11 completed, 3 recruiting, 2 terminated, 1 active not recruiting. 19 Phase 3, 4 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 19 | Completed 11, Recruiting 3, Terminated 2, Active not recruiting 1, Unknown 1, Withdrawn 1 |
| Phase 2 | 4 | Completed 1, Recruiting 1, Unknown 1, Withdrawn 1 |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 |
|---|---|---|---|---|---|---|
| Treatment With Full-spectrum Cannabis Extract of Refractory Epilepsy Associated With Tuberous Sclerosis Complex (TSC) YCJ-01 Full Spectrum cannbis Extract, Placebo - Tuberous Sclerosis Complex (TSC) | Phase 3 | Recruiting | Oils4Cure | 84 | 2024-06-01 | 2026-02-11 |
| Placebo Controlled Study to Assess the Efficacy and Safety of Rapamycin in Drug Resistant Epilepsy Associated With Tuberous Sclerosis Complex Rapamycin, Placebo - Tuberous Sclerosis Complex | Phase 3 | Recruiting | Katarzyna Kotulska | 200 | 2023-01-23 | 2026-10-22 |
| Clinical Study of NPC-12Y Gel in Patients With Skin Lesions Associated With TSC NPC-12Y gel, NPC-12Y placebo gel - Tuberous Sclerosis Complex | Phase 3 | Completed | Nobelpharma | 43 | 2022-06-01 | 2024-11-22 |
| Open-label Study of Adjunctive GNX Treatment in Children and Adults With TSC-related Epilepsy Ganaxolone - Tuberous Sclerosis Complex | Phase 3 | Terminated | Marinus Pharmaceuticals | 117 | 2022-05-16 | 2025-10-01 |
| Adjunctive GNX Treatment Compared With Placebo in Children and Adults With TSC-related Epilepsy Ganaxolone, Placebo - Tuberous Sclerosis Complex | Phase 3 | Completed | Marinus Pharmaceuticals | 129 | 2022-04-01 | 2025-07-11 |
| Assessment of Adjunctive Cannabidiol Oral Solution (GWP42003-P) in Children With Tuberous Sclerosis Complex (TSC), Dravet Syndrome (DS), or Lennox-Gastaut Syndrome (LGS) Who Experience Inadequately-controlled Seizures GWP42003-P - Seizure in Participants With Tuberous Sclerosis Complex, Seizure in Participants With Dravet Syndrome | Phase 3 | Terminated | Jazz Pharmaceuticals | 3 | 2021-05-19 | 2025-10-31 |
| 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 |
| Topical Everolimus in Patients With Tuberous Sclerosis Complex Everolimus, Placebo - Facial Angiofibromas | Phase 2 / Phase 3 | Withdrawn | Hospices Civils de Lyon | 2020-12-01 | 2021-05-03 | |
| 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 |
| Roll-over Study to Collect and Assess Long-term Safety of Everolimus in Patients With TSC and Refractory Seizures Who Have Completed the EXIST-3 Study [CRAD001M2304] and Who Are Benefitting From Continued Treatment everolimus - Tuberous Sclerosis Complex | Phase 3 | Active not recruiting | Novartis Pharmaceuticals | 206 | 2017-08-23 | 2026-08-21 |
| An Open-label Extension Trial of Cannabidiol (GWP42003-P, CBD) for Seizures in Tuberous Sclerosis Complex (GWPCARE6) GWP42003-P - Tuberous Sclerosis Complex, Seizures | Phase 3 | Completed | Jazz Pharmaceuticals | 199 | 2016-08-31 | 2022-07-14 |
| A Randomized Controlled Trial of Cannabidiol (GWP42003-P, CBD) for Seizures in Tuberous Sclerosis Complex (GWPCARE6) GWP42003-P, Placebo - Tuberous Sclerosis Complex, Seizures | Phase 3 | Completed | Jazz Pharmaceuticals | 224 | 2016-04-06 | 2022-09-28 |
| Phase III Trial of Topical Formulation of Sirolimus to Skin Lesions in Patients With Tuberous Sclerosis Complex (TSC) NPC-12G gel, Placebo gel - Tuberous Sclerosis, Angiofibroma | Phase 3 | Completed | Nobelpharma | 62 | 2015-12-01 | 2017-02-02 |
| Long-term Trial of Topical Sirolimus to Angiofibroma in Patient With Tuberous Sclerosis Complex NPC-12G gel - Tuberous Sclerosis, Angiofibroma | Phase 3 | Completed | Nobelpharma | 94 | 2015-12-01 | 2019-02-20 |
| Efficacy and Safety of Circadin® in the Treatment of Sleep Disturbances in Children With Neurodevelopment Disabilities Circadin 2/5/10 mg, Placebo - Sleep Disorders | Phase 3 | Completed | Neurim Pharmaceuticals Ltd. | 125 | 2013-10-01 | 2024-04-23 |
| Topical Rapamycin and Calcitriol for Angiofibroma of Tuberous Sclerosis Rapamycin, Calcitriol, Rapamycin-calcitriol combination - Facial Angiofibroma | Phase 3 | Completed | National Taiwan University Hospital | 52 | 2013-09-05 | 2017-05-04 |
| 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 |
| Efficacy and Safety of Everolimus (RAD001) in Patients of All Ages With Subependymal Giant Cell Astrocytoma Associated With Tuberous Sclerosis Complex (TSC)(EXIST-1) Everolimus, Placebo - Tuberous Sclerosis, Subependymal Giant Cell Astrocytoma | Phase 3 | Completed | Novartis Pharmaceuticals | 117 | 2009-08-01 | 2016-02-03 |
| Efficacy and Safety of RAD001 in Patients Aged 18 and Over With Angiomyolipoma Associated With Either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM) Everolimus (RAD001), Everolimus Placebo - Tuberous Sclerosis Complex (TSC), Lymphangioleiomyomatosis (LAM) | Phase 3 | Completed | Novartis Pharmaceuticals | 118 | 2009-04-01 | 2017-02-17 |
Seizures (5) · Epilepsy (3) · Angiofibroma (2) · Astrocytoma (2) · Plaque, Amyloid (2) · Tuberous Sclerosis 2 (2) · Angelman Syndrome (1) · Angiomyolipoma (1) · Autism Spectrum Disorder (1) · Autistic Disorder (1) · Drug Resistant Epilepsy (1) · Epilepsies, Myoclonic (1) · Hereditary Sensory and Autonomic Neuropathies (1) · Intellectual Disability (1)
Congenital Abnormalities (19) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (19) · Genetic Diseases, Inborn (19) · Hamartoma (19) · Heredodegenerative Disorders, Nervous System (19) · Malformations of Cortical Development (19) · Malformations of Cortical Development, Group I (19) · Neoplasms, Multiple Primary (19) · Neoplastic Syndromes, Hereditary (19) · Nervous System Diseases (19) · Nervous System Malformations (19) · Neurocutaneous Syndromes (19) · Neurodegenerative Diseases (19) · Neoplasms (18) · Pathological Conditions, Signs and Symptoms (9)
Everolimus (5) · Sirolimus (4) · Cannabidiol (3) · ganaxolone (2) · Calcitriol (1) · Fluid Therapy (1) · Melatonin (1) · Vigabatrin (1)
Jazz Pharmaceuticals (3) · Nobelpharma (3) · Novartis Pharmaceuticals (3) · Katarzyna Kotulska (2) · Marinus Pharmaceuticals (2) · Dermatology Specialties Limited Partnership (1) · Erasmus Medical Center (1) · Hospices Civils de Lyon (1) · National Taiwan University Hospital (1) · Neurim Pharmaceuticals Ltd. (1) · Oils4Cure (1)
United States (10) · Spain (8) · United Kingdom (7) · Australia (6) · France (6) · Italy (6) · Poland (6) · Canada (5) · Japan (5) · Netherlands (5) · Germany (4) · Russia (3) · Belgium (2) · China (2) · Hungary (2)
Ioana Stanescu (1) · Katarzyna Kotulska-Jozwiak (1) · Mari Wataya-Kaneda, MD, PhD (1) · Mari Wataya-Kaneda,, MD, PhD (1) · Paul Gringras, PhD (1) · Robert Findling, MD (1)
PMID 29023494, PMID 30053159, PMID 34957550, PMID 23158522, PMID 23733802, PMID 24729041, PMID 25456370, PMID 25682485, PMID 26156073, PMID 26858193, PMID 27351628, PMID 29096777, PMID 29800026, PMID 30192751, PMID 31217257, PMID 31982581, PMID 32385845, PMID 33346789, PMID 35175622, PMID 37463422, PMID 38441854
2015 (2), 2008 (1), 2018 (1)