NYU Langone Health (1) · Noema Pharma AG (1) · Takeda (1) · The University of Texas Health Science Center, Houston (1) · University of Pennsylvania (1)
United States (20) · Australia (5) · Spain (3) · Israel (2) · Poland (2) · Canada (1) · China (1) · Czechia (1) · Hungary (1) · India (1) · Italy (1) · New Zealand (1) · Portugal (1) · Serbia (1) · Slovakia (1)
Dennis J Dlugos, MD (2) · John Bissler, MD (2) · Darcy Krueger, M.D. Ph.D (1) · Darcy Krueger, MD, PhD (1) · David Kwiatkowski, MD, PhD (1) · David N Franz, M.D. (1) · Frank McCormack, MD (1) · Hope Northrup, MD (1) · Ioana Stanescu (1) · John J. Bissler, M.D. (1) · Martina Bebin, MD, MPA (1) · Mary Kay Koenig, MD (1) · Maryl Kreider, MD, MSCE (1)
PMID 18184959, PMID 21047224, PMID 21482669, PMID 21915260, PMID 22262746, PMID 23325902, PMID 23437092, PMID 23798472, PMID 26381530, PMID 29023494, PMID 29800048, PMID 30424962, PMID 31912454, PMID 35587487, PMID 35841234, PMID 37463422, PMID 37638552, PMID 39331837
2018 (1), P30CA006516 (1), R01CA107164 (1), R21CA103486 (1), U01NS092595 (1)