54 completed, 21 recruiting, 11 unknown, 10 active not recruiting. 35 Phase 2, 19 Phase 3, 16 Phase 1, 14 Not applicable, 6 Phase 4.
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 |
|---|---|---|---|---|---|---|
| Neurocutaneous Syndromes in Children: Prevalence and Clinical Characteristics Sturge-Weber Syndrome, Neurocutaneous Syndromes | Not yet recruiting | Assiut University | 100 | 2026-09-20 | 2026-09-28 | |
| 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 |
| Safety, Tolerability, and Pharmacokinetics of SVG103 (Paxalisib) in Focal Cortical Dysplasia Type II (FCD-II), Tuberous Sclerosis Complex (TSC) or Hemimegalencephaly (HME) SVG103 - Focal Cortical Dysplasia, Tuberous Sclerosis Complex (TSC) | Phase 1 / Phase 2 | Recruiting | Sovargen | 12 | 2026-07-14 | 2026-08-10 |
| Periodontal Disease in Rare Renal Disorders (PERIO-RA-RE) Observational assessment - Periodontal Disease, Periodontitis | Recruiting | Stefan Lujinschi | 100 | 2026-05-04 | 2026-06-26 | |
| A Smart Phone Application to Improve Adoption of the 2024 Kidney Disease Improving Global Outcomes (KDIGO) Chronic Kidney Disease (CKD) Guidelines Use of an electronic digital Application - Chronic Kidney Disease, Proteinuria | Not applicable | Recruiting | St. James's Hospital, Ireland | 80 | 2026-01-14 | 2026-05-01 |
| Sapu003 in Advanced mTOR-sensitive Solid Tumors Sapu003, Exemestane 25 MG - Breast Cancer Metastatic, Renal Cell Carcinoma (RCC) | Phase 1 | Recruiting | SAPU NANO (US) LLC | 27 | 2025-12-15 | 2026-01-27 |
| TANDem-2: Closing the Gap to Interventions for TAND Well-Beans for Caregivers Program - Adult Caregivers of Individuals With TSC, Tuberous Sclerosis Complex (TSC) | Not applicable | Enrolling by invitation | Vrije Universiteit Brussel | 30 | 2025-07-15 | 2025-09-22 |
| 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 Evaluate the Pharmacokinetics and Safety Effects Following Coadministration of Carbamazepine or Itraconazole With Radiprodil in Healthy Adults Radiprodil with Carbamazepine, Radiprodil with Itraconazole - Tuberous Sclerosis Complex (TSC), Focal Cortical Dysplasia | Phase 1 | Terminated | GRIN Therapeutics, Inc. | 30 | 2025-05-08 | 2025-11-21 |
| Epidyolex® in Lennox Gastaut, Dravet Syndrome and Tuberous Sclerosis Complex: an Observational Study in ITALY Epidiolex 100 mg/mL Oral Solution - Lennox Gastaut Syndrome, Dravet Syndrome | Active not recruiting | Jazz Pharmaceuticals | 111 | 2025-02-24 | 2026-06-03 | |
| Sertraline vs. Placebo in the Treatment of Anxiety in Children and AdoLescents With NeurodevelopMental Disorders Sertraline, Placebo - Neurodevelopmental Disorders, Autism | Phase 2 | Recruiting | Holland Bloorview Kids Rehabilitation Hospital | 130 | 2024-09-16 | 2026-07-27 |
| 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 |
| 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 |
| Reproductive Options in Inherited Skin Diseases Ichthyosis, Palmoplantar Keratoses | Enrolling by invitation | Maastricht University Medical Center | 650 | 2024-01-01 | 2025-05-18 | |
| Qualitative Study in Patients With Genodermatoses and Healthcare Professionals on Reproductive Counselling Qualitative interview - Quality of Life, Ichthyosis | Recruiting | Maastricht University Medical Center | 25 | 2024-01-01 | 2025-05-18 | |
| Web Intervention for Parents of Youth With Genetic Syndromes (WINGS) Functional Behavioral Training (FBT), Positive Parenting Strategies-Treatment as Usual - Telomeric 22Q13 Monosomy Syndrome, Tuberous Sclerosis | Not applicable | Active not recruiting | Rush University Medical Center | 79 | 2023-09-15 | 2026-09-18 |
| Tuberous Sclerosis Complex and Lymphangioleiomyomatosis Pregnancy Registry (TSC-LAM Registry) Tuberous Sclerosis Complex, Lymphangioleiomyomatosis | Recruiting | David M. Ritter | 300 | 2023-07-01 | 2026-01-23 | |
| A Study to Investigate Behavioral and Other Co-Occurring Outcomes With Epidiolex as Add-On Therapy in Participants Aged 1 to 65 Years of Age With Tuberous Sclerosis Complex Cannabidiol Oral Solution [Epidiolex] - Tuberous Sclerosis Complex Associated Neuropsychiatric Disease | Phase 4 | Completed | Jazz Pharmaceuticals | 79 | 2023-06-29 | 2026-04-27 |
| A Phase IV Study of Safety and Efficacy of Everolimus in Taiwanese Patients With Tuberous Sclerosis Complex Who Have Renal Angiomyolipoma (TSC-AML) Everolimus - Renal Angiomyolipoma | Phase 4 | Active not recruiting | Novartis Pharmaceuticals | 4 | 2023-05-01 | 2026-09-17 |
| 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 |
Showing 20 of 114. Sign in to see all of them.
Epilepsy (22) · Lymphangioleiomyomatosis (16) · Seizures (14) · Autistic Disorder (8) · Epilepsies, Myoclonic (8) · Lennox Gastaut Syndrome (8) · Angiomyolipoma (7) · Focal Cortical Dysplasia (7) · Angiofibroma (5) · Neurofibromatosis 1 (5) · Angelman Syndrome (4) · Astrocytoma (4) · CDKL5 deficiency disorder (4) · Hereditary Sensory and Autonomic Neuropathies (4)
Congenital Abnormalities (114) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (114) · Genetic Diseases, Inborn (114) · Hamartoma (114) · Heredodegenerative Disorders, Nervous System (114) · Malformations of Cortical Development, Group I (114) · Neoplasms, Multiple Primary (114) · Nervous System Malformations (114) · Neurodegenerative Diseases (114) · Neoplastic Syndromes, Hereditary (113) · Nervous System Diseases (113) · Malformations of Cortical Development (112) · Neoplasms (112) · Neurocutaneous Syndromes (111) · Neoplasms by Histologic Type (39)
Everolimus (18) · Sirolimus (15) · Cannabidiol (6) · Activator Appliances (3) · Vigabatrin (3) · ganaxolone (3) · radiprodil (3) · Melatonin (2) · 2-chloro-4-(1-(4-fluorophenyl)-2,5-dimethyl-1H-imidazol-4-ylethynyl)pyridine (1) · Acceptance and Commitment Therapy (1) · Actigraphy (1) · Anticonvulsants (1) · Aspirin (1) · Calcitriol (1) · Carbamazepine (1)
Children's Hospital Medical Center, Cincinnati (7) · Jazz Pharmaceuticals (6) · Novartis Pharmaceuticals (6) · National Heart, Lung, and Blood Institute (NHLBI) (5) · University of California, Los Angeles (5) · Beijing Children's Hospital (3) · GRIN Therapeutics, Inc. (3) · Longboard Pharmaceuticals (3) · Marinus Pharmaceuticals (3) · Nobelpharma (3) · Boston Children's Hospital (2) · Cardiff University (2) · Dana-Farber Cancer Institute (2) · Erasmus Medical Center (2) · Katarzyna Kotulska (2)
United States (62) · Australia (17) · United Kingdom (15) · France (13) · Canada (12) · Italy (12) · Netherlands (12) · Spain (12) · Poland (11) · China (9) · Belgium (6) · Germany (5) · Japan (5) · Israel (4) · Russia (3)
Darcy Krueger, MD, PhD (3) · Joel Moss, M.D. (3) · Anna Jansen, MD, PhD (2) · Antoni Gostynski, MD, PhD (2) · Dennis J Dlugos, MD (2) · Hope Northrup, MD (2) · John Bissler, MD (2) · Martina Bebin, MD, MPA (2) · Mary Kay Koenig, MD (2) · Mustafa Sahin, MD, PhD (2) · Alessandra Pereira,, PhD, MD (1) · Alessandro Flavio Ferri (1) · Andrea Pasini, MD (1)
PMID 29023494, PMID 30053159, PMID 30291925, PMID 31912454, PMID 34957550, PMID 38996393, PMID 18184959, PMID 19141364, PMID 21047224, PMID 21482669, PMID 21525172, PMID 21915260, PMID 22113127, PMID 22262746, PMID 22934754, PMID 23158522, PMID 23325902, PMID 23437092, PMID 23732715, PMID 23733802, PMID 23798472, PMID 24729041, PMID 25456370, PMID 25682485, PMID 26156073, PMID 26381530, PMID 26858193, PMID 27222591, PMID 27351628, PMID 29096777, PMID 29572283, PMID 29797247, PMID 29800026, PMID 29800048, PMID 30192751, PMID 30424962, PMID 31217257, PMID 31237569, PMID 31444548, PMID 31969108, PMID 31982581, PMID 32385845, PMID 33346789, PMID 33407896, PMID 33434304, PMID 33549601, PMID 33834539, PMID 35175622, PMID 35587487, PMID 35841234
2015 (2), 2018 (2), 2023 (2), 2008 (1), 2025 (1), P30CA006516 (1), R01CA107164 (1), R01HD090138 (1), R21CA103486 (1), U01NS082320 (1), U01NS092595 (1), U54NS092090 (1)