11 completed. 11 Phase 1, 7 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 11 | Completed 11 |
| Phase 2 | 7 | Completed 7 |
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 |
|---|---|---|---|---|---|---|
| Phase 1, Open-label, Drug-drug Interaction Study to Assess Effect Between Radiprodil and the Co-administered Drugs. Radiprodil + co-administered drugs - Tuberous Sclerosis Complex (TSC), Focal Cortical Dysplasia | Phase 1 | Completed | GRIN Therapeutics, Inc. | 18 | 2025-05-20 | 2025-11-18 |
| 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 |
| 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 |
| 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 |
| Tuberous Sclerosis Complex: Facial Angiofibroma Skin Cream Rapamune - Facial Angiofibroma | Phase 1 | Completed | Le Bonheur Children's Hospital | 11 | 2013-10-01 | 2016-08-19 |
| Assessment of the Pharmacokinetics of Circadin® in Children With Neurodevelopmental Disorders and Sleep Disturbances Circadin 2 mg, Circadin 10 mg - Sleep Problems | Phase 1 | Completed | Neurim Pharmaceuticals Ltd. | 16 | 2013-03-01 | 2014-04-04 |
| 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 |
| Topical Rapamycin Therapy to Alleviate Cutaneous Manifestations of Tuberous Sclerosis Complex (TSC) and Neurofibromatosis I (NF1) Skincerity, Skincerity plus sirolimus/rapamycin, Skinercity plus sirolimus/rapamycin - Tuberous Sclerosis, Neurofibromatoses | Phase 1 | Completed | The University of Texas Health Science Center, Houston | 52 | 2009-12-01 | 2012-02-27 |
| 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 |
| 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 |
Epilepsy (3) · Hereditary Sensory and Autonomic Neuropathies (3) · Angiofibroma (2) · Lymphangioleiomyomatosis (2) · Angelman Syndrome (1) · Angiolipoma (1) · Astrocytoma (1) · Autism Spectrum Disorder (1) · CDKL5 deficiency disorder (1) · Epilepsies, Myoclonic (1) · Focal Cortical Dysplasia (1) · Lennox Gastaut Syndrome (1) · Neurofibroma (1) · Neurofibromatoses (1)
Congenital Abnormalities (11) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (11) · Genetic Diseases, Inborn (11) · Hamartoma (11) · Heredodegenerative Disorders, Nervous System (11) · Malformations of Cortical Development (11) · Malformations of Cortical Development, Group I (11) · Neoplasms (11) · Neoplasms, Multiple Primary (11) · Neoplastic Syndromes, Hereditary (11) · Nervous System Diseases (11) · Nervous System Malformations (11) · Neurocutaneous Syndromes (11) · Neurodegenerative Diseases (11) · Neoplasms by Histologic Type (6)
Everolimus (5) · Sirolimus (4) · Melatonin (1) · Simvastatin (1) · radiprodil (1)
Children's Hospital Medical Center, Cincinnati (5) · GRIN Therapeutics, Inc. (1) · Le Bonheur Children's Hospital (1) · Longboard Pharmaceuticals (1) · Neurim Pharmaceuticals Ltd. (1) · The University of Texas Health Science Center, Houston (1) · University of Pennsylvania (1)
United States (9) · Australia (2) · Netherlands (1)
Darcy Krueger, M.D. Ph.D (1) · Darcy Krueger, MD, PhD (1) · David N Franz, M.D. (1) · Dennis J Dlugos, MD (1) · Frank McCormack, MD (1) · Hope Northrup, MD (1) · James W Wheless, MD (1) · John Bissler, MD (1) · John J. Bissler, M.D. (1) · Mary Kay Koenig, MD (1) · Maryl Kreider, MD, MSCE (1) · Vera P Krymskaya, PhD, MBA (1)
PMID 21047224, PMID 21482669, PMID 22262746, PMID 22934754, PMID 23325902, PMID 23798472, PMID 26381530, PMID 29023494, PMID 30424962, PMID 39331837